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. 2021 Jan 4;4(1):e2034750.
doi: 10.1001/jamanetworkopen.2020.34750.

Evaluation of Ruxolitinib for Steroid-Refractory Chronic Graft-vs-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Hengwei Wu  1   2 , Jimin Shi  1   2 , Yi Luo  1   2 , Yamin Tan  1   2 , Mingming Zhang  1   2 , Xiaoyu Lai  1   2 , Jian Yu  1   2 , Lizhen Liu  1   2 , Huarui Fu  1   2 , He Huang  1   2 , Yanmin Zhao  1   2
Affiliations

Evaluation of Ruxolitinib for Steroid-Refractory Chronic Graft-vs-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Hengwei Wu et al. JAMA Netw Open. .

Abstract

Importance: 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).

Objectives: 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.

Design, setting, and participants: 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.

Exposure: Ruxolitinib.

Main outcomes and measures: Treatment responses, factors associated with response, and adverse effects during ruxolitinib administration.

Findings: 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).

Conclusions and relevance: 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.

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Conflict of interest statement

Conflict of Interest Disclosures: None reported.

Figures

Figure 1.
Figure 1.. Treatment Response and Prednisone Dose Among 41 Patients with Steroid-Refractory Chronic Graft-vs-Host Disease (cGVHD)
Median (range) treatment duration was 8.6 (1.1-24.9) months in responders. CR indicates complete remission; GI, gastrointestinal tract; PD, progressive disease; PR, partial remission; and SD, stable disease.
Figure 2.
Figure 2.. Overall Survival, Relapse Incidence, and Cumulative Flare Incidence During the Follow-up Period
cGVHD indicates chronic graft-vs-host disease; NRM indicates nonrelapsed mortality.
Figure 3.
Figure 3.. Survival Probabilities
cGVHD indicates chronic graft-vs-host disease; CR indicates complete remission; and HSCT indicates hematopoietic stem cell transplantation.
See this image and copyright information in PMC

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