Synonyms: Familial AML | Inherited AML | Pure familial AML | Pure familial acute myeloid leukemia
Inherited acute myeloid leukemia (AML) is a rare malignant hematopologic disease characterized by clonal proliferation of myeloid blasts primarily involving the bone marrow in association with congenital disorders (e.g. Fanconi anemia dyskeratosis congenita Bloom syndrome Down syndrome congenital neutropenia neurofibromatosis etc.) and genetic defects predisposing to AML. Patients present with signs and symptoms related to ineffective hematopoesis (fatigue bleeding and bruising recurrent infections bone pain) and/or extramedullary site involvement (gingivitis splenomegaly etc.). Depending on the underlying genetic defect there may be additional cancer risks and other health problems present.
Data from Orphanet are used to provide information on a disease's name, synonym(s), and overview. Reference: Access aggregated data from Orphanet at Orphadata. Orphadata: Free access data from Orphanet. © INSERM 1999. Available on http://www.orphadata.org. Data version May 2026
Newly diagnosed with
Inherited acute myeloid leukemia?
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Advocacy Organizations
Cache DNA
At Cache, we are on a mission to store the biomolecules of today in order to unlock the possibilities of tomorrow. Our vision is to create a scalable, sustainable, and standardized platform for reliable storage and access of biomolecules by combining chemistry, biology, automation, and computational tools to support patient advocacy groups worldwide and better connect samples to insights.
RUNX1 Research Program
At the RUNX1 Research Program, our mission is to improve the quality of life and prevent cancer in patients with RUNX1-FPD. We do this by promoting awareness and funding world-class collaborative research with an empowered patient community.
Team Telomere
A Community for Telomere Biology Disorders Our mission is to provide information and support services to families worldwide affected by Dyskeratosis Congenita and Telomere Biology Disorders, to encourage the medical community’s research in finding causes and effective treatments, and to facilitate improved diagnosis by educating medical providers.
Uganda Alliance of Patients Organization
Supporting patients to access quality, safe and patient-centered healthcare services.
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Clinical Trials
For a list of clinical trials in this disease area, please click here.