Waldenström macroglobulinemia

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ORPHA:33226OMIM:153600C88.0
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9Active trials19Specialists8Treatment centers1Financial resources

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Waldenström macroglobulinemia (WM), also called lymphoplasmacytic lymphoma, is a rare type of slow-growing blood cancer. It starts in white blood cells called B-cells, which normally help fight infections. In WM, these cells become abnormal and multiply out of control in the bone marrow — the spongy tissue inside your bones where blood cells are made. These abnormal cells produce a large amount of a protein called IgM (immunoglobulin M), sometimes called a 'macroglobulin.' When too much IgM builds up in the blood, it can make the blood thicker than normal, a condition called hyperviscosity syndrome. Common symptoms include extreme tiredness, unexplained weight loss, night sweats, and swollen lymph nodes. The thick blood can cause headaches, blurred vision, dizziness, and even bleeding from the nose or gums. Some people develop numbness or tingling in their hands and feet because the IgM protein can damage nerves. Anemia — a low red blood cell count — is also very common and contributes to fatigue. Many people with WM are watched closely without treatment at first, a strategy called 'watchful waiting.' When treatment is needed, doctors use chemotherapy, targeted drugs, and immunotherapy. Key FDA-approved and commonly used treatments include ibrutinib (Imbruvica), rituximab, bendamustine, and zanubrutinib (Brukinsa). WM is generally not curable with standard treatments, but many people live for many years with good quality of life when the disease is well managed.

Key symptoms:

Extreme tiredness and low energy (fatigue)Unexplained weight lossNight sweatsSwollen lymph nodes in the neck, armpits, or groinHeadaches and dizzinessBlurred or double visionNumbness or tingling in the hands and feetNosebleeds or bleeding gumsPale skin due to anemiaShortness of breathFeeling full quickly or a swollen belly (enlarged spleen or liver)Confusion or difficulty thinking clearlySensitivity to cold (Raynaud's phenomenon)Recurrent infections

Clinical phenotype terms (45)— hover any for plain English
Retinal hemorrhageHP:0000573Abnormality of neutrophilsHP:0001874Normocytic anemiaHP:0001897LeukemiaHP:0001909
Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Late onset

Begins later in life, typically after age 50

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

9 events
Mar 2026Study to Assess Change in Disease Activity of Oral Venetoclax in Adult Participants With Recurring Relapsed or Refractory (R/R) Waldenström Macroglobulinemia (WM)/Lymphoplasmacytic Lymphoma (LPL)

AbbVie — PHASE2

TrialRECRUITING
Mar 2025Efficacy of Venetoclax in Combination With Rituximab in Waldenström's Macroglobulinemia

Christian Buske — PHASE2

TrialRECRUITING
Dec 2024Zanubrutinib, Bendamustine, Rituximab Prev. Untreated WM

Massachusetts General Hospital — PHASE2

TrialRECRUITING
Feb 2022Loncastuximab Tesirine in WM

Shayna Sarosiek, MD — PHASE2

TrialRECRUITING
Oct 2021Spanish Registry of Patients With IgM Monoclonal Gammopathies

Fundación Española de Hematología y Hemoterapía

TrialRECRUITING
Mar 2021Bendamustine, Rituximab and Acalabrutinib in Waldenstrom's Macroglobulinemia

Sunnybrook Health Sciences Centre — PHASE2

TrialACTIVE NOT RECRUITING
Jul 2020Ibrutinib + Venetoclax in Untreated WM

Dana-Farber Cancer Institute — PHASE2

TrialACTIVE NOT RECRUITING
Feb 2020Rituximab and Ibrutinib (RI) Versus Dexamethasone, Rituximab and Cyclophosphamide (DRC) as Initial Therapy for Waldenström's Macroglobulinaemia

University College, London — PHASE2, PHASE3

TrialRECRUITING
Feb 2015Vaccine Therapy in Treating Patients With Lymphoplasmacytic Lymphoma

M.D. Anderson Cancer Center — PHASE1

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Waldenström macroglobulinemia.

9 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

9 recruitingView all trials with filters →
Phase 26 trials
Loncastuximab Tesirine in WM
Phase 2
Actively Recruiting
PI: Shayna Sarosiek, MD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Boston, Massachusetts +2 more · Age: 1899 yrs
Study to Assess Change in Disease Activity of Oral Venetoclax in Adult Participants With Recurring Relapsed or Refractory (R/R) Waldenström Macroglobulinemia (WM)/Lymphoplasmacytic Lymphoma (LPL)
Phase 2
Actively Recruiting
PI: ABBVIE INC. (AbbVie) · Sites: Higashiibaraki-gun, Ibaraki; Chuo-Ku, Tokyo +1 more · Age: 1899 yrs
Ibrutinib + Venetoclax in Untreated WM
Phase 2
Active
PI: Jorge J Castillo, MD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Boston, Massachusetts · Age: 1899 yrs
Zanubrutinib, Bendamustine, Rituximab Prev. Untreated WM
Phase 2
Actively Recruiting
PI: Andrew Branagan, MD, PhD (Massachusetts General Hospital) · Sites: Denver, Colorado; Boston, Massachusetts +3 more · Age: 1899 yrs
Efficacy of Venetoclax in Combination With Rituximab in Waldenström's Macroglobulinemia
Phase 2
Actively Recruiting
PI: Christian Buske, Prof. Dr. (University Hospital Ulm Department of Internal Med) · Sites: Bielefeld; Chemnitz +9 more · Age: 1899 yrs
Bendamustine, Rituximab and Acalabrutinib in Waldenstrom's Macroglobulinemia
Phase 2
Active
PI: Neil L Berinstein, MD (Sunnybrook Research Institute) · Sites: Calgary, Alberta; Edmonton, Alberta +7 more · Age: 1899 yrs
Phase 11 trial
Vaccine Therapy in Treating Patients With Lymphoplasmacytic Lymphoma
Phase 1
Active
PI: Sheeba Thomas (M.D. Anderson Cancer Center) · Sites: Houston, Texas · Age: 1899 yrs
Other1 trial
Spanish Registry of Patients With IgM Monoclonal Gammopathies
Actively Recruiting
· Sites: Mérida, Badajoz; Palma, Balearic Islands +49 more · Age: 1899 yrs

Specialists

19 foundView all specialists →
JM
Jorge J Castillo, MD
Specialist
PI on 3 active trials
VP
Véronique LEBLOND, Professor
Specialist
PI on 1 active trial
PM
Paolo Caimi, MD
CLEVELAND, OH
Specialist
PI on 5 active trials
TM
Thomas E. Witzig, MD
ROCHESTER, MN
Specialist
PI on 9 active trials
VP
Véronique LEBLOND, MD, PhD
Specialist
PI on 1 active trial
SK
Shaji Kumar
Anchorage, Alaska
Specialist

Rare Disease Specialist

PI on 7 active trials
SM
Shaji K. Kumar, MD
ROCHESTER, MN
Specialist
PI on 5 active trials
JP
James L. Rubenstein, MD, PhD
Specialist
PI on 2 active trials
AW
Ann Woolfrey
SEATTLE, WA
Specialist
PI on 5 active trials
JM
Jonathan Friedberg, MD
HOUSTON, TX
Specialist
PI on 2 active trials
C(
Contact Lilly at 1-800-LillyRx (1-800-545-5979)
Specialist
PI on 2 active trials
SS
Shayna R. Sarosiek
BOSTON, MA
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources

BRUKINSA

BeiGene

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Travel Grants

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Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.Do I need to start treatment now, or is watchful waiting appropriate for me?,What are my MYD88 and CXCR4 mutation results, and how do they affect my treatment options?,Which treatment do you recommend for me, and what are the main side effects I should expect?,How will we know if the treatment is working, and what happens if it stops working?,Are there any clinical trials I might be eligible for?,How often will I need blood tests and check-ups, and what symptoms should prompt me to call you right away?,Are any of my family members at higher risk, and should they be tested or monitored?

Common questions about Waldenström macroglobulinemia

What is Waldenström macroglobulinemia?

Waldenström macroglobulinemia (WM), also called lymphoplasmacytic lymphoma, is a rare type of slow-growing blood cancer. It starts in white blood cells called B-cells, which normally help fight infections. In WM, these cells become abnormal and multiply out of control in the bone marrow — the spongy tissue inside your bones where blood cells are made. These abnormal cells produce a large amount of a protein called IgM (immunoglobulin M), sometimes called a 'macroglobulin.' When too much IgM builds up in the blood, it can make the blood thicker than normal, a condition called hyperviscosity syn

How is Waldenström macroglobulinemia inherited?

Waldenström macroglobulinemia follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Waldenström macroglobulinemia typically begin?

Typical onset of Waldenström macroglobulinemia is late onset. Age of onset can vary across affected individuals.

Are there clinical trials for Waldenström macroglobulinemia?

Yes — 9 recruiting clinical trials are currently listed for Waldenström macroglobulinemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Waldenström macroglobulinemia?

19 specialists and care centers treating Waldenström macroglobulinemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Waldenström macroglobulinemia?

1 patient support program are currently tracked on UniteRare for Waldenström macroglobulinemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.