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Acute megakaryoblastic leukemia (AMKL) is a form of acute myeloid leukemia (AML) that occurs predominantly in childhood and particularly in children with Down syndrome (DS-AMKL). Nonspecific symptoms may be irritability, weakness, and dizziness while specific symptoms include pallor, fever, mucocutaneous bleeding, hepatosplenomegaly, neurological manifestations and rarely lymphadenopathy. Acute panmyelosis with myelofibrosis may also be associated with AMKL. In contrast to DS-AMKL (around 80 % survival), non-DS-AMKL is an AML subgroup associated with poor prognosis.
Biomarker and diagnostic research for acute megakaryoblastic leukemia has been reported in the published literature.
Estimated prevalence: <1 in 1,000,000 (VERY_RARE).
3 clinical trials registered. Interventions under study include drug therapy, other interventions, and gene therapy. Pipeline includes 1 PHASE2, 1 PHASE1. Research is primarily sponsored by academic and government institutions.
57 publications have been identified in PubMed for acute megakaryoblastic leukemia. Research spans Case Report / Case Series (35%), Basic Science / Preclinical (35%), and Gene Therapy / Novel Therapeutics (11%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 20 |
Data assembled from 4 of 12 sources · Last updated Oct 3, 2026, 11:26 PM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Common questions about acute megakaryoblastic leukemia
Laboratory research | 20 | 35% |
New treatment approaches | 6 | 11% |
Testing and diagnosis research | 5 | 9% |
Research summaries | 3 | 5% |
Disease patterns and progression | 2 | 4% |
Clinical study results | 1 | 2% |
Yang W (2026). [PMID: 41767736](https://pubmed.ncbi.nlm.nih.gov/41767736/). *Open medicine (Warsaw, Poland)*. [Case Report / Case Series]
Yang XQ (2026). [PMID: 41888737](https://pubmed.ncbi.nlm.nih.gov/41888737/). *BMC Pediatr*. [Case Report / Case Series]
Lin S (2026). [PMID: 41991315](https://pubmed.ncbi.nlm.nih.gov/41991315/). *Zhonghua Xue Ye Xue Za Zhi*. [Review / Meta-Analysis]
Li T (2026). [PMID: 41806317](https://pubmed.ncbi.nlm.nih.gov/41806317/). *Cytometry. Part B, Clinical cytometry*. [Diagnostic / Biomarker]
Choudhury SR (2026). [PMID: 41141393](https://pubmed.ncbi.nlm.nih.gov/41141393/). *Genes & diseases*. [Case Report / Case Series]
Komori T (2026). [PMID: 41631723](https://pubmed.ncbi.nlm.nih.gov/41631723/). *British journal of haematology*. [Clinical Trial Publication]
Munakata M (2026). [PMID: 40769884](https://pubmed.ncbi.nlm.nih.gov/40769884/). *Fukushima journal of medical science*. [Case Report / Case Series]
Su N (2025). [PMID: 41211316](https://pubmed.ncbi.nlm.nih.gov/41211316/). *PeerJ*. [Basic Science / Preclinical]
Crispino JD (2025). [PMID: 40875550](https://pubmed.ncbi.nlm.nih.gov/40875550/). *Blood*. [Basic Science / Preclinical]
Sudunagunta VS (2025). [PMID: 41000678](https://pubmed.ncbi.nlm.nih.gov/41000678/). *bioRxiv : the preprint server for biology*. [Basic Science / Preclinical]