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Treosulfan vs busulfan conditioning for allogeneic bmt in children with nonmalignant disease: a randomized phase 2 trial

Optimal conditioning prior to allogeneic hematopoietic stem cell transplantation for children with non-malignant diseases is subject of ongoing research. This prospective, randomized, phase 2 trial compared safety and efficacy of busulfan with treosulfan based preparative regimens. Children with non...

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Main Authors: Sykora, Karl-Walter (Author) , Beier, Rita (Author) , Schulz, Ansgar Stephan (Author) , Cesaro, Simone (Author) , Greil, Johann (Author) , Gozdzik, Jolanta (Author) , Sedlácek, Petr (Author) , Bader, Peter (Author) , Schulte, Johannes (Author) , Zecca, Marco (Author) , Locatelli, Franco (Author) , Gruhn, Bernd (Author) , Reinhardt, Dirk (Author) , Styczyński, Jan (Author) , Piras, Simona (Author) , Fagioli, Franca (Author) , Bonanomi, Sonia (Author) , Caniglia, Maurizio (Author) , Li, Xieran (Author) , Baumgart, Joachim (Author) , Kehne, Jochen (Author) , Mielcarek-Siedziuk, Monika (Author) , Kalwak, Krzysztof (Author)
Format: Article (Journal)
Language:English
Published: 04 November 2023
In: Bone marrow transplantation
Year: 2023, Pages: 1-10
ISSN:1476-5365
DOI:10.1038/s41409-023-02135-9
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1038/s41409-023-02135-9
Verlag, kostenfrei, Volltext: https://www.nature.com/articles/s41409-023-02135-9
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Author Notes:Karl-Walter Sykora, Rita Beier, Ansgar Schulz, Simone Cesaro, Johann Greil, Jolanta Gozdzik, Petr Sedlacek, Peter Bader, Johannes Schulte, Marco Zecca, Franco Locatelli, Bernd Gruhn, Dirk Reinhardt, Jan Styczynski, Simona Piras, Franca Fagioli, Sonia Bonanomi, Maurizio Caniglia, Xieran Li, Joachim Baumgart, Jochen Kehne, Monika Mielcarek-Siedziuk and Krzysztof Kalwak
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Summary:Optimal conditioning prior to allogeneic hematopoietic stem cell transplantation for children with non-malignant diseases is subject of ongoing research. This prospective, randomized, phase 2 trial compared safety and efficacy of busulfan with treosulfan based preparative regimens. Children with non-malignant diseases received fludarabine and either intravenous (IV) busulfan (4.8 to 3.2 mg/kg/day) or IV treosulfan (10, 12, or 14 g/m2/day). Thiotepa administration (2 × 5 mg/kg) was at the investigator’s discretion. Primary endpoint was freedom from transplantation (treatment)-related mortality (freedom from TRM), defined as death between Days -7 and +100. Overall, 101 patients (busulfan 50, treosulfan 51) with at least 12 months follow-up were analyzed. Freedom from TRM was 90.0% (95% CI: 78.2%, 96.7%) after busulfan and 100.0% (95% CI: 93.0%, 100.0%) after treosulfan. Secondary outcomes (transplantation-related mortality [12.0% versus 3.9%]) and overall survival (88.0% versus 96.1%) favored treosulfan. Graft failure was more common after treosulfan (n = 11), than after busulfan (n = 2) while all patients were rescued by second procedures except one busulfan patient. CTCAE Grade III adverse events were similar in both groups. This study confirmed treosulfan to be an excellent alternative to busulfan and can be safely used for conditioning treatment in children with non-malignant disease.
Item Description:Gesehen am 13.12.2023
Physical Description:Online Resource
ISSN:1476-5365
DOI:10.1038/s41409-023-02135-9

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