HCT for Wiskott-Aldrich Syndrome: An EBMT Inborn Errors Working Party Analysis

Albert MH, Slatter MA, Gennery AR, et al. Hematopoietic Stem Cell Transplantation for Wiskott-Aldrich Syndrome: An EBMT Inborn Errors Working Party Analysis. Blood. 2022; (doi: 10.1182/blood.2021014687).

Allogeneic hematopoietic stem cell transplantation (HSCT) led to nearly 90% survival among patients with Wiskott-Aldrich syndrome (WAS) regardless of donor type or stem cell source, when administered with conditioning regimens recommended by the EBMT Inborn Errors Working Party. Investigators evaluated 197 patients treated at European Society for Blood and Marrow Transplantation (EBMT) centers between 2006 and 2017, including 103 who received busulfan and 94 who received treosulfan plus fludarabine ± thiotepa. After an average follow-up of 44.9 months post-HSCT, 176 patients were still alive. The rates of three-year overall survival and chronic graft versus host disease (GVHD)-free survival (CRFS) were 88.7% and 81.7%, respectively. Neither conditioning regimen nor donor type significantly affected overall survival or CRFS. Overall survival was significantly better for patients younger than 5 years at HSCT. The overall cumulative incidence of grade III-IV acute GVHD was 6.6%, while the incidence of extensive/moderate/severe chronic GVHD was 2.1%. Graft failure, mixed donor chimerism, and secondary procedures were more common in patients who received treosulfan-based conditioning.

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