Abstract
Ruxolitinib, a selective inhibitor of the Janus kinases 1/2 signaling pathway, has shown a significant response in steroid-refractory chronic graft-vs-host disease (SR-cGVHD), a major cause of morbidity and mortality in individuals who have undergone allogeneic hematopoietic stem cell transplantation (HSCT). To investigate the clinical response to ruxolitinib in patients with SR-cGVHD after allogeneic HSCT and to evaluate its safety profile during the treatment course. This single-center case series included 41 consecutive patients who were treated with ruxolitinib for SR-cGVHD after allogeneic HSCT between August 2017 and December 2019. Data were collected from each patient's medical record at the First Affiliated Hospital of Zhejiang University School of Medicine. Data analysis was conducted from March to May 2020. Ruxolitinib. Treatment responses, factors associated with response, and adverse effects during ruxolitinib administration. Overall, 41 patients (median [range] age, 31 [17-56] years; 14 [34.1%] women) were treated with ruxolitinib and included in this study. A total of 15 patients (36.6%) had a complete remission, and 14 (34.1%) had a partial remission, with an overall response rate of 70.7% (29 patients; 95% CI, 56.2%-85.3%). Lung involvement (odds ratio, 0.112; 95% CI, 0.020-0.639; P = .01) and matched related donors (odds ratio, 0.149; 95% CI, 0.022-0.981; P = .048) were associated with less favorable treatment response. Major adverse events associated with ruxolitinib were cytopenias and infectious complications. The median (range) follow-up for this cohort was 14.9 (1.4-32.5) months. Prolonged survival was observed in patients with a male donor (P = .006), complete remission before transplantation (P = .02), baseline moderate cGVHD (P = .02), and skin cGVHD (P = .001). In this small, single-site case series, ruxolitinib demonstrated a significant response in heavily pretreated patients with SR-cGVHD and a reasonably well-tolerated safety profile. The results add to the body of literature suggesting ruxolitinib as a promising treatment option in SR-cGVHD.
Highlights
Chronic graft-vs-host disease is the leading cause of late morbidity and mortality as well as impaired quality of life after allogeneic hematopoietic stem cell transplantation (HSCT).1 Despite the use of standard prophylaxis, 35% to 70% of recipients develop cGVHD.2 Established first-line therapy for cGVHD still comprises corticosteroids and calcineurin inhibitors.3 Approximately half of patients with cGVHD are refractory to corticosteroid therapy
Prolonged survival was observed in patients with a male donor (P = .006), complete remission before transplantation (P = .02), baseline moderate cGVHD (P = .02), and skin cGVHD (P = .001). In this small, single-site case series, ruxolitinib demonstrated a significant response in heavily pretreated patients with SR-cGVHD and a reasonably well-tolerated safety profile
Based on the limited preclinical and clinical outcomes of ruxolitinib in GVHD published to date, the present study investigated the clinical response to ruxolitinib and its safety profile in patients with SR-cGVHD after allogeneic HSCT
Summary
Chronic graft-vs-host disease (cGVHD) is the leading cause of late morbidity and mortality as well as impaired quality of life after allogeneic hematopoietic stem cell transplantation (HSCT). Despite the use of standard prophylaxis, 35% to 70% of recipients develop cGVHD. Established first-line therapy for cGVHD still comprises corticosteroids and calcineurin inhibitors. Approximately half of patients with cGVHD are refractory to corticosteroid therapy. Chronic graft-vs-host disease (cGVHD) is the leading cause of late morbidity and mortality as well as impaired quality of life after allogeneic hematopoietic stem cell transplantation (HSCT).. Despite the use of standard prophylaxis, 35% to 70% of recipients develop cGVHD.. Established first-line therapy for cGVHD still comprises corticosteroids and calcineurin inhibitors.. Half of patients with cGVHD are refractory to corticosteroid therapy. For various second-line therapies or interventions, the response rates range from 30% to 60%,4,5 and no consensus has been reached regarding the optimal salvage treatment for steroid-refractory (SR)–cGVHD. It is essential to identify a promising therapeutic drug for the adequate therapy of SR-cGVHD
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