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09.01.2022 | Original Article

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

verfasst von: Roswitha Lüftinger, Natalia Zubarovskaya, Jacques-Emmanuel Galimard, Annamaria Cseh, Elisabeth Salzer, Franco Locatelli, Mattia Algeri, Akif Yesilipek, Josu de la Fuente, Antonella Isgrò, Amal Alseraihy, Emanuele Angelucci, Frans J. Smiers, Giorgia La La Nasa, Marco Zecca, Tunc Fisgin, Emel Unal, Katharina Kleinschmidt, Christina Peters, Arjan Lankester, Selim Corbacioglu, on behalf of the EBMT Pediatric Diseases, Inborn Errors Working Parties

Erschienen in: Annals of Hematology | Ausgabe 3/2022

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Abstract

Significant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients’ life expectancy. However, transfusion-associated iron overload remains a significant 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–fludarabine-based and treosulfan–fludarabine-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 first HSCT between 2010 and 2018. Four hundred ten patients received busulfan–fludarabine-based conditioning (median age 8.6 years) and 362 patients received treosulfan–fludarabine-based conditioning (median age 5.7 years). Patient outcomes were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confidence interval: 89.3–95.1%) after busulfan–fludarabine-based conditioning and 94.7% (95% confidence interval: 91.7–96.6%) after treosulfan–fludarabine-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.

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Titel: Busulfan–fludarabine- or treosulfan–fludarabine-based myeloablative conditioning for children with thalassemia major
verfasst von: Roswitha Lüftinger
Natalia Zubarovskaya
Jacques-Emmanuel Galimard
Annamaria Cseh
Elisabeth Salzer
Franco Locatelli
Mattia Algeri
Akif Yesilipek
Josu de la Fuente
Antonella Isgrò
Amal Alseraihy
Emanuele Angelucci
Frans J. Smiers
Giorgia La La Nasa
Marco Zecca
Tunc Fisgin
Emel Unal
Katharina Kleinschmidt
Christina Peters
Arjan Lankester
Selim Corbacioglu
on behalf of the EBMT Pediatric Diseases, Inborn Errors Working Parties
Publikationsdatum: 09.01.2022
Verlag: Springer Berlin Heidelberg
Erschienen in: Annals of Hematology / Ausgabe 3/2022
Print ISSN: 0939-5555
Elektronische ISSN: 1432-0584
DOI: https://doi.org/10.1007/s00277-021-04732-4

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