Excellent outcomes arising from haploidentical hematopoietic cell transplantation in 64 children with chronic granulomatous disease: a retrospective study from a single-center

Abstract Background: Allogeneic hematopoietic stem cell transplantation is a radical treatment for certain refractory patients with CGD. Haplo-HSCT have been used as treatments for CGD, however, only a few cases have been reported from a single center. There have been no previous publications relating to the combination of Haplo-HSCT combined with tp-UCB for the treatment of CGD. Here, we present the outcomes of 64 pediatric patients with CGD who underwent Haplo-HSCT combined with tp-UCB in our center.Methods: From May 2014 to November 2020, 64 consecutive children with CGD underwent allo-HSCT from Haplo-HSCT combined with tp-UCB in our center. We retrospectively collected their clinical data and analyzed the TRM, OS, and EFS survival.Results: The median days of neutrophil and platelet engraftment were 13 days and 22 days respectively. The cumulative incidence of grade III-IV aGVHD was 15.6% and that of cGVHD was 7.8%. In total, 60 patients achieved EFS during a median follow-up period of 885 days. The cumulative incidence of TRM, OS, and EFS were 6.9%, 93.1%, and 84.6%, respectively. Conclusion: Collectively, our data indicate that Haplo-HSCT combined with tp-UCB, can both provide good therapeutic effects for children with CGD.