Bhatt Recaps Trial of Ruxolitinib in Steroid Refractory Sclerotic cGVHD

Vijaya Raj Bhatt, MBBS, MS, associate professor & medical director of the leukemia program in the division of hematology & oncology at the University of Nebraska Medical Center, discusses background and findings from a multicenter, phase 2 study (NCT03616184) of ruxolitinib (Jakafi) for the treatment of patients with steroid refractory sclerotic chronic graft-versus-host disease (cGVHD).

According to Bhatt, the phase 2 trial met its primary end point as half of the adult patients with moderate-severe sclerotic cGVHD had partial responses, as well as overall cGVHD responses. Overall, ruxolitinib correlated with high rates of failure-free survival and results in a high rate of responses in this type of cGVHD.

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0:10 | We did a multicenter phase 2 trial using ruxolitinib for the management of sclerotic chronic graft-versus-host disease, which is a highly morbid form of chronic graft-versus-host disease. Eligible patients included adults with sclerotic chronic graft-versus-host disease who had refractory chronic graft-versus-host disease and had received either steroids for 12 months and 1 additional line of treatment, or steroids and 2 additional lines of treatment. Eligible patients were treated with ruxolitinib 10 mg twice a day for 6 months, and at the end of 6 months, we assessed response to treatment. Patients who had at least partial response and stable disease were allowed to receive ruxolitinib for an additional 6 months.

1:13 | We enrolled a total of 47 patients who received ruxolitinib and we saw a partial response of 49% at 6 months, a treatment failure rate of 15% at 6 months, and 22% at 12 months. We had 1 death which meant a non-relapse mortality of 2%. There were no relapses of underlying malignancies, overall survival was 98%, and failure-free survival was close to 80%.

1:54 | This means that we have demonstrated ruxolitinib is an effective treatment for sclerotic chronic graft-versus-host disease, and our response rate compared favorably with results of a prior trial that compared rituximab vs imatinib (Gleevec) and had a clinical benefit of 27%.