• Pediatr Hematol Oncol · May 2021

    Ruxolitinib for steroid-refractory graft versus host disease in pediatric HSCT: high response rate and manageable toxicity.

    • Yasmina Mozo, David Bueno, Luisa Sisinni, Alba Fernández-Arroyo, Blanca Rosich, MartínezAntonio PérezAPhttps://orcid.org/0000-0002-6436-9195Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain., María Isabel Benítez-Carabante, Laura Alonso, María Luz Uría, HerediaCristina Díaz deCDPediatric Hemato-Oncology Department, Hospital Universitari Vall d´Hebron, Barcelona, Spain., Carmen Mestre-Duran, Cristina Ferreras Bárbara Pascual, Juan Torres, Itsaso Losantos, Adela Escudero, and Beatriz Ruz-Caracuel.
    • Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain.
    • Pediatr Hematol Oncol. 2021 May 1; 38 (4): 331-345.

    AbstractRuxolitinib, a selective Janus Kinase (JAK) 1/2 inhibitor, is a promising treatment for the steroid-refractory graft-vs-host disease (GvHD) after hematopoietic stem cell transplantation (HSCT). Most studies have been performed in the adult population showing efficacy against GvHD. In this retrospective study, we evaluated the outcomes of 19 children who received ruxolitinib for refractory acute or chronic GvHD (cGvHD) after HSCT from two Pediatric Hemato-Oncology Departments in Spain between March 2017 and December 2018. Patients received a median number of 4 (IQR 2) previous lines of treatment before starting ruxolitinib. The overall response rate in acute GvHD (aGvHD) and cGvHD was 87% and 91%, respectively. Complete response (CR) was observed in 37% of aGvHD and 8.3% of cGvHD. Remarkably, 43% and 40% of patients with steroid-refractory gastrointestinal aGvHD and lung cGvHD achieved CR. During ruxolitinib treatment, there were 36%, 31%, and 10% infections caused by viruses, bacteria, and fungi, respectively. Overall, four patients interrupted ruxolitinib due to infectious complications, hematological, and liver toxicity. The 2-year overall survival was 71.9% (CI 95% 58.6-85.2). Our experience supports the use of ruxolitinib as an effective treatment for steroid-refractory acute and cGvHD in children with a moderate toxicity profile.

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