Successful Haematopoietic Stem Cell Transplantation for LRBA Deficiency with Fludarabine, Treosulfan, and Thiotepa-Based Conditioning.

Autor: Shadur B; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel. bella.shadur@gmail.com.; Garvan Institute of Medical Research, Sydney, Australia. bella.shadur@gmail.com.; School of Clinical Medicine, Faculty of Medicine and Health, UNSW, Sydney, NSW, Australia. bella.shadur@gmail.com., NasserEddin A; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel., Zaidman I; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel., Schejter YD; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel., Even-Or E; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel., Berkun Y; Department of General Paediatrics, Hadassah University Medical Centre, Jerusalem, Israel., Meyts I; Inborn Errors of Immunity, Department of Microbiology, Immunology and Transplantation, KU Leuven, Leuven, Belgium.; Pediatric Immunodeficiency, Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium., Hmedat H; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel., Sulaiman A; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel., Tangye SG; Garvan Institute of Medical Research, Sydney, Australia.; School of Clinical Medicine, Faculty of Medicine and Health, UNSW, Sydney, NSW, Australia., Stepensky P; Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Medical Centre, Jerusalem, Israel.
Jazyk: angličtina
Zdroj: Journal of clinical immunology [J Clin Immunol] 2024 Sep 12; Vol. 45 (1), pp. 3. Date of Electronic Publication: 2024 Sep 12.
DOI: 10.1007/s10875-024-01770-1
Abstrakt: LRBA deficiency is an inborn error of immunity defined by autoimmunity, lymphoproliferation, recurrent infections, cytopenia, and inflammatory bowel disease. Despite recent advances in managing this disease with targeted biologic therapy, haematopoietic stem cell transplant (HSCT) remains the only cure. However, great variability exists between protocols used to transplant patients with LRBA deficiency. We describe a cohort of seven patients with LRBA deficiency who underwent HSCT using a myeloablative, reduced toxicity regime of fludarabine, treosulfan, and thiotepa at two transplantation centres from 2016 to 2019. Data were collected both retrospectively and prospectively, measuring time to engraftment, infectious complications, incidence of graft versus host disease, and post-transplantation chimerism. Six of seven patients survived transplantation, and four of six surviving patients achieving treatment-free survival. We thus recommend that HSCT with fludarabine, treosulfan, and thiotepa-based conditioning be considered in patients with LRBA deficiency.
(© 2024. The Author(s).)
Databáze: MEDLINE
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