Effect of haplo-allogeneic hematopoietic stem cell transplantation timing on patients with severe aplastic anemia without histocompatible matched sibling donor

This is the authors' abstract. We don't add key points for this paper.

Abstract To evaluate the impact of transplantation timing, specifically frontline versus salvage haplo-HSCT, on the outcomes of patients with SAA. We conducted this retrospective study of 82 SAA patients who underwent haplo-HSCT following the "Beijing protocol". Patients were categorized into the frontline HSCT group (n = 40, 48.8%) and the salvage HSCT group (n = 42, 51.2%) based on transplantation timing. All 82 patients received grafts from related haploidentical donors. Multivariate analysis identified salvage HSCT (HR: 5.344, 95% CI: 1.904–14.995), ferritin levels > 1000 (HR: 5.588, 95% CI: 1.696–18.414), and CMV infection (HR: 11.909, 95% CI: 2.335–60.725) as independent risk factors for graft failure. The overall survival rate was significantly higher in the front HSCT group (90%, 36/40) compared to the salvage HSCT group (71.4%, 30/42) with mortality rates of 10.0% (4/40) and 28.6% (12/42), respectively (p = 0.029). The expected 5-year OS was significantly higher in the frontline HSCT group compared to the salvage group. Salvage HSCT, ECOG score ≥ 1, and ferritin levels > 1000 were identified as independent risk factors for prognosis. For young patients with primary SAA who lack a MSD, frontline haplo-HSCT demonstrates superior efficacy and safety, making it an effective treatment option deserving further clinical promotion.