Superior long-term survival with acceptable safety of ATG/G-CSF–based haplo-HSCT with intensified BU+MEL/TT conditioning in CR Pediatric Non-DS–AMKL - Summary - MDSpire
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Superior long-term survival with acceptable safety of ATG/G-CSF–based haplo-HSCT with intensified BU+MEL/TT conditioning in CR Pediatric Non-DS–AMKL
To compare the long-term efficacy and safety of standard BU/CY conditioning versus intensified BU+MEL/TT in pediatric non–DS–AMKL patients undergoing haplo-HSCT, highlighting the clinical significance of this comparison.
Key Findings:
The BU+MEL/TT group had significantly higher graft CD34+ and CD3+ cell doses, which may contribute to improved outcomes.
5-year OS was 75.2% for BU+MEL/TT vs 50.0% for BU (P = 0.045), indicating a significant survival advantage.
5-year LFS was 76.3% for BU+MEL/TT vs 50.0% for BU (P = 0.039), suggesting better disease control.
No significant differences in TRM, engraftment, GVHD, or viral reactivations between groups, indicating safety.
Pre-transplant NR/PR status was the strongest adverse factor affecting outcomes, underscoring the need for effective pre-transplant management.
Interpretation:
Intensified conditioning with BU+MEL/TT improves OS and LFS in pediatric non–DS–AMKL patients transplanted in CR without increasing early NRM, highlighting its potential as a preferred treatment approach.
Limitations:
Retrospective design may introduce bias, affecting the reliability of the findings.
Limited sample size may affect generalizability, necessitating further studies to confirm results.
Conclusion:
Intensified conditioning is beneficial for improving survival outcomes in pediatric non–DS–AMKL, emphasizing the critical need for achieving remission prior to transplantation to maximize treatment efficacy.
