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Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Whether you’re newly diagnosed or managing ongoing care, learn how the latest findings impact your myeloproliferative neoplasms (MPN) treatment options and quality of life.

Dr. John Mascarenhas of The Tisch Cancer Institute at Mount Sinai and patient advocate Andrew Schorr share the latest breakthroughs in MPN care. Explore personalized treatments, cutting-edge therapies, and groundbreaking research that are changing how MPNs are treated. Learn how new discoveries can improve your treatment options, help manage side effects, and enhance your quality of life.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care
Hosted by The Patient Story Team
Dr. John Mascarenhas (Mount Sinai) and patient advocate Andrew Schorr share the latest breakthroughs in MPN care. Explore personalized treatments, cutting-edge therapies, and groundbreaking research that’s changing how MPNs are treated. Learn how new discoveries can improve your treatment options, help manage side effects, and enhance your quality of life.
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Hear about cutting-edge research and new therapies presented at the 2024 American Society of Hematology meeting. Learn how individualized treatments can improve your outcomes and quality of life. Get practical strategies for handling common side effects of MPN treatments. Discover innovative therapies, including JAK inhibitors and combination treatments. Find out how to stay informed and participate in promising clinical trials. Learn the key questions to ask your healthcare team to ensure you’re receiving the best, most current care.


The Leukemia & Lymphoma Society is here for you with information about clinical trials, resources, and support.

Thank you to The Leukemia & Lymphoma Society for their partnership. The Leukemia & Lymphoma Society is here for you with information about clinical trials, resources, and support.


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Thank you to Sobi and Incyte for supporting our patient education program. The Patient Story retains full editorial control over all content.

This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make treatment decisions.



Introduction

Tiffany Drummond: As a clinical researcher and patient advocate, I am excited to talk about some very exciting developments in MPN treatment, including important breakthroughs and promising new combination therapies. Many of these advancements were highlighted at the 2024 American Society of Hematology meeting, better known as ASH, where leading doctors and researchers from around the world gather to share the latest findings.

Our goal is to provide patients and care partners with valuable information to help in their healthcare journey. We want to empower you to have informed conversations with your medical team so you can better understand your treatment options and how to balance effective care with maintaining your quality of life.

Tiffany Drummond patient advovate

We want to thank our sponsors, Sobi and Incyte, for their support, which helps us host more of these programs for free to our audience. The Patient Story retains full editorial control over all content as always. We also thank all of our promotional partners for their support. It is because of you our programs reach the audience who needs it. While we hope you find this program helpful, please keep in mind that the information provided is not a substitute for medical advice.

Let’s kick off another engaging conversation with amazing patient advocate Andrew Schorr and leading hematologist-oncologist Dr. John Mascarenhas.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Andrew Schorr: Welcome to this program about the latest in MPNs. I’m with a friend and leading scientist-physician Dr. John Mascarenhas at The Tisch Cancer Institute at Mount Sinai in New York. John, you have many titles. You’re a noted hematologist and subspecialist in MPNs. Thanks for joining us.

Dr. John Mascarenhas: Andy, thanks for inviting me. I always enjoy connecting with you.

There has been a lot of interest as we understand the disease biology even greater than we did in 2005 when the JAK mutation was first discovered.

Dr. John Mascarenhas

Is There Encouraging Progress for Myelofibrosis Patients?

Andrew: It’s very personal for me. I’ve been living with primary myelofibrosis since 2011 and it’s somewhat progressed. I’ve been on two JAK inhibitors and maybe I’ll switch to a third. Will I have combination therapy with a JAK inhibitor and something else? We all wonder about that.

Some of us are concerned. Should we have a transplant or can medical therapies take the place of a transplant? If you have polycythemia vera or essential thrombocythemia, you ask if you’re going to progress to myelofibrosis and at what rate. How is our situation different from the next person?

John, you were a speaker at the 2024 ASH meeting in San Diego, and you were involved in lots of studies. I want to talk about what’s significant for patients. We have the current therapies and a lot of drugs that many of us have never heard of that are in development. You’re involved in a lot of the development. Which way is the wind blowing? Are you encouraged for us? We saw progress in other blood cancers. Is it now starting to blossom in MPNs, specifically for myelofibrosis?

Dr. Mascarenhas: The short answer is yes, I am encouraged. That’s a fundamental defect that I have, continuing to be optimistic no matter what we’re looking at. That optimism has been maintained over almost 20 years that we continue to move in the right direction, but unfortunately, often not fast enough for our patients.

What I’ve seen is the evolution of the JAK inhibitor era, which you alluded to. We have four JAK inhibitors that are approved that allow us to tailor and personalize the therapy to patients based on their profile and blood counts, and even provide serial sequencing of JAK inhibitors. But that isn’t enough.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

There has been a lot of interest as we understand the disease biology even greater than we did in 2005 when the JAK mutation was first discovered. We now recognize that there’s a greater degree of complexity and heterogeneity among patients. There are a lot of different pathways that appear to be very important and relevant to the physiology and pathophysiology of this disease.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

There is a real interest in targeting the grandfather/grandmother cell in which the disease originated. We are looking for vulnerabilities in those pathways in that cell population that would allow us to ultimately delete that cell to provide deeper responses and even curative approaches. Outside of transplantation, the therapies we have don’t cure patients. They address issues that are not unimportant, like spleen size and cytopenia or low blood counts, and improve how patients do and ultimately prolong survival. But we’re looking to leverage these findings from the lab to find therapies that change the disease course and improve outcomes like survival and progression-free survival.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Many agents are leading us in that direction. These agents turn on the p53 pathway, like navtemadlin, an oral drug that binds a protein called MDM2 and relieves repression on p53, allowing for the natural cell processes to be induced, which is cell death of the malignant cell. It’s a fascinating concept. Navtemadlin is at the forefront of doing that. We showed data in the relapsed/refractory setting of using that as a single agent. We’re now going to move it up to combinations.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Drugs like that are telling. They’re hitting pathways that can induce malignant cells at their core to die, to synergize, and be practical with it. We want to create therapies and approaches that capitalize off the benefits we have, like JAK inhibitors, which can be well-tolerated but can provide deeper responses than what we’ve seen thus far. Navtemadlin is a great example of that.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

What Have We Learned from MPN Gene Mutations?

Andrew: We’re going to go through a laundry list as we dig into different drugs, but I want to go over what you said. You’re trying to go back to the very basics of cancer, what went haywire in a patient who ends up with a bone marrow problem that leads to one of the MPNs. Can you shut it down at the earliest stage by understanding it?

Over the last few years, your scientific community has identified different oncogenes (cancer genes) that have been responsible for that. You talked about the heterogeneity or the differences. Some of us have CALR, some have JAK2, and some have MPL. Is that understanding helping?

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Dr. Mascarenhas: I do think it helps because we recognize that it’s not a monolithic disease. The driver mutations and the different amounts of those mutations that are understood to be present in the bone marrow cells as well as the sequence in which the mutations arose all tell a picture. They paint a picture of complexity at the molecular and cellular level that explains why there’s heterogeneity at the clinical level — why some patients have very high white counts and some patients have very low platelet counts; why some patients have very big spleens and some patients have a lot more anemia and transfusion dependence. It can all be explained relative to the biology, these mutations, and the effects of these mutations.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Once we’ve realized that, the next step is how to take all of that complex data and distill that to help us understand how best to target those cells based on that genomic complexity. That’s where things like artificial intelligence and machine learning will help us move the field forward as it’s doing with other sciences. We’re moving in this direction of a deeper understanding of the biology from the molecular standpoint that informs us with prognostication, which is important, but most importantly, therapeutic implications.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

There is substantial data that would suggest that certain mutations likely influence outcomes and responses to treatment. Most patients will have had next-generation sequencing. You look at these gene panels and see if mutations exist in any given individual and what they mean. We know that some of these mutations can have influenced prognosis and outcome. Some of these mutations and the presence of more than one mutation could even predict a lesser response to drugs like ruxolitinib, a less robust spleen, a shorter duration of response, and a quicker time to failure of drugs. Knowing that upfront may be strategic in understanding how better to approach diseases rather than give the same drug to everybody.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

The same drug for everyone is not going to be the right answer. At the forefront, drugs that target CALR, for example, are exciting. We’re taking out a subset of patients with myelofibrosis and ET with the mutant CALR protein expressed on the surface of the cell and saying these patients may be best served by a drug that specifically targets that protein on the surface. That wouldn’t make sense for a JAK2-mutated patient. A JAK2-mutated patient may be best served with a small molecule inhibitor that specifically and selectively targets the JAK2 mutation, which is under investigation. You’re seeing these mutations inform the clinical development of very selective and specific drugs.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Andrew: I almost think of next-generation sequencing like a modern art painting with red and blue splattered. Hopefully, with some consultation with an MPN specialist, you can find out how current therapies or investigational ones apply to your specific situation.

The genes that are driving our illness may evolve, so what the story is today may not be the same story in a year, two, or three.

Andrew Schorr

How Do Doctors Choose the Right JAK Inhibitor for the Patient?

Andrew: You also mentioned how the genes that are driving our illness may evolve, so what the story is today may not be the same story in a year, two, or three. I’ve been living with myelofibrosis since 2011. It has been pretty stable and has been driven by JAK2 V617F. You mentioned the four current approved JAK inhibitors. They have nuances and it would seem like the choice of which one or sequencing, as you said, may vary by patient based on their situation. How do you know where to start?

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Dr. Mascarenhas: We are blessed that we have choices today because it wasn’t always the case. We have opportunities to select drugs that may be best suited for different subsets of patients. For example, patients with low platelets or those who have cytopenic profiles may be best served by drugs that are easily delivered and have rationale in that patient population, namely pacritinib over ruxolitinib, in which we know platelets often limit the ability to dose up on ruxolitinib. Fedratinib, even more recently, had some data providing some more security there. We know that platelets could be a determinant of which one of the JAK inhibitors you’re going to select.

Most patients will start with ruxolitinib. It’s the oldest, most familiar, and probably still one of the best drugs that I’ve ever seen in this field

Dr. John Mascarenhas

Anemia is another one that’s gotten a lot of attention. It can be a major issue for patients at presentation and can worsen over time. Ruxolitinib is a great drug, but it can worsen anemia for some patients, so it may not be the best therapy in that setting. However, drugs like momelotinib or even pacritinib that inhibit ACVR1, a different protein, can improve anemia in some patients.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

We use patient profiles to help us understand which drugs to choose from, but for the most part, most patients will start with ruxolitinib. It’s the oldest, most familiar, and probably still one of the best drugs that I’ve ever seen in this field in terms of achieving its goals. But again, not every patient fits the bill, so you can tweak that as needed.

Is Combination Therapy the Next Step in Building on JAK Inhibitors?

Andrew: A lot of studies talk about building on ruxolitinib and I’m sure there’s discussion about building on the other JAK inhibitors as well. Is a one-two punch necessarily better? Is that where you’re headed?

Dr. Mascarenhas: It’s definitely what we’re interested in asking. We have JAK inhibitors that are disposable and beneficial, but they’re not sufficient. We have other drugs that have demonstrated clinical activity and are rational and validated in preclinical models, which are systems that help us understand whether it makes sense to take it into a patient. These are drugs like pelabresib, imetelstat, navtemadlin, and selinexor, but each drug has a rationale and is active even as a single agent in these diseases.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

The question being asked now is: is it better to combine the two drugs? If you look throughout oncology, most of oncology is treated with combinations of therapy. It’s very rare to find an oncologic disease, whether it’s of the blood or of the solid malignancy, where we use one agent and then if it fails, we go to a single subsequent agent. Usually, combinations of therapy are more potent together.

If you put agent X — whether it’s selinexor, navtemadlin, pelabresib, or imetelstat — together with ruxolitinib, which tends to be the first drug you pick, you tend to see better efficacy and, in some cases, even better safety profile when the two are combined.

Dr. John Mascarenhas

A term we often use is synergize. If you take either drug alone in a lab and expose them to malignant cells, the combination of the two drugs works better than one plus one — it’s almost one plus one plus one. You get better effects in terms of killing or limiting malignant cells. We hope to replicate in humans what we see in the lab or in mice that are engineered to have these diseases.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

The data in the field has taken us towards the route of combining novel agents that have shown activity in the relapsed/refractory setting with a single agent as combination therapy upfront. Most of the data would point that if you put agent X — whether it’s selinexor, navtemadlin, pelabresib, or imetelstat — together with ruxolitinib, which tends to be the first drug you pick, you tend to see better efficacy and, in some cases, even better safety profile when the two are combined. The natural question is: if we take two active agents, can we get deeper responses? What does deeper response mean?

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

At the most superficial, it could mean more spleen reduction — that’s a regulatory endpoint — and deeper symptom improvements, but we’re looking at other biomarkers to understand if we’re hitting the target that we want. Are we getting deeper reductions in the driver mutation amounts of the variant allele frequency (VAF)? Are we reducing those numbers to suggest that we’re reducing the burden of disease in the bone marrow? Since we can’t measure it with a CT scan, are we reducing the disease in the bone marrow from other viewpoints like fibrosis? Is the amount of circulating abnormal cells reduced, something called the CD34+ cell number? Are we also reducing cytokines or inflammatory markers to a deeper extent?

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

We look at all of these biological aspects and hallmarks of the disease. Are we getting even more profound effects on these biomarkers, suggesting that we’re modulating the disease more effectively? At the end of the day, MPN patients want to live better and longer, but we look for markers early in trials to understand if we’re getting there.

Andrew: These are very powerful medicines. Will the quality of life be diminished if you add this other big gun? We want to live longer, for sure, but we want to live well. Could you talk about the power of the combinations but the worry about additional side effects?

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care

Dr. Mascarenhas: I’ll give you a prime example where one plus one can equal three from an efficacy standpoint but might still equal one from a safety standpoint. A great example is the MANIFEST-2 study. We took JAK inhibitor-naive patients with myelofibrosis and randomized them to the standard of care, which would be ruxolitinib, plus a placebo and ruxolitinib plus the study drug pelabresib, which is an oral BET inhibitor, a very rational drug that modeling has shown us should synergize very nicely with ruxolitinib. It’s a double-blinded study, so the patients and investigators don’t know what the person is getting; only a computer knows.

The answer is it did. Efficacy-wise, if you look at 24 weeks, there were very profound reductions in spleen size and very profound reductions in symptoms. If you look at the biomarkers — the bone marrow fibrosis, the inflammatory cytokines, and the JAK2 mutation — the reductions were far more significant. Everything aligned with the superiority of the combination.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

But most intriguingly, if you looked at the safety profile, there were fewer grade 3 and 4 treatment-emergent side effects with the combination than with the single agent. I’ve never seen that before where two active agents combined got almost double the clinical activity and less toxicity. I hope it’s reassuring for patients that double the action doesn’t mean double the trouble. You can combine some of these drugs, get good activity, and not make patients feel worse but even make patients feel better and have less toxicity.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

We have to acknowledge that we add toxicity sometimes when we add combinations. Sometimes that toxicity is in the form of gastrointestinal (GI) toxicity or lower blood counts. Sometimes it’s a trade-off. Are we getting deeper responses that could lead to better outcomes where we could be getting more cytopenia and more need for monitoring, or even transfusions? Is that reasonable for a given individual? Could we be adding some nausea and diarrhea by doing that?

What’s key to this conversation is: are we adding these toxicities continuously or periodically when some of these (MPN treatments) are dosed? For example, navtemadlin is a very active drug. When we looked at the data, it was very clear that you could get some GI toxicity. It was mostly low-grade and easy to manage, but it’s there. The drug is dosed for seven days in a row out of 28 days. The toxicity was mostly relegated to days two and nine.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

This is an esoteric or personal question: for any given individual, if that deeper response could lead to better outcomes, is that period where you may have GI toxicity worth it? From a human perspective, I’m not sure. From a clinical investigation, we’re interested in trying to understand: are we providing full good at a price or is it going to be good and no price? Nothing comes for free, but these are important questions.

We rely on the patient community to tell us. We don’t simply ask patients how they’re feeling. There’s also a much-validated tool that we use called Patient Global Impression of Change (PGIC), which is simple. It asks: if you put everything together, all the toxicities that might ensue and benefits that you’re noticing or being told by the physician that you’re getting, do you feel like you are the same, a little better, a lot better, a hell of a lot better, or a little worse? The PGIC is a very valuable tool because patients will tell you if the whole thing is worth it or not. It’s key to making sure that globally, they believe that they feel that what they’re doing and what they’re going through is a net benefit at the end of the day.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Transplant vs. Medication: Where Do We Stand?

Andrew: You’ve mentioned a number of these drugs that are investigational on top of the four approved JAK inhibitors. I’m 74, so I don’t plan to do a transplant, but some patients are younger and it’s been recommended they have a transplant. As you say, it can be curative. It’s a big gun. I lived in Seattle for a long time where they developed it originally and I knew about the morbidity and mortality, and that continues for some people. Where are we now with transplant versus all these other treatments?

My hope and my goal in my lifetime and my career would be that we ultimately develop therapies that make transplants unnecessary.

Dr. John Mascarenhas

Dr. Mascarenhas: Fortunately or unfortunately, transplant remains the only modality that we have for a cure and, as you’ve pointed out, it’s not for everybody. If you’re advanced in age or have too many comorbidities, a transplant may be more dangerous and detrimental than it ever will be helpful. I’m an advocate for transplant, but it’s for a select group of patients. Patients who go into transplant are moving into an aggressive type of therapy, but it has to meet the aggressive nature of the disease. You would never take someone who has a low-risk version of the disease right into transplant because you could cause more harm earlier on than good.

It’s a complicated discussion that involves understanding where the patient is from a disease perspective, the nature of their disease, their goals, understandings, and expectations, and making sure they have a donor and support system to do a transplant. I encourage that conversation. It’s important, but it’s not going to be for everyone.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

My hope and my goal in my lifetime and my career would be that we ultimately develop therapies that make transplants unnecessary. At its core, transplant is taking immune cells and using them to ultimately get rid of the grandfather or grandmother cell, which is what we call the stem cell that started the disease process, and that’s an immune-mediated elimination of the cell. If we can figure out how best to do that with medicinal therapies that may not be as intense, then we could get to a point where transplant may become historic.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

At Mount Sinai, that’s what a lot of our translational research has been based on, and Ron Hoffman and others have taught me this. It’s a stem cell-directed approach to shut down or eliminate that pool of cells that allows the disease to persist. Even after you wipe out cells with a transplant, those cells can come back. Using science and collaborating with patients, targeting stem cells with rational therapies is the only way we’re going to do that.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

If it weren’t for patients who show up at tertiary care centers like ours, meet physicians like me, and sign consent forms to allow us to take their blood, bank it, and use it to understand the biology, then we wouldn’t be able to move the field forward. It’s the science that we derive from our patient’s cells and their generosity, and allowing us access to their data that help us understand how to make the next generation of therapies that will target that stem cell.

Andrew: I’m a big believer in that. I go to a tertiary center as well. I’m willing to give the blood and I’d recommend that to people.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Will PV or ET Progress to Myelofibrosis? What Can Be Done?

Andrew: We have people who may not have myelofibrosis. They may have polycythemia vera or essential thrombocythemia. As they learn, they know that there can be a progression from one to another. They’re not on a JAK inhibitor, but they might be someday. What do we know about slowing progression or even knowing who will progress?

Dr. Mascarenhas: We know that the disease is chronic and progressive. Progression is not just a fear of the patient; it’s a reality that we as physicians try to risk stratify. When we meet patients, we try to understand if there are risk variables that may help us predict what that timeline might be to make treatment decisions.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

We believe the rate of progression and the reason patients progress is due to clonal evolution. Blood cells acquire more mutations and alterations that allow that cell population to behave differently and change the clinical picture. We use variables, like age, anemia, white blood cell count elevation, presence of circulating blasts, low platelet count, and high molecular mutations or chromosomal abnormalities, and enter them into prognostic scoring systems, which can be found online.

Many patients will find one of these prognostic scoring systems, plug their information in, and get a sense of where they fall in prognosis. But I will caution patients: if you ever do that or speak to a physician, you must understand statistics. Please don’t make the mistake of assuming that the median survival you see is what your lifespan is. It doesn’t work that way. It’s a framework to understand where in the spectrum of patients you fall and help us make treatment decisions about the most appropriate therapy.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Our approach will evolve over time to a more refined, risk-adapted approach where we use mutations to guide us not just in the selection of therapies but the timing of when to use those therapies. From seeing enough patients, I understand that progression is a real concern for ET, PV, and myelofibrosis patients.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Progression to Acute Myeloid Leukemia (AML)

Dr. Mascarenhas: The ultimate concern about progression is the potential to evolve into acute myeloid leukemia (AML). Sometimes you might hear it called the blast phase. That’s an aggressive form of leukemia that is problematic and is a fear factor for patients and physicians treating patients with MPNs.

To see if that’s a concern, we look at mutations like p53, a type of mutation, or the presence of circulating blasts or blasts, which are immature cells in the bone marrow. That information can help us get a sense of what the risk may be of a patient transforming into an acute myeloid leukemia.

MPN-related AML is molecularly distinct from de novo AML… Unfortunately, it’s often more resistant or refractory to the traditional types of therapies that we give in that setting.

Dr. John Mascarenhas

Are We Making Progress with Secondary AML in MPN Patients?

Andrew: There’s a percentage of MF patients who traditionally would progress to AML. There’s been progress in what you’d call primary AML and there have been a number of drugs developed, but secondary AML that would come out of myelofibrosis, I understand, has been more difficult. Where are we with that now?

Dr. Mascarenhas: You’re right. For what we call de novo AML, we have a plethora of different agents. They’re still not enough, but we have agents that can be quite effective in trying to control that type of AML and induce a response. Those agents typically are not as effective and don’t have a very significant impact on the disease process in secondary AML or AML that arises out of an antecedent myeloproliferative neoplasm, whether that’s ET, PV, or myelofibrosis.

MPN-related AML is molecularly distinct from de novo AML. It looks different from a mutation profile and behaves differently. Unfortunately, it’s often more resistant or refractory to the traditional types of therapies that we give in that setting. There’s no benefit to an AML patient who had an MPN previously by giving them induction chemotherapy unless that’s followed by a transplant. Every study tells us that doesn’t improve patient outcomes, so we know that’s not effective.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

We rely on drugs like hypomethylating agents, like decitabine, decitabine+cedazuridine, or azacitidine. These epigenetic therapies try to induce maturation of these immature cells, which is what leukemia is, or immature cells that don’t know grow up. We try to induce that maturation process so that they die a more natural death. It’s less intense, but it’s more effective with this type of AML.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

There are a lot of other therapies under clinical investigation. Some of them are molecularly directed therapies. We’ve run trials with IDH inhibitors, oral drugs that specifically go after mutations that can be present in AML that arose out of an MPN, which can be quite effective. Understanding the molecular profile of that AML could inform treatment decisions. The reality is that’s an AML that’s problematic. Our real goal is to stop the process from evolving to AML because we know that treating that is complicated.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Role of Interferon for the Treatment of MPNs

Andrew: We’ve had interferon for a long time. Where does that fall in?

Dr. Mascarenhas: Interferon, which is a biologic that has been around a long time, is an interesting compound. There is a lot of data in the lab and in patients who’ve been treated on trials, particularly polycythemia vera and essential thrombocythemia, that this drug can be anti-cloning. You can see changes in blood count numbers with ET and PV, and reductions in mutation levels that are driving mutations like the JAK2 mutation. Groups have shown that a reduction in those levels correlates with better event-free survival (EFS). Events are clotting, bleeding, progression, and death in ET and PV. We see the value in that setting. Interferon’s increasing in its utility and use throughout the world.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Myelofibrosis is a little bit different. There’s probably some value there, particularly in treatment-naive early forms of the disease or prefibrotic forms. Interferon is under active clinical investigation in a global study, which is taking patients with lower-risk diseases that don’t have so much complexity and fibrosis in their bone marrow and have seen so many different types of therapies. Interferon works best early on in the disease course. Once the disease gets too complicated and too advanced, it may not be that effective.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

New Drugs Being Studied in MPN Treatments Clinical Trials

Andrew: We mentioned the ASH conference. There are the European Hematology Association (EHA) meetings and other meetings that you attend as well. You mentioned selinexor and navtemadlin. There’s nuvisertib, elritercept, and DISC-071, an anti-hemojuvelin antibody. Help us understand this constellation of what’s going on.

Dr. Mascarenhas: You’ll notice certain themes. For the uninitiated, these names are somewhat frustrating because they don’t resonate. If one were to think about themes, they make sense.

What we’re trying to do in translating the understanding of the biology from the laboratory to the clinic is targeting signaling pathways. These pathways are inappropriately activated in malignant cells that continue to tell them to proliferate, secrete inflammation, and do things that they’re not supposed to do.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

We have very intricate, complex, and overlapping signaling pathways in many malignancies, including myelofibrosis. Multiple signaling pathways are inappropriately activated or hyperactivated. For example, JAK inhibitors inhibit the JAK-STAT signaling pathway and you’ll notice those drugs end with “nib.” Those are small molecule inhibitors that inhibit enzymes in those signaling pathways.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Nuvisertib is a small molecule inhibitor which is a selective PIM-1 kinase inhibitor. It inhibits an enzyme in a signaling pathway that is very relevant to the biology of the disease. It has nice data so far that shows a reduction in symptoms, particularly spleen size and cytokines, as a single agent in patients who’ve already been on a JAK inhibitor.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Other drugs may try to turn on mechanisms that have been turned off, like navtemadlin and selinexor. These drugs are focused on different pathways but with one unifying theme, which is the p53 pathway. It’s the master regulator of cell fate. In a normal situation, your cells would get cues to commit suicide if they were infected or corrupted in some way and that is governed by the p53 pathway, an intrinsic pathway that limits the cell’s life. The problem is malignant cells have co-opted that system and turned it off. They turn it off in different ways. Navtemadlin and selinexor, through having different mechanisms, act on trying to turn that pathway on and encourage that cell to die.

You’ll see drugs that get into the cell that affect pathways, drugs that get into the cell that try to turn on pathways, and drugs that try to use the immune system in different ways to go after the abnormal cell.

Dr. John Mascarenhas

Then you might see a drug like a calreticulin antibody, which inevitably will get a name. You’ll notice early on that it’s just letters and numbers and as the development goes on, it becomes funny names that don’t make any sense and then ultimately, when it gets to the commercial space, it will be a catchy name that could be marketed. The mutant CALR antibody drug by Incyte is a perfect example of what will be a “mab,” a monoclonal antibody, which is targeted at the CALR mutant protein expressed aberrantly on the surface of the cell and is a marker for those abnormal cells. What better way to attack an abnormal cell than to have a selective marker? It’s like a handshake. It’s not even a drug; it’s a peptide. It’s a protein that binds that and by doing that, internalizes that protein complex, and that leads to the death of that cell selectively.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Approaches like that will be very fascinating. Those are immune-based approaches. They have BiTEs (bispecific T-cell engagers) that multiple companies are developing, which introduce T cells in a myelofibrosis patient to the abnormal cell by having two ends of that antibody — one that goes after the CALR, for example, and the other one that goes after CD3 on a T cell — and introduces the two cells together so that that T cell does what it should have done in the first place: recognize and create a response to that abnormal cell.

You’ll see drugs that get into the cell that affect pathways, drugs that get into the cell that try to turn on pathways, and drugs that try to use the immune system in different ways to go after the abnormal cell. These are the general themes.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Considering a Clinical Trial

Andrew: Some drugs are being studied at different levels. What would you say about considering enrolling a patient in a trial once there’s a clear picture of their case? Is there one of these that could line up with that? Take us through the thinking and discussion between a doctor and a patient about considering being in a trial.

Dr. Mascarenhas: I have to acknowledge that being on a trial is scary. I hear this all the time: guinea pig. You feel as though you may be experimented on. I always try to caution patients from that mentality because if you have a disease like myelofibrosis that is frightening to have in itself and can limit the quality of life and maybe even the time that you have, then it warrants consideration to do better than the MPN treatment standard of care. The standard of care, which is JAK inhibitors, has benefits for sure, but it’s incomplete for many patients.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

The consideration for trials hinges on the patient’s goals and desires. Clinical trials may not be reasonable for every patient. Geography influences clinical trials. If you live in a rural area, the distance to a center offering a clinical trial may preclude you from participating. We know other factors may also get in the way of joining clinical trials, like language barriers, cultural barriers, and financial barriers.

I would encourage any patient who sees a specialist and goes to a tertiary care center to at least seek a consultation. This will help them understand what someone who does this full-time thinks about their disease, to clarify or classify their disease, and what clinical trial options exist.

Trials should make sense. You have to ask questions so that you understand what you’re getting involved in.

Dr. John Mascarenhas

I’ve been around long enough to know ruxolitinib as INCB18424 before it was Jakafi. The drug was used in the clinic and we prayed that it was going to make a big difference and it did. It wasn’t enough of a difference, but it made a big difference. I remember those brave patients who were the first patients to get on that study. They set the tone for the whole field and the development of other therapies because of what they put themselves into. Those patients needed the therapy, but what they did allowed us to prescribe the drug to a lot of patients. It has a profound impact way beyond that individual.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

The reality is patients go on a clinical trial for themselves and that is what it should be, but you end up helping the greater good at the same time. You have to feel comfortable with joining a clinical trial. You have to read the consent form. You have to ask questions. What does it involve? What are the potential toxicities? What would be the consequences of participating in a trial?

I feel very comfortable saying that in 2025, any trial I’m aware of that’s offered to patients is an informed trial. These are not trials where we’re taking a random agent off a shelf, throwing it over there, and hoping that it works. These are drugs and agents that are vetted, have a rationale, and go through many layers of pre-testing before they go into humans. You have to trust your physician because if your physician is working with you, then the trial that’s offered to you would hopefully make sense. Trials should make sense. You have to ask questions so that you understand what you’re getting involved in.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Getting the Test Drug vs. a Placebo

Andrew: You mentioned that people ask if they’re going to be a guinea pig. They also ask if they’re going to get the “good stuff.” In other words, they want to know if they’re getting a sugar pill or a placebo. Could you talk about that concerning these trials for drugs that are investigational now?

Dr. Mascarenhas: If you’re in a phase 3 study that’s randomized, a computer randomizes you to arm A or arm B. Arm A may be the active clinical trial agent and arm B could be a placebo, but that has to be disclosed in a consent form. That’s essential. You can ask the physician, “Do I get the study drug or is it going to be randomized? Is it going to be a placebo?”

But a placebo is not always bad. For example, in the MANIFEST-2 study or what’s currently enrolling in the SENTRY study with selinexor, you get randomized as a JAK inhibitor-naive patient with myelofibrosis to either Jakafi plus a placebo, so you’re still getting active therapy, versus Jakafi plus selinexor.

It would be unethical to give nothing to someone who needs treatment.

Dr. John Mascarenhas

The idea is: can we build upon the success of the standard of care? That becomes important. It would be unethical to give nothing to someone who needs treatment. It’s not unethical and it’s practical to give someone who needs treatment the standard of care plus a sugar pill, which is blinded, versus the standard of care plus the study drug to ask which treatment regimen is better.

You can ask your physician, but you’ll notice that most trials will allow crossover and that becomes important. If you get the placebo, which only the computer knows, at some interval, most trials will allow you to cross over to the active compound. It becomes a question of: do I get the active combination upfront or is it delayed? That’s a nuance that I want to stress. If you’re participating in a phase 1 or phase 2 study, you’re going to get the drug. Those are not placebo-controlled studies. You should always be getting the drug in those settings.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

What If I Have Other Chronic Conditions?

Andrew: Some of us have other conditions, like diabetes, high blood pressure, or a heart condition — for me, it’s chronic lymphocytic leukemia, which is fortunately pretty well-controlled— but we’re often excluded from trials depending on the condition. We understand the worry of the investigators. Is our data clean? Can you extrapolate from that? Can you talk about exclusion criteria and compassionate use?

Dr. Mascarenhas: The reality is there are biases in the way we do trials. We avoid patients who may be inappropriate and these are real patients who might be in need, but the trial may not be appropriate. These can be patients who have very significant kidney or heart dysfunction or a competing malignancy. Interestingly, CLL, which is more frequently seen in patients with MPNs, has different stages. Sometimes a study will allow patients who have a very indolent type of CLL to go on the study. But often, trials will be very particular, especially registration studies where they’re going for approval.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

The last thing a study needs is to have confounding data where they’re unclear whether they’re making something unrelated to the myelofibrosis worse or that condition is making the assessment of the drug harder to appreciate. There is a tendency to exclude patients who have extremes of comorbidities so not all comorbidities. If you have grade 1 heart failure, that’s often allowed in a study because that’s a reality of life.

There is a very strict inclusion and exclusion criteria, what we call eligibility criteria, which determine the ability to put someone on a study. Most of those are there for safety reasons to avoid causing additional problems with a drug because it’s not intended for that reason. Another reason why they’re there is if you have a very messy, patient population with lots of diversity and heterogeneity in organ function, etc., it can be very difficult to assess whether the treatment we’re giving is safe or effective. To some extent, out of necessity, we create some homogeneity out of the heterogeneity.

Compassionate use… requires a lot of regulatory oversight, approval, and effort, which is not provided by a company or anyone.

Dr. John Mascarenhas

Andrew: How about compassionate use? If somebody would otherwise be excluded, could their doctor make a plea for a late-stage patient?

Dr. Mascarenhas: Compassionate use is often not that compassionate. It’s trying to get access to a drug under an investigational new drug (IND) and creating a protocol for one individual. What is not always appreciated is while that sounds great, many steps go into that and it requires a lot of regulatory oversight, approval, and effort, which is not provided by a company or anyone. It’s the physician and whatever team members he can assemble to try to do that.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Although compassionate use is an opportunity to get a drug, it’s often an opportunity plagued by the realities of a very cumbersome bureaucratic system that doesn’t make it very easy and timely to get to that drug. Although it’s there and in some cases helpful, it’s not always practical or realistic for people to get compassionate use and sometimes the access to those drugs is restricted by the FDA and/or the sponsor.

But compassionate use can sometimes be an opportunity to get a drug that wouldn’t otherwise be eligible and that’s key. If there’s a trial that allows the patient to get access, they often will not provide compassionate use because it would be felt unethical to allow someone to get access to a drug while all other patients would have to go through the normal routine of the clinical trial. This has to fit very strict criteria and is not always achievable.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

The Future of Treatments for MPN Patients

Andrew: We have ASH in the rearview mirror. You have other conferences during the year and you’re speaking at a number of them. You’re actively involved in research and seeing patients as well. I want to get where your head’s at as far as promise in the field, how you feel about it, and what people can do about it.

Dr. Mascarenhas: We are way further ahead than we were in 2005, 2010, and 2015. I’ve watched the field grow in terms of our understanding, the amount of attention and support that’s provided to this rather small area of hematology from the federal government in terms of NIH grants — in which we indulge in getting to try to help move the field forward independent of pharmaceutical interests — but as well as pharmaceutical interest and philanthropy. They all help grease this machinery of translational research and understanding. We have cadres of smart, well-intentioned PhDs, MDs, and MD-PhDs that are helping us understand the biology and that is directly translating into the clinic to help us fine-tune our treatment.

We’re trying to understand how to get access to and develop multiple therapies that could be used in different subsets of patients or different combinations.

Dr. John Mascarenhas

You look at ASH or EHA and see endless abstracts of agents that we didn’t even know about or targets that we didn’t even understand 10 years ago. I have to believe that’s going to translate ultimately to better therapies and multiple therapies. I don’t think it’s going to be one therapy fits all. We’re trying to understand how to get access to and develop multiple therapies that could be used in different subsets of patients or different combinations and that is something that I do feel optimistic about. I would say we’re moving forward in 2025 and beyond in rational combinations and allowing science to drive the advancements.

You’ve known me for a long time. I always feel positive. I walk to work and I’m enthusiastic every day I come in because I believe in the mission. We’re making strides. I see it in the patients that I treat, but I believe it for the future.

What Patients Can Do to Alleviate Symptom Burden

Andrew: For a patient who has significant anemia, declining platelet counts, an enlarged spleen, or whatever symptom they’re experiencing, what can they do that could make a difference in the short term?

Dr. Mascarenhas: If you’re having symptom burden, particularly if it’s interfering with your quality of life and activities of daily living, you should see a physician who specializes in hematology and, ideally, someone who has expertise in this area.

The longer one delays starting therapy, the less likely that therapy will be effective and that the effects of that therapy will be durable.

Dr. John Mascarenhas

A JAK inhibitor is the front-line therapy to try to address those aspects. It doesn’t preclude one from looking at clinical trials that might even be combinations upfront of a JAK inhibitor plus an active agent to see if one can get even better, deeper, and earlier responses. But at the very least, a JAK inhibitor to try to address symptoms because you don’t get extra points for suffering through symptoms. It’s harder to rescue if one delays treatment. The data is very clear about that. The longer one delays starting therapy, the less likely that therapy will be effective and that the effects of that therapy will be durable.

Andrew: You may live at a distance from one of the major centers where there is an MPN specialist, but there are MPN specialists. You refer to cadres of researchers and physicians who are working in this area and it makes sense to have a consultation with somebody like Dr. Mascarenhas at Mount Sinai or my doctor, Dr. Jamieson at UC San Diego. Fortunately, many others are studying this and working on it and understand these nuances, so you get personalized care for where you are now and where you may be headed.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Final Takeaways on MPN Treatments

Andrew: This has been a helpful discussion. Is there anything you wanted to add, John, that we didn’t cover?

Dr. Mascarenhas: We covered all the major points and I conveyed my continued optimism. One boon that we have is it’s a very collaborative field. When you look at the abstracts, trials, and studies, you will see all the names that are listed, which reflects our collaborative nature. These are rare diseases. You can’t work in a silo. We’re all friends and colleagues. We all have a unified mission. We’re focused on that as an international team and that’s why I remain enthusiastic that we’re going to make the progress.

Andrew: Thank you for your collaboration and people. I’m seeing a lot of younger physicians and scientists interested in this area, so that gives us a great deal of hope. Dr. Mascarenhas, thank you for your devotion to us. We appreciate your time. We’re all bonded in this together. Remember: knowledge can be the best medicine of all.

Your MPN, Your Journey - How New Discoveries Will Impact Personalized Care

Conclusion

Tiffany: That discussion [ on MPN treatments ] was the definition of news you can use. Thank you, Dr. Mascarenhas and our patient advocate and moderator Andrew, for taking the time out of your busy schedules to keep The Patient Story community informed. If you are a patient, caregiver, partner, or advocate, consider being a voice leader in your community or with us at The Patient Story.

To be empowered is to be inspired. We want you to make informed decisions about your care and that includes being educated about the latest treatment options. Thank you again to our sponsors, Sobi and Incyte, for their support of our independent patient program and to all of our promotional partners. Until next time, I’m Tiffany Drummond, signing off and, on behalf of The Patient Story, thank you for watching.

Your MPN, Your Journey: How New Discoveries Will Impact Personalized Care
Hosted by The Patient Story Team
Dr. John Mascarenhas (Mount Sinai) and patient advocate Andrew Schorr share the latest breakthroughs in MPN care. Explore personalized treatments, cutting-edge therapies, and groundbreaking research that’s changing how MPNs are treated. Learn how new discoveries can improve your treatment options, help manage side effects, and enhance your quality of life.
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Incyte

Special thanks again to Sobi and Incyte for supporting our independent patient education content. The Patient Story retains full editorial control.


MPN Patient Stories

Andrea S. feature profile

Andrea’s Myelofibrosis & Essential Thrombocythemia Story

Andrea S., essential thrombocythemia (ET) progressing to Myelofibrosis Symptoms: Fatigue, anemia Treatments: Targeted therapy (JAK inhibitor)...

Holly’s Myelofibrosis Story

Holly S., Myelofibrosis Symptoms: Severe fatigue, throbbing pain in left calf, significant weight loss, itching and...
Ben H.

Ben’s Myelofibrosis with CALR Mutation Story

Ben H., Myelofibrosis Symptoms: None; caught at a routine blood testTreatments: Hydroxyurea & aspirin, ruxolitinib...
Doug A. feature profile

Doug’s Myelofibrosis Story

Doug A., Myelofibrosis Symptom: FatigueTreatments: ruxolitinib, selinexor (clinical trial)...
Kristin D.

Kristin’s Myelofibrosis Story

Kristin D., Myelofibrosis Symptoms: None; caught at routine blood workTreatment: Stem cell transplant...
Joseph C. feature profile

Joseph’s Myelofibrosis Story

Joseph C., Myelofibrosis Symptoms: None; caught at routine blood workTreatment: Clinical trial: VONJO (pacritinib)...

Categories
Multiple Myeloma Patient Events

Together in Treatment: Strengthening Your Myeloma Care Team

Together in Treatment: Strengthening Your Myeloma Care Team

Edited by: Katrina Villareal

Together in Treatment: Strengthening Your Myeloma Care Team
Hosted by The Patient Story Team
The relationship between a patient and their doctor can make all the difference. A strong partnership leads to more informed decisions, personalized care, and a greater sense of control. Join myeloma patient advocate Michelle and her doctor and myeloma expert, Dr. Caitlin Costello, as they discuss what makes their patient-doctor teamwork truly effective.
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The relationship between a patient and their doctor can make all the difference. A strong partnership leads to more informed decisions, personalized care, and a greater sense of control. Myeloma patient advocate Michelle and her doctor and myeloma expert Dr. Caitlin Costello discuss what makes their patient-doctor teamwork truly effective.

Learn how to build trust and open communication with your healthcare team. Understand the role of shared decision-making in multiple myeloma care. Hear first-hand experiences of navigating a chronic cancer with your doctor by your side. Discover practical tips for advocating for yourself or a loved one in the treatment process. Explore how teamwork fosters a supportive environment for long-term care.


LLS

We would like to thank The Leukemia & Lymphoma Society for its partnership.

This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.


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Thank you to Sanofi for supporting our patient education program. The Patient Story retains full editorial control over all content



Building Bonds Episode 1 - Patient-Doctor Partnerships for Better Waldenstrom Care

Introduction

Tiffany Drummond: I’m an advocate who has worked in cancer research for 20 years, but more importantly, I became a care partner advocate when my mom was diagnosed with endometrial cancer in 2014. Her journey led me to find out as much as I could to help with her care. Information wasn’t easy to find, so I’m honored to join The Patient Story in putting on programs to help people navigate life after diagnosis.

This program is hosted by The Patient Story, where we aim to help people navigate life before, at, and after diagnosis through empowering patient stories and educational discussions where we focus on how patients, caregivers, and their partners can best communicate with their doctors as they go from diagnosis through treatment with myeloma.

Tiffany Drummond

We want to thank The Leukemia & Lymphoma Society for its partnership. The LLS offers incredible free resources, like their Information Specialists, to help you communicate with members of your healthcare team and provide information about treatment options.

We want to thank our sponsor, Sanofi, for its support, which helps us host these programs for free. The Patient Story retains full editorial control over all content. I hope you find this information helpful, but please keep in mind that this program is not a substitute for medical advice.

We have two special guests from whom we can learn more about the patient-physician partnership. We have Dr. Caitlin Costello, an associate professor of medicine at UC San Diego Health and a hematologist-oncologist specializing in blood cancers, including myeloma, lymphoma, and leukemia. We also have our patient advocate, Michelle, who is a multiple myeloma fighter and a survivor.

Together in Treatment - Strengthening Your Myeloma Care Team
Michelle C.

Initial Multiple Myeloma Symptoms and Diagnosis

Tiffany: Let’s start back to your initial diagnosis. What was that experience like for you?

Michelle: I was diagnosed in 2016 at the age of 35. I had a five-year-old and a one-year-old at the time. The year leading up to my diagnosis, I had on-and-off back pain, which they said was because my muscles weren’t strong after having a baby. I was starting to lose weight, which they attributed to breastfeeding. I ended up having other symptoms that led me to go to the doctor more.

But then I ended up having severe stomach pain, which at times made me want to drive myself to the ER. I also ended up having itchy scabs all over my body. I went to the dermatologist and my general physician. They both started digging, and my general physician started ordering lab work, which eventually led to the diagnosis of multiple myeloma.

Tiffany: You knew in your gut that something wasn’t right. What did it take for you to advocate for yourself? And when you were referred to a specialist or a hematologist-oncologist, what was your initial experience like?

Michelle: My husband travels for work. was at home with my one-year-old and five-year-old when I was having stomach pains. I didn’t know what to do. I almost drove myself to the ER one day. I took a breath, pulled over, and the pain stopped. I called my doctor and told him he needed to see me the following day. I had already seen the dermatologist about the scabs all over my body. She looked into things and I didn’t know if any of my symptoms were related.

The doctor called and said, “You need to come in right now.” My husband was home, so we picked up our one-year-old and drove to the doctor’s office. My husband stayed with our one-year-old so I could listen to what the doctor was saying.

Michelle C.

When we got home, we started making calls…. They all led us to Dr. Caitlin Costello… She fit me in right away, and we never looked back.

Michelle

He said, “You have multiple myeloma.” I asked, “Is that cancer?” He said yes, explained what it was, and sent me to a hematologist-oncologist in their group, who was not at UCSD then. They got me in that day, and the hematologist-oncologist did a bone marrow biopsy.

When we got home, we started making calls. I called my boys’ pediatrician, who I loved and whose opinion I valued, and we reached out to several other people. They all led us to Dr. Caitlin Costello, so we gave her a call. She fit me in right away, and we never looked back.

Together in Treatment - Strengthening Your Myeloma Care Team
Michelle C.

Explaining Multiple Myeloma to Patients

Tiffany: Doctors all explain myeloma differently, so Dr. Costello, how do you explain myeloma to your patients?

Dr. Caitlin Costello: For many people, when they hear of and think of cancer, they think of a lump, like breast cancer, or there are symptoms associated with a tumor. But when you’re talking about blood cancers, no tumor can be picked up on routine screening tests, of which there aren’t any for myeloma.

With more “typical cancers,” we’re so used to having public health initiatives for screenings like mammograms and colonoscopies. Multiple myeloma, however, while it’s the third most common blood cancer, doesn’t come anywhere near number-wise in terms of how many patients are affected in the United States each year. For that reason, for better or for worse, I don’t think we have good epidemiologic advances to say that everyone should have a blood test screening looking for this.

There’s ongoing interest in screening wide groups of populations. In Iceland, they’re screening the entire country for all patients over the age of 40 to see if they can figure out how many patients have what looks like the beginnings of multiple myeloma. And if they’re not myeloma at that point, they want to understand if it makes sense to screen patients.

Patients are going to live with this for the rest of their lives, so there’s no greater importance than understanding and knowledge.

Dr. Caitlin Costello

For the most part, that means that most patients are diagnosed with multiple myeloma when they develop a symptom of some sort. Many people have not heard of myeloma. When a doctor says to a patient that they have multiple myeloma, they ask, “What does that mean?”

When I describe multiple myeloma to a patient, I say, “What we have identified is a form of a blood cancer called multiple myeloma based on your blood tests and your bone marrow tests.” A lot of my consultations with patients are to explain that diagnosis because.

We have a wonderful problem with myeloma. This has turned into a chronic illness. Patients are going to live with this for the rest of their lives, so there’s no greater importance than understanding and knowledge so that patients know what it is that we’re talking about, know how to follow their blood tests, understand the successes of therapy or perhaps early signs of failures of therapy, and advocate for themselves.

There’s so much to say of the educated patient, which doesn’t mean Doctor Google. There are very good patient advocacy groups and platforms like The Patient Story, where patients can get great information to help them understand their disease and their journey with it.

Michelle C.

When I explain myeloma, I explain that it’s a form of blood cancer that comes from a plasma cell, which is part of your immune system. The plasma cell is designed to produce the weapons needed to protect our body. The bone marrow is like the armed forces. We have an army, a navy, an air force, and all these different branches with different weapons designed to protect you. One branch went rogue when one plasma cell went rogue and started producing extra bad guy weapons that don’t work well and cannot protect the body and cause damage.

Together in Treatment - Strengthening Your Myeloma Care Team

Bones are the most common way that myeloma can affect a person’s body, and therefore, that’s one of the most common ways that patients are eventually diagnosed because they come seeking help for pain. As Michelle experienced, back pain is one of the most common ones. We think the bones in the middle of our body are more often affected than elsewhere. The classic story is someone had back pain, their doctor did X-rays, but they didn’t see anything, or they were referred to physical therapy, but the pain got worse.

Everyone’s allowed to hurt, but pain that doesn’t go away, came on for no good reason, and is persistent needs to be evaluated. Michelle did all the right things because she had what we can say are typical symptoms with pain, some atypical symptoms with the skin and the belly discomfort, but she asked all the right questions to get her to the right people who could help her.

Together in Treatment - Strengthening Your Myeloma Care Team
Michelle C.

Common Questions After a Multiple Myeloma Diagnosis

Tiffany: I love that you talk about patient education. I believe that part of patient education is learning all the medical terms you probably weren’t familiar with before. How receptive are your patients in terms of wanting to learn all that? What are the top three common questions you get after someone is diagnosed with myeloma?

Dr. Costello: The top three questions are: How did I get this? What can I do to make it go away? Is it genetic?

Everyone wants to feel empowered that they can make some lifestyle change, for example, to make things better or help rationalize this to some degree to say X caused Y. It would be fair if there was some culprit, but it’s unfair because there is no culprit. We think this is a random thing that happened for no good reason, by no fault of anybody’s. Nature changed the makeup of your bone marrow and the part of your immune system.

Getting Involved in the Decision-Making Process

Tiffany: Michelle, when you first met with Dr. Costello and as she was explaining your treatment options, how involved were you in the decision-making process? What did that experience look like?

Michelle: Dr. Costello presented me with what the standard of care was for multiple myeloma at the time. I was very receptive. I sought a second opinion and reached out to another multiple myeloma specialist in the vicinity. She confirmed the same thing, so we went with what everyone was recommending.

Michelle C.
Together in Treatment - Strengthening Your Myeloma Care Team

Factors to Consider in Shared Decision-Making

Tiffany: Dr. Costello, when you approach your patients about their treatment options, what are your thoughts? How do you approach shared decision-making with your patients? What factors do you consider to help them come to that process with you?

Michelle C.

Dr. Costello: Every person is different. People process information differently. People hear information differently. Some people want to know more, and some people don’t want to know more.

An important part of any conversation is to level set and say, “What do you want to get out of this conversation? What is it that you want to know?” More often than not, the patient has something in mind, and the family members have something different in mind. It’s important to gauge the group to determine what it is that they’re hoping to get out of the appointment so that I’m not overstepping boundaries.

Once we are able to establish how much will be shared, my job is to give the information. What is the standard of care? What is the typical approach? Once we’ve laid the groundwork, then we can determine treatment recommendations based on the patient and the details of their health, caregiver support, and the biology of their disease. Very specific details can make treatment recommendations slightly nuanced for any individual person.

My job is to help them be as informed as possible so that they can make the best decision for themselves.

Dr. Caitlin Costello

I love that Michelle got a second opinion. I have no ego. When you are diagnosed with something life-changing, you need to feel very confident in what your next approach is going to be. The more people think about you, the better. Like many myeloma specialists, this is what we do day in and day out, but it’s nice to have a fresh set of eyes so nothing’s missed. Yesterday’s information may have been different from a month ago’s research.

The approach is standard, but the shared decision-making is where things may be slightly different. I can make all of my recommendations and say, “This is what’s standard. These are the slight modifications I would make for you.” Sometimes, the patients will take that information and say, “I’ll get back to you.” Some people will say, “Let’s do this. Whatever you say, doc.” Some people will say, “No.”

I’m not in their body. I’m not making decisions for them. My job is to help them be as informed as possible so that they can make the best decision for themselves. I may not agree with their decision, but that’s not my job. My job is to help them arrive at the best decision that’s for them.

Michelle C.

Tiffany: You said everything that I wanted to hear personally, especially when it comes to seeking a second opinion. For a lot of patients, especially if they like their physician in the first meeting, they feel like they’re turning their back on their physician. Thank you so much for encouraging patients to seek a second opinion.

Michelle C.

Multiple Myeloma Treatment Journey

Tiffany: Michelle, where are you in your treatment journey?

Michelle: I did four months of the initial treatment regimen and went into an autologous stem cell transplant. Unfortunately, it wasn’t successful and the myeloma returned within the 100-day mark. We regrouped, went back on one treatment for the summer, regrouped again, and did a more aggressive approach.

The MRD testing at the beginning of 2024 showed that the myeloma was slightly coming back. It was affecting my quality of life and I was ready for something different, so we regrouped again. The doctors agreed that I could take the summer off. We did a repeat bone marrow biopsy, so I would have some initial data to compare against when I start my new treatment plan.

Tiffany: Did the pandemic affect your treatment at all?

Michelle: It gave me a lot of anxiety, but I had to be persistent. Even when everything closed down initially, we were on the phone asking, “Am I coming in?” She said yes, so I went in. I went in every other week all through the pandemic.

Coordinating Care with Multiple Healthcare Providers

Tiffany: When it comes to cancer, you have more than one healthcare provider. Your healthcare team is very vast. Is that specific to UC San Diego or do you have local providers that you also go to? You’re always receiving a lot of information, so how do you coordinate that among yourself and your medical team?

Michelle: I don’t live in San Diego anymore, so I have a local hematologist-oncologist in Sacramento where I now live. This is my disease and my choice. I’ve always sought second opinions, especially when making big decisions about changing treatment plans and what to do next.

Thankfully, the multiple myeloma world is small, so they all know each other. I’ve always been able to discuss with each physician. Even if I don’t agree with a treatment plan, I can seek a different opinion. They have been very kind and take into account my quality of life and what I would like.

Getting the information is best. I seek opinions, weigh out what I want to do and how the treatment is going to affect my life, especially with raising two active boys, what I can handle as far as raising them and having a great quality of life, and then make my decision of how I want to proceed based on their recommendations.

Michelle C.
Together in Treatment - Strengthening Your Myeloma Care Team
Michelle C.

Managing Patient Care From Afar

Tiffany: Dr. Costello, for someone like Michelle who doesn’t live in your area, how do you approach seeing patients from afar? Is that something that you do? I had a conversation recently about how large academic centers are more specialized and how you share information with local providers who may not have the same knowledge that you would have. How does that work for you?

Dr. Costello: I don’t feel like Michelle gives herself enough credit. From everything that she said, while raising two young children amid a pandemic, I told her to jump, and she said, “How high?” She has such a commitment to her health and her family.

She got her care at UCSD where we have myeloma-dedicated physicians, but most myeloma patients are taken care of in the community. They see an oncologist, who is possibly a general oncologist who’s seeing them right after they see someone with breast cancer and right before they see someone with lung cancer. Often, they’re good with myeloma, but it’s hard to be a jack of all trades also, and that’s where the importance of a myeloma specialist comes in. I don’t know how they do what they do, seeing so many different cancers. I have difficulty keeping track of one, let alone all of them.

The importance of the connection between the community oncologist and the academic myeloma specialists can’t be underlined enough because we have different tools at our disposal.

Dr. Caitiln Costello

Myeloma is a team sport. Your team includes the patient, myeloma specialist, general oncologist, nurse navigator, nurse, and social worker. There’s a whole team of people who are trying to come together to hold hands with our patients to get them through this whole process.

I can’t speak for other places, but a lot of that can be a little insurance-driven, especially in California. Some insurers will require you to stay with your community oncologist and if that’s the case, the patients get referred for their stem cell transplant, CAR T-cell therapy, or whatever treatment we have to offer at the academic center that perhaps the local oncologist cannot offer. That allows us to maintain that relationship with our patients as well. I have a list of phone numbers of all my local community oncologists down here because we are constantly talking about our patients behind the scenes.

Michelle C.

The importance of the connection between the community oncologist and the academic myeloma specialists can’t be underlined enough because we have different tools at our disposal. Myeloma is complicated. There are so many drugs, which is a wonderful problem, but that means that it can be complicated to understand which drug to use and in what order. The connection between the oncologist in the community and the myeloma specialist is absolutely paramount to navigate this whole thing.

I want to be well and healthy to see my boys’ future, and I want to do that with a great quality of life.

Michelle
Michelle C.

Importance of Quality of Life in Driving Treatment Decisions

Tiffany: I used to be a caregiver, so I understand the importance of quality of life. Michelle, you were able to take some time off treatment. How important was your quality of life in driving treatment decisions?

Michelle: I want to be around for my children. That’s my top priority and however I’m going to get there, I will get there. I will cross that finish line no matter what it takes. I want to be well and healthy to see my boys’ future, and I want to do that with a great quality of life.

I started not feeling well after treatments. I was dragging myself and making myself nauseous before even getting to treatment. It was psychosomatic. I realized this wasn’t good and I needed to switch things.

I’m very fortunate where I have a lot of flexibility in my time, so I’m able to make doctor’s appointments and do my treatments during the day when my boys are at school. I try to lead my life in a way that doesn’t affect my children. I’m not in the infusion center when they’re home and going to bed, and I’m very blessed to have that opportunity.

Together in Treatment - Strengthening Your Myeloma Care Team

Data That Looks at Quality of Life for Multiple Myeloma Patients

Tiffany: Dr. Costello, is there increasing data that looks at quality of life when it comes to myeloma and treatment options?

Dr. Costello: I don’t even know how to emphasize quality of life enough. People ask, “Is it quantity of life? Is it quality of life? Is it both?” I ask that to some degree to find out about their goals. People’s goals are different. It’s realistic though to say that some of our treatments are not that great. They’re inconvenient and take up a lot of time even though they work. We need to have a conversation to find out their deal breakers. Some patients don’t want to be in an infusion center and only want to take a pill, even if it means it’s not as effective because that’s what’s meaningful to them.

Together in Treatment - Strengthening Your Myeloma Care Team

As far as research goes, fortunately, a lot of different studies are looking at new drugs or new drug combinations, including what we call patient-related outcomes. People may hate it because there’s a lot of surveys that happen in the midst of clinical trials asking them, “How’s today? How’s your body image? How do you feel like this? Are you content with this treatment? How do you perceive the side effects of treatment? How much time has this taken out of your day to do this?”

There is more interest in expanding on what we’ve always relied on to evaluate the safety and efficacy of drugs, to incorporate how these drugs can change people’s lives positively and negatively, and to help guide doctors in making treatment decisions and help patients understand if that’s a deal breaker.

Together in Treatment - Strengthening Your Myeloma Care Team

Approaching the Clinical Trial Conversation

Tiffany: You brought up clinical trials, which is one of my favorite topics. Oftentimes, I’ll hear patients say they don’t want to go on a clinical trial because they think that’s the last resort. Dr. Costello, how do you approach the clinical trial conversation with your patients?

Dr. Costello: I first dispel the myth that placebos still exist. Some people still have it embedded in their mind. I always say that it isn’t ethical. We don’t do that anymore. Clinical trials are designed to give you what we consider the best available treatment right now and/or compare it to something that we think is as good or potentially better. I tell patients they’re potentially getting the best of both worlds no matter what they get assigned to at clinical trials.

Part of clinical trials is to help patients understand that there are various phases. Some are randomized where we don’t get to say in what treatment the patient will get. They get assigned to one or the other, but both are great options. There’s an earlier phase trial, which evaluates the safety or efficacy of these treatments and every patient will get the exact same treatment.

There are great benefits that can be reaped by participating in trials, which include getting access to cutting-edge therapies.

Dr. Caitlin Costello

There’s a thought that participating in a clinical trial is purely altruism and to some degree, yes. You are helping the future of myeloma therapies, but you’re getting the benefit yourself from it. Clinical trials are not always designed to be testing the next best thing when a patient has no other options. It’s improving all the steps of treatment that currently exist because we can always do better.

When the conversation about clinical trials comes up, a lot of it is dispelling myths and helping people recognize that it’s not just for others. There are great benefits that can be reaped by participating in trials, which include getting access to cutting-edge therapies that I otherwise cannot write a prescription for.

Seek other opinions and make the best informed decision for yourself.

Michelle

Key Takeaways

Tiffany: Michelle, what would you tell a patient who is newly diagnosed? Honestly, it seems like you did everything right, so I want to commend you.

Michelle: You are your best advocate. Do your research. Seek other opinions and make the best informed decision for yourself.

Tiffany: Dr. Costello, how do you help a provider to be an active participant and proponent of informed decision-making and shared decision-making, especially junior providers who are coming into the fold? What advice would you give providers to be the kind of person who has relationships with their patients like you and Michelle have?

Michelle C.

Dr. Costello: Thank goodness that the paternalistic approach to medicine is a thing of the past. While there may still be a bit of it out there, there has been such an important message about customer service with medicine. You have to understand that this is a give-and-take relationship to some degree. The patient deserves to hear all the information and it’s the physician’s role to give all that information.

There’s been such an important emphasis on compassion and communication. If the physician can put themselves in the shoes of a 35-year-old newly diagnosed mom walking into a cancer center, we can all step out of our bodies and our egos to understand that there is more that can be improved in terms of developing that relationship and the importance of communication. We need to understand that we have much to offer, but our patients have so much to offer us as well.

Together in Treatment - Strengthening Your Myeloma Care Team

Conclusion

Tiffany: Thank you, Michelle and Dr. Costello, for such an engaging and empowering conversation. I learned a lot about both of you personally and professionally. What you’ve had to say is going to resonate with our audience.

Dr. Costello is truly a testament to what makes a great physician partner. Witnessing her and Michelle interact was refreshing since we know the patient-physician conversation isn’t always light-hearted when it comes to cancer care. It is important to be empowered so that you and your caregivers can make informed decisions about your care.

Thanks again to our sponsor, Sanofi, for its support of our independent patient program and to our partner The Leukemia & Lymphoma Society. Check out the links on their website, including their information resource center, which provides free one-on-one support.


LLS

Special thanks to The Leukemia & Lymphoma Society for its partnership.


Sanofi logo

Special thanks again to Sanofi for supporting our independent patient education content. The Patient Story retains full editorial control.


Multiple Myeloma Patient Stories

Clay

Clay D., Relapsed/Refractory Multiple Myeloma



Symptoms: Persistent kidney issues, nausea

Treatments: Chemotherapy (CyBorD, KRd, VDPace), radiation, stem cell transplant (autologous & allogeneic), targeted therapy (daratumumab), immunotherapy (elotuzumab)
...
Melissa

Melissa V., Multiple Myeloma, Stage 3



Symptoms: Frequent infections

Treatments: IVF treatment & chemotherapy (RVD) for 7 rounds
...

Elise D., Refractory Multiple Myeloma



Symptoms: Lower back pain, fractured sacrum

Treatments: CyBorD, Clinical trial of Xpovio (selinexor)+ Kyprolis (carfilzomib) + dexamethasone
...
Marti P multiple myeloma

Marti P., Multiple Myeloma, Stage 3



Symptoms: Dizziness, confusion, fatigue, vomiting, hives



Treatments: Chemotherapy (bortezomib & velcade), daratumumab/Darzalex, lenalidomide, revlimid, & stem cell transplant
...
Ray H. feature

Ray H., Multiple Myeloma, Stage 3



Symptoms: Hemorrhoids, low red blood cell count

Treatments: Immunotherapy, chemotherapy, stem cell transplant
...

Categories
Acute Myeloid Leukemia (AML) Chemotherapy Leukemia Patient Stories Treatments

Grace’s Acute Myeloid Leukemia Story

Grace’s Acute Myeloid Leukemia Story

Interviewed by: Taylor Scheib
Edited by: Chris Sanchez

Grace hails from California and is a proud mother to her 3 children. She was diagnosed with acute myeloid leukemia (AML) in 2022. Her cancer was revealed by a blood test requested by her doctor after she suffered a headache that persisted for a week.

Grace’s diagnosis blindsided her and filled her with anger and confusion, as she has lived an active lifestyle and has stayed away from tobacco, alcohol, and recreational drugs. She admits, though, that her diet may have contained too many processed foods, and suspects that this may have contributed to her cancer.

Grace was completely unfamiliar with leukemia and had to learn all about it from her doctors. She also had to steel herself for all her tests and treatments, including her first bone marrow biopsy of a total of 6, which she describes as being more painful even than childbirth. 

Grace ended up taking 6 chemotherapy treatments for her AML. She also had to undergo a stem cell transplant—and was lucky enough to find a compatible donor who could help.

Grace’s cancer is now in remission. To try and make sure she stays healthy from now on, she has resolved to buy and consume only organic products. Moreover, her brush with cancer has made her realize that she needs to stop taking things in her life for granted, from her family to “little things” such as hummingbirds and flowers.

Grace shares her story with us today to help others realize the importance of living a healthy lifestyle as well as having the right attitude and taking time to truly appreciate life.


  • Name: Grace A.
  • Diagnosis:
    • Acute myeloid leukemia (AML)
  • Initial Symptoms:
    • Headache that lasted 1 week
  • Treatment:
    • Chemotherapy
    • Stem cell transplant

This interview has been edited for clarity. This is not medical advice. Please consult with your healthcare provider to make treatment decisions.


Thank you for sharing your story, Grace!

Inspired by Grace's story?

Share your story, too!


Related Cancer Stories

More Acute Myeloid Leukemia (AML) Stories
Sasha

Sasha T., Acute Myeloid Leukemia (AML)



Symptoms: Easily bruised and swollen, painful hip
Treatments: Chemotherapy, bone marrow transplant

Luisa L., Acute Myeloid Leukemia (AML) with FLT3 Mutation



Symptoms: Painful hives on legs, migraines
Treatments: Chemotherapy, total body radiation, stem cell transplant
Hayley

Hayley A., Acute Monocytic Leukemia (AML-M5)



Symptoms: Severe fatigue, excessive bleeding after oral surgery
Treatments: Chemotherapy, bone marrow transplant
Nicole

Nicole T., Acute Myeloid Leukemia (AML)



Symptoms: Severe itchiness, night sweats, fatigue
Treatments: Chemotherapy, bone marrow transplant
Mary Clare

Mary Clare B., Acute Myeloid Leukemia (AML)



Symptoms: Extreme fatigue, upset stomach, bad & persistent headaches
Treatments: Chemotherapy, radiation, bone marrow transplants
Categories
Chronic Myeloid Leukemia (CML) Leukemia Patient Stories

Mark’s Chronic Myeloid Leukemia Story

Mark’s Chronic Myeloid Leukemia Story

Interviewed by: Taylor Scheib
Edited by: Chris Sanchez

Mark discovered that he had Chronic Myeloid Leukemia (CML) at the age of 47.

Mark and his family had just moved to a new city where he was going to start a new job. He had been experiencing weight loss and low energy, which had been easy to attribute to stress from the move. However, a visit to a new doctor for a routine checkup revealed that his spleen was quite enlarged and his white blood cell count alarmingly high. He had also been experiencing other symptoms such as heavy night sweats and a frequent need to urinate after bedtime.

Tests conducted by an oncologist confirmed that Mark had CML. Mark quickly began a range of treatments aimed at treating his spleen through lowering his white blood counts, as well as addressing his leukemia through tyrosine kinase inhibitors or TKIs. His doctors also helped him become more familiar and comfortable with living with CML.

Mark’s story underscores the importance of listening to one’s body and not shrugging off symptoms, and of finding a supportive community that includes doctors, family members, and colleagues.


  • Name: Mark K.
  • Diagnosis:
    • Chronic Myeloid Leukemia (CML)
  • Initial Symptoms:
    • Weight loss
    • Low energy
    • Night sweats
    • Frequent need to urinate after bedtime
    • Enlarged spleen
    • Elevated white blood cell count
  • Treatment:
    • Tyrosine kinase inhibitors (TKIs) including Sprycel

This interview has been edited for clarity. This is not medical advice. Please consult with your healthcare provider to make treatment decisions.


Thank you for sharing your story, Mark!

Inspired by Mark's story?

Share your story, too!


Related Cancer Stories

More Chronic Myeloid Leukemia Stories

Michele T., Chronic Myeloid Leukemia (CML)



Symptoms: Trouble breathing, rash, bruising



Treatments: Sprycel and Bosulif
Mark K's story of his Chronic Myeloid Leukemia (CML) diagnosis

Mark K., Chronic Myeloid Leukemia (CML)



Initial Symptoms: Weight loss, low energy, night sweats, enlarged spleen, elevated WBC count, frequent need to urinate

Treatment: Tyrosine kinase inhibitors (TKIs)
Categories
Classical Hodgkin Lymphoma Patient Stories

Brescia’s Hodgkin Lymphoma Story

Brescia’s Hodgkin Lymphoma Story

Interviewed by: Taylor Scheib
Edited by: Chris Sanchez

Brescia was only 20 when she was found to have early-stage Hodgkin’s Lymphoma.

When her cancer was caught, Brescia was returning to America from a study stint in Italy and was on the verge of returning to college. Her primary care physician discovered a suspicious swelling in the side of her neck during a routine physical examination, and she was scheduled for an ultrasound. A subsequent biopsy confirmed her cancer.

Brescia’s diagnosis was unsettling, to say the least, but she found refuge in the things that bring her joy and fulfillment, including being creative, making movies, and enjoying nature.

Brescia and her family sat down with her team of doctors at the Mayo Clinic in Arizona to discuss her treatment options. They considered a combination of chemotherapy and radiation, but decided to go for 6 rounds of ABVD chemotherapy and no radiation, given her youth and the fact that radiation can have long-term side effects. Brescia ended up taking the whole semester off to focus on her treatment.

Brescia has been cancer-free for 5 years now. She is now a photographer and videographer and runs her own boutique agency. Looking back at the time she was ill, she notes how she gained a new appreciation for school as well as the simple things in her life, appreciates the perspective she gained after her illness, and shares the lessons she learned from her experience.

In addition to Brescia’s narrative, The Patient Story offers a diverse collection of stories about Hodgkin Lymphoma. These empowering stories provide real-life experiences, valuable insights, and perspectives on symptoms, diagnosis, and treatment options for cancer.


  • Name: Brescia D.
  • Diagnosis:
    • Hodgkin Lymphoma
  • Initial Symptoms:
    • Swelling in the side of her neck
  • Treatment:
    • Chemotherapy: 6 rounds of ABVD

This interview has been edited for clarity. This is not medical advice. Please consult with your healthcare provider to make treatment decisions.


Thank you for sharing your story, Brescia!

Inspired by Brescia's story?

Share your story, too!


Related Cancer Stories

More Hodgkin Lymphoma Stories

Madi J., Nodular Sclerosis Hodgkin’s Lymphoma, Stage 1B



Symptom: Shortness of breath
Treatment:
Chemotherapy

Danielle D., Hodgkin’s Lymphoma, Stage 2



Symptom: Swollen lump on right side of neck and chest area that continued to grow
Treatment: Chemotherapy

Lani S., Hodgkin’s Lymphoma, Stage 2



Symptom: None; appendicitis led to a CT scan that found tumor
Treatment: Chemotherapy
Jason

Jason F., Hodgkin’s Lymphoma, Stage 2A



Symptoms: Itchy legs, bloated face and “upper trunk,” slow-healing wounds, asthma worsened
Treatments: Chemotherapy, radiation
Logan

Logan A., Hodgkin’s Lymphoma, Stage 2A



Symptoms: Lump in neck, fatigue
Treatment:
Chemotherapy
Categories
Ovarian Patient Stories

Tiffany’s Stage 3A Ovarian Cancer Story

Tiffany’s Stage 3A Ovarian Cancer Story

Interviewed by: Taylor Scheib
Edited by: Chris Sanchez

33 year-old Tiffany is undergoing treatment for stage 3A ovarian cancer.

Tiffany was about to embark on a yearlong trip when she received life-altering news, a cancer diagnosis. Her diagnosis not only forced her to postpone her trip, but also convinced her to undergo IVF egg retrieval in order to preserve her ability to have children later on.

Tiffany is in the midst of her ovarian cancer treatment and, as of her interview, was steeling herself to undergo major surgery. But she has many reasons to be positive about her future. She shares her story with us to help others in the same situation.

In addition to Tiffany’s narrative, The Patient Story offers a diverse collection of stories about ovarian cancer. These empowering stories provide real-life experiences, valuable insights, and perspectives on symptoms, diagnosis, and treatment options for cancer.


 
  • Name: Tiffany L.
  • Diagnosis:
    • Ovarian cancer
  • Staging:
    • Stage 3A
  • Initial Symptoms:
    • Severe bleeding after insertion of IUD
    • Discomfort and pain after working out
  • Treatment:
    • Chemotherapy: Carboplatin and Taxol
    • Surgery: Total hysterectomy

This interview has been edited for clarity. This is not medical advice. Please consult with your healthcare provider to make treatment decisions.


My biggest advice is to be really honest with yourself first.

Like, really checking in, are you feeling something or not?

Introduction

My name is Tiffany. I’m based in Austin, Texas. I’m 33 years old. 

I love pole dancing, contortion, and I’m learning how to draw for fun, as well as watercolor in my free time.

I’ve been diagnosed with ovarian cancer, stage 3A.

Discovery and diagnosis

I was supposed to go travel abroad for a year in 2024, and was supposed to leave the first weekend of January. Didn’t want to carry a year’s supply of birth control with me, so I decided to get an IUD. 

I had an annual exam at the gynecologist, just standard stuff, and then that was when I mentioned an IUD, and so we got that inserted. We did the normal stuff that you would do during the annual exam. The gynecologist didn’t find anything weird then. 

At first, the IUD was fine, it wasn’t too painful or anything. About a week after it was inserted, I started bleeding like crazy.

I had my strings checked a month later, and that was okay, too, but I was bleeding a lot. It was like I was soaking through a heavy pad every hour or so. And so I just left it because it went away eventually. 

But a couple weeks after the string check, I started bleeding a lot again, and I didn’t really stop bleeding until they took it out. I was having heavy bleeding, and then it would it would kind of slow down a bit.

It just went on for weeks. So that’s why I called them to double check to make sure it was okay. 

I called them probably mid December or so. Because usually what happens is like, you call them and they just tell you, oh, it’s normal. It’s an adjustment period for the IUD, you’ll be okay. But I’m leaving in a few weeks. I need you to make sure this is okay before I go abroad.

So finally, they got back to me and they’re like, okay, we’re gonna do an ultrasound to check. I remember going in, I was like, I’m pretty sure this IUD is misplaced. I remember the sonographer going through everything. And then I looked at the screen and I saw this huge hole. 

I can’t read the graphics, but I saw her face and I was like, what is going on? I’m pretty sure there’s something more than just this IUD problem. But she’s not able to tell me what’s happening because I don’t think she’s allowed to by law. 

I almost was able to talk to the doctor right away, but then I actually had a work meeting, so I had to schedule it for I think the next day or something. I was just really anxious for  24 hours or so. And then the next day I went in and the doctor was like, hey, look, we found a gigantic cyst.

We don’t know what it is, the doctor said. I’m going to need to refer you to a gyno oncologist. Like I’m supposed to leave next week. Can I get an appointment right away? And she’s like, yeah, I’m gonna try to see if we can bump you up. And then she ran this blood test for the tumor marker, CA 125, that same day. So the new doctor can get all the results and stuff. 

So I had an appointment I think the next week, right before I was supposed to leave. I was like, well, there’s a huge thing in me, regardless of it being cancer or not, I need to take this out. So I probably do have to postpone my trip. There’s no way, you know, this person can just take it out in a week. So, yeah, I postponed my trip.

In my head I was like, okay, maybe it’s just postponed for a few weeks or a month. Maybe I can head out in February or something, which is not a big deal. But by that time, I had already quit my job because that was the plan. 

My last day of work was right before I was supposed to leave. It it just put me in a weird position because, I was like, well, I might leave soon anyway, so I guess it’s just like a month, I don’t need to get my job back. So I didn’t really bother with it. But also, it was just like a strange, in-between time where everything was up in the air. 

So I went to the new doctor, the gyno oncologist. And she basically was like, I need to see an MRI. So I had to get that done the next week.

And then after that, the follow up appointment, she didn’t really say it was cancer or anything, actually, even though my blood work was over the chart, my CA 125 was like 4000 and the standard was like 35. So it was crazy high. But, different things can affect your CA 125, such as endometriosis, which I wasn’t diagnosed with. All I knew was that I had PCOS. [Polycystic ovary syndrome (PCOS) is a hormonal disorder in reproductive-age women, causing infrequent or prolonged periods and elevated androgen levels.]

So when I talked to her after the MRI, she wasn’t definitive that it was cancer, but she wasn’t saying that it wasn’t either, because they don’t really tell you until they really go in there anyways. So she basically she knew that she had to take out the ovary that was attached to the tumor. But she asked me, if it’s cancer, what are your thoughts about having kids? Um, because if it’s cancer, usually they take everything out.

It took me a few days to get back to her because it was just so shocking to think about. We ended up deciding that we would keep the other ovary in there, even if it’s cancer, because we wanted to preserve my fertility and see if we could go through the IVF route.

Going into surgery, the doctor thought it’s probably borderline because it’s rare that someone in their 30s would have ovarian cancer. But, I mean, it could happen. She said that if it is borderline, then I probably wouldn’t need chemo. It ended up not being borderline. 

What happened during surgery is that she took the tumor out with the ovary, and she said the tumor was hard to take out. It wasn’t like a solid tumor, where, she could just pick it up with the robot and take it away. It was mushy and weird, gross-sounding.

The surgery took five hours, I believe, which was way longer than I expected because, besides taking it out, she also had to wait for a biopsy to see if it’s cancer and then after it’s cancerous, she does surgical staging. So she would swipe samples of different areas of my pelvis. I think it was bladder, bowels, uterus. 

She took the omentum out for biopsy to see if there’s any cancer cells there. So that’s also probably why it took five hours. I took up pretty much the rest of her afternoon, so I didn’t really see her until the next day. And that’s when she told me that, hey, it is cancer. 

But we didn’t know the staging; we just know that it’s probably more than likely, more than stage two, based on what I saw in there, but not definitive. Two weeks later, during my post-op appointment, I got the report. 

So the original report says there were cancer cells in the omentum. So that would put me in stage 3A. But there was another part of the report where they weren’t exactly sure where the origin site is for the cancer.

So they were suspecting that it’s either primary ovarian or possibly could be from the uterus or it’s synchronized cancer, because the type that I have was called an endometrioid ovarian cancer. That might come from endometriosis. So they are thinking that it could be synchronized, but for now they are treating it as a primary ovarian cancer. 

So it’s like still not definitive in a way. A little strange because I think the staging changes depending on the primary side. 

So if it’s ovarian primary, it’s like stage three, if it’s uterine primary and it’s in the omentum, that could put me in stage four. But then if it’s synchronized then it could be stage one for both. So it’s very confusing. The staging kind of tells you the prognosis, but then all the information data that they have are for like older women. So then it’s not like that information is for my population. 

At this rate, like honestly, I don’t look at my prognosis because none of the data is very specific to my population.

So I went ahead and got a second opinion too, just because why not? Insurance covers it. And I got a second biopsy at MD Anderson. And the report came back the same. 

So that’s a good thing, that it’s the same, I guess. Ultimately they’re not really going to be able to really find out exactly what it is until they take everything out. 

Treatment plan

After the doctor told me about my ovarian cancer diagnosis, she gave me a treatment plan. 

So I was going to be having three rounds of chemo, surgery to take everything out, and then three rounds again. 

So the midway point for the surgery, they just wanted to get the chemo in me first, but because I already would have had three rounds of chemo by then, they might not be able to really biopsy enough. There might not be enough cancer cells there for them to find out what is really going on. 

IVF

About a week after the report and discussing it with her, I started my IVF cycle, so I started freezing my embryos. 

That wasn’t fun. It was a lot of injections, doctor’s appointments., and going back and forth, trying to get a discount.

My insurance didn’t cover the IVF cycle, so I had to look into different organizations that would provide discounts on the clinic. And there are discounts for cancer patients, I think in most clinics, at least mine did. And I was able to get help from Livestrong and the Heart Beat program with Walgreens

The Heart Beat program basically gave me all the medication for free, which was really helpful because the medication itself, it’s like $10,000. And then the Livestrong also helped with the medication as well. It was like a 20% discount on certain things through the clinic. 

I think we were fortunate in a sense, because I know a lot of women, they have to go through multiple cycles to freeze 1 or 2 embryos. We were lucky in a sense that we only had 1 shot, just 1 cycle before chemo started. We were able to freeze 8 embryos, which was amazing. 

The clinic helped. They were the ones who told me about Livestrong. And they were the ones who applied on my behalf for the Walgreens Heart Beat program. 

I think the complicated part, at least with my clinic, it was more like I wasn’t really sure who should be doing what part of the application process. It might have just been a clinic issue, but they weren’t very clear on who is starting? Am I the one applying directly or are they doing it on my behalf? So I had to do a little bit of work in that regards. But ultimately they were the ones who found the programs. 

My doctor was the one who referred me to this clinic, so it seemed like she already had a relationship with the reproductive endocrinologist. So before I even went on my consultation, she already knew what was going on.

In terms of figuring out financially, I think we were fortunate in a way, because we had saved up for a trip and so we already had that nest there. If it wasn’t going to cost like that, we would have just done it anyways. 

My husband and I, this has been a lot of discussion between like having kids or not. He’s the one that really wanted the kids. I was more like, could be maybe. Maybe not. It was just like, okay, might as well do this because if we don’t do it, we might regret it later.

This is a little bit morbid. If I, you know, pass away in 2 years, I felt at least I left something for my husband—a part of me there for him.

So, the IVF process. The first appointment was just discussing the different options. So I had the option of just freezing the eggs or embryos. But you still have to go through the IVF process. It essentially means they’re just taking the eggs out, the embryo part comes later. 

During the process, they would monitor your follicles to see how they’re growing. Usually they want you to start, I think, like day 1 or 2 of your period. Um, I didn’t. I was on a timeline, so it didn’t pertain to me But I also luckily had my period the first day I saw her. So it kind of worked out in that sense.

So the first appointment, the doctor would check to see how many eggs are already there. In a way it was fortunate I had PCOS, so I had extra eggs. Apparently, if you have PCOS, it’s better for the process because you have more eggs. 

Once she thinks you’re ready, you will start doing your injections. I did two medications, on my abdomen. The first injection was just so scary because you guess your spouse or someone could help you, but I just did it on my own. The medication helps grow the follicles. 

I only had one ovary to do this, for others they might have two. But for my one ovary, she saw like 18 or something on there already. So they’re trying to grow everything at the same time essentially with the medication, but they don’t want you to grow it so fast, your ovary will get too big and then you will get hyper stimulating ovaries, which will cause a lot of pain.

They want to monitor you, every other day or so with blood work to check your estrogen level and also ultrasound. So I had to go to the clinic every other day. They gave me a different medication to start, to kind of balance it out so it doesn’t overgrow.

And then after about ten days, they decided that it was time for the trigger shot. They’re checking the size of each follicle. They want them all to be as big as possible. I think I had three that were like 20mm or something, I can’t remember. And that was when they decided it was time. 

So then I did the trigger shot, and then the day after, they did the egg retrieval. During egg retrieval, they put me under, and then the process took like ten minutes. 

And then they woke me up and they were like, we took out 22 eggs or something.

Treatment

Chemotherapy: Carboplatin and Taxol

Right after, a week after IVF, I started chemotherapy to deal with my ovarian cancer.

I got a week break between IVF and the chemo cycle. And I went back to Jersey for a wedding.

Okay, so the chemo regimen I’m on, it’s carboplatin and taxol. My chemo regimen is every 3 weeks. I would say it’s only bad the first week, and then it’s pretty much back to normal the next two weeks. 

I did so much research before, I feel like I already knew everything before I started. And also, before chemo, they did like a chemo teach where I met with one of the physician’s assistants and they gave me a binder of information. So I already had an idea of what to expect.

Side effects

In terms of side effects, I think the major one is the hair loss. That pretty much happened after cycle 1 and throughout cycle 2 as well. But I think after cycle 1, I was just so anxious, I was like, I’m going to shave it off anyways. Just get it over with. 

When I was reading online, I wanted to know when my hair loss would start. And most people said it would start about the second week. So I thought, I wish I knew that because I remember after the first, for like a week, I was just obsessive about my hair. 

I would wake up and be like, are you going? Are you leaving me yet? And then when it was still here, I was like, oh my gosh, maybe I’m one of those lucky people. I wish I would have known that it really would start like after the second week.

I was never too attached to my hair. Or at least I thought so. Some women love their hair and they want it to look a certain way. I guess if I cut it and it’s ugly, I didn’t care too much, would be upset for a minute, and then I’d be like, okay, it’s gonna grow back. You’ll be fine. 

When my hair started falling off, when I started seeing strands of hair on my pillow, It was just horrific to see that much hair coming out. I did get a little bit emotional. I think because I didn’t have that much time to process everything. Just seeing the hair kind of hit me in the face a little bit.

And then I felt really concerned about how I looked like without hair. I wasn’t sure if I would still feel attractive or if my husband was still find me attractive. 

Ultimately, when I shaved my hair off, I was I was kind of surprised that, I thought, I still look good without the hair. I went on a lot of shopping sprees, just for new styles and wigs and makeup stuff. So I think that kind of helped. 

Right now, I have days where I’m like, I really miss my hair. Especially like when I go out and I see people with beautiful hair, I’m like, oh, I really miss having hair. 

But then most of the other days I’m just like, oh, how would this look on me now? I feel like I get to play a little bit with a different style. And then sometimes I’m also kind of like, I wonder what style is going to stick after this is over. Maybe I’ll adopt some of these new things.

So other side effects have mostly been swollen hands. I’m also having a slight neuropathy, only on my index finger. Very strange. And then a little bit of brain fog, but that tends to fade away after the first week.

Surgery: total hysterectomy

So my next milestone in my ovarian cancer journey, I guess, is my surgery. That’s coming up on May 20th. I get 4 weeks in between surgery and chemo, which means I get an extra week to play.

So the surgery I’m getting will be a total hysterectomy. They’re going to be taking out my remaining ovary, my uterus, the fallopian tube that attaches to the ovary, and my cervix as well. 

I am incredibly anxious about it, because once they take out that ovary, that puts me in surgical menopause. And for women that are in forced menopause, I’m high-risk later on for osteoporosis as well as heart disease. 

Not to mention, I’ve been reading a lot of other women’s experiences that are around my age, mostly breast cancer survivors. It sounds like there’s like a thing called vaginal atrophy that will happen or might happen as well. 

Also, all the other stuff that comes with menopause, like hot flashes. I’m very, very scared just about what might happen. 

Also they say that sometimes you don’t get those symptoms right away. So it’s not like I’m gonna wake up and it happens. It might take a few weeks before it happens.

They haven’t really talked to me about post-surgery. I just know I get a 4-week break and then I go back for chemo, but I do believe it’s the same chemo.

… don’t brush away that little voice that’s telling you something is probably wrong.

Shifts and learnings

Support

I felt like because of my ovarian cancer situation, everyone was extra nice to me. I would say I’m very fortunate, because everyone is being so supportive. 

My mom lives in Jersey and flies here every cycle to help me out, and my husband has been very supportive, too. My friends like giving me rides and everything, too.

I think it’s going pretty well for what it is.

Realizations

So when I look back, I do feel like I had the symptoms of ovarian cancer, I definitely ignored them. I do crazy workouts, so, yeah, I’m going to be like, there’s some tightness there or I just kind of brushed it off. but when I look back, I was kind of in pain. 

I was remembering how there were days after I trained and I would be like, wow. Training really hurt today. And I really don’t want to do this post because it’s putting a lot of pressure in my abdomen and it’s really pretty uncomfortable. But I just brushed it away. 

And so I think that if I did not get that IUD, I think it would have gotten me eventually, Because I’m young and healthy. I didn’t think of it as anything. I mean, I didn’t really bring it to the doctor or anything, but even, like, with the whole IUD thing, I felt like I had to really push for them to, take a look at it. And so, it is very important, don’t brush away that little voice that’s telling you something is probably wrong.

I think it’s mostly realizing, this sucks, but I still get to do so much cool stuff later on. Hopefully. 

It sucks that I didn’t get to travel, but after this, maybe I can I can travel again, but it’s gonna look a little bit different than I planned because I’m not going to be able to just leave for a year now because of all the monitoring that they do. Kind of realizing that I’m still going to be able to see everything. 

But I honestly think what has really gotten me through this is just me on the pole. Because I’m still able to pole dance, and contort, which I don’t understand how sometimes. So that has been pretty incredible, very helpful as well. Like for my mental health, too.

But I think what shifted in the way I’m thinking about it is to just appreciate what I can do, versus going after certain things and being frustrated with it. So I kind of see it in like a new perspective. Oh, I just took a class, and then I never looked back. Yeah. 

Pole dancing is like a challenge. I love it because as long as you put the work or the training, the time into it, you will see results. And it’s like a way to express yourself in whatever way you want it to be.

So it doesn’t have to be sexy. It could be like emotional. It could be very athletic or just like, whatever you want. And I really love it for the art form and also for the physical challenge. And also the pole community is incredible. I made so many friends off of it, and it’s just a great place.

Advice

My biggest advice from everything that I’ve learned in my ovarian cancer journey is to be really honest with yourself first. Like, really checking in, are you feeling something or not? Even if the doctor thinks you’re being a little too much, just push them to do something because you never know.

And then really do your research before going to the doctor, because, hopefully you have a good doctor, but, if you don’t, you have the knowledge to back up what you are suspecting. And then be very assertive as to what you’re asking them for. 

I would say something like, hey, I have a concern with this. Can we do a scan or something, instead of having them lead the way? You kind of want to almost lead the conversation instead. 

And then also be honest with them as to what you’re feeling, too, because I think sometimes people brush away symptoms because they’re scared of what it could be. It’s better to know what it is and deal with it earlier versus later.


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