Defibrotide in hematopoietic stem cell transplantation: A multicenter survey study of the Spanish Hematopoietic Stem Cell Transplantation Group (GETH).
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González Vicent Marta, Díaz de Heredia Cristina, González de Pablo Jesús, Molina Blanca, Regueiro Alexandra, Pérez Martinez Antonio, Palomo Pilar, López Corral Lucía, García Estefanía, Fernández José María, Pérez Ariadna, Jiménez María José, Guerreiro Manuel, Vallejo Carlos, Gallardo Ana Isabel, López Oriana, Benito Ana, Marsal Julia, Duarte Mónica, Bento Leyre, Badell Isabel, Pedraza Alexandra, Jiménez Ubieto Ana, González Pedro, Gómez Centurión Ignacio, Costilla Lissette, Beléndez Cristina, Esquirol Albert, Espigado Ildefonso, Lavilla Esperanza, Díaz Miguel Ángel
Аннотация
METHODS: This multicenter, retrospective study included patients treated with defibrotide for any indication at 28 HSCT centers of the Grupo Español de Trasplante Hematopoyetico (GETH) including the pediatric subgroup Grupo Español de Trasplante de Medula en Niños (GETMON).
RESULTS: Three hundred and eighty eight patients treated with defibrotide between January 2011 and December 2018 were included. 253 patients were children, and 135 patients were adults. In total, 332 transplants were allogeneic, and the remainder were autologous. Main indications for defibrotide use were severe/very severe SOS in 173 patients, SOS prophylaxis in 135 patients, moderate SOS in 41 patients, TAM in six patients and suspected SOS in 33 patients. Overall survival (OS) at day +100 in the SOS prophylaxis group was 89% (95% CI, 87%-91%). In the group of patients with moderate and severe/very severe SOS, the OS at day +100 was 80% (95% CI, 74%-86%) and 62% (95% CI, 59%-65%), respectively (P = .0015). With a longer follow-up, median of 2 years (4 months-7 years), OS was 63% (95% CI, 59%-67%) in the SOS prophylaxis patients. OS for patients with moderate and severe/very severe SOS groups was 53% (95% CI, 47%-61%) and 26% (95% CI, 22%-30%), respectively (P = .006). 191 patients died, and SOS was the main cause of death in 23 patients (12%).
CONCLUSIONS: Defibrotide has an acceptable safety profile with an improved response in severe/very severe SOS compared with historical controls, mainly in pediatric patients. Use of defibrotide for prophylaxis may improve prognosis of patients at high risk of complications due to endothelial damage such as those who receive a second transplant. SOS has an important impact on the HSCT long-term survival, as can be concluded from our study.