Development of a curative treatment within the AML-BFM studies

Area of Science:

• Pediatric Oncology
• Hematology
• Clinical Trials

Background:

• The first multicenter treatment study for childhood acute myeloid leukemia (AML) in Germany began in 1978.
• Therapy plans were initially modeled after acute lymphoblastic leukemia (ALL) protocols.

Purpose of the Study:

• To assess the efficacy of combined cytarabine and anthracyclines in induction and consolidation for childhood AML.
• To evaluate the role of preventive cranial irradiation in CNS relapse prevention.
• To optimize treatment schedules and improve long-term survival rates in pediatric AML.

Main Methods:

• Combined induction and consolidation therapy with cytarabine and anthracyclines.
• Preventive cranial irradiation followed by maintenance therapy.
• Sequential optimization of treatment protocols across multiple AML-BFM studies.

Main Results:

• Initial remission rate was 80%, with a 5-year survival rate increasing from <10% to 40%.
• Subsequent trials improved 5-year survival to 70%, and 90% in core-binding factor leukemias.
• Cranial irradiation showed a reduced risk of CNS and/or bone marrow relapses in study -87.

Conclusions:

• The AML-BFM study sequence demonstrated a significant stepwise increase in long-term survival for childhood AML.
• Optimized treatment schedules, improved supportive care, and staff experience were crucial for survival gains.
• Cranial irradiation at 12 Gy was found to be as effective as 18 Gy in preventing relapses.