A new study has revealed that a pill taken once a day helped restore skin color in some people with vitiligo, an autoimmune condition that causes white patches to appear on the skin.
This development may provide an easier treatment option, especially for people with extensive patches or in areas that are difficult to reach with topical creams, as the pill works throughout the body rather than targeting specific patches.
Vitiligo occurs when the immune system mistakenly attacks the cells responsible for skin pigmentation. Although the condition is not contagious, the visible patches can significantly impact the lives and mental health of those affected, potentially leading to social stigma, anxiety, and exclusion. Therefore, restoring skin color goes beyond cosmetic concerns to improving quality of life. Current treatments include creams and light therapy, which can help restore color but require repeated treatments over several months. Furthermore, applying creams can be challenging when vitiligo covers large areas.
The new study, published in The Lancet, tested the drug upadacitinib on 614 adults and adolescents (aged 12 and older) in two clinical trials conducted at 138 institutions in 18 countries. All participants had the most common form of vitiligo, affecting both the face and body. They were randomly assigned to receive either 15 mg of the drug or a placebo daily for 48 weeks, without either the participants or the researchers knowing who was receiving the actual treatment
The results showed that in the first trial, 25% of patients who took the drug regained at least 75% of their lost facial skin color, compared to only 6% in the placebo group. The second trial replicated almost identical results (23% versus 7%). Color also returned to other parts of the body, with 19% and 21% of patients in the two trials, respectively, regaining at least half of the lost color in the affected areas, compared to 6% in each placebo group.
Some patients experienced even stronger results, with 14% of those treated regaining at least 90% of their lost facial color, compared to 2-3% in the placebo groups. Even after the trials concluded, skin color continued to return, suggesting that longer treatment may offer additional benefits.
Upadacitinib works by suppressing part of the immune response that causes vitiligo, potentially reducing the attack on healthy skin cells and giving affected areas a chance to regain their color. The drug belongs to the JAK inhibitor class, which has also been studied for other conditions where an overactive immune system damages the skin. Because the pill works throughout the body, it may be particularly beneficial for people with widespread vitiligo or patches in areas where creams are difficult to apply, and it may be more convenient than frequent visits to clinics for phototherapy.
The European Union has approved the use of upadacitinib for eligible adults and adolescents (aged 12 and over) with vitiligo, with different approval statuses in other regions.
However, significant uncertainties remain, as approximately three-quarters of patients did not achieve the primary goal of facial improvement, and about 80% did not regain half of the lost body pigmentation. The most common side effects included upper respiratory infections, acne, headaches, and nasal and throat irritation. Seven patients developed shingles, and serious health problems were reported in 4% of patients in the first trial and 2% in the second. The 48-week period was insufficient to rule out rare or long-term risks, as the drug already carries warnings regarding serious infections, cancer, heart problems, and blood clots when used for other conditions.
The participants are followed up for an additional 112 weeks, which may reveal the extent of further possible improvement, and it remains unclear whether the restored color will remain after treatment is stopped.
The trials compared the pill to a placebo, not to current creams or light therapy, and most participants were white with limited representation of people with dark skin.
The study was funded by AbbVie, the drug manufacturer, which helped design the trials, analyze the data, and participate in preparing the research paper.
