Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

免疫学 医学 环磷酰胺 人类白细胞抗原 CD19 Blinatumoab公司 HLA-B抗原 内科学 免疫系统 抗原 化疗
作者
Su Han Lum,Michael H. Albert,Patrick Gilbert,Tiarlan Sirait,Mattia Algeri,R. Muratori,Benjamin Fournier,Alexandra Laberko,Musa Karakükçü,Ekrem Ünal,Mouhab Ayas,Satya Prakash Yadav,Tunç Fışgın,Reem Elfeky,Juliana Folloni Fernandes,Maura Faraci,Theresa Cole,Ansgar Schulz,Roland Meisel,Marco Zecca,Marianne Ifversen,Alessandra Biffi,Jean‐Sébastien Diana,Tanja C. Vallée,Stefano Giardino,Gizem Zengin Ersoy,Despina Moshous,Andrew R. Gennery,Dmitry Balashov,Carmem Bonfim,Franco Locatelli,Arjan C. Lankester,Bénédicte Neven,Mary Slatter
出处
期刊:Blood [Elsevier BV]
被引量:2
标识
DOI:10.1182/blood.2024024038
摘要

HLA-mismatched transplants with either in vitro depletion of CD3+TCRαβ/CD19 (TCRαβ) cells or in vivo T-cell depletion using post-transplant cyclophosphamide (PTCY) have been increasingly used for patients with inborn errors of immunity (IEI). We performed a retrospective multicenter study via the EBMT registry on 306 children with IEI undergoing first transplant between 2010-2019 from an HLA-mismatched donor using TCRαβ (n=167) or PTCY (n=139). Median age at HSCT was 1.2 years (range, 0.03-19.6 years). The 3-year overall survival (OS) was 78% (95% confidence interval (CI), 71-84%) after TCRαβ and 66% (57-74%) after PTCY (p=0.013). Pre-HSCT morbidity score (hazard ratio (HR) 2.27, 1.07-4.80, p=0.032) and non-Busulfan/Treosulfan conditioning (HR 3.12, 1.98-4.92, p<0.001) were the only independent predictors of unfavorable OS. The 3-year event-free survival (EFS) was 58% (50-66%) after TCRαβ and 57% (48-66%) after PTCY (p=0.804). Cumulative incidence of severe acute GvHD was higher after PTCY (15%, 9-21%) than TCRαβ (6%, 2-9%, p=0.007), with no difference in chronic GvHD (PTCY, 11%, 6-17%; TCRαβ, 7%, 3-11%, p=0.173). The 3-year GvHD-free EFS was 53% (44-61%) after TCRαβ and 41% (32-50%) after PTCY (p=0.080). PTCY had significantly higher rates of veno-occlusive disease (14.4% versus TCRαβ 4.9%, p=0.009), acute kidney injury (12.7% versus 4.6%, p=0.032) and pulmonary complications (38.2% versus 24.1%, p=0.017). Adenoviraemia (18.3% versus PTCY 8.0%, p=0.015), primary graft failure (10%, versus 5%, p=0.048), and second HSCT (17.4% versus 7.9%, p=0.023) were significantly higher in TCRαβ. In conclusion, this study demonstrates that both approaches are suitable options in IEI patients, although characterized by different advantages and outcomes.

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