Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

作者：Michael H. Albert, Tiarlan Sirait, Dirk‐Jan Eikema, Katerina Bakunina, Claudia Wehr, Felipe Suárez, María Laura Fox, Nizar Mahlaoui, Andrew Richard Gennery, Arjan C. Lankester, Rita Beier, Maria Ester Bernardo, Venetia Bigley, Caroline A. Lindemans, Siobhan Oisin Burns, Ben Carpenter, Jarosław Dybko, Tayfun Güngör, Fabian H. Hauck, Su Han Lum, Dmitry Balashov, Roland Meisel, Despina Moshous, Ansgar Schulz, Carsten Speckmann, Mary Anne Slatter, Brigitte Strahm, Duygu Uçkan, Isabelle Meyts, Tanja C. Vallée, Robert Wynn, Bénédicte Neven, Emma C. Morris, Alessandro Aiuti, Alexei Maschan, Mahmoud Deeb Aljurf, Tobias Gedde‐Dahl, Günhan Gürman, Victoria Bordon, Gergely Kriván, Franco Locatelli, Fulvio Porta, David Valcárcel, Yves P. Beguin, Maura Faraci, Nicolaus Martin Kröger, Aleksandr Kulagin, Peter John Shaw, Joan Hendrik Veelken, Cristina Díaz de Heredia, Franca Fagioli, Matthias Felber, Bernd Gruhn, Wolfgang Holter, Claudia Rössig, Petr Sedláček, Jane Apperley, Mouhab Fakhreddine Ayas, Ivana Boďová, Goda Choi, Jan J. Cornelissen, Anne Sîrvent, Anjum Bashir Khan, Alphan Küpesiz, Stig Lenhoff, Hakan Özdoğu, Nicolas von der Weid, Montserrat Rovira, Rik Schots, Donald Cuong Vinh · 发表于：Blood · 年份：2022 · DOI：10.1182/blood.2022015506 · 被引用次数：57 · 研究领域：Immunodeficiency and Autoimmune Disorders、Hematopoietic Stem Cell Transplantation、Blood disorders and treatments

Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard curative therapy for infants and children with many inborn errors of immunity (IEI), but adolescents and adults with IEI are rarely referred for transplant. Lack of published HSCT outcome data outside small, single-center studies and perceived high risk of transplant-related mortality have delayed the adoption of HSCT for IEI patients presenting or developing significant organ damage later in life. This large retrospective, multicenter HSCT outcome study reports on 329 IEI patients (age range, 15-62.5 years at HSCT). Patients underwent first HSCT between 2000 and 2019. Primary endpoints were overall survival (OS) and event-free survival (EFS). We also evaluated the influence of IEI-subgroup and IEI-specific risk factors at HSCT, including infections, bronchiectasis, colitis, malignancy, inflammatory lung disease, splenectomy, hepatic dysfunction, and systemic immunosuppression. At a median follow-up of 44.3 months, the estimated OS at 1 and 5 years post-HSCT for all patients was 78% and 71%, and EFS was 65% and 62%, respectively, with low rates of severe acute (8%) or extensive chronic (7%) graft-versus-host disease. On univariate analysis, OS and EFS were inferior in patients with primary antibody deficiency, bronchiectasis, prior splenectomy, hepatic comorbidity, and higher hematopoietic cell transplant comorbidity index scores. On multivariable analysis, EFS was inferior in those with a higher n...