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Ruxolitinib for treatment of sclerotic cGvHD: Results from a phase II study
By Ella Dixon
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Sclerotic chronic graft-versus-host (cGvHD) is a highly-refractory form of cGvHD associated with high morbidity.1 The biology of sclerotic cGvHD differs to other forms of cGvHD; however, there is a lack of clinical trials investigating treatments for sclerotic cGvHD. A multicenter phase II trial assessing the efficacy and safety of ruxolitinib in 47 adult patients with sclerotic cGvHD refractory to corticosteroid and other systemic therapies was published in the Journal of Clinical Oncology by Bhatt et al.1 |
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At 6 months, partial response in skin and/or joints was achieved in 49% of ruxolitinib-treated patients, with 45% showing a response in joints/fascia and 19% in the skin. |
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The overall partial response rate was 47%, with 77% of patients maintaining skin/joint response at 12 months. The failure-free survival rate at 12 months was 77.1%. |
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38% of patients showed improvement in the Lee Symptom Scale summary and skin subscale scores, reflecting enhanced symptom management. |
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Grade ≥3 adverse events occurred in 40% of patients, with 13% of these related to ruxolitinib. At 12 months, the cumulative incidence of non-relapse mortality and treatment failure was 2.2% and 20.8%, respectively. No cases of recurrent malignancy were observed. |
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These findings suggest that ruxolitinib was associated with high response rates and was well tolerated, offering a promising treatment option for refractory sclerotic cGvHD. Its favorable safety and efficacy profile may support its use in earlier treatment lines and in combination with other emerging therapies. |
- Bhatt VR, Shostrom VK, Choe HK, et al. A multicenter phase II trial of ruxolitinib for treatment of corticosteroid refractory sclerotic chronic graft-versus-host disease. J Clin Oncol. Online ahead of print. DOI: 10.1200/JCO.24.00205
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