The impact of Treosulfan-based conditioning for inborn errors of immunity: Is dose monitoring crucial?

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dc.authoridZengin Ersoy, Gizem/0000-0002-8968-9849
dc.authorwosidBaşoğlu Öner, Özlem/JBI-7610-2023
dc.authorwosidBozkurt, Ceyhun/AAL-5043-2021
dc.contributor.authorErsoy, Gizem Zengin
dc.contributor.authorCipe, Funda
dc.contributor.authorFisgin, Tunc
dc.contributor.authorAksoy, Basak Adakli
dc.contributor.authorOner, Ozlem Basoglu
dc.contributor.authorHashemi, Nazli
dc.contributor.authorAydogdu, Selime
dc.date.accessioned2024-05-19T14:40:57Z
dc.date.available2024-05-19T14:40:57Z
dc.date.issued2023
dc.departmentİstinye Üniversitesien_US
dc.description.abstractIntroductionIn children with inborn errors of immunity (IEI) who will receive a hematopoietic stem cell transplant (HSCT) treosulfan-based conditioning is currently preferred. The aim of this study was to investigate early and late outcomes in pediatric IEI patients receiving pre-HSCT treosulfan and to examine the effect of treosulfan dose monitoring on outcomes. MethodsSeventy-three pediatric patients receiving this management between 2015 and 2022 were included. ResultsOverall survival rate was 80%, and event-free survival was 67.8%. A larger treosulfan dose AUC after first application increased the rate of early toxicity (p = .034) and slowed lymphocyte engraftment (r = .290; p = .030). Underlying disease, treosulfan AUC, donor type, stem cell type, number of immunosuppressive agents, the dose of anti-thymocyte globulin, and post-transplantation cyclophosphamide did not to increase risk of acute graft-versus-host disease. The risk of mixed chimerism (MC) in patients with autoimmune lymphoproliferative syndrome and leukocyte adhesion deficiency were higher than those with severe combined immunodeficiency (p = .021 and p = .014, respectively). The risk of MC was lower in those receiving peripheral blood stem cells (SC) compared with bone marrow derived SC (OR = .204, p = .022). ConclusionThe AUC of the treosulfan dose was not associated with poorer late outcomes. Treosulfan is an agent that can be used safely in the IEI patient group, level measurement appears essential to identify early toxicities. Prospective studies with more extended follow-up periods are needed.en_US
dc.identifier.doi10.1111/ctr.15083
dc.identifier.issn0902-0063
dc.identifier.issn1399-0012
dc.identifier.issue9en_US
dc.identifier.pmid37534623en_US
dc.identifier.scopus2-s2.0-85166653693en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.urihttps://doi.org10.1111/ctr.15083
dc.identifier.urihttps://hdl.handle.net/20.500.12713/5040
dc.identifier.volume37en_US
dc.identifier.wosWOS:001041668900001en_US
dc.identifier.wosqualityN/Aen_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.language.isoenen_US
dc.publisherWileyen_US
dc.relation.ispartofClinical Transplantationen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.snmz20240519_kaen_US
dc.subjectDose Monitoringen_US
dc.subjectInborn Errors Of Immunityen_US
dc.subjectStem Cell Transplantationen_US
dc.subjectTreosulfan Conditioningen_US
dc.titleThe impact of Treosulfan-based conditioning for inborn errors of immunity: Is dose monitoring crucial?en_US
dc.typeArticleen_US

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