Inherited acute myeloid leukemia

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Inherited acute myeloid leukemia

ORPHA:319465Disease

Also called Familial AML · Inherited AML · Pure familial AML · Pure familial acute myeloid leukemia

What it is

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.

Key facts

Inheritance
Autosomal dominant
Classified as
Disease

Recorded for the broader condition

Prevalence
1-5 / 10 000 (Europe)Acute myeloid leukemia
Age of onset
All agesAcute myeloid leukemia

Orphanet records these for the broader condition rather than for this specific form. They are a starting point, not a figure for this subtype — subtypes often differ, and some are defined by being rarer than the condition they sit under.

Genes

CEBPADisease-causing germline mutation(s)
ERCC6L2Disease-causing germline mutation(s)
TGM6Disease-causing germline mutation(s)

ICD-10 codes

C92.0filed under a broader ICD-10 category — shared with 13 other rare diseases

ICD-10 has no distinct code for most rare diseases, so many are filed under the nearest available one. A shared code identifies the group, not this disease — the ORPHAcode above is what identifies it.

Related health topics

Reached through the ICD-10 categories this disease is filed under, so these describe the broader group rather than this disease specifically.

Cross-references

MONDO 0017893OMIM 601626UMLS C4707228

Orphadata Science: Free access data from Orphanet. © INSERM 1999. Data version 2026-06-23. Available under CC BY 4.0. This is a reference, not medical advice.

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