Meissner, Barbara and Lang, Peter and Bader, Peter and Hoenig, Manfred and Mueller, Ingo and Meisel, Roland and Greil, Johann and Sauer, Martin G. and Metzler, Markus and Corbacioglu, Selim and Burkhardt, Birgit and Woelfl, Matthias and Strahm, Brigitte and Kafa, Kinan and Basu, Oliver and Lode, Holger N. and Gruhn, Bernd and Cario, Holger and Ozga, Ann-Kathrin and Zimmermann, Martin and Jarisch, Andrea and Beier, Rita (2024) Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+cell count and immunosuppression. BONE MARROW TRANSPLANTATION, 59 (5). pp. 587-596. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
We performed a retrospective analysis on 124 patients with transfusion-dependent thalassemia who were registered in the German pediatric registry for stem cell transplantation. All patients underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 2011 and 2020 and belonged mainly to Pesaro risk class 1-2. Four-year overall (OS) and thalassemia-free survival (TFS) were 94.5% +/- 2.9% and 88.0% +/- 3.4% after treosulfan-fludarabine-thiotepa- and 96.9% +/- 3.1% (P = 0.763) and 96.9% +/- 3.1% (P = 0.155) after busulfan-fludarabine-based conditioning. Mixed chimerism below 75% occurred predominantly in treosulfan-based regimens (27.5% versus 6.2%). OS and TFS did not differ significantly between matched sibling, other matched family and matched unrelated donor (UD) HSCTs (OS: 100.0%, 100.0%, 96.3% +/- 3.6%; TFS: 96.5% +/- 2.4%, 90.0% +/- 9.5%, 88.9% +/- 6.0%). However, mismatched UD-HSCTs performed less favorable (OS: 84.7% +/- 7.3% (P = 0.029); TFS: 79.9% +/- 7.4% (P = 0.082)). We generated a scoring system reflecting the risk to develop mixed chimerism in our cohort. The main risk-reducing factors were a high CD3+ cell count (>= 6 x 107/kg) in the graft, busulfan-conditioning, pre-conditioning therapy and low-targeted ciclosporin A trough levels. Acute GvHD grade III-IV in treosulfan-based concepts predominantly occurred in patients with UD and reduced GvHD prophylaxis but not in the context of high CD3+ cell doses. Taken together, this information might be used to develop more risk-adapted HSCT regimens for thalassemia patients.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; TRANSFUSION; EXPERIENCE; DISEASE; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 17 Nov 2025 10:22 |
| Last Modified: | 17 Nov 2025 10:22 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65495 |
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