Ruxolitinib Cream Dose Safe, Effective in Children With Atopic Dermatitis, Vitiligo

Children and adolescents with atopic dermatitis (AD) and vitiligo were found to have significant responses to 1.5% ruxolitinib cream in phase 3 trials, according to posters presented at the Society for Pediatric Dermatology (SPD) Annual Meeting held in Toronto, Ontario, from July 11 to July 14, 2024.

According to the American Academy of Dermatology,¹ AD is a skin condition that usually starts in childhood. AD can cause dry, itchy, and inflamed skin. Although AD can often fade in a patient’s teenage years, many patients live with the condition for the rest of their life. Because of its prevalence among children and adolescents, treatment for the disease can focus on these demographics in particular.

Investigating the safety of ruxolitinib cream in patients aged 2 to 11 years was the aim of one poster presented at the annual meeting.² This poster presented findings from the TRuE-AD3 phase 3 study, a randomized, double-blind, vehicle-controlled study that highlighted the hematologic laboratory safety results of 1.5% ruxolitinib cream applied twice per day in children with mild to moderate AD. Children were included if they had a diagnosis of AD for 3 months or longer, had an Investigator’s Global Assessment score of 2 or 3, and had body surface area of 3% to 20% affected by AD.

Ruxolitinib 1.5% was effective and safe for children with vitiligo and atopic dermatitis | Image credit: ChayTee - stock.adobe.com

The 330 participants were randomized 2:2:1 into ruxolitinib 0.75%, ruxolitinib 1.5%, and vehicle groups. These participants were encouraged to take the treatment for 8 weeks with hematologic assessments at baseline, week 2, and week 8, and plasma concentration analysis done at week 2 or 4 and week 8.

The researchers found that, after 8 weeks of treatment, there were no clinically meaningful changes in the hematologic parameters of the children. Age subgroups also had no significant changes from baseline. The steady-state ruxolitinib concentration and mean changes in hemoglobin, neutrophil, or platelet counts from baseline were found not to have any correlation.

The researchers concluded that hematologic laboratory changes that were indicative of systemic Janus kinase (JAK) inhibition were found in these patients and that limited plasma concentrations of ruxolitinib persisted in children with AD aged 2 to 11 years.

Ruxolitinib 1.5% can likewise help adolescents who are diagnosed with vitiligo. Vitiligo is a skin condition that causes the patient to lose color and pigmentation in their skin.³ This can occur in small spots or can cause widespread loss of color. The most common types of vitiligo are nonsegmental, which is when patches of color loss occur on both sides of the body, and segmental, where color loss occurs on 1 side or part of the body. Nonsegmental is the more common of these 2 types.³

The TRuE-V1 and TRuE-V2 phase 3 studies were double-blind, randomized studies conducted in patients 12 years and older with nonsegmental vitiligo,⁴ which aimed to assess the efficacy of ruxolitinib 1.5% on facial vitiligo. Participants were encouraged to apply either a ruxolitinib 1.5% dose or a vehicle dose twice per day for 24 weeks before all patients were given the ruxolitinib 1.5% dose.

A poster presented at the SPD Annual Meeting relayed the results of a post hoc analysis of the TRuE-V1 and TRuE-V2 trials.⁵ The analysis focused on the efficacy data from the TRuE-V studies to evaluate rates of complete or near-complete repigmentation by adolescents’ and adults’ body regions. The Vitiligo Area Scoring Index (VASI) was used to evaluate repigmentation, with thresholds of at least 50%, at least 75%, at least 90%, and 100%. This study was specifically focused on facial VASI100 (F-VASI100), as well as total VASI75 (T-VASI75) and total VASI90 (T-VASI90). VASI50 was used to assess 6 body regions, excluding the face.

The post hoc analysis found that patients 12 years and older with nonsegmental vitiligo had significant repigmentation when applying 1.5% ruxolitinib cream twice per day compared with patients who applied the vehicle through 24 weeks. This finding was also observed through week 52 of the trial when measured through the VASI. Adolescents had a higher rate of complete facial repigmentation compared with adults when both applying 1.5% ruxolitinib cream after 24 weeks (5.7% vs 2.9%), but this separation decreased by week 52 (8.0% vs 8.0%).

More adolescents had a greater response for T-VASI75 at week 24 (13.2% vs 5.6%) and week 52 (22.0% vs 20.3%) compared with adults. T-VASI90 also had a greater response in adolescents at week 24 (3.8% vs 0.3%) and week 52 (12.0% vs 4.0%). VASI50 was also more likely to be achieved in adolescents within 52 weeks for the lower extremities (67.3% vs 51.8%) and feet (37.5% vs 27.9%) compared with adults.

The researchers noted that, based on this research, adolescents were more likely to achieve VASI50 for lower extremities and feet as well as repigment faster. However, the population of adolescents in this analysis was small and shorter disease duration could have been a confounding factor, which indicates future studies into this topic should be considered.

Both of these posters demonstrate both the safety and efficacy of 1.5% ruxolitinib cream both in children aged 2 to 11 years diagnosed with AD and in adolescents 12 years and older who are diagnosed with vitiligo.

References

1. Ludmann P. Eczema types: atopic dermatitis overview. American Academy of Dermatology Association. Updated October 10, 2023. Accessed July 11, 2024. https://www.aad.org/public/diseases/eczema/types/atopic-dermatitis
2. Paller AS, Stein Gold LF, Simpson EL, et al. Hematologic laboratory safety of ruxolitinib cream in children aged 2 to 11 years with mild to moderate atopic dermatitis: results from a randomized, double-blind, vehicle-controlled phase 3 study (TRuE-AD3). Presented at: Society for Pediatric Dermatology Annual Meeting; July 11-July 14, 2024; Toronto, Ontario, Canada. Abstract 17.
3. Ludmann P. Vitiligo: overview. American Academy of Dermatology Association. Updated June 29, 2022. Accessed July 11, 2024. https://www.aad.org/public/diseases/a-z/vitiligo-overview
4. Rosmarin D, Passeron T, Pandya AG, et al. Two phase 3, randomized, controlled trials of ruxolitinib cream for vitiligo. N Engl J Med. 2022;387:1445-1455. doi:10.1056/NEJMoa2118828
5. Gooderham MJ, Grimes P, Passeron T, et al. Adolescents with vitiligo achieved higher rates of more complete repigmentation than adults: post hoc analysis from the phase 3 TRuE-V studies of ruxolitinib cream. Presented at: Society for Pediatric Dermatology Annual Meeting; July 11-July 14, 2024; Toronto, Ontario, Canada. Abstract 4.