Overview
Chronic mast cell leukemia (also sometimes called chronic MCL) is a very rare and serious blood cancer that involves abnormal mast cells. Mast cells are a type of immune cell normally found in tissues throughout the body. They play an important role in allergic reactions and fighting infections. In chronic mast cell leukemia, these cells grow out of control and build up in the bone marrow, blood, and organs like the liver, spleen, and skin. Unlike the acute form of this disease, the chronic form tends to progress more slowly, but it is still considered a life-threatening condition. People with this disease often experience a wide range of symptoms because mast cells release powerful chemicals that affect many parts of the body. These chemicals can cause severe allergic-like reactions, skin changes, bone pain, and problems with the digestive system. The spleen and liver may become enlarged, causing discomfort and a feeling of fullness. Treatment is focused on controlling the growth of abnormal mast cells and managing symptoms. Targeted therapies, including drugs that block a protein called KIT (such as midostaurin and avapritinib), have improved care for some patients. Stem cell transplant may be considered in certain cases. Because this disease is so rare, care at a specialized center with experience in mast cell diseases is strongly recommended.
Also known as:
Key symptoms:
Severe allergic-like reactions (flushing, hives, swelling)Bone pain or tendernessFatigue and weaknessEnlarged spleen causing abdominal discomfort or fullnessEnlarged liverSkin lesions or reddish-brown spots (urticaria pigmentosa)Nausea, vomiting, or diarrheaLow blood counts leading to anemia or increased infectionsUnexplained weight lossNight sweatsItching (especially after heat, exercise, or stress)Low blood pressure or fainting episodes
Sporadic
Usually appears on its own, not inherited from a parent
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
8 eventsAscentage Pharma Group Inc. — PHASE1
Ankara City Hospital Bilkent — NA
iOMEDICO AG
National Heart, Lung, and Blood Institute (NHLBI)
Northwell Health — NA
Medical College of Wisconsin — PHASE1, PHASE2
Memorial Sloan Kettering Cancer Center — PHASE2
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA) — PHASE1, PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableKrystexxa
indicated for the treatment of chronic gout in adult patients refractory to conventional therapy
Clinical Trials
View all trials with filters →No actively recruiting trials found for Chronic mast cell leukemia at this time.
New trials open frequently. Follow this disease to get notified.
Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
2 resourcesKetalar
Par
Chronic Pain
Travel Grants
No travel grants are currently matched to Chronic mast cell leukemia.
Community
No community posts yet. Be the first to share your experience with Chronic mast cell leukemia.
Start the conversation →Latest news about Chronic mast cell leukemia
Disease timeline:
New recruiting trial: CAR-20/19-T Cells in Patients With Relapsed Refractory B Cell Malignancies
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: Evaluation of Hypertension Management and Cardiovascular Adverse Event Prevention in Patients With B-cell Malignancies Undergoing Treatment With Bruton Tyrosine Kinase Inhibitors, the HALT Study
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: A Safety and Efficacy Study Evaluating CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: A Study of AZD0486 Monotherapy or in Combination With Other Anti-Cancer Agents for Mature B-Cell Malignancies
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: Study of Zanubrutinib, Obinutuzumab, and Venetoclax in Patients With Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Leukemia (SLL)
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: Implanted Loop Recorders for Detection and Management of Arrhythmia With Bruton Tyrosine Kinase Inhibitors
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: Observational Study of Cardiac Arrhythmias During Treatment With BTK Inhibitors or Venetoclax
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: Registry Platform Hematologic Malignancies (RUBIN) - Extension of Tumor Registry Lymphatic Neoplasms
A new clinical trial is recruiting patients for Chronic mast cell leukemia
New recruiting trial: The Effects of Intra-articular and Peri-articular Platelet-rich Plasma (PRP) Injections ın Chronic Knee Osteoarthritis
A new clinical trial is recruiting patients for Chronic mast cell leukemia
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.Which form of mast cell leukemia do I have, and how does that affect my treatment options?,Do I have the KIT D816V mutation, and does that change which drugs will work best for me?,Am I a candidate for avapritinib or midostaurin, and what side effects should I expect?,Should I consider a stem cell transplant, and what would that process involve?,Are there any clinical trials I should know about?,What triggers should I avoid to reduce the risk of a severe allergic reaction?,How often will I need bone marrow biopsies and blood tests to monitor the disease?
Common questions about Chronic mast cell leukemia
What is Chronic mast cell leukemia?
Chronic mast cell leukemia (also sometimes called chronic MCL) is a very rare and serious blood cancer that involves abnormal mast cells. Mast cells are a type of immune cell normally found in tissues throughout the body. They play an important role in allergic reactions and fighting infections. In chronic mast cell leukemia, these cells grow out of control and build up in the bone marrow, blood, and organs like the liver, spleen, and skin. Unlike the acute form of this disease, the chronic form tends to progress more slowly, but it is still considered a life-threatening condition. People wit
How is Chronic mast cell leukemia inherited?
Chronic mast cell leukemia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Chronic mast cell leukemia typically begin?
Typical onset of Chronic mast cell leukemia is adult. Age of onset can vary across affected individuals.
Which specialists treat Chronic mast cell leukemia?
25 specialists and care centers treating Chronic mast cell leukemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Chronic mast cell leukemia?
2 patient support programs are currently tracked on UniteRare for Chronic mast cell leukemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.