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myeloid leukemia associated with down syndrome - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

myeloid leukemia associated with down syndrome

MONDO:0850271

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Overview

An acute megakaryocytic leukemia occurring in children with Down syndrome and that has material basis in mutation in the GATA1 gene.

Clinical Trials4 registered · 2 recruiting

ResearchLimited published research (7 articles)

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

4 clinical trials registered, 2 recruiting. Interventions under study include drug therapy, other interventions, and procedural interventions. Pipeline includes 1 PHASE3, 2 PHASE1. Research is primarily sponsored by academic and government institutions.

7 publications have been identified in PubMed for myeloid leukemia associated with down syndrome. Research spans Clinical Trial Publication (43%), Review / Meta-Analysis (14%), and Case Report / Case Series (14%).

Recent Publications

Thibodeau J (2026). [PMID: 41692349](https://pubmed.ncbi.nlm.nih.gov/41692349/). *Biochem Pharmacol*. [Basic Science / Preclinical]
Laszig S (2026). [PMID: 41118594](https://pubmed.ncbi.nlm.nih.gov/41118594/). *Blood*. [Clinical Trial Publication]
Verboon L (2026). [PMID: 41945753](https://pubmed.ncbi.nlm.nih.gov/41945753/). *Blood Adv*. [Gene Therapy / Novel Therapeutics]
Deana A (2026). [PMID: 42077118](https://pubmed.ncbi.nlm.nih.gov/42077118/). *Pediatr Blood Cancer*. [Clinical Trial Publication]
Kaleta K (2024). [PMID: 39062461](https://pubmed.ncbi.nlm.nih.gov/39062461/). *Biomolecules*. [Review / Meta-Analysis]

Data assembled from 3 of 12 sources · Last updated Sep 20, 2026, 11:13 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

Questions for Your Care Team

→Are any of the 4 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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myeloid leukemia associated with down syndrome

Overview

Research Landscape

Recent Publications

Medical References

Related Diseases

Questions for Your Care Team

Questions for Your Care Team