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Impact of treosulfan exposure on clinical outcome after allogeneic stem cell transplantation in children: A substudy of 2 phase 2 trials

K. Kalwak, A. Vora, P. Bader, B. Burkhardt, S. Corbacioglu, K. Drabko, J. Gozdzik, J. Greil, B. Gruhn, K. Patrick, A. Schulz, P. Sedlacek, J. Styczynski, M. Mielcarek-Siedziuk, F. Locatelli, D. Reinhardt, PG. Schlegel, J. Baumgart, J. Kehne, X....

. 2025 ; 91 (3) : 882-893. [pub] 20241119

Jazyk angličtina Země Anglie, Velká Británie

Typ dokumentu časopisecké články, klinické zkoušky, fáze II, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc25009749

Grantová podpora
medac GmbH

AIMS: Allogeneic 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. METHODS: We 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. RESULTS: Our 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. CONCLUSION: These findings suggest that treosulfan exposure after standardized body surface area-based dosing is appropriate in paediatric allo-HSCT.

Citace poskytuje Crossref.org

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$a Burkhardt, Birgit $u Peaditric Haematology, Oncology and BMT, University Hospital Muenster, Muenster, Germany
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$a Corbacioglu, Selim $u Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, University of Regensburg, Regensburg, Germany
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