Case Study Points to Potential New Treatment for Patients with Vitiligo

Patients with vitiligo have a choice of many treatment options, which run the gamut from applying make-up to depigmented areas, to light treatment, all the way up to surgery to move pigmented skin cells to affected areas. Yet, many patients elect not to treat the condition at all, despite the fact that the condition can be emotionally devastating and progressive.

The choice to not treat at all is due, in part, to the fact that many of the current treatments can be ineffective or temporary at best, and potentially harmful to the skin at worst. Light therapy, for example, has been shown to be effective in up to 70% of patients in clinical research trials, but within a year, almost half of those patients see the results disappear.

A recent case study in JAMA Dermatology offers some hope that citrate, an oral Janus kinase 1/3 inhibitor (JAK inhibitor), may result in significant repigmentation. JAK inhibitors work by blocking cytokine signaling that can result in certain types of cancer and inflammatory diseases such as rheumatoid arthritis. Recent research work has suggested that tofacitinib shows promise in conditions including plaque psoriasis and alopecia universalis.

“Recent advances in the understanding of the pathogenesis of vitiligo suggest that Janus kinase inhibitors may be a therapeutic option [for vitiligo],” the authors note.

A woman in her 50s who had vitiligo on her face and hands for approximately one year had tried other therapies — including ointments and narrowband UV-B phototherapy. She indicated no family history of vitiligo. Given the recent advances in the understanding of vitiligo, the clinicians opted to try tofacitinib citrate at a dosage of 5 mg every other day. After 3 weeks, the dosage was increased to 5 mg per day. Partial repigmentation occurred within 3 months of therapy and was nearly complete after 5 months of therapy. No adverse effects were noted.

Although the results of the trial are encouraging, the study authors noted a few limitations, including the short duration of the patient’s condition and the fact that, although uncommon, tofacitinib can present serious adverse effects, including malignant disease. Beyond providing some evidence for an additional treatment for vitiligo patients, the case study is another example of the promising direction of medical treatments based on understanding clinical pathways.

“To our knowledge, this report is the first to demonstrate effective pathogenesis-based therapy for a patient with vitiligo,” the case study authors noted. “As we better understand the pathomechanisms of different diseases, targeted therapy becomes possible, and existing medications can be repurposed and/or new medications created for diseases with limited, if any, treatment options.”