Clinical Studies Supporting Ruxolitinib Cream for Frontline Vitiligo Treatment
Dr Rosmarin describes the impacts of ruxolitinib on the treatment strategies for vitiligo.
David Rosmarin, M.D.: Let’s review the TRuE-V1 and TRuE-V2 phase 3 program, which evaluated ruxolitinib cream 1.5% twice a day compared with vehicle in repigmenting patients with vitiligo. Adolescents and adults with 10% or less vitiligo on their body but at least a half a hand’s worth of vitiligo on their face and 3% of vitiligo on their body enrolled in the study. The study had a 2:1 ratio of patients on ruxolitinib cream to vehicle. The primary end point was at six months, evaluating patients for F-VASI75, which is 75% or more repigmentation on the face, which is a meaningful outcome to patients.
The reason it’s designed such a way is because it’s important to have a vehicle control when we’re evaluating the efficacy of a medication, and the face is earlier to respond than the body. It’s hard to keep a patient on a vehicle for more than six months. It’s also important to evaluate how well it works on the body, which is an important key secondary end point. But because vitiligo repigmentation takes a long time and we want to best evaluate that efficacy end point, we use F-VASI75 as the primary end point. Key secondary end points are how many patients are achieving facial repigmentation at the year mark, as well as whole-body repigmentation.
In that study, the average surface area for patients with vitiligo was about 7%. We had patients of all ages and duration. Most of the patients had previously tried other treatments, such as calcineurin inhibitors, corticosteroids, and phototherapy. With the key end point, a third of patients hit F-VASI75 at six months. But the important part is that at the year mark, half the patients achieved that end point, because repigmentation takes time. Half the patients in the study achieved 50% or more repigmentation on their whole body at the year mark. I want to emphasize that this is monotherapy. This is ruxolitinib cream on its own, not in combination, where it may even work better and faster.
In terms of safety profile, the medicine was very well tolerated. Six percent of patients get acne, which is usually mild in nature, and less than 2% of patients stopped the treatment for adverse events. Some patients get a little itching or redness at the site, but we don’t have any burning sensations, which we commonly see with calcineurin inhibitors. The safety profile looks quite good. Laboratory values were checked, and there were no clinically meaningful changes for our patients. In clinical practice, most of us don’t check laboratory values for patients we’re initiating on ruxolitinib cream.
When we do subgroup analysis from this study—races, ethnicities, ages, duration of disease—all respond similarly well on the face, and there was about 30% response for all those different demographics. In alopecia areata, it’s different. If a patient has alopecia areata for more than 10 years, it becomes harder to treat. That isn’t true in vitiligo. Patients who have the disease for over 50 years can still get a response. We see good responses for adolescents, especially on the body, but all age groups can get that same primary end point of facial repigmentation. It’s helpful for us to counsel patients and for insurance companies to understand that we’re targeting a broad swath of the population. There shouldn’t be limits based on those subgroups.
There’s a difference in response depending on anatomic site. The face is easiest to repigment, followed by the trunk and extremities. But the acral sites, the hands and the feet, are harder to repigment. We can see some responses, but it’s much more challenging. If a patient comes in and has vitiligo only on their fingertips, and they aren’t going to be happy unless they get all their pigment back, I advise them that maybe this isn’t the right treatment for them, and that that’s going to be challenging to do.
Transcript edited for clarity.
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