Myeloproliferative Neoplasms Treatments

While researchers are still working on a cure for myeloproliferative neoplasms, originally referred to as myeloproliferative disorders, several treatments exist to help improve the symptoms and prevent further complications of MPNs. While the course of treatment varies by MPN type, there are generally ten different treatment options for a patient with an MPN.

The commonly considered treatment options for Myeloproliferative Neoplasms include:

• Phlebotomy
• Platelet apheresis
• Transfusion therapy
• Chemotherapy
• Radiation therapy
• Drug therapy
• Surgery
• Immunotherapy
• Targeted therapy
• Stem Cell Transplant

This article looks at how each treatment is used for myeloproliferative neoplasms and the common treatments for each of the six different MPNs.

What are the Treatments for Myeloproliferative Neoplasms?

A care team typically considers one or a combination of the following ten treatment options for patients with MPNs.

Cancer treatment decisions can be difficult to make. Therefore, it is important to understand the benefits and side effects of all treatment options.

Phlebotomy

Phlebotomy is simply the process of taking blood from your vein to run tests on. This test may be one of the ways that your doctor to see if you have an MPN; however, it can also be part of the treatment plan.

Frequent phlebotomies may help manage the negative impacts of having too many red blood cells. Therefore, your doctor may prescribe reoccurring phlebotomies as a way to manage MPN symptoms.

Platelet Apheresis

Platelet apheresis involves the usage of a plasma collection machine (PCM) to draw blood, separate the blood products, and then return the blood cells at normal levels back into the patient’s body.

The use of a PCM is part of a typical treatment plan for those patients with an MPN that does involve the overproduction of blood cells other than red blood cells.

Transfusion Therapy

Transfusion therapy is used in MPNs where blood cell production is limited. A blood transfusion can replace blood cells destroyed by the body or, in some cases, another form of cancer treatment.

Chemotherapy

Chemotherapy is a common treatment for several different forms of cancer. However, it is particularly effective in treating MPNs because it is a systemic treatment.

The chemotherapy is administered through an IV or via a pill and then enters the bloodstream. The drug then kills cells throughout the entirety of the body.

Radiation Therapy

Radiation therapy targets cancer cells to either kill them or prevent them from growing. There are two types of radiation therapy:

• External radiation therapy – a machine outside of the body produces a beam of radiation, typically aimed towards a specific part of the body.
• Radioactive drug therapy – the drug P32 is administered intravenously to help lower the number of blood cells produced by the bone marrow.

Both types of radiation therapy can be dangerous because you are exposing your body to high radiation levels.

Drug Therapy

Aside from P32, your doctor may recommend drug therapy to help manage the symptoms of an MPN or even prevent the further spread of cancer. Some commonly prescribed drugs for the treatment of MPNs include:

• Anagrelide or aspirin to help reduce the risk of blood clots
• Thalidomide, lenalidomide, or pomalidomide to prevent blood vessels from growing into areas with tumorous cells.
• Prednisone or danazol to help treat the underproduction of red blood cells.

For a complete list of drugs used to treat myeloproliferative neoplasms, visit cancer.gov.

Targeted Therapy

While targeted therapy can be considered a form of drug therapy, the intention of targeted therapy differs enough from typical drug therapy to warrant its own section. 

Targeted therapy makes use of drugs that attack specific cancer cells. In theory, this kills cancerous cells without harming the other healthy cells in your body. There are currently two targeted therapy drugs used to treat MPNs:

• Tyrosine kinase inhibitor (TKI) – blocks the signals cancer cells need to grow.
• Ruxolitinib – Blocks the JAK2 mutation, which is linked to MPNs.

 While there are only a limited number of targeted therapies for MPNs, further research is sure to produce other forms.

Immunotherapy

Immunotherapy, also known as biological therapy, is a way to help boost or restore your body’s immune system by creating substances that are typically produced by the body in a laboratory.

Surgery

In extreme cases of spleen enlargement, your care team may suggest a splenectomy.

Stem Cell Transplant

A stem cell transplant is the only form of treatment that can cure certain kinds of MPNs. A stem cell transplant involves a three-part process:

• Stem cells, also known as immature blood cells, are removed from the blood or bone marrow of the patient or donor, frozen, and stored.
• The patient completes chemotherapy to rid the body of any cancerous cells.
• The stem cells are then thawed and infused into the body to help the body grow healthy blood cells.

Stem cell transplants aren’t always an option for all patients with MPNs. It depends on your age, current symptoms, and MPN subtype.

Do treatments vary based on type of myeloproliferative neoplasms?

Each type of myeloproliferative neoplasms comes with its unique symptoms. Therefore, each form of MPN includes its treatment plan.

Correctly diagnosing the type of MPN is essential because the diagnosis can dramatically impact the treatment plan.

Treatments for chronic myelogenous leukemia (CML)?

Chronic myelogenous leukemia is a rare form of cancer in the bone marrow. Overproduction of myeloblasts leads to an increased number of white blood cells.

Common treatments for CML include:

• Targeted drug therapy
• Stem cell transplant (sometimes referred to as a bone marrow transplant)
• Chemotherapy

Targeted drug therapy for CML involves the use of drugs that block the action of tyrosine kinase. Some commonly prescribed medicines include:

• Gleevec (Imatinib)
• Sprycel (Dasatinib)
• Tasigna (Nilotinib)
• Bosulif (Bosutinib)
• Iclusig (Ponatinib)

Treatments for polycythemia vera

Polycythemia vera is an MPN that results in the overproduction of all blood cells, most commonly red blood cells. As a result, your blood ends up being much thicker than usual.

Treatments for polycythemia vera include:

• Frequent phlebotomies
• Medication or radiation therapy to reduce itching
• Medication to reduce the number of red cells
• Heart medications or even low doses of aspirin to minimize the risk of blood clots

In most circumstances, doctors will not prescribe medicine to treat polycythemia vera unless the phlebotomies are inefficient in reducing red blood cell counts.

Treatments for primary myelofibrosis

Primary myelofibrosis is characterized by the buildup of scar tissue in the bone marrow. As a result, the bone marrow cannot produce enough blood cells, or blood is made in organs like the liver and spleen.

Common treatments for primary myelofibrosis include:

• Blood transfusions
• Androgen therapy (use of male hormones to encourage blood cell production)
• Targeted drug therapy (specifically targeting the JAK2 gene mutation)
• Splenectomy
• Radiation therapy
• Stem cell transplant

The course of treatment may differ depending on if you are experiencing anemia or spleen complications.

Treatments for essential thrombocythemia

Essential thrombocythemia is when the body overproduces blood platelets resulting in abnormal clotting or bleeding.

Common treatments for essential thrombocythemia include:

• Prescribed medications including hydroxyurea, anagrelide, interferon alfa-2b, or peginterfereon alfa-2a.
• Platelet apheresis

Plate apheresis is only used in emergency situations where there is a high risk of a blood clot.

Treatments for chronic neutrophilic leukemia

Chronic neutrophilic leukemia (CNL) is when the neutrophil, a type of white blood cell, multiplies uncontrollably. 

There is no standard treatment for CNL due to the disease being so uncommon. Additionally, since CNL patients can remain stable for years, the disease often is not treated until it becomes acute.

However, there have been instances of attempts at stem cell transplantation and the use of drug therapy, specifically hydroxyurea.

Treatments for chronic eosinophilic leukemia

Chronic eosinophilic leukemia (CEL) is similar to CNL, only the body overproduces the white blood cell eosinophils instead of neutrophils.

Much like CNL, there is no standard treatment due to the rarity of the disease and its relatively stable nature. However, some known treatments have included chemotherapy and drug therapy.

What clinical trials are there for myeloproliferative neoplasms?

While there are plenty of options available to help treat the symptoms of myeloproliferative neoplasms, continuous research is constantly happening to find new ways to treat and even cure MPNs.

If have interest in participating in a clinical trial that may help advance research on MPNs, check out the MPN Research Foundation’s MPN Clinical Trial Pipeline.

Learn More About Myeloproliferative Neoplasms

The Patient Story is committed to sharing the stories and experiences of those who’ve had cancer.

To learn more about myeloproliferative neoplasms and hear the experiences of those who have battled MPNs, visit the myeloproliferative home page.