- Tytuł:
- Treosulfan vs busulfan conditioning for allogeneic bmt in children with nonmalignant disease : a randomized phase 2 trial
- Autorzy:
-
Goździk, Jolanta
Kalwak, Krzysztof
Greil, Johann
Locatelli, Franco
Schulz, Ansgar
Beier, Rita
Mielcarek-Siedziuk, Monika
Li, Xieran
Sykora, Karl-Walter
Zecca, Marco
Reinhardt, Dirk
Baumgart, Joachim
Gruhn, Bernd
Caniglia, Maurizio
Cesaro, Simone
Bonanomi, Sonia
Styczynski, Jan
Kehne, Jochen
Bader, Peter
Piras, Simona
Sedlacek, Petr
Schulte, Johannes
Fagioli, Franca - Opis:
- 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/m$_{2}$ /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.
- Dostawca treści:
- Repozytorium Uniwersytetu Jagiellońskiego
Artykuł