Ruxolitinib add-on in corticosteroid-refractory graft- vs-host disease after allogeneic stem cell transplantation: Results from a retrospective study on 38 Chinese patients

Si-Hua Dang; Qin Liu; Rong Xie; Na Shen; Shu Zhou; Wei Shi; Wen Liu; Ping Zou; Yong You; Zhao-Dong Zhong

doi:10.12998/wjcc.v8.i6.1065

Ruxolitinib add-on in corticosteroid-refractory graft- vs-host disease after allogeneic stem cell transplantation: Results from a retrospective study on 38 Chinese patients

World J Clin Cases. 2020 Mar 26;8(6):1065-1073. doi: 10.12998/wjcc.v8.i6.1065.

Authors

Si-Hua Dang¹, Qin Liu², Rong Xie¹, Na Shen¹, Shu Zhou¹, Wei Shi¹, Wen Liu¹, Ping Zou¹, Yong You¹, Zhao-Dong Zhong³

Affiliations

¹ Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, Hubei Province, China.
² Department of Gynecology and Obstetrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, Hubei Province, China.
³ Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, Hubei Province, China. whxhzzd008@126.com.

Abstract

Background: Graft-vs-host disease (GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation. Some patients have steroid-refractory (SR) GVHD.

Aim: To evaluate the effect and safety of ruxolitinib add-on in the treatment of patients with SR acute (a) and chronic (c) GVHD.

Methods: We retrospectively analyzed 38 patients administered ruxolitinib add-on to standard immunosuppressive therapy for SR-aGVHD or SR-cGVHD following allogeneic hematopoietic stem cell transplantation. Ruxolitinib was administered 5-10 mg/d depending on disease severity, patient status, and the use of anti-fungal drugs. Overall response rate, time to best response, malignancy relapse rate, infection rate, and treatment-related adverse events were assessed.

Results: The analysis included 10 patients with SR-aGVHD (grade III/IV, n = 9) and 28 patients with SR-cGVHD (moderate/severe, n = 24). For the SR-aGVHD and SR-cGVHD groups, respectively: Median number of previous GVHD therapies was 2 (range: 1-3) and 2 (1-4); median follow-up was 2.5 (1.5-4) and 5 (1.5-10) mo; median time to best response was 1 (0.5-2.5) and 3 (1-9.5) mo; and overall response rate was 100% (complete response: 80%) and 82.1% (complete response: 10.7%) with a response observed in all GVHD-affected organs. The malignancy relapse rates for the SR-aGVHD and SR-cGVHD groups were 10.0% and 10.7%, respectively. Reactivation rates for cytomegalovirus, Epstein-Barr virus, and varicella-zoster virus, respectively, were 30.0%, 10.0%, and 0% for the SR-aGVHD group and 0%, 14.3%, and 7.1% for the SR-cGVHD group.

Conclusion: Ruxolitinib add-on was effective and safe as salvage therapy for SR-GVHD.

Keywords: Allogeneic hematopoietic stem cell transplantation; Antifungal drugs; Graft-vs-host disease; Graft-vs-leukemia effect; Ruxolitinib; Treatment.