Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which donor immune cells attack the recipient’s tissues. The graft cells recognize the recipient’s tissues as foreign, so they mount an immune response. GVHD can be acute (happening within a few months after the transplant) or chronic (developing several months later and can last a lifetime). Its severity varies based on transplant type, donor-recipient match, and conditioning regimen. Cutaneous GVHD refers to graft-versus-host disease affecting the skin. It is a common GVHD manifestation and may occur in either the acute or chronic form. People with cutaneous GVHD may experience an itchy red rash (that sometimes blisters) which begins on the palms or soles before spreading elsewhere. Skin texture changes, ulceration, and discoloration may also occur.

According to Josh Friedman of Healio, researchers presented on topical ruxolitinib for cutaneous GVHD during two separate meetings. The results came from a randomized Phase 2 clinical study and compared ruxolitinib with vehicle cream.

Evaluating the Study: Ruxolitinib for Cutaneous GVHD

Ruxolitinib is already approved as an oral formulation for both acute and chronic graft-versus-host disease. In topical form, ruxolitinib is approved for eczema and vitiligo. Researchers wanted to understand whether topical ruxolitinib could confer benefits for cutaneous GVHD. In the Phase 2 study, researchers evaluated ruxolitinib 1.5% cream. 24 individuals enrolled.

Individuals had one half of their body treated with vehicle cream and the other half with ruxolitinib. The findings show that:

• Ruxolitinib reduced affected body surface area by 57% by the 28th day of the study. This was compared to just 28% reduction when using vehicle cream.
• Additionally, ruxolitinib reduced the severity of cutaneous graft-versus-host lesions by 30%.
• Ruxolitinib was more effective in reducing lesions (size, scaliness, itchiness, redness) in people with acute leukemia and classic chronic graft-versus-host disease, and less effective in individuals with nonsclerotic cutaneous chronic graft-versus-host disease. These individuals often required additional systemic therapy.
• While some side effects occurred, they were relatively mild in nature. For example, one individual experienced a headache after treatment.