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Abstract Post-transplant cyclophosphamide, sirolimus and mycophenolate mofetil (PTCy/siro/MMF) constitutes an innovative and well-tolerated acute graft-versus-host disease (aGVHD) prophylaxis after allogeneic stem cell transplantation (allo-HSCT), but risk factors for aGVHD incidence and therapy failure in this setting are scarce. The study prospectively registered all consecutive adult patients with hematologic malignancies who received an allo-HSCT using PTCy/siro/MMF prophylaxis at our institution between 2017 and 2023. A total of 439 patients were included, of whom 40% were transplanted from matched sibiling donors, 34% from matched unrelated donors (MUD) and 26% from haploidentical donors. The 100-day cumulative incidence of grade II-IV and grade III-IV aGVHD was 22% (95% confidence interval CI 18–26%) and 11% (95% CI 8–14%), respectively. The use of MUD was associated with decreased risk of severe aGVHD while a diagnosis of myelodysplastic or myeloproliferative neoplasms (MDS/MPN) was deleterious. Among 92 patients receiving first-line systemic corticosteroids, 51% achieved a sustained complete response, while 22% and 23% developed steroid-dependent (SD-aGVHD) and steroid-refractory aGVHD (SR-aGVHD), respectively. Only grade III-IV aGVHD was a predictor of steroid failure. SR-aGVHD was associated with worse salvage treatment response and overall survival compared to SD-aGVHD. The 1-year cumulative incidence of aGVHD-related mortality was 5.7% (95% CI, 3.7–8.2). Risk factors for aGVHD-related mortality included haploidentical donors, older donors, diagnosis of MDS/MPN, and grade IV aGVHD. This study confirms a low incidence aGVHD with PTCy/siro/MMF prophylaxis. SR-aGVHD showed poorer response to salvage therapies and worse survival, while haploidentical donors and older donor age were negative predictors for aGVHD-related deaths.
Gómez‐Seguí et al. (Fri,) studied this question.