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The Latest on a Novel Topical Therapy for Vitiligo

David Rosmarin, MD, shares the results of recent studies into ruxolitinib cream for the treatment of vitiligo. A recent Lancet report,1 of which Dr Rosmarin was the principal investigator, was named a finalist for the Top 10 Clinical Research Achievement Awards from the Clinical Research Forum.2 

Dr Rosmarin is the vice chair of research and education in the department of dermatology at Tufts Medical Center in Boston, MA.



Reference
1. Rosmarin D, Pandya AG, Lebwohl M, et al. Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial. Lancet. 2020;396(10244):110-120. doi:10.1016/S0140-6736(20)30609-7

2. Clinical Research Forum presents Moderna and Pfizer with award for extraordinary impact on health. Press release. Clinical Research Forum; March 30, 2021. Accessed April 22, 2021. https://www.businesswire.com/news/home/20210330005777/en/Clinical-Research-Forum-Presents-Moderna-and-Pfizer-With-Award-for-Extraordinary-Impact-on-Health

Transcript
Dr Rosmarin:  Our next study, we evaluated different doses of ruxolitinib cream for repigmenting vitiligo. The primary result is that about half the patients on the two highest doses of ruxolitinib cream achieved 50% or more repigmentation on the face after 6 months compared to only 3% on a control vehicle, which had no active medication.

Patients started to see repigmentation rapidly as early as 8 weeks. With longer use, patients saw more benefit. At the 1-year mark, slightly more than half the patients on the highest dose of ruxolitinib cream achieved 75% or more repigmentation on the face. Furthermore, we know ruxolitinib cream doesn't just work on the face. When evaluating patients who had up to 20% of their body affected by vitiligo, almost half the patients achieved 50% or more repigmentation of their body.

In terms of safety, the medication's well tolerated. There can be some mild redness at the site of applying the cream. Also, they can get acne, which is usually mild. Overall, ruxolitinib cream holds significant promise as a treatment.

Vitiligo affects up to 1% of the world's population, and there are estimated to be over 1 million people in the US that suffer with vitiligo. Despite how common it is, there's not a single medicine that is FDA approved to repigment vitiligo.

I want to emphasize that vitiligo is not a cosmetic issue but a medical disease. It can have a high impact on one's quality of life. Ruxolitinib cream is in a phase 3 trial right now, where we will hopefully have some initial results in several months.

As a topical that has a very favorable safety profile, I'm optimistic ruxolitinib cream will be the first treatment that will be FDA-approved to repigment vitiligo. If a patient wants to repigment their disease, they will hopefully be able to do so.

At the AAD VMX, there's some more exciting data that shows continued improvement of patients up to two years after starting on ruxolitinib cream and repigmenting the face and total body. The longer we treat, the better the response seems to be.

Additionally, the safety profile is looking good through 2 years as well. When we look at different subgroups, even patients who had vitiligo for more than 20 years are still repigmenting well. We also are seeing robust responses from our younger patients.

In a study evaluating different anatomical locations, even in difficult-to-treat areas, such as the hands and feet, we're seeing some responses with ruxolitinib cream monotherapy. What would I say to my colleagues? Help is on the way.

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