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Hematology AdvisorLow-Dose Chemo Noninferior to Standard-Dose Chemo in Children with AML

Low-Dose Chemotherapy in Pediatric AML: A New Standard in Treatment Efficacy and Tolerance

In a pivotal phase 3 trial, a low-dose chemotherapy regimen has demonstrated noninferior outcomes compared to the standard-dose regimen in treating children and adolescents with acute myeloid leukemia (AML). This groundbreaking research, presented at the ASH Annual Meeting 2023, marks a significant stride in pediatric oncology, offering a regimen that not only matches the efficacy of traditional methods but also boasts improved tolerability. The findings are particularly noteworthy as they suggest a potential paradigm shift in AML treatment protocols, prioritizing patient well-being without compromising treatment effectiveness.

Key Points:

  • The phase 3 trial included 497 pediatric and adolescent patients with de novo AML, treated across 11 institutions in China.
  • Patients were randomly assigned to either a low-dose chemotherapy regimen (246 patients) or a standard-dose chemotherapy regimen (251 patients), with similar baseline characteristics in both groups.
  • The low-dose regimen involved subcutaneous cytarabine and intravenous mitoxantrone or idarubicin, plus granulocyte colony-stimulating factor, while the standard-dose included intravenous cytarabine, daunorubicin, and etoposide.
  • Post-first induction, CR/CRi rates were comparable between low-dose (72.8%) and standard-dose (70.3%) arms; minimal residual disease (MRD) levels below 1% were also similar.
  • Post-second induction, CR/CRi rates remained comparable; MRD levels below 0.01% were 85.9% for low-dose and 84.4% for standard-dose.
  • Three-year event-free survival rates were nearly identical (61.9% low-dose, 62.0% standard-dose), as were the overall survival rates (81.0% low-dose, 83.2% standard-dose).
  • Notably, the low-dose regimen resulted in significantly fewer non-hematologic toxicities such as febrile neutropenia, colitis, and gastrointestinal bleeding.
  • Time to neutrophil and platelet recovery was significantly shorter in the low-dose arm during both induction courses.

According to the American Cancer Society, the 5-year survival rate for children with AML has increased significantly over the past few decades, from under 30% in the 1970s to about 70% recently.

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