Busulfan–fudarabine- or treosulfan–fudarabine-based myeloablative conditioning for children with thalassemia major

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dc.contributor.authorLüftinger, Roswitha
dc.contributor.authorZubarovskaya, Natalia
dc.contributor.authorGalimard, Jacques‑Emmanuel
dc.contributor.authorCseh, Annamaria
dc.contributor.authorSalzer, Elisabeth
dc.contributor.authorLocatelli, Franco
dc.contributor.authorAlgeri, Mattia
dc.contributor.authorYeşilipek, Akif
dc.contributor.authorFuente, Josu de la
dc.contributor.authorIsgro, Antonella
dc.contributor.authorAlseraihy, Amal
dc.contributor.authorAngelucci, Emanuele
dc.contributor.authorSmiers, Frans J.
dc.contributor.authorNasa, Giorgia La La
dc.contributor.authorZecca, Marco
dc.contributor.authorFışgın, Tunç
dc.contributor.authorÜnal, Emel
dc.contributor.authorKleinschmidt, Katharina
dc.contributor.authorPeters, Christina
dc.contributor.authorLankester, Arjan
dc.contributor.authorÇorbacıoğlu, Selim
dc.date.accessioned2022-01-27T06:26:58Z
dc.date.available2022-01-27T06:26:58Z
dc.date.issued2021en_US
dc.departmentFakülteler, Tıp Fakültesien_US
dc.description.abstractSignifcant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients’ life expectancy. However, transfusion-associated iron overload remains a signifcant barrier to long-term survival with good quality of life. Today, allogeneic hematopoietic stem cell transplantation (HSCT) is the current curative standard of care. Alongside selection of the best available donor, an optimized conditioning regimen is crucial to maximize outcomes for patients with TDT undergoing HSCT. The aim of this retrospective analysis was to investigate the role of busulfan– fudarabine-based and treosulfan–fudarabine-based conditioning in TDT patients undergoing HSCT. We included 772 patients registered in the European Society for Blood and Marrow Transplantation (EBMT) database who underwent frst HSCT between 2010 and 2018. Four hundred ten patients received busulfan–fudarabine-based conditioning (median age 8.6 years) and 362 patients received treosulfan–fudarabine-based conditioning (median age 5.7 years). Patient outcomes were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confdence interval: 89.3–95.1%) after busulfan–fudarabine-based conditioning and 94.7% (95% confdence interval: 91.7–96.6%) after treosulfan–fudarabine-based conditioning. There was a very low incidence of second HSCT overall. The main causes of death were infections, graft-versus-host disease, and rejection. In conclusion, use of busulfan or treosulfan as the backbone of myeloablative conditioning for patients with TDT undergoing HSCT resulted in comparably high cure rates. Long-term follow-up studies are warranted to address the important issues of organ toxicities and gonadal function.en_US
dc.identifier.citationLüftinger, R., Zubarovskaya, N., Galimard, J. E., Cseh, A., Salzer, E., Locatelli, F., ... & Corbacioglu, S. (2022). Busulfan–fludarabine-or treosulfan–fludarabine-based myeloablative conditioning for children with thalassemia major. Annals of Hematology, 1-11.en_US
dc.identifier.endpage11en_US
dc.identifier.scopus2-s2.0-85122828552
dc.identifier.scopusqualityQ2
dc.identifier.startpage1en_US
dc.identifier.urihttps://hdl.handle.net/20.500.12939/2229
dc.indekslendigikaynakScopus
dc.indekslendigikaynakPubMed
dc.institutionauthorFışgın, Tunç
dc.language.isoen
dc.publisherAnnals of Hematologyen_US
dc.relation.ispartofAnnals of Hematology
dc.relation.isversionof10.1007/s00277-021-04732-4en_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.subjectHematopoietic Stem Cell Transplantationen_US
dc.subjectConditioningen_US
dc.subjectThalassemia Majoren_US
dc.subjectBusulfanen_US
dc.subjectTreosulfanen_US
dc.titleBusulfan–fudarabine- or treosulfan–fudarabine-based myeloablative conditioning for children with thalassemia major
dc.typeArticle

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