Long-Term Follow-Up of Ruxolitinib in the Treatment of Steroid-Refractory Chronic Graft-versus-Host Disease.

Autor: Ferreira AM; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil. Electronic address: ali_ferreira@hotmail.com., Szor RS; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil., Molla VC; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil; Division of Hematology, Federal University of São Paulo, São Paulo, Brazil., Seiwald MC; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil., de Moraes PA; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil; Division of Hematology, Federal University of São Paulo, São Paulo, Brazil., da Fonseca ARBM; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil., Xavier EM; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil., Serpa MG; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil., Tucunduva L; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil., Novis Y; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil., Arrais-Rodrigues C; Bone Marrow Transplant, Hospital Sirio Libanes, São Paulo, Brazil; Division of Hematology, Federal University of São Paulo, São Paulo, Brazil.
Jazyk: angličtina
Zdroj: Transplantation and cellular therapy [Transplant Cell Ther] 2021 Sep; Vol. 27 (9), pp. 777.e1-777.e6. Date of Electronic Publication: 2021 Jun 09.
DOI: 10.1016/j.jtct.2021.06.002
Abstrakt: Chronic graft-versus-host disease (cGVHD) remains a major barrier to successful hematopoietic stem cell transplantation (HSCT). In cases refractory to first-line therapy with steroids, there is no standard of care for second-line therapy. As such, ruxolitinib is a promising drug in this scenario. We retrospectively analyzed the efficacy and safety of ruxolitinib in treating steroid-refractory cGVHD in 35 patients from 2 transplantation centers, with the longest follow-up described to date. The evaluated patients had a median of 3 organs affected (range, 1 to 7 organs), with most (64%) having moderate cGVHD. The median number of previous therapy lines was 2 (range, 1 to 6). The overall response rate was 89% (complete response, 26%) after a median of 4 weeks of therapy. The median follow-up was 43 months (range, 11 to 59 monts). At follow-up, of the 27 patients still alive, 18 (67%) were free of any immunosuppression, and 6 (22%) were receiving ruxolitinib as their sole immunosuppressive drug. Failure-free survival was 77.1% at 6 months, 68.6% at 12 months, 54% at 24 months, and 51.4% at 36 months. The median overall survival was not reached. Toxicities were mostly hematologic and resolved after dosage reduction in most cases. Overall, our data, which represent the cohort of patients with cGVHD treated with ruxolitinib with the longest follow-up to date, support the use of this drug as a safe and effective option for refractory cGVHD.
(Copyright © 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE