Luftinger, Roswitha and Zubarovskaya, Natalia and Galimard, Jacques-Emmanuel and Cseh, Annamaria and Salzer, Elisabeth and Locatelli, Franco and Algeri, Mattia and Yesilipek, Akif and de la Fuente, Josu and Isgro, Antonella and Alseraihy, Amal and Angelucci, Emanuele and Smiers, Frans J. and Nasa, Giorgia La La and Zecca, Marco and Fisgin, Tunc and Unal, Emel and Kleinschmidt, Katharina and Peters, Christina and Lankester, Arjan and Corbacioglu, Selim (2022) Busulfan-fludarabine- or treosulfan-fludarabine-based myeloablative conditioning for children with thalassemia major. ANNALS OF HEMATOLOGY, 101 (3). pp. 655-665. ISSN 0939-5555, 1432-0584
Full text not available from this repository. (Request a copy)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.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | STEM-CELL TRANSPLANTATION; BONE-MARROW-TRANSPLANTATION; BETA-THALASSEMIA; REGIMEN; DISEASE; RISK; BLOOD; MANAGEMENT; SCT; Hematopoietic stem cell transplantation; Conditioning; Thalassemia major; Busulfan; Treosulfan |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 29 Jan 2024 12:21 |
| Last Modified: | 29 Jan 2024 12:41 |
| URI: | https://pred.uni-regensburg.de/id/eprint/58636 |
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