571 - Case series of topical 1.5% ruxolitinib cream for pediatric vitiligo
Authors: Nanette Silverberg, Harry M Meister, Mark Lebwohl
Journal: British Journal of Dermatology (2024)

Abstract Introduction/Background Pediatric Vitiligo affects 1.52% of 4-11-year-olds within the United States and 2.16% of 12-17-year-olds with non-segmental disease affecting 65% and 69% respectively (1). The FDA has not approved any medications for vitiligo for children younger than 12 years of age. Additionally, about one-third of pediatric vitiligo is segmental (1), and there is no FDA-approved topical agent for segmental vitiligo in any pediatric age group (2). Objectives To identify the efficacy of 1.5% ruxolitinib cream for pediatric vitiligo in clinical practice. Methods An IRB-exempted pediatric chart review was conducted addressing the usage of 1.5% ruxolitinib cream, currently FDA-approved for non-segmental vitiligo affecting 10% of body surface area or less for 12 years of age or older. All children and adolescents using topical 1.5% ruxolitinib cream including segmental, non-segmental, and mixed vitiligo including individuals up to age 17 years. Results Twelve children were identified with topical usage of 1.5% ruxolitinib cream for vitiligo ages 3-16 years (average age: 10.75 years). Demographics included 9 males, 3 females and race/ ethnicity included Black (n=1), Hispanic/ Latin X (n=4), Indian (n=1), and White (n=6). Vitiligo subtype included: Non-segmental vitiligo (n=7), Mixed type vitiligo (n=1), and segmental vitiligo (n=4). Regarding treatment subgroup of Non-segmental vitiligo and the non-segmental component of the Mixed type included 8 children, average age of 10.4 years (range 3-15 years), including 5 children under the age of 12 years (one 3-year-old, two 9 year-olds, and two 10 year-olds). Treatment was once daily in 1 child and twice daily in 7 children. One was lost to follow-up. Of the 6 children with documented response, average maximum repigmentation occurred at 6.14 months (range 3-12 months), with five achieving complete repigmentation, one partial repigmentation with only a three-month trial. The average length of disease before treatment began was 1.25 years (1 month to 6 years). The onset of response was 1.57 months on average (range 1-3 months). The initial location of response was the face (n=3), arms (n=2), and abdomen (n=1). No adverse events were reported. Four children had a complete blood count while on medication, with no laboratory abnormalities, including the 3-year-old. Two had a parent with vitiligo. Four patients previously failed tacrolimus topically, two patients failed topical class 2 corticosteroids, and one previously failed narrowband UVB, with no notable difference in response in these patients. Regarding treatment subgroup of Segmental vitiligo including five SV patients average age 11.2 years (range 5-16 years). One had facial segmental, one face and neck lesion (solitary stripe), one chest/back, and one on the lower abdomen. The five year-old did not repigment after 6 months of neck application, but never developed poliosis. The ten-year-old with segmental disease for 6 years had no response. The eleven-year-old SV of the chest/ back failed topical tacrolimus and responded with 40% repigmentation over 3 months, increasing to 90% after 5 months and 12 concurrent sessions of 308-nm laser. The 14-year-old developed slight poliosis, but started repigmenting within 3 weeks, with continuous usage and subtotal repigmentation at 14 months. The 16-year-old began to repigment after 1 month, with a concurrent weekly 308-nm laser, and subtotal repigmentation after 5 months. Repigmentation was One facial SV (12 treatments), one chest/back SV (12 treatments), and one NSV had concurrent 308-nm laser (24 treatments) with pulsed dexamethasone, without any notable adverse events. Two teenage males (ages 15 and 16 years) developed acne on sites of application on the face. Laboratory screening including complete blood count was normal during treatment for 2 patients evaluated. Conclusions NSV responds to 1.5% topical ruxolitinib consistently, with a solitary patient responding to once daily, and efficacy in children as young as three years of age with no notable lab abnormalities in screened patients. Topical 1.5% ruxolitinib cream appears very effective in repigmenting SV in children if used twice-daily and in combination with 308-nm laser sessions in the first 6 months. Topical ruxolitinib can cause acne in teenagers with facial segmental vitiligo. Early institution of topical 1.5% ruxolitinib cream appears to have a rapid onset of repigmentation (average 1.57 months) and rapid complete response (6.14 months). Clinical trials of 1.5% topical ruxolitinib in children are needed to identify outliers and long term outcomes. Early institution of therapy appears to speed up the response. Adjunctive 308-nm laser is promising but requires placebo-controlled trials.