Literature Review Assesses Use of Ruxolitinib in Chronic Steroid-Refractory GVHD

A study presented at the 2022 American Society of Clinical Oncology Annual Meeting reported on results of a literature review compiling outcomes of ruxolitinib in steroid-refractory chronic graft-versus-host disease (cGVHD).

A retrospective cohort study by Yang et al, which comprised 36 patients with steroid-refractory cGVHD, found that 27.8% of patients had complete disappearance of disease symptoms, and 52.7% of patients had partial response and symptom relief after being treated with ruxolitinib. Patients were treated with ruxolitinib 2.5 mg to 10 mg twice daily, tapering 1 week after symptoms had improved.

In a phase III trial conducted by Zeiser et al, 165 patients observed a 6.7% complete response rate as well as a 43% partial response rate associated with treatment with ruxolitinib 10 mg daily for 6 cycles at 28 days per cycle.

A pilot prospective study conducted by Mozo et al included 12 pediatric patients with steroid-refractory cGVHD who were also treated with ruxolitinib 2.5 mg to 10 mg twice daily. Complete response of symptoms was seen in 8.3% of patients, and a partial response occurred in 82.7% of patients.

In a pilot prospective study conducted by Morozova et al, a pre-transplant dose of ruxolitinib 45 mg daily was distributed to 20 patients with primary or secondary myelofibrosis. The drug was administered from 7 days before transplant to 2 days before transplant and was then tapered down to 15 mg from day 5 to day 100 post-transplant. Partial response was observed in 47.1% of patients at 6 months.

The most common all-grade adverse events that occurred throughout the 4 studies include mucositis, liver toxicity, and infections, among others. Overall, ruxolitinib has been found to be an effective and well-tolerated treatment for steroid-refractory cGVHD, according to the authors.

Kovalenko I, Saleem T, Laek B, et al. Ruxolitinib in chronic steroid-refractory graft-versus-host-disease. Abstract #e19053. Presented at the 2022 American Society of Clinical Oncology Annual Meeting; June 3-7, 2022; Chicago, IL