Post-transplant cyclophosphamide as graft-versus-host disease prophylaxis for matched related donor hematopoietic stem cell transplantation in acute leukemia: a systematic review and meta-analysis.

In 30 seconds

This systematic review and meta-analysis assessed post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis in patients with acute leukemia undergoing matched related donor hematopoietic stem cell transplantation. Across 21 studies involving 10,860 patients, PTCy-based regimens were associated with lower odds of grade II-IV acute GVHD (OR 0.54, 95% CI 0.35-0.86) and chronic GVHD (OR 0.47, 95% CI 0.37-0.60) than calcineurin inhibitor-based regimens. GVHD-free/relapse-free survival was also higher, while disease-free survival was similar. Trends toward lower non-relapse mortality and higher overall survival were not statistically significant.

Key findings

• PTCy was associated with lower grade II-IV acute GVHD than calcineurin inhibitor-based prophylaxis (OR 0.54, 95% CI 0.35-0.86).
• Chronic GVHD odds were lower with PTCy (OR 0.47, 95% CI 0.37-0.60).
• GVHD-free/relapse-free survival was higher with PTCy (OR 2.29, 95% CI 1.42-3.67), while disease-free survival was similar (OR 1.05, 0.75, 1.49).
• Reported adverse events included delayed engraftment, cytomegalovirus reactivation (29%), and hemorrhagic cystitis (20%).

Why it matters

GVHD remains an important obstacle to successful transplantation. These pooled findings suggest PTCy may reduce acute and chronic GVHD in matched related donor transplantation without a detected difference in disease-free survival. The results help characterize a prophylaxis option in this setting, while the reported adverse events and need for randomized trials underscore that benefits and risks require further evaluation.

What to keep in mind

The analysis pooled 21 studies, and the abstract does not specify their designs or establish randomized comparisons. The authors state that further randomized trials are warranted.