MINNEAPOLIS — The first data from a retrospective cohort registry that evaluated disease manifestations, comorbidities, and treatments in 351 children with vitiligo suggest that responses to common therapies used alone or in combination differ in head and neck vs trunk involvement and in segmental vs nonsegmental disease.

"We need prospective studies for confirmation, but the registry data are helping us to address some of the unknowns about treatment of vitiligo when evaluated by subtype, location, and other variables," said Nanette Silverberg, MD, chief of pediatric dermatology for Mount Sinai Health Systems in New York City.

There are four soon-to-be-published papers coming from the data generated from the registry, but Silverberg provided a preview of the initial findings at the Society for Pediatric Dermatology (SPD) 2026 Annual Meeting.

The good news is that sustained rates of clearance are achieved in the majority of patients treated long-term, but the bad news is that optimal responses might not be reached for 3 or more years, according to Silverberg. The registry data also suggest that there are several groups, particularly those with evidence of poliosis, who do considerably less well. 

Poliosis Is Adverse Risk Factor for Vitiligo 

"Poliosis is a risk factor for never repigmenting," said Silverberg, who estimated that poor response to common therapies for vitiligo is several times higher in patients with poliosis relative to those without. 

In one head and neck substudy of 202 patients aged 1-18 years with adequate follow-up to compare responses, the 163 patients with nonsegmental vitiligo typically did better on therapy than the 39 with segmental disease, with a mean body surface area (BSA) vitiligo reduction at 12 months of nearly 90% in the former group and 55.0% in the latter.

In this substudy, which was summarized in a poster at the meeting as well as cited in Silverberg's oral presentation, topical ruxolitinib was only offered to 27 of the 202 patients because it had recently been approved for treating vitiligo. There was a signal that ruxolitinib improved control in patients with an inadequate response to topical tacrolimus, which was the most common agent employed, but the small patient number prevented confirmation. 

Conversely, topical corticosteroids did not appear to provide a substantial further reduction in BSA when added to tacrolimus, but again the numbers were small. Even fewer patients received both topical ruxolitinib and corticosteroids on top of tacrolimus, but this combination signaled a cumulative advantage over time. The BSA reduction climbed to 74.0% at 24 months from 51.0% at 12 months. All eight patients in this subgroup had nonsegmental vitiligo. 

When segmental and nonsegmental disease involvement in the head and neck were compared, the majority — 62% in each group — had facial involvement. The clinical significance of variations in involvement at other sites was greater for segmental disease in the eyelids (42% vs 31%) but lower for the lips/perioral area (26% vs 33%) and scalp (10% vs 18%) is not yet clear.

Excimer Laser Improves Response in Vitiligo 

In the same substudy for head and neck vitiligo, treatment with excimer laser produced mean reductions in BSA that were high relative to tacrolimus alone, but excimer laser plus additional agents did not appear to provide a further incremental benefit, according to Silverberg, who reported that more details will be presented in the papers already submitted for publication.

For body involvement, unlike head and neck vitiligo, the addition of topical corticosteroids to topical tacrolimus did provide a reduction in BSA whether measured at 6 months (47.3% vs 25.0%) or 12 months (50.0% vs 29.2%). There was also a further gain when excimer laser was added to tacrolimus alone at 6 months (56.7% vs 25.0%) and 12 months (66.7% vs 29.2%).

The relationship between vitiligo and thyroid disease, a potentially modifiable risk factor, was also evaluated in the registry and will be the subject of one of the four upcoming publications. Due to the frequency with which thyroid disease is present in children with vitiligo, Silverberg said thyroid screening should be a routine part of workup.

"If you are only testing for [thyroid stimulating hormone], you will be missing half of the kids who have a risk factor for severe disease," she said.

One of the messages from the registry data is that response to treatment is slow but cumulative, meaning the patients and families must be provided with realistic expectations even if the majority of children can expect a good clinical result.

"In the head and neck, it can take 3 months or more to see anything happen and the maximum response might not be seen for 36 months," said Silverberg, noting that repigmentation is still progressing on combination therapies 2 or more years after treatment is started.

She said that a short course of pulse steroids can help, but "we can no longer keep children on steroids in this moment in time when we have other options." 

Vitiligo Typically Requires Combination Therapy 

Optimizing therapies by ensuring adequate vitamin D, adding topical to oral agents, offering narrow band phototherapy, and adding excimer laser or even skin grafting when indicated can all be part of algorithm to work toward clear or almost clear skin over the course of treatment, according to Silverberg.

"You need to do as much as you can as quickly as you can," she said, meaning that therapy should be routinely escalated when response is inadequate. But she also urged patience. 

"Take photographs so you can assess progress with the patient," said Silverberg, suggesting that although progress is gradual "you will eventually get there in most patients."

Relative to single agent trials, the registry data are expected to be a valuable source of information on the natural history of the disease among real-world patients receiving multiple therapies. Senior author of the 2024 vitiligo pediatric guidelines, Silverberg expects these data to inform the next guideline deliberations. 

One of the coauthors of the 2024 pediatric guidelines, Lawrence F. Eichenfield, MD, chief of pediatric and adolescent dermatology, Rady Children's Hospital, and professor of dermatology and pediatrics, University of California, San Diego, agreed. He is optimistic that registry data can be part of the effort to better understand how to prioritize therapeutic strategies.

"Registries are very different from randomized trials in that they can provide practical information about strategies used clinically in patients that have not been rigorously screened for a formal study," Eichenfield told Medscape Medical News at the meeting. "They provide different types of information, but both can be helpful."

Based on the preliminary results, Eichenfield believes that the publications generated by the registry, which are expected within the next several months, will have a clinical impact.

Silverberg reported financial relationships with AbbVie, Alphyn, Incyte, Pelthos, Pfizer, Regeneron/Sanofi, Verrica, and Zensoderm. Eichenfield reported financial relationships with AbbVie, Acrotech, Almirall, Amgen, Apogee Therapeutics, Arcutis, Attovia, BMS, Castle Biosciences, CorEvitas, Dermavant, Dermira, Forte Biosciences, Galderma, Incyte, Janssen, Johnson & Johnson, LEO Pharma, Lilly, Novartis, Ortho Dermatologics, Pfizer, Regeneron, Sanofi-Genzyme, Target RWE, TRex Bio, and UCB. 

Ted Bosworth, a career medical writer based in New York City, has been covering advances in clinical medicine, including dermatology and oncology, for several decades.