Ruxolitinib demonstrates efficacy in patients with refractory sclerotic chronic graft-versus-host disease (cGVHD), according to a study published in the Journal of Clinical Oncology. The phase 2 trial (NCT03616184) evaluated the safety and efficacy of ruxolitinib in patients with sclerotic cGVHD who had failed prior systemic therapies. Sclerotic cGVHD is often associated with significant morbidity and resistance to standard treatments.
The multicenter, single-arm trial enrolled 47 patients with a median age of 62 years. The majority (60%) were male, and a significant proportion had undergone myeloablative conditioning prior to transplant (47%). Most patients (85%) had severe cGVHD, and the median number of prior therapies was 3. Patients received 10 mg of ruxolitinib orally twice daily for at least 6 months.
Efficacy and Outcomes
After a median follow-up of 11 months, 49% (95% CI, 34-64) of patients achieved a partial response in skin and/or joints at 6 months. Specifically, 45% had a joint and fascia response, while 19% had a skin response. At 12 months, 77% (95% CI, 48-91) of these patients maintained an ongoing response. The overall partial response rate for cGVHD was 47% (95% CI, 32-61).
The 12-month failure-free survival rate was 77.1% (95% CI, 61.3-87), and the nonrelapse mortality rate was 2.2% (95% CI, 0.17-10.3). These results suggest that ruxolitinib can provide meaningful clinical benefit in a population with limited treatment options.
Safety and Tolerability
Ruxolitinib was generally well-tolerated, with no new safety signals identified. Common adverse events included diarrhea (11%), nausea (19%), vomiting (13%), and fatigue (15%). Serious adverse events (grade 3 or higher) occurred in 40% of patients, including lung infection and hypertension. One-third of patients reported improvement in symptom burden.
Clinical Implications
The study authors suggest that ruxolitinib represents an effective treatment option for sclerotic cGVHD, addressing a significant unmet need. They also propose that future studies should explore the use of ruxolitinib and other novel agents, such as belumosudil or axatilimab, earlier in the treatment course to potentially improve outcomes. "Until such studies are conducted, on the basis of the results of this trial, single agent ruxolitinib offers an effective option for sclerotic cGVHD, thus meeting a hitherto unmet need," the authors concluded.
