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Acute myeloid leukemia, disease

Last edited: 4/16/2026

Overview

Acute myeloid leukemia (AML) is a hematologic malignancy characterized by the uncontrolled proliferation of myeloid progenitor cells in the bone marrow, leading to impaired hematopoiesis and often extramedullary involvement 1.

Diagnosis

  • Bone marrow biopsy showing ≥20% blasts with specific cytogenetic or molecular abnormalities 1.
  • Peripheral blood smear may reveal leukocytosis, blasts, or dysplastic changes 1.
  • Flow cytometry essential for immunophenotyping blasts 1.
  • Cytogenetic analysis and molecular testing (e.g., FLT3, NPM1, CEBPA mutations) crucial for risk stratification 1.
  • Management

  • Induction therapy: Anthracycline (e.g., daunorubicin 60-90 mg/m2) plus cytarabine (Ara-C, 100-200 mg/m2) for 7 days 1.
  • Consolidation: Multiple cycles of high-dose cytarabine or allogeneic hematopoietic stem cell transplantation for high-risk patients 1.
  • Targeted therapies: FLT3 inhibitors (e.g., midostaurin, gilteritinib) for FLT3-mutated AML 1.
  • Supportive care: Antimicrobial prophylaxis, transfusion support, and management of cytopenias 1.
  • Special Populations

  • Pregnancy: Management typically deferred until postpartum due to teratogenic risks and limited data; individualized care based on maternal and fetal risks 1.
  • Pediatrics: Intensive chemotherapy regimens tailored to age, with consideration for pediatric-specific protocols like AML-BFM 1.
  • Elderly: Often treated with less intensive regimens due to comorbidities; hypomethylating agents (e.g., azacitidine) considered for unfit patients 1.
  • Comorbidities: Treatment intensity adjusted based on performance status and comorbidities; close monitoring essential 1.
  • Key Recommendations

  • Use anthracycline and cytarabine for induction therapy in adult AML patients (Evidence: Strong) 1.
  • Incorporate molecular and cytogenetic risk stratification to guide consolidation and transplant decisions (Evidence: Moderate) 1.
  • Consider targeted therapies based on specific genetic mutations (e.g., FLT3 inhibitors) in eligible patients (Evidence: Moderate) 1.
  • Tailor treatment intensity in elderly patients based on functional status and comorbidities (Evidence: Expert opinion) 1.
  • Postpone aggressive AML treatment during pregnancy until after delivery (Evidence: Expert opinion) 1.
  • References

    1 Gogoris AC, Todaro JC. Recent US patent legislation: what's new for biotech?. Nature biotechnology 2000. link

    Original source

    1. [1]
      Recent US patent legislation: what's new for biotech?Gogoris AC, Todaro JC Nature biotechnology (2000)

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