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Vitiligo : Episode 3

First FDA-Approved Therapy for Repigmentation Is a Breakthrough for Vitiligo, Says Dr Brett King


Prior to this first therapy for repigmentation being approved, the only approved therapy was a depigmenting agent, which is usually the opposite of what providers are trying to accomplish with treatment.

While the diagnosis of vitiligo is fairly straightforward, identifying the goal of treatment can vary among patients, explained Brett King, MD, PhD, associate professor of dermatology, Yale School of Medicine.

The good news is there is finally an approved therapy for repigmentation, which represents a remarkable breakthrough in treatment. However, despite having an FDA-approved therapy, vitiligo is often considered a cosmetic condition, which can lead to issues with insurance coverage of treatment.

In an interview with The American Journal of Managed Care® (AJMC®), King also discussed the challenges of how long treatment can take at a time when treatment for other conditions show improvements much faster.

AJMC®: How do you approach diagnosing a patient with suspected vitiligo? Are there certain cases that are not straightforward when diagnosing?

King: The diagnosis is usually straightforward. We're looking for white patches of skin, a certain kind of characteristic distribution—that is usually straightforward. However, we sometimes have a little bit of doubt. There is certainly a differential diagnosis for vitiligo. We always want to be sure that somebody has actual depigmentation and not hypopigmentation. We rarely will entertain that somebody might have a condition called hypopigmented mycosis fungoides, which is a form of cutaneous T-cell lymphoma.

But, again, the vast majority of the time you look, you might use a Wood’s lamp to examine a patient, again, to help differentiate hypopigmentation from depigmentation. The diagnosis is fairly straightforward.

AJMC®: With the diagnosis straightforward, what are the goals of treatment for patients with vitiligo, and how are the responses to therapy defined?

King: This is somewhat complex, because there is the vitiligo that somebody has at a moment in time—the moment that we are meeting them. Some of those patients that we're meeting say, “These are the spots that I've had, and this is what it has been like.” Meaning it's been stable for some period of time: months or years. Then other people come in and say, “This is what it looks like today but a month ago, it wasn't as bad as today. And 6 months before that it wasn't as bad was a month ago.” They're progressing.

In those 2 cases, we have theoretically different goals. In the one case of the person with rapidly or progressing disease, the first goal is to halt disease progression and keep it from evolving further. Then, in everybody—both the first example and the second example—we want to repigment. We want to help people to get back the pigment that they've lost.

Broadly speaking, those are 2 goals: halt progression and then repigmentation. We may try to achieve each of those aims with different therapeutic modalities.

AJMC®: Could you please discuss the current treatment landscape for patients with vitiligo?

King: We are really coming from a time of, I would say, very limited treatment options for vitiligo. Indeed, up until the FDA approval of ruxolitinib 1.5% cream in the last few months, the only approved therapy for vitiligo was monobenzyl ether of hydroquinone, or MBEH, a depigmenting agent.

Imagine for the longest time, the one approved therapy was almost the opposite of what we're always trying to achieve in our patients with vitiligo. It was a depigmenting agent. That doesn't mean that we didn't have therapeutics that we used sometimes with success. Those therapies are phototherapy—in particular, narrowband ultraviolet B-phototherapy—and topical calcineurin inhibitors—for instance, pimecrolimus cream or tacrolimus ointment. Sometimes we use topical corticosteroids, as well. It's kind of amazing that, again, we've had nothing approved for repigmentation until ruxolitinib 1.5% cream, which signals a remarkable breakthrough in both our understanding of disease pathogenesis and also marking an era when we can say, “I know something about your disease, and we're not going to just randomly choose an agent because people say it works, but rather, we have clinical trials with hundreds of patients that shows exactly how well this medicine works. We're going to try to bring that medicine to the treatment of your condition.”

Those are the big discussion points for a kind of vitiligo called segmental vitiligo. Most of our discussion is around the most common form of vitiligo, which is nonsegmental vitiligo. For a form of vitiligo called segmental vitiligo, which is typically vitiligo affecting a small area of, typically, one side of the body, for some of those people, we will recommend a procedure called MKTP [melanocyte-keratinocyte transplantation], which is a procedure in which we take melanocytes from one part of the body and transplant them to the affected area sometimes with success.

Then, lastly, for patients with rapidly evolving or progressive vitiligo, for their treatment, to halt progression of their disease will sometimes use systemic corticosteroids. That really rounds out the treatment landscape for vitiligo treatment across the different kinds and folks with stable versus progressive disease.

AJMC®: What challenges are associated with these treatments, such as adverse events, treatment administration schedule, or out-of-pocket costs?

King: I think the biggest challenges for treatment are really that vitiligo takes a long time to treat. We have a little bit of a short attention span in dermatology. The biologics for psoriasis and our treatments for atopic dermatitis have only exacerbated our short attention span, because we can make psoriasis go away literally in 1 month or 2 months. We can make atopic dermatitis go away in weeks or a few months.

Vitiligo takes time. We cannot give somebody pigment throughout their entire epidermis in days or weeks. It is necessarily a months-long process to treat it. In my mind, this really is one of the biggest challenges—even therapies at work take time to work. We've struggled to even communicate among ourselves that this is a disease that takes a long time to treat and so often we as dermatologists are undertaking treatment failing to recognize that it takes time. If we don't appreciate that, we're not communicating that with our patients, and then our patients go home. We just gave them a medicine to make their disease better, but 4 weeks later, they're not better, and they're saying, “Obviously, this doesn't work.”

One of the big gaps in my mind is really that we all need a better understanding of disease pathogenesis, but also what it takes to make the disease better, including just the time scale required, the amount of time necessary to make the disease better. Of course, beyond that we would like medicines that work twice as well as the medicines that we have. We would like to be able to use combination therapy—narrowband ultraviolet B phototherapy plus topical calcineurin inhibitors or narrowband ultraviolet B phototherapy plus ruxolitinib 1.5% cream. There are barriers to that in terms of insurance approval, in terms of access to these treatment modalities. There are all the usual challenges with access and insurance.

One of the really important things to mention here is that with vitiligo, payers have failed to understand the significant burden of disease on patients. Insurance rarely questions that psoriasis merits treatment. Payers rarely question that atopic dermatitis merits treatment. But vitiligo is still often considered a cosmetic condition. I mentioned we would like to use narrowband UVB phototherapy plus ruxolitinib 1.5% cream. Well, if insurance is failing to recognize that this is a condition that merits treatment, we're certainly going to have a hard time getting patients everything that they need to get better.

There are some of the usual challenges of everything in medicine or dermatology, but then also some special considerations for vitiligo, specifically.

AJMC®: What additional barriers exist for providers and payers in the management of vitiligo? What are considerations in managing medical versus pharmacy benefits for the discussed treatments?

King: I’m hopeful that the approval of ruxolitinib 1.5% cream really is going to signal a market change in everything. All of the challenges that we just discussed, I hope are going to get better because now we have something that's well-studied—the benefits are clear, they're well-delineated, there are great statistics. It means that we all need to sit down at the table and have a conversation. Prior to ruxolitinib 1.5% cream, what I said, “Oh, no, really and truly phototherapy is great.” Somebody could say, “Well, show me the data?” Well, here's a study of 30 patients. What's the data that topical calcineurin inhibitors work? Here's a systematic review. That's not high-quality data.

I'm hopeful that now that we have an approved agent, everybody's going to sit down at the table. Payers are going to sit down and say, “Oh, geez, we have to contend with this disease. What does that mean? How many people have it? What's the cost of treatment? How are we going to manage this?”

There are the challenges that I mentioned earlier. Even challenges with a lot of my colleagues, a lot of dermatologists. I get denial letters for appeals that are being reviewed by my dermatology colleagues saying it's a cosmetic disease. Holy cow. If dermatologists are struggling to understand that this is not a cosmetic disease, well then how can we blame the payers? We can't blame the payers if we in the community aren't universally appreciating that it's a disease that merits treatment.

Again, I think that we're going to start breaking down these barriers now that we have an approved therapy and things are going to get better. Slowly, but surely, things are going to get better.

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