Outcome of poor response paediatric AML using early SCT

Area of Science:

• Pediatric Hematology Oncology
• Hematopoietic Stem Cell Transplantation
• Acute Myeloid Leukemia Research

Background:

• Acute myeloid leukemia (AML) in children with poor treatment response is associated with unfavorable outcomes.
• The efficacy of allogeneic stem cell transplantation (SCT) for pediatric AML with poor response remains uncertain.
• This study evaluates survival in pediatric AML patients with poor response undergoing SCT.

Purpose of the Study:

• To assess the survival outcomes of children with poor response acute myeloid leukemia (AML) treated with allogeneic stem cell transplantation (SCT).
• To determine the prognostic impact of intensive induction chemotherapy and SCT in this high-risk pediatric AML cohort.

Main Methods:

• Patients were treated under the NOPHO-AML 2004 protocol, receiving induction chemotherapy (AIET and AM).
• Poor response was defined by blast percentage (>15% on day 15 post-AIET or >5% post-AM).
• Eligible poor responders underwent intensive induction followed by SCT using various donor types.

Main Results:

• Out of 267 patients, 31 (12%) exhibited poor response to induction therapy.
• Twenty-five patients proceeded to SCT, utilizing matched unrelated, matched sibling, cord blood, or haploidentical donors.
• The 3-year survival probability for these 31 poor responders was 70% (95% CI 59-77%) with a median follow-up of 2.6 years.

Conclusions:

• Pediatric AML patients with poor response to induction therapy demonstrated a favorable prognosis when treated with intensive induction followed by SCT.
• Allogeneic SCT is a viable and effective treatment strategy for improving survival in this challenging pediatric AML subgroup.