Summary

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• This summary is based on the review of systematic reviews or meta-analyses. ^[1]
• Grafapex (treosulfan) is indicated for use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients 1 year of age and older with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).
• In pediatric patients undergoing hematopoietic stem cell transplantation (HSCT), treosulfan-based conditioning showed a marginally better survival rate compared to busulfan (OR: 1.57; 95% CI: 1.00, 2.44), though this finding was unstable in sensitivity analysis.
• No significant differences were observed between treosulfan and busulfan in the incidence of acute graft versus host disease (GVHD) (OR: 0.96; 95% CI: 0.57, 1.61), grade II to IV acute GVHD (OR: 1.19; 95% CI: 0.83, 1.72), chronic GVHD (OR: 1.18; 95% CI: 0.70, 2.00), or transplant-related mortality (OR: 0.70; 95% CI: 0.34, 1.42).
• The effectiveness outcomes were derived from studies exclusively involving pediatric patients undergoing HSCT, with no additional subgroups identified.
• No significant safety concerns or adverse effects were identified for treosulfan compared to busulfan in the reviewed pediatric population.
• The included studies focused exclusively on pediatric patients undergoing HSCT. No significant differences in effectiveness or safety outcomes were identified between treosulfan and busulfan in this population, and the marginal survival benefit observed with treosulfan (OR: 1.57; 95% CI: 1.00, 2.44) was not stable upon sensitivity analysis.