Overview
Acute promyelocytic leukemia (APL), also known as acute myeloid leukemia M3 or AML-M3, is a fast-moving type of blood cancer that affects the bone marrow — the spongy tissue inside your bones where blood cells are made. In APL, immature white blood cells called promyelocytes build up in the bone marrow and blood instead of developing into healthy, mature cells. This buildup crowds out normal blood cells, leading to serious problems with bleeding and infection. APL is caused by a specific chromosomal change in the leukemia cells: a swap of genetic material between chromosomes 15 and 17, written as t(15;17). This creates an abnormal gene called PML-RARA, which blocks blood cell development. The most dangerous feature of APL is a life-threatening bleeding disorder that can develop very quickly, making early diagnosis and treatment absolutely critical. The good news is that APL has become one of the most treatable forms of leukemia. With modern therapy using all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), most patients achieve complete remission. Cure rates now exceed 80-90% in many patients, making APL a remarkable success story in cancer medicine. Early recognition and urgent treatment are key to the best outcomes.
Also known as:
Key symptoms:
Unusual or easy bruisingHeavy or prolonged bleeding from cuts or minor injuriesNosebleeds that are hard to stopBleeding gumsTiny red or purple spots on the skin (petechiae)Extreme tiredness and weaknessPale skinFrequent infections or feversShortness of breathSwollen lymph nodes (less common than in other leukemias)Bone or joint painHeadache or confusion (if bleeding occurs in the brain)
Clinical phenotype terms (36)— hover any for plain English
Sporadic
Usually appears on its own, not inherited from a parent
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
10 eventsThe First Affiliated Hospital of Soochow University — PHASE3
Quetzal Therapeutics — PHASE3
SDK Therapeutics, Inc. — PHASE3
Stichting Hemato-Oncologie voor Volwassenen Nederland — PHASE3
Syndax Pharmaceuticals — PHASE3
Anhui Medical University — NA
Janssen Research & Development, LLC — PHASE3
Gruppo Italiano Malattie EMatologiche dell'Adulto — PHASE2
First Affiliated Hospital Xi'an Jiaotong University — PHASE3
Moleculin Biotech, Inc. — PHASE2, PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableTrisenox
For induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is…
For induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is characterized by the presence of the t(15;17) translocation of PML/RAR-alpha gene expression.
Vesanoid
Induction of remission in patients with acute promyelocytic leukemia who are refractory to or unable to tolerate anthracycline based cytotoxic chemotherapeutic regimens.
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 resourcesTravel Grants
No travel grants are currently matched to Acute promyelocytic leukemia.
Community
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1 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.What is my risk category (low, intermediate, or high risk), and how does that affect my treatment plan?,Will I need chemotherapy in addition to ATRA and arsenic trioxide?,How will you monitor whether my treatment is working, and what does a PCR test result mean for me?,What are the signs of the dangerous bleeding disorder (DIC), and what should I do if I notice them?,What are the short-term and long-term side effects of arsenic trioxide and ATRA that I should watch for?,What is my expected chance of cure, and what happens if the leukemia comes back?,Are there any clinical trials I should consider, especially if I am high-risk or if treatment doesn't work?
Common questions about Acute promyelocytic leukemia
What is Acute promyelocytic leukemia?
Acute promyelocytic leukemia (APL), also known as acute myeloid leukemia M3 or AML-M3, is a fast-moving type of blood cancer that affects the bone marrow — the spongy tissue inside your bones where blood cells are made. In APL, immature white blood cells called promyelocytes build up in the bone marrow and blood instead of developing into healthy, mature cells. This buildup crowds out normal blood cells, leading to serious problems with bleeding and infection. APL is caused by a specific chromosomal change in the leukemia cells: a swap of genetic material between chromosomes 15 and 17, writte
How is Acute promyelocytic leukemia inherited?
Acute promyelocytic leukemia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Acute promyelocytic leukemia typically begin?
Typical onset of Acute promyelocytic leukemia is adult. Age of onset can vary across affected individuals.
Are there clinical trials for Acute promyelocytic leukemia?
Yes — 20 recruiting clinical trials are currently listed for Acute promyelocytic leukemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Acute promyelocytic leukemia?
25 specialists and care centers treating Acute promyelocytic 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 promyelocytic leukemia?
2 patient support programs are currently tracked on UniteRare for Acute promyelocytic leukemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.