Acute myelomonocytic leukemia | UniteRare Disease Library

Overview

Acute myelomonocytic leukemia (AMML) is a type of blood cancer that affects the bone marrow — the soft tissue inside your bones where blood cells are made. In AMML, the bone marrow starts producing too many abnormal, immature white blood cells called myeloblasts and monocytes. These abnormal cells crowd out the healthy blood cells your body needs to fight infection, carry oxygen, and stop bleeding. AMML is classified as a subtype of acute myeloid leukemia (AML), sometimes referred to as AML-M4 under the older FAB classification system. Because healthy blood cells are replaced by leukemia cells, people with AMML often feel very tired, get infections easily, and bruise or bleed more than usual. The disease can also affect organs like the spleen, liver, and lymph nodes, causing swelling and discomfort. In some cases, leukemia cells can spread to the skin or gums. Treatment usually involves intensive chemotherapy to destroy the leukemia cells, often followed by a stem cell transplant for eligible patients. While treatment can lead to remission in many people, AMML can be aggressive and may come back. Research into targeted therapies and immunotherapy is ongoing, offering hope for better outcomes in the future.

Also known as:

AMMoL AML M4

Key symptoms:

Extreme tiredness and weaknessFrequent or severe infectionsEasy bruisingUnusual or prolonged bleeding (such as from the gums or nose)Pale skinFever without a clear causeNight sweatsUnexplained weight lossSwollen lymph nodesEnlarged spleen or liver causing belly discomfortSwollen or bleeding gumsSkin rashes or lumps caused by leukemia cells (leukemia cutis)Bone or joint painShortness of breath

Clinical phenotype terms (9)— hover any for plain English

Abnormality of the gingivaHP:0000168

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events

Feb 2026TACrolimus Targeted Immunosuppression Cessation in ALlogeneic HCT

Stanford University — PHASE1

TrialNOT YET RECRUITING

Jul 2025A Study of CBX-250 in Participants With Relapsed or Refractory Myeloid Leukemias

Crossbow Therapeutics, Inc. — PHASE1

TrialRECRUITING

Apr 2025Selinexor Combined With Azacitidine Therapy in High-Risk Myeloid Neoplasms Patients

Institute of Hematology & Blood Diseases Hospital, China — PHASE2

TrialNOT YET RECRUITING

Feb 2025ABBA CORD: dCBT w/ Abatacept for aGVHD Prophylaxis

Leland Metheny — PHASE2

TrialRECRUITING

Feb 2025Oral-ATO for TP53-mutated Myeloid Malignancies

The University of Hong Kong — PHASE2

TrialRECRUITING

Feb 2025A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy

Children's Oncology Group — PHASE1

TrialRECRUITING

Jan 2024Intensive Conditioning withTHI/Bu/Flu/Ara-C in Allo-HSCT for Myeloid Malignancies With Extramedullary Involvement

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

TrialRECRUITING

Jun 2023Venetoclax in Addition to Sequential Conditioning With Fludarabine / Amsacrine / Ara-C (FLAMSA) + Treosulfan for Allogeneic Blood Stem Cell Transplantation in Patients With MDS, CMML or sAML

Heinrich-Heine University, Duesseldorf — PHASE1, PHASE2

TrialRECRUITING

Jan 2023Study of CPX-351 (VYXEOS) in Individuals < 22 Years With Secondary Myeloid Neoplasms

St. Jude Children's Research Hospital — PHASE2

TrialACTIVE NOT RECRUITING

Dec 2022Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS

Clinical Hub for Interventional Research (CHOIR) — PHASE1

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Acute myelomonocytic leukemia.

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

View clinical trials →

Clinical Trials

20 recruitingView all trials with filters →

Phase 27 trials

Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome

Phase 2

Active

PI: Yesid Alvarado-Valero, MD (M.D. Anderson Cancer Center) · Sites: Houston, Texas · Age: 18–99 yrs

Oral-ATO for TP53-mutated Myeloid Malignancies

Phase 2

Actively Recruiting

PI: Harinder Gill, MD (The University of Hong Kong) · Sites: Hong Kong · Age: 18–99 yrs

Topotecan Hydrochloride and Carboplatin With or Without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia

Phase 2

Active

PI: Keith W Pratz (JHU Sidney Kimmel Comprehensive Cancer Center LAO) · Sites: Los Angeles, California; Los Angeles, California +4 more · Age: 18–99 yrs

Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome

Phase 2

Actively Recruiting

PI: Courtney DiNardo (M.D. Anderson Cancer Center) · Sites: Baltimore, Maryland; Cleveland, Ohio +1 more · Age: 12–99 yrs

ABBA CORD: dCBT w/ Abatacept for aGVHD Prophylaxis

Phase 2

Actively Recruiting

PI: Leland Metheny, MD (University Hospitals Seidman Cancer Center, Case C) · Sites: Cleveland, Ohio · Age: 18–65 yrs

CPX-351 as a Novel Approach for the Treatment of Older Patients With AML and MDS

Phase 2

Active

PI: Sudipto Mukherjee, MD, PhD (Cleveland Clinic, Case Comprehensive Cancer Center) · Sites: Cleveland, Ohio · Age: 60–99 yrs

Combining Active and Passive DNA Hypomethylation

Phase 2

Actively Recruiting

PI: Kirsten Grønbæk, Prof., MD (Rigshospitalet, Denmark) · Sites: Aalborg; Aarhus +8 more · Age: 18–99 yrs

Phase 15 trials

FHD-286 as Monotherapy or Combination Therapy in Subjects With Advanced Hematologic Malignancies

Phase 1

Active

PI: Sarah Reilly, MD (Foghorn Therapeutics) · Sites: Duarte, California; Boston, Massachusetts +3 more · Age: 16–99 yrs

Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MDS, MF and CMML (HSCT 002)

Phase 1

Actively Recruiting

PI: Karen Ballen (UVA) · Sites: Philadelphia, Pennsylvania; Charlottesville, Virginia · Age: 18–75 yrs

Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS

Phase 1

Actively Recruiting

PI: John Pimanda, Professor (University of New South Wales) · Sites: Sydney, New South Wales; Sydney, New South Wales +3 more · Age: 18–99 yrs

Study of Oral LY3410738 in Patients With Advanced Hematologic Malignancies With IDH1 or IDH2 Mutations

Phase 1

Active

PI: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) (Eli Lilly and Company) · Sites: Duarte, California; Los Angeles, California +35 more · Age: 18–99 yrs

A Phase 1 Trial of CIML NK Cell Infusion for Myeloid Disease Relapse After Hematopoietic Cell Transplantation

Phase 1

Actively Recruiting

PI: Susanne Baumeister, MD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Boston, Massachusetts · Age: 12–99 yrs

Other1 trial

Intensive Conditioning withTHI/Bu/Flu/Ara-C in Allo-HSCT for Myeloid Malignancies With Extramedullary Involvement

Actively Recruiting

PI: Xianmin Song, MD (Shanghai General Hospital, Shanghai Jiao Tong Univ) · Sites: Shanghai, Shanghai Municipality · Age: 18–55 yrs

Specialists

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KP

Keith W Pratz

PHILADELPHIA, PA

Specialist

Acute myeloid leukemia with minimal differentiation

PI on 2 active trials

Overview

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