Reduced-Dose Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis in Matched Donor Allogeneic Stem Cell Transplantation: A Systematic Review of Efficacy and Toxicity.
Areez Shafqat, Malaz El-Sibai, Abdulrahman Nasiri et al.
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In 30 seconds
This systematic review assessed reduced-dose post-transplant cyclophosphamide (PTCy) for graft-versus-host disease prophylaxis in matched-related or matched-unrelated donor allogeneic stem cell transplantation. Eighteen studies evaluated doses from 40 mg/kg to 80 mg/kg. Overall, reduced-dose PTCy was associated with similar acute and chronic GVHD rates to controls, alongside lower reported cytomegalovirus reactivation and hemorrhagic cystitis and faster neutrophil and platelet engraftment. The evidence was limited by observational studies at high risk of bias; the single randomized trial combined 40 mg/kg PTCy with reduced-dose ATG.
Key findings
- Across the included studies, reduced-dose PTCy (40 mg/kg to 80 mg/kg) showed similar acute and chronic GVHD rates to controls.
- Recipients of reduced-dose PTCy had lower reported rates of cytomegalovirus reactivation and hemorrhagic cystitis.
- Reduced-dose PTCy was associated with accelerated neutrophil and platelet engraftment.
- Five of 8 non-randomized comparative studies had critical risk of bias; the single randomized trial had some concerns of bias.
Why it matters
PTCy dose reduction may offer a way to limit treatment-related toxicity while maintaining GVHD control in matched-donor transplantation. The reported toxicity and engraftment findings are clinically relevant, but the evidence does not establish comparative benefit reliably. Better-designed randomized studies could clarify which reduced dose, if any, balances GVHD prevention and toxicity.
What to keep in mind
Evidence was largely observational, with 5 of 8 non-randomized comparative studies at critical risk of bias. Only one randomized trial was included, and there was no consensus on an appropriate reduced dose.