Kalwak, Krzysztof and Vora, Ajay and Bader, Peter and Burkhardt, Birgit and Corbacioglu, Selim and Drabko, Katarzyna and Gozdzik, Jolanta and Greil, Johann and Gruhn, Bernd and Patrick, Katharine and Schulz, Ansgar and Sedlacek, Petr and Styczynski, Jan and Mielcarek-Siedziuk, Monika and Locatelli, Franco and Reinhardt, Dirk and Schlegel, Paul-Gerhardt and Baumgart, Joachim and Kehne, Jochen and Li, Xieran and Beier, Rita (2025) Impact of treosulfan exposure on clinical outcome after allogeneic stem cell transplantation in children: A substudy of 2 phase 2 trials. BRITISH JOURNAL OF CLINICAL PHARMACOLOGY, 91 (3). pp. 882-893. ISSN 0306-5251, 1365-2125
Full text not available from this repository. (Request a copy)Abstract
AimsAllogeneic haematopoietic stem cell transplantation (allo-HSCT) is a vital treatment for various paediatric malignant and nonmalignant diseases. The conditioning treatment before allo-HSCT is crucial for successful engraftment. Treosulfan, a cytotoxic prodrug, has gained popularity due to its lower toxicity compared to traditional alkylating agents used for conditioning treatment.MethodsWe investigated the relationship between pharmacokinetics and pharmacodynamics of treosulfan in paediatric patients, in a substudy pooling from 2 multicentre phase 2 clinical trials. A total of 83 children with malignant and nonmalignant diseases received treosulfan-based conditioning. Treosulfan exposure and its relationship with clinical outcomes, including survival, graft failure and graft-vs.-host disease, were investigated.ResultsOur findings reveal no significant association between treosulfan exposure and the key clinical outcomes or toxicity (P-values between .22 and .99), if the dosing is based on the approved product information.ConclusionThese findings suggest that treosulfan exposure after standardized body surface area-based dosing is appropriate in paediatric allo-HSCT.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | ; treosulfan; paediatrics; stem cell transplantation |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 31 Mar 2026 08:57 |
| Last Modified: | 31 Mar 2026 08:57 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65397 |
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