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Significant Disparity: Myeloma Real-World Results Show Striking Contrast with Clinical Trials

Worse Outcomes for Myeloma Patients in Real-World Results vs. Clinical Trials

Multiple myeloma patient outcomes are strikingly different in the real-world versus controlled environments according to data presented at the ASH Conference 2023 (Abstract 541). In the real world, multiple myeloma patients demonstrated a 44% reduction in progression-free survival (PFS) and a 75% decrease in overall survival (OS) compared to participants in clinical trials.

The study titled “Comparison of the Efficacy in Clinical Trials Versus Effectiveness in the Real-World of Treatments for Multiple Myeloma: A Population-Based Cohort Study” explores the gap between clinical trial efficacy and real-world effectiveness of treatments for multiple myeloma.

Conducted by a team led by Dr. Alissa Visram, the research focused on assessing the outcomes of patients treated with standard-of-care multiple myeloma regimens in routine practice compared to those in registration phase III randomized controlled trials (RCTs).

Key Findings:

  1. Efficacy-Effectiveness Gap: Real-world (RW) patients experienced a 44% worse progression-free survival (PFS) and a 75% worse overall survival (OS) compared to RCT patients across various multiple myeloma regimens.
  2. Patient Characteristics: RW patients were generally older, and for relapsed regimens, there was a longer time between multiple myeloma diagnosis and the start of the regimen in the real-world compared to RCTs.
  3. Regimen Performance: Most multiple myeloma regimens evaluated showed worse PFS and OS in the real-world setting, except for pomalidomide/dex (Pd), which demonstrated a trend towards better performance.
  4. Safety Profile: The safety profile, measured by inpatient hospitalization rates during treatment, was comparable between the real-world cohort and reported serious adverse events (AEs) in RCTs.

Implications: The study emphasizes the significant efficacy-effectiveness gap between registration RCTs and real-world usage of multiple myeloma regimens. The findings underscore the importance of ongoing evaluation of real-world data to inform clinicians and patients for shared treatment decision-making.

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Patient Stories by Multiple Myeloma Type

Explore our multiple myeloma stories below, where patients describe things like:

  • First myeloma symptoms
  • What treatments they underwent
  • Living with multiple myeloma

Active myeloma


Clay D.

Diagnosis: Multiple myeloma
1st Symptoms:
Persistent kidney issues, nausea
chemo, radiation, stem cell transplant

Melissa V.

Diagnosis: Multiple myeloma, stage 3
1st Symptoms:
Frequent infections
IVF treatment & Chemotherapy (RVD) for 7 rounds

Elise D.

Diagnosis: Multiple myeloma, refractory
1st Symptoms: Lower back pain, fractured sacrum
Treatment: CyBorD, Clinical trial of Xpovio (selinexor)+ Kyprolis (carfilzomib) + dexamethasone
Marti P multiple myeloma

Marti P.

Diagnosis: Multiple myeloma, stage 3

1st Symptoms: Dizziness, confusion, fatigue, vomiting, hives

Treatment: Chemotherapy (Bortezomib/Velcade), Daratumumab/ Darzalex, Lenalidomide, Revlimid) and stem cell transplant
Ray H. feature

Ray H.

1st signs: Hemorrhoids, low red blood cell count
Treatment: Immunotherapy, Chemotherapy, Stem Cell Transplant
Valarie T. feature profile

Valarie T.

Symptoms: Nose bleeds, fatigue, back pain
Treatment: Chemotherapy, stem cell transplant
Jenny A. feature profile

Jenny A.

Symptoms: Nose bleeds, fatigue, back pain
Treatment: Chemotherapy, stem cell transplant
Keith G.

Keith G.

Symptoms: High levels of protein
Treatment: Chemotherapy, stem cell transplant
Julie C.

Julie C.

Symptoms: Queasiness, food aversions, lack of appetite, fatigue
Treatment: Stem cell transplant, chemotherapy (D+PD), bispecific antibodies (talquetamab & cevostamab)
Erin H. feature profile

Erin H.

Symptoms: Back pains
Treatment: Chemotherapy, stem cell transplant
Gregory P. feature profile

Gregory P.

Symptoms: Back pains
Treatment: Chemotherapy, stem cell transplant
Laura E. feature profile

Laura E.

Symptom: Increasing back pain
Treatments: Chemotherapy, stem cell transplant, bispecific antibodies

Typical myeloma

The majority of people diagnosed with myeloma fall under this category:

  • IgG k (kappa)
  • IgG λ (lambda)
  • IgA k (kappa)
  • IgA λ (lambda)

Tim H.

Diagnosis: Multiple myeloma
1st Symptoms: None that could be identified; cancer found through CT scan for gallbladder removal
Treatment: Chemotherapy: Revlimid, Velcade, and Dexamethasone; Darzalex, Kyprolis, and Dexamethasone; Stem cell transplant)


Scott C.

Diagnosis: Multiple myeloma, relapsed/refractorySubtype: IgG lambda (majorityof myelomas)
1st Symptoms: Pain in hips and ribs, night sweats, weight loss, nausea
Clinical trial, chemo, kyphoplasty, stem cell transplant

Jude A.

Diagnosis: Multiple myeloma, stage 3
1st Symptoms:
Pain in back, hips and ribs; difficulty walking
Bilateral femoral osteotomy, reversal due to infection; chemotherapy

Light chain myeloma

It’s estimated that light chain myeloma makes up about 15% of all myeloma diagnoses. There are times when malignant plasma cells produce only the light chain component of the antibody. Patients diagnosed with these cases have what’s known as “light chain myeloma.”

Carlos C.

Diagnosis: Multiple myeloma, Light Chain, Stage 2
1st Symptoms:
Back pain and spasms
Back surgery to fuse T1 and T2, chemotherapy (RVD) and stem cell transplant

Non-secretory myeloma

Beth A.

Diagnosis: Multiple myeloma, relapsed/refractory
Subtype: Non-secretory (1-5% of myelomas)
1st Symptoms: Extreme pain between shoulder blades, sternum, head, burning sensation
1st Line Treatment: VAD chemo, radiation, stem cell transplant
RR Treatment: 8 chemo regimens, successful combo→selinexor+bortezomib+dexamethasone

Inactive myeloma

Smoldering myeloma

Maui B.

Diagnosis: Smoldering myeloma
Cancer Details:
Smoldering myeloma is pre-symptomatic or pre-treatment multiple myeloma
1st Symptoms:
Inflammatory eye disease, uterine bleeding

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