At the 65th American Society of Hematology (ASH) Annual Meeting in San Diego, California, Alina Markova, MD, Dermatology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York, shared results from the first study to characterize the effect of topical JAK1/2 blockade on cutaneous chronic graft-versus-host disease (GVHD).

Results demonstrated that ruxolitinib 1.5% cream was safe and effective compared to placebo in treating cutaneous nonsclerotic and superficially sclerotic GVHD.

Transcript:

Hi, I am Alina Markova. I'm a dermatologist at Memorial Sloan Kettering Cancer Center, and I'm thrilled to be here at ASH 2023 to present the final results of my investigator-initiated study on ruxolitinib cream for chronic cutaneous GVHD. This study is examining a topical [Janus kinase] (JAK) ½ inhibitor for the safety and efficacy of treating chronic cutaneous GVHD in patients who had received an allogeneic stem cell transplantation. What we did in this study is we enrolled 24 patients and we randomized 1 to 1 to assign a split face/body application of a cream. In half of the patients, patients applied a ruxolitinib cream to the left side of their face or body, and in the other half, they applied a vehicle cream.  The other group had the contralateral application. The opposite side received placebo cream and the opposite side received ruxolitinib cream. 

This way, any patients who were on other systemic medications, the effect we were able to account for that effect because patients had an internal control. What we looked at was the day 28 outcome of applying the ruxolitinib cream to one side of the body and the vehicle cream to the other side of the body. What we found in our primary outcome, which was body surface area, was that [on] day 28, patients had significantly reduced body surface area in the ruxolitinib-treated side versus the vehicle-treated side. This effect was seen starting from day 14. We also found other scales that we used that were physician's global assessment, scale of improvement also, that were statistically significantly improved starting from day 14 to day 28, and another scale called the Kale Scale, which looked at erythema scale and body surface area and itch also improved from day 14 and 28 in the ruxolitinib treated arm versus the placebo arm. Furthermore, we looked at skin tape stripping to look at RNA analysis in the skin, and we were able to detect differences in signaling of PD1 and TH17 in the treated and untreated arms, and then differences in signaling in IL12 in the responder and non-responder arms. So, very interesting. We really need this study to be verified in a larger fashion, prospectively. We look forward to using these results to treat more patients with chronic cutaneous GVHD.

Source:

Markova A, Papadopoulos E, Dusza S, et al. Topical ruxolitinib for chronic cutaneous GvHD: Promising results of a phase 2 clinical trial. Presented at the ASH 65th Annual Meeting & Exposition; December 9-12 2023; San Diego, California. Abstract 777