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

•Ruxolitinib as therapy for steroid-refractory chronic graft-versus-host disease is associated with high rates of overall response.•Long-term follow-up demonstrates that 67% of patients are free of any immunosuppression and 22% are receiving ruxolitinib as the sole immunosuppressive drug.•GVHD relap...

Full description

Saved in:
Bibliographic Details
Published in:Transplantation and cellular therapy 2021-09, Vol.27 (9), p.777.e1-777.e6
Main Authors: Ferreira, Aliana Meneses, Szor, Roberta Shcolnik, Molla, Vinicius Campos, Seiwald, Maria Cristina, de Moraes, Pedro Arruda, da Fonseca, Ana Rita Brito Medeiros, Xavier, Erick Menezes, Serpa, Mariana Gomes, Tucunduva, Luciana, Novis, Yana, Arrais-Rodrigues, Celso
Format: Article
Language:English
Subjects:
Allogeneic
Graft-versus-host disease
Hematopoietic stem cell transplantation
Ruxolitinib
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:•Ruxolitinib as therapy for steroid-refractory chronic graft-versus-host disease is associated with high rates of overall response.•Long-term follow-up demonstrates that 67% of patients are free of any immunosuppression and 22% are receiving ruxolitinib as the sole immunosuppressive drug.•GVHD relapse/progression rates after ruxolitinib are moderate and most of the patients respond to retreatment.•Ruxolitinib is associated with a favorable safety profile in patients with refractory chronic GVHD. 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.
ISSN:2666-6367
2666-6367
DOI:10.1016/j.jtct.2021.06.002