In a phase II study reported in the Journal of Clinical Oncology, Vijaya Raj Bhatt, MS, MBBS, and colleagues found that ruxolitinib produced responses in patients with corticosteroid-refractory sclerotic chronic graft-vs-host disease (GVHD) after transplantation for hematologic malignancies.
Vijaya Raj Bhatt, MS, MBBS
Study Details
In the U.S. multicenter study, 47 patients with sclerotic chronic GVHD refractory to corticosteroids and at least one additional line of systemic therapy for chronic GVHD received ruxolitinib at 10 mg twice daily for 6 months; those with partial response or better could have treatment extended for an additional 6 months. The primary endpoint of the study was complete or partial response in skin and/or joints according to the 2014 National Institutes of Health chronic GVHD Consensus Criteria.
Key Findings
The median duration of ruxolitinib treatment was 11 months. Chronic GVHD responses (all partial) in skin and/or joints were observed in 49% (95% confidence interval [CI] = 34%–64%) of patients at 6 months, with 45% having joint and fascia responses and 19% having skin responses.
At 12 months, the overall chronic GVHD partial response rate was 47% (95% CI = 32%–61%). Among responders, skin/joint response was maintained at 12 months in 77% (95% CI = 48%–91%).
At 12 months, improvement in Lee Symptom Scale summary and skin subscale scores was observed in 38% of patients. Cumulative incidence of treatment failure was 20.8% (95% CI = 10.0%–34.1%); nonrelapse mortality occurred in 2.2% of patients (95% CI = 0.17%–10.3%); no recurrent malignancies were observed; and failure-free survival was 77.1% (95% CI = 61.3%–87.0%).
The most common adverse events of any grade associated with ruxolitinib were nausea (19%), upper respiratory infection (17%), and fatigue (15%). Grade ≥ 3 adverse events occurred in 40% of patients, most commonly lung infection (9%) and hypertension (6%).
The investigators concluded: “The use of ruxolitinib was associated with relatively high rates of skin/joint responses and overall chronic GVHD responses, improvement in patient-reported outcomes, low nonrelapse mortality, and high failure-free survival in patients with refractory sclerotic chronic GVHD.”
Dr. Bhatt, of the Division of Oncology/Hematology, University of Nebraska Medical Center, Omaha, is the corresponding author for the Journal of Clinical Oncology article.
Disclosure: The study was supported by Incyte. For full disclosures of the study authors, visit ascopubs.org.
