Published online Mar 26, 2020. doi: 10.12998/wjcc.v8.i6.1065
Peer-review started: November 22, 2019
First decision: December 12, 2019
Revised: February 19, 2020
Accepted: February 28, 2020
Article in press: February 28, 2020
Published online: March 26, 2020
Processing time: 124 Days and 13.7 Hours
Graft-vs-host disease (GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation. Some patients have steroid-refractory (SR) GVHD.
To evaluate the effect and safety of ruxolitinib add-on in the treatment of patients with SR acute (a) and chronic (c) GVHD.
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.
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.
Ruxolitinib add-on was effective and safe as salvage therapy for SR-GVHD.
Core tip: This study aimed to investigate the effect and safety of ruxolitinib add-on in the treatment of patients with steroid-refractory (SR) acute (a) and chronic (c) graft-vs-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation. An important finding of this retrospective case series was that the use of ruxolitinib as a salvage therapy for SR-GVHD resulted in an overall response rate of 100% in patients with SR-aGVHD (complete response: 80%) and 82.1% in patients with SR-cGVHD (complete response: 10.7%). In addition, this was achieved with a lower dose (5-10 mg/d) of ruxolitinib than previous reports, indicating that the dose of ruxolitinib could be lowered when used in combination with antifungal drugs (CYP2C9/CYP3A4 inhibitors).