Kleinschmidt, Katharina and Penkivech, Gina and Troeger, Anja and Foell, Juergen and Hanafee-Alali, Tarek and Leszczak, Stefanie and Jakob, Marcus and Kramer, Sonja and Kietz, Silke and Hoffmann, Petra and Behrendt-Boehm, Claudia and Kaess, Carina and Brosig, Andreas M. and Offner, Robert and Wolff, Daniel and Corbacioglu, Selim (2025) αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia. BONE MARROW TRANSPLANTATION, 60 (5). pp. 682-689. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
Life expectancy of patients with severe transfusion-dependent beta-thalassemia (TDT) remains below that of the general population. Allogenic hematopoietic stem cell transplantation (HSCT) is the standard curative treatment. Due to the paucity of matched donor (MD) availability, haploidentical HSCT (haplo-HSCT) is a reasonable alternative. Twenty patients with TDT (median age 10 years; range 2-23) received either a matched sibling donor (MSD; n = 7) or a haplo-HSCT (n = 13) in a single center (Regensburg, Germany) between 2016 and 2022, including two patients referred for a haplo-HSCT as rescue failing prior MD- and haplo-HSCT, respectively. The conditioning regimen consisted of anti-thymocyte globulin (ATG; Grafalon (R)), treosulfan, thiotepa, and fludarabine (FTT). Immunosuppression consisted of a calcineurin inhibitor and mycophenolate mofetil (MMF). At a median follow-up of 37 months (range 6-90), overall survival (OS) was 100% with a disease-free survival (DFS) of 100% in MSD and 92% in haplo-HSCT, respectively. Two patients in haplo-HSCT experienced graft failure, one achieving DFS after a second haplo-HSCT. No acute graft-versus-host disease (aGvHD) >= degrees III or severe chronic GvHD (cGvHD) were observed. No sinusoidal obstruction syndrome (SOS) was observed in this high-risk population. Treosulfan-based T-cell depleted haplo-HSCT can achieve comparable OS and DFS even in young adult TDT patients with no SOS/VOD.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | CHILDREN; DONORS; BLOOD; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie) Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation Medicine > Lehrstuhl für Klinische Chemie und Laboratoriumsmedizin |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 26 Aug 2026 10:05 |
| Last Modified: | 26 Aug 2026 10:05 |
| URI: | https://pred.uni-regensburg.de/id/eprint/66060 |
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