Efficacy and Safety of Ruxolitinib in Steroid-Refractory/Dependent Chronic Graft-versus-Host Disease

Steroid-refractory (SR) chronic graft-versus-host disease (cGVHD) is a major obstacle in recipients of allogeneic stem cell transplantation (HCT). Ruxolitinib is the first agent to demonstrate superior efficacy to the best available therapy, but real-life data are still lacking. Here we describe the...

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Autores: Redondo, Sara|||0000-0001-8967-7410, Esquirol, Albert|||0000-0002-6062-5980, Novelli, Silvana|||0000-0001-8750-0195, Caballero, Ana Carolina|||0000-0002-4950-5259, Garrido Diaz, Ana|||0000-0002-5297-8946, Oñate, Guadalupe|||0000-0003-2180-2371, López, Jordi, Moreno, Carol|||0000-0003-3275-0271, Saavedra, Silvana|||0000-0001-5699-0531, Granell, Miquel|||0000-0002-0366-3673, Briones Meijide, Javier|||0000-0003-2750-3735, Sierra, Jorge|||0000-0002-7966-0356, Martino Bofarull, Rodrigo|||0000-0001-5143-4042, García Cadenas, Irene|||0000-0002-2994-9055
Tipo de recurso: artículo
Fecha de publicación:2022
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:289908
Acceso en línea:https://ddd.uab.cat/record/289908
https://dx.doi.org/urn:doi:10.1016/j.jtct.2021.10.015
Access Level:acceso abierto
Palabra clave:Steroid-refractory-chronic GvHD
Ruxolitinib
Steroids
GVHD-flare
Toxicity-profile
Descripción
Sumario:Steroid-refractory (SR) chronic graft-versus-host disease (cGVHD) is a major obstacle in recipients of allogeneic stem cell transplantation (HCT). Ruxolitinib is the first agent to demonstrate superior efficacy to the best available therapy, but real-life data are still lacking. Here we describe the results of ruxolitinib compassionate use for the treatment of SR/steroid-dependent cGVHD in a tertiary care university hospital. In this retrospective single-center study, we evaluated the outcomes of 48 patients diagnosed with SR-cGVHD who were treated with ruxolitinib. Forty-seven (98%) had moderate-severe disease, and 27 (56%) had received ≥2 lines of prior therapy (excluding steroids). Results were analyzed using SPSS version 26.0.01 and R version 3.4.3. The overall response rate was 77% (37 of 48), with 15% (7 of 37) in complete remission. The median time to response was 2 months (range, 0.5 to 8 months). Steroid tapering was achieved in 26 patients (54%) and definitive discontinuation was achieved in 10 patients (21%) after a median of 20 months (range, 1.5 to 60 months). Toxicity was predominantly hematologic, including a 33% rate of anemia and a 17% rate of thrombocytopenia. Overall survival at 2 years was significantly higher in responders compared with nonresponders (88% [95% confidence interval (CI), 65% to 96%] versus 49% [95% CI, 12% to 78%]; P =.01). At last follow-up, tapering of ruxolitinib had been started in 8 of 37 responders (22%). Our experience supports the efficacy of ruxolitinib in the treatment of SR-cGVHD, along with its steroid-sparing effect and manageable toxicity. Gradual tapering of ruxolitinib seems feasible without cases of GVHD flare. More studies and longer follow-up are needed to confirm these data, as well as to identify the ideal dose adjustments in cases of toxicity.

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