Outcomes of pediatric patients with therapy-related myeloid neoplasms

Area of Science:

• Hematology
• Pediatric Oncology
• Transplantation Immunology

Background:

• Long-term outcomes for pediatric therapy-related myeloid neoplasms (tMNs) following allogeneic hematopoietic cell transplantation (HCT) remain challenging.
• Limited multicenter data exists on prognostic factors influencing survival in this specific pediatric population.

Purpose of the Study:

• To identify prognostic factors for long-term survival in pediatric patients with tMNs undergoing HCT.
• To analyze the largest cohort to date for predictive factors of survival after HCT for tMNs.

Main Methods:

• Multivariate analysis of 401 pediatric patients who underwent HCT for tMNs.
• Comparison of outcomes based on conditioning intensity (myeloablative vs. reduced-intensity).
• Assessment of factors including conditioning regimen, graft-versus-host disease (GVHD), and tMN characteristics.

Main Results:

• No significant difference in overall survival (OS) or event-free survival (EFS) based on conditioning intensity.
• Higher treatment-related mortality in myeloablative conditioning (MAC) regimens, particularly from acute GVHD and end-organ failure.
• Total body irradiation (TBI) and grade III/IV acute GVHD were associated with worse OS.
• Therapy-related myelodysplastic syndrome and complex karyotype predicted worse EFS.

Conclusions:

• Reduced-toxicity conditioning regimens, distinct from reduced-intensity, may offer a strategy to reduce relapse while mitigating mortality associated with TBI-based HCT conditioning.
• Further research into optimized conditioning is crucial for improving pediatric tMN HCT outcomes.