Non-TBI hematopoietic stem cell transplantation in pediatric AML patients: a single-center experience

Area of Science:

• Pediatric Hematology
• Oncology
• Stem Cell Transplantation

Background:

• Hematopoietic stem cell transplantation (HSCT) is a key treatment for pediatric acute myeloid leukemia (AML).
• Minimizing HSCT toxicity without compromising efficacy in children is a critical research area.
• This study reports long-term outcomes of HSCT in pediatric AML using a non-total body irradiation conditioning regimen.

Purpose of the Study:

• To evaluate the long-term efficacy and safety of HSCT in pediatric AML patients.
• To assess the impact of conditioning regimens on outcomes.
• To identify prognostic factors influencing survival and relapse in pediatric AML post-HSCT.

Main Methods:

• A cohort of 133 pediatric AML patients (age < 15 years) underwent autologous (auto-) or allogeneic (allo-) HSCT.
• Conditioning regimens included oral busulfan with etoposide (auto-HSCT) or cyclophosphamide (allo-HSCT).
• Long-term outcomes including overall survival (OS), leukemia-free survival (LFS), relapse, and transplantation-related mortality were analyzed.

Main Results:

• Three-year OS and LFS were 67.6% and 62.2%, respectively, with a relapse rate of 27.3% and transplantation-related mortality of 10.1%.
• No significant differences in outcomes were observed between allo-HSCT and auto-HSCT groups.
• Male sex was linked to improved OS and LFS, while age ≤3 years correlated with higher relapse rates and worse OS/LFS.

Conclusions:

• The study demonstrates the feasibility and long-term effectiveness of non-TBI HSCT for pediatric AML.
• The role of allogeneic HSCT in pediatric AML patients in first complete remission requires further investigation.
• Randomized trials are recommended to determine the optimal postremission therapy for pediatric AML.