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JOURNAL ARTICLE

Ruxolitinib in refractory acute and chronic graft-versus-host disease: a multicenter survey study

Virginia Escamilla Gómez, Valentín García-Gutiérrez, Lucía López Corral, Irene García Cadenas, Ariadna Pérez Martínez, Francisco J Márquez Malaver, Teresa Caballero-Velázquez, Pedro A González Sierra, María C Viguria Alegría, Ingrid M Parra Salinas, Cristina Calderón Cabrera, Marta González Vicent, Nancy Rodríguez Torres, Rocío Parody Porras, Christelle Ferra Coll, Guillermo Orti, David Valcárcel Ferreiras, Rafael De la Cámara LLanzá, Paula Molés, Kyra Velázquez-Kennedy, María João Mende, Dolores Caballero Barrigón, Estefanía Pérez, Rodrigo Martino Bofarull, Silvanna Saavedra Gerosa, Jorge Sierra, Marc Poch, María T Zudaire Ripa, Miguel A Díaz Pérez, Blanca Molina Angulo, Isabel Sánchez Ortega, Jaime Sanz Caballer, Juan Montoro Gómez, Ildefonso Espigado Tocino, José A Pérez-Simón
Bone Marrow Transplantation 2019 November 7
31700138
Graft-versus-host disease is the main cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation. First-line treatment is based on the use of high doses of corticosteroids. Unfortunately, second-line treatment for both acute and chronic graft-versus-host disease, remains a challenge. Ruxolitinib has been shown as an effective and safe treatment option for these patients. Seventy-nine patients received ruxolitinib and were evaluated in this retrospective and multicenter study. Twenty-three patients received ruxolitinib for refractory acute graft-versus-host disease after a median of 3 (range 1-5) previous lines of therapy. Overall response rate was 69.5% (16/23) which was obtained after a median of 2 weeks of treatment, and 21.7% (5/23) reached complete remission. Fifty-six patients were evaluated for refractory chronic graft-versus-host disease. The median number of previous lines of therapy was 3 (range 1-10). Overall response rate was 57.1% (32/56) with 3.5% (2/56) obtaining complete remission after a median of 4 weeks. Tapering of corticosteroids was possible in both acute (17/23, 73%) and chronic graft-versus-host disease (32/56, 57.1%) groups. Overall survival was 47% (CI: 23-67%) at 6 months for patients with aGVHD (62 vs 28% in responders vs non-responders) and 81% (CI: 63-89%) at 1 year for patients with cGVHD (83 vs 76% in responders vs non-responders). Ruxolitinib in the real life setting is an effective and safe treatment option for GVHD, with an ORR of 69.5% and 57.1% for refractory acute and chronic graft-versus-host disease, respectively, in heavily pretreated patients.

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