Overview
Acute megakaryoblastic leukemia (AMKL) is a rare and aggressive type of blood cancer. It is also sometimes called acute myeloid leukemia, M7 subtype, or AML-M7. In this disease, the bone marrow — the spongy tissue inside bones that makes blood cells — starts producing large numbers of abnormal, immature cells called megakaryoblasts. These are early versions of the cells that normally make platelets, which help blood clot. When these abnormal cells multiply out of control, they crowd out healthy blood cells, leading to serious problems throughout the body. Because healthy red blood cells, white blood cells, and platelets are reduced, people with AMKL often feel very tired, get infections easily, and bleed or bruise more than normal. The disease can affect people of any age, but it is especially seen in young children, particularly those under 3 years old. Children with Down syndrome (trisomy 21) have a significantly higher risk of developing this type of leukemia. Treatment typically involves intensive chemotherapy, and in some cases a bone marrow transplant (stem cell transplant) may be recommended. Children with Down syndrome who develop AMKL often respond better to chemotherapy than children without Down syndrome. Research into targeted therapies is ongoing, and outcomes vary depending on age, genetics, and how well the disease responds to initial treatment.
Key symptoms:
Extreme tiredness or fatiguePale skinEasy bruising or unusual bruisingBleeding that is hard to stop (such as nosebleeds or bleeding gums)Frequent infections or feversSwollen lymph nodes (lumps in the neck, armpits, or groin)Enlarged spleen or liver causing belly pain or fullnessBone or joint painShortness of breathLoss of appetite and unexplained weight lossNight sweatsSmall red or purple spots on the skin (petechiae)
Sporadic
Usually appears on its own, not inherited from a parent
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
4 eventsIn combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy
XOSPATA: FDA approved
XOSPATA is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation as detected by an FDA-approved test.
DAURISMO: FDA approved
DAURISMO is indicated, in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are >75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Roswell Park Cancer Institute — PHASE1
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
4 availableIdamycin
IDAMYCIN PFS is indicated for the treatment of adult patients with acute myeloid leukemia (AML) as a component of a combination chemotherapy regimen
Tibsovo
In combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-…
In combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy
XOSPATA
XOSPATA is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation as detected by an FDA-approved t…
XOSPATA is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation as detected by an FDA-approved test.
DAURISMO
DAURISMO is indicated, in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are >75 years old or who have comorbidities that…
DAURISMO is indicated, in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are >75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Clinical Trials
View all trials with filters →No actively recruiting trials found for Acute megakaryoblastic leukemia at this time.
New trials open frequently. Follow this disease to get notified.
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
1 resourcesTravel Grants
No travel grants are currently matched to Acute megakaryoblastic leukemia.
Community
No community posts yet. Be the first to share your experience with Acute megakaryoblastic leukemia.
Start the conversation →Latest news about Acute megakaryoblastic leukemia
5 articlesCaregiver 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.Does my child or I have the Down syndrome-related form of AMKL, and how does that affect the treatment plan?,What specific genetic changes were found in the leukemia cells, and what do they mean for prognosis?,Is a bone marrow transplant recommended, and if so, when and from what type of donor?,Are there any clinical trials available that might be appropriate for our situation?,What are the most important warning signs of infection or bleeding that should prompt an emergency room visit?,What are the possible long-term side effects of the chemotherapy drugs being used?,What support services — such as social work, psychology, or school support — are available to help our family during treatment?
Common questions about Acute megakaryoblastic leukemia
What is Acute megakaryoblastic leukemia?
Acute megakaryoblastic leukemia (AMKL) is a rare and aggressive type of blood cancer. It is also sometimes called acute myeloid leukemia, M7 subtype, or AML-M7. In this disease, the bone marrow — the spongy tissue inside bones that makes blood cells — starts producing large numbers of abnormal, immature cells called megakaryoblasts. These are early versions of the cells that normally make platelets, which help blood clot. When these abnormal cells multiply out of control, they crowd out healthy blood cells, leading to serious problems throughout the body. Because healthy red blood cells, whit
How is Acute megakaryoblastic leukemia inherited?
Acute megakaryoblastic leukemia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Which specialists treat Acute megakaryoblastic leukemia?
25 specialists and care centers treating Acute megakaryoblastic 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 Acute megakaryoblastic leukemia?
2 patient support programs are currently tracked on UniteRare for Acute megakaryoblastic leukemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.