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

KW. Sykora, R. Beier, A. Schulz, S. Cesaro, J. Greil, J. Gozdzik, P. Sedlacek, P. Bader, J. Schulte, M. Zecca, F. Locatelli, B. Gruhn, D. Reinhardt, J. Styczynski, S. Piras, F. Fagioli, S. Bonanomi, M. Caniglia, X. Li, J. Baumgart, J. Kehne, M....

. 2024 ; 59 (1) : 107-116. [pub] 20231104

Jazyk angličtina Země Anglie, Velká Británie

Typ dokumentu randomizované kontrolované studie, klinické zkoušky, fáze II, časopisecké články, práce podpořená grantem

Perzistentní odkaz   https://www.medvik.cz/link/bmc24007810
E-zdroje Online Plný text

NLK Free Medical Journals od 1997 do Před 1 rokem
Freely Accessible Science Journals od 1997 do Před 1 rokem
ProQuest Central od 2000-01-01 do Před 1 rokem
Open Access Digital Library od 1997-01-01
Health & Medicine (ProQuest) od 2000-01-01 do Před 1 rokem

Odkazy

PubMed 37925531
DOI 10.1038/s41409-023-02135-9
Knihovny.cz E-zdroje

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.

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