Outcomes of HLA-mismatched HSCT with TCR?ß/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity
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Tarih
2024
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
Erişim Hakkı
info:eu-repo/semantics/closedAccess
Özet
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.
Açıklama
Anahtar Kelimeler
HLA, Inborn errors of immunity (IEI)
Kaynak
Blood
WoS Q Değeri
Q1
Scopus Q Değeri
Q1
Cilt
144
Sayı
5
Künye
Lum, S. H., Albert, M. H., Gilbert, P., Sirait, T., Algeri, M., Muratori, R., Fournier, B., Laberko, A., Karakükcü, M., Ünal, E., Ayas, M. F., Yadav, S. P., Fışgın, T., Elfeky, R., Fernandes, J. F., Faraci, M., Cole, T., Schulz, A. S., Meisel, R., Zecca, M., Ifversen, M., Biffi, A., Diana, J.-S., Vallee, T. C., Giardino, S., Ersoy, G. Z., Moshous, D., Gennery, A. R., Balashov, D., Bonfim, C. M. S., Locatelli, F., Lankester, A. C., Neven, B., Slatter, M. A. (2024). Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity. Blood, 144(5), 565-580. 10.1182/blood.2024024038
