Second allogeneic hematopoietic stem cell transplantation in patients with inborn errors of immunity

Abstract

Graft failure (GF) remains a serious issue of hematopoietic stem cell transplantation (HSCT) in inborn errors of immunity (IEI). Second HSCT is the only salvage therapy for GF. There are no uniform strategies for the second HSCTs and limited data are available on the second HSCT outcomes. 48 patients with various IEI received second allogeneic HSCT from 2013 to 2020. Different conditioning regimens were used, divided into two main groups: containing myeloablative doses of busulfan/treosulfan (n = 19) and lymphoid irradiation 2–6 Gy (n = 22). Irradiation-containing conditioning was predominantly used in suspected immune-mediated rejection of the first graft. Matched unrelated donor was used in 28 patients, mismatched related in 18, and matched related in 1. 35 patients received TCRαβ/CD19 graft depletion. The median follow-up time was 2.4 years post-HSCT. One patient died at conditioning. The OS was 0.63 (95% CI: 0.41–0.85) after busulfan/treosulfan and 0.68 (95% CI: 0.48–0.88) after irradiation-based conditioning, p = 0.66. Active infection at HSCT significantly influenced OS: 0.43 (95% CI: 0.17–0.69) versus 0.73 (95% CI: 0.58–0.88) without infection, p = 0.004. The cumulative incidence of GF was 0.15 (95% CI: 0.08–0.29). To conclude, an individualized approach is required for the second HSCT in IEI. Low-dose lymphoid irradiation in suspected immune-mediated GF may be a feasible option.

Allogeneic Hematopoietic Stem Cell Transplantation Activity in Inborn Errors of Immunity in Russian Federation

“…Of note, the number of second HSCT procedures in Russia remains consistent (about 13% of all HSCTs). This corresponds to a rejection rate in IEI patients reported for IEI by one of the participating centers [ 34 ].…”

EBMT-2023: overview in brief

“…Incidence of chronic GvHD was 9%. The 5-year OS was 73% [85]. center Study of JAK1/2 inhibitor Ruxolitinib added to corticosteroids in pediatric patients with treatment-Naïve or Steroid-Refractory acute GvHD.…”