Ruxolitinib Demonstrates Efficacy as Treatment for Refractory Sclerotic Chronic Graft-Versus-Host Disease

Ruxolitinib was associated with relatively high rates of skin/joint responses and overall chronic graft-versus-host disease (GVHD) responses, including improvement in patient-reported outcomes, low non-relapse mortality (NRM), and high failure-free survival (FFS) among patients with refractory sclerotic chronic GVHD, according to results from a phase 2 trial.

“Sclerotic chronic graft-versus-host disease represents a highly morbid and refractory form of c[hronic] GVHD, and novel therapies for sclerotic c[hronic] GVHD are critically needed,” said Vijaya Raj Bhatt, MS, MBBS, University of Nebraska Medical Center, Omaha, Nebraska, and colleagues.

In this single-arm, multicenter phase 2 trial, 47 adults with sclerotic chronic GVHD refractory to corticosteroids and ≥1 additional line of systemic therapy for chronic GVHD received ruxolitinib for ≥6 months. Investigators noted the primary end point was defined as complete or partial response (PR) in skin and/or joint defined according to the 2014 National Institute of Health chronic GVHD Consensus Criteria.

Following ruxolitinib use for a median of 11 months, study results demonstrated a PR in skin and/or joints of 49% (95% [confidence interval] CI, 34 to 64) at 6 months, with 45% of patients having joint and fascia response and 19% having skin response. Additionally, the duration of skin/joint response was 77% (95% CI, 48 to 91) at 12 months. Overall, a chronic GVHD PR was noted in 47% (95% CI, 32 to 61) of patients. Improvement in Lee Symptom Scale summary and skin subscale scores was also measured in 38% of patients. With a cumulative incidence of treatment failure of 20.8% (95% CI, 10 to 34.1), NRM of 2.2% (95% CI, 0.17 to 10.3), and no recurrent malignancy, study authors determined that FFS was 77.1% (95% CI, 61.3 to 87) at 12 months.

“The use of ruxolitinib was associated with relatively high rates of skin/joint responses and overall c[hronic] GVHD responses, improvement in patient-reported outcomes, low NRM, and high FFS in patients with refractory sclerotic c[hronic] GVHD,” concluded Dr Bhatt and colleagues.

“Ruxolitinib offers an effective treatment option for refractory sclerotic c[hronic] GVHD,” they added.

Source:

Bhatt V, Shostrom V, Choe H, et al. A multicenter phase 2 trial of ruxolitinib for treatment of corticosteroid refractory sclerotic chronic graft-versus-host disease. Published online August 16, 2024. doi: 10.1200/JCO.24.00205