What is the treatment for acute leukemia?

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Treatment for Acute Leukemia

The standard treatment for acute leukemia consists of induction chemotherapy with an anthracycline and cytarabine, followed by consolidation therapy, with treatment approaches varying based on leukemia subtype and risk factors. 1

Types of Acute Leukemia and Treatment Approach

  • Acute leukemia is classified into two main types: Acute Myeloid Leukemia (AML) and Acute Lymphoblastic Leukemia (ALL), each requiring different treatment approaches 1
  • Acute Promyelocytic Leukemia (APL) is a distinct subtype of AML requiring specific treatment with all-trans retinoic acid (ATRA) 2

Treatment Phases for AML

Induction Therapy

  • For AML patients under 60 years, standard induction consists of the "3+7" regimen: an anthracycline (e.g., daunorubicin 45 mg/m²/day IV for 3 days) and cytarabine (100 mg/m²/day IV infusion for 7 days) 2, 3
  • For patients 60 years and older, a reduced dose of daunorubicin (30 mg/m²/day IV for 3 days) with cytarabine (100 mg/m²/day IV for 7 days) is recommended 3
  • Higher doses of daunorubicin (90 mg/m² x 3 days versus 45 mg/m² x 3 days) have shown improved complete remission rates and overall survival in patients under 60 years 2
  • Patients with excessive leukocytosis may require emergency leukapheresis before starting induction chemotherapy 2

Consolidation Therapy

  • Patients achieving complete remission should receive one to several cycles of post-remission therapy 2
  • Good-risk AML patients (including NPM-mutated AML without FLT3-ITD, CBF AML, and bi-allelic mutant CEBPα AML) should receive at least one cycle of intensive consolidation chemotherapy with intermediate or high-dose cytarabine 2
  • Patients with intermediate and poor-risk AML with an HLA-identical sibling are candidates for allogeneic stem cell transplantation in first remission 2
  • Patients without a family donor may qualify for allogeneic transplantation with an unrelated HLA-matched donor 2

Treatment for APL

  • APL requires a distinctive treatment approach with ATRA plus an anthracycline given simultaneously during induction 2
  • Non-high-risk APL patients (WBC count <10 x 10⁹/l) should be treated with arsenic trioxide (ATO) and ATRA 2
  • Four 8-week consolidation cycles with ATO/ATRA are recommended for non-high-risk APL patients 2
  • If ATO is not available, the classical combination of ATRA and anthracycline-based chemotherapy is acceptable, but requires 2-year maintenance therapy 2

Treatment of Relapsed or Refractory Disease

  • Patients in second or subsequent remission may qualify for allogeneic transplantation with an unrelated HLA-matched donor 2
  • In relapsed APL, arsenic trioxide can induce remission even in patients who have become refractory to ATRA 2

Response Evaluation

  • Response to induction is monitored through clinical examination, serial peripheral blood counts, and bone marrow aspirates 2
  • During induction-induced aplasia, a bone marrow aspirate should be obtained to monitor for early marrow response 2
  • Complete remission requires normal cellularity of the bone marrow, blast levels <5% in bone marrow smears, and morphologically normal hematopoiesis 2

Important Considerations

  • Treatment should be conducted at experienced leukemia centers with multidisciplinary expertise 2
  • Patients with poor performance status, significant comorbidities, and advanced age may not tolerate standard induction therapy and require alternative approaches 2
  • Untreated acute leukemia is uniformly fatal with a median survival of less than 3 months, making prompt treatment essential 4
  • Recent data suggest that a short delay in treatment start (to complete diagnostic testing) does not negatively impact survival outcomes 5
  • Supportive care including transfusion support, antimicrobial prophylaxis, and management of complications is crucial throughout treatment 1

Common Pitfalls and Caveats

  • Failure to identify APL can be fatal as these patients require immediate ATRA treatment upon suspicion of diagnosis 2
  • Patients with APL are at high risk for developing coagulopathy and require careful monitoring of coagulation status 2
  • Tumor lysis syndrome can occur in patients with high blast counts (>50,000/mcL) and requires preventive measures 2
  • Dose adjustments are necessary for patients with hepatic or renal impairment (e.g., reduce daunorubicin dose by 25% for serum bilirubin 2-3 mg% and by 50% for bilirubin >3 mg%) 3
  • Anthracyclines can cause cardiotoxicity, which may be more frequent in elderly patients and children, requiring cardiac risk assessment at diagnosis 2, 3

References

Guideline

Acute Leukemia Treatment Guidelines

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

Adult acute leukemia.

Current problems in cancer, 1997

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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