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

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dc.contributor.authorLum, Su Han
dc.contributor.authorAlbert, Michael H.
dc.contributor.authorGilbert, Patrick
dc.contributor.authorSirait, Tiarlan
dc.contributor.authorAlgeri, Mattia
dc.contributor.authorMuratori, Rafaella
dc.contributor.authorFournier, Benjamin
dc.contributor.authorLaberko, Alexandra
dc.contributor.authorKarakükcü, Musa
dc.contributor.authorÜnal, Ekrem
dc.contributor.authorAyas, Mouhab F.
dc.contributor.authorYadav, Satya Prakash
dc.contributor.authorFışgın, Tunç
dc.contributor.authorElfeky, Reem
dc.contributor.authorFernandes, Juliana Folloni
dc.contributor.authorFaraci, Maura
dc.contributor.authorCole, Theresa
dc.contributor.authorSchulz, Ansgar S.
dc.contributor.authorMeisel, Roland
dc.contributor.authorZecca, Marco
dc.contributor.authorIfversen, Marienne
dc.contributor.authorBiffi, Alessandra
dc.contributor.authorDiana, Jean-Sebastien
dc.contributor.authorVallee, Tanja C.
dc.contributor.authorGiardino, Stefano
dc.contributor.authorErsoy, Gizem Zengin
dc.contributor.authorMoshous, Despina
dc.contributor.authorGennery, Andrew R.
dc.contributor.authorBalashov, Dmitry
dc.contributor.authorBonfim Carmem M. S.
dc.contributor.authorLocatelli, Franco
dc.contributor.authorLankester, Arjan C.
dc.contributor.authorNeven, Benedicte
dc.contributor.authorSlatter, Mary A.
dc.date.accessioned2024-05-06T12:49:33Z
dc.date.available2024-05-06T12:49:33Z
dc.date.issued2024en_US
dc.departmentFakülteler, Tıp Fakültesien_US
dc.description.abstractHLA-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.en_US
dc.identifier.citationLum, 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.2024024038en_US
dc.identifier.endpage580en_US
dc.identifier.issn0006-4971
dc.identifier.issn1528-0020
dc.identifier.issue5en_US
dc.identifier.pmid38669631
dc.identifier.scopus2-s2.0-85196417938
dc.identifier.scopusqualityQ1
dc.identifier.startpage565en_US
dc.identifier.urihttps://hdl.handle.net/20.500.12939/4681
dc.identifier.volume144en_US
dc.identifier.wosWOS:001293213800001
dc.identifier.wosqualityQ1
dc.indekslendigikaynakWeb of Science
dc.indekslendigikaynakScopus
dc.indekslendigikaynakPubMed
dc.institutionauthorFışgın, Tunç
dc.institutionauthorErsoy, Gizem Zengin
dc.language.isoen
dc.relation.ispartofBlood
dc.relation.isversionof10.1182/blood.2024024038en_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.subjectHLAen_US
dc.subjectInborn errors of immunity (IEI)en_US
dc.titleOutcomes of HLA-mismatched HSCT with TCR?ß/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity
dc.typeArticle

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