Late Endocrine and Organ Toxicities After Treosulfan-Based Conditioning in Pediatric Hematopoietic Cell Transplantation: A Systematic Review.
Pediatr Blood CancerSep 10, 2026 (epub)
Takuto Takahashi, Miki Nishitani, Elizabeth Weiskopf et al.
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In 30 seconds
This systematic review evaluated late endocrine and organ toxicities following treosulfan-based conditioning in pediatric hematopoietic cell transplantation (HCT). Sixteen retrospective studies were analyzed, revealing that treosulfan is associated with significantly less gonadal toxicity compared to busulfan. Notably, no patients required sex hormone replacement therapy.
Key findings
- Treosulfan was associated with significantly less gonadal toxicity compared to busulfan in several cohorts.
- No patients receiving treosulfan required sex hormone replacement therapy.
- Non-gonadal endocrine or non-endocrine late effects showed no significant differences compared to busulfan.
Why it matters
Understanding the long-term effects of conditioning agents like treosulfan is crucial for optimizing patient care in pediatric HCT. This review highlights the potential for reduced gonadal toxicity, which may influence treatment decisions and follow-up strategies.