Erschienen in:
03.11.2020 | Original Article
Outcome of Non-hematological Autoimmunity After Hematopoietic Cell Transplantation in Children with Primary Immunodeficiency
verfasst von:
Su Han Lum, Reem Elfeky, Federica R. Achini, Adriana Margarit-Soler, Bianca Cinicola, Inigo Perez-Heras, Zohreh Nademi, Terry Flood, Tim Cheetham, Austen Worth, Waseem Qasim, Rakesh Amin, Kanchan Rao, Robert Chiesa, Robbert G. M. Bredius, Persis Amrolia, Mario Abinun, Sophie Hambleton, Paul Veys, Andrew R. Gennery, Arjan Lankester, Mary Slatter
Erschienen in:
Journal of Clinical Immunology
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Ausgabe 1/2021
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Abstract
Purpose
Knowledge of post-hematopoietic cell transplantation (HCT) non-hematological autoimmune disease (AD) is far from satisfactory.
Method
This multicenter retrospective study focuses on incidence, risk factors, and outcomes of post-HCT AD in 596 children with primary immunodeficiency (PID) who were transplanted from 2009 to 2018.
Results
The indications of HCT were severe combined immunodeficiency (SCID, n = 158, 27%) and non-SCID PID (n = 438, 73%). The median age at HCT was 2.3 years (range, 0.04 to 18.3 years). The 5-year overall survival for the entire cohort was 79% (95% cumulative incidence (CIN), 74–83%). The median follow-up of surviving patients was 4.3 years (0.08 to 14.7 years). The CIN of post-HCT AD was 3% (2–5%) at 1 year post-HCT, 7% (5–11%) at 5 years post-HCT, and 11% (7–17%) at 8 years post-HCT. The median onset of post-HCT AD was 2.2 years (0.12 to 9.6 years). Autoimmune thyroid disorder (n = 19, 62%) was the most common post-HCT AD, followed by neuromuscular disorders (n = 7, 22%) and rheumatological manifestations (n = 5, 16%). All patients but one required treatment for post-HCT AD. After multivariate analysis, age at transplant (p = 0.01) and T cell–depleted graft (p < 0.001) were significant predictors of post-HCT AD. None of the T cell–depleted graft recipients developed post-HCT AD. Patients with a lower CD3+ count at 6 months post-HCT had a significant higher incidence of post-HCT AD compared to disease controls. Graft-versus-host disease, viral infection, and donor chimerism had no association with post-HCT AD.
Conclusion
Post-HCT AD occurred in 11% at 8 years post-HCT and its occurrence was associated with older age at HCT and unmanipulated graft.