Ruxolitinib Cream Demonstrates Long-Term Safety and Disease Control in Young Patients With Atopic Dermatitis

Ruxolitinib cream maintained disease control and demonstrated similar safety profiles over 52 weeks in children aged 2-6 years and 7-11 years with atopic dermatitis, with patients spending nearly half of the long-term treatment period off medication due to lesion clearance, according to results from the phase 3 TRuE-AD3 study.

Weilly Soong, MD

Researchers, led by Weily Soong, MD, medical director of the Clinical Research Center of Alabama, in Birmingham, reported no cases of folliculitis, herpes zoster, major cardiovascular events, malignancy, or thrombosis among the 282 pediatric patients who completed the long-term safety evaluation. More than 70% of participants in both age subgroups achieved clear or almost clear skin Investigator’s Global Assessment (IGA) of 0 or 1 at week 52, while mean affected body surface area remained at approximately 2% or less.

Findings were presented as a poster during the Revolutionizing Atopic Dermatitis meeting, June 6-7, 2025, in Nashville, TN.

The TRuE-AD3 study enrolled children aged 2-11 years with mild to moderate atopic dermatitis in a randomized, double-blind, vehicle-controlled trial. Investigators randomly assigned the children 2:2:1 to apply ruxolitinib cream twice daily at concentrations of 0.75%, 1.5%, or vehicle for 8 weeks of continuous treatment, followed by 44 weeks of as-needed treatment.

In the long-term safety-evaluable population, the median age was 7.0 years (range 2-11), with 49.3% aged 2-6 years and 50.7% aged 7-11 years. At baseline, participants had a mean affected body surface area of 10.4%, and 77.0% presented with IGA score of 3.

Participants initially assigned to the 1.5% ruxolitinib cream group spent substantial periods off treatment as a result of lesion clearance. Children aged 2-6 years remained off treatment for a mean of 155.7 days (SD 88.8, range 2-309), while those aged 7-11 years stayed off treatment for 130.7 days (SD 88.5, range 7-288). Soong and colleagues reported similar results in children receiving 0.75% cream.

The study protocol required participants to self-evaluate lesion recurrence between visits and treat active atopic dermatitis affecting ≥20% body surface area. They discontinued treatment 3 days after lesion disappearance and contacted investigators if new lesions became extensive.

Among those initially assigned to receive ruxolitinib cream, disease control as measured by IGA 0/1 either maintained or improved during the as-needed treatment period in both age subgroups. Children who crossed over from vehicle treatment generally achieved similar disease control levels as those initially randomized to ruxolitinib cream.

Safety analysis revealed both ruxolitinib cream concentrations were well tolerated with few application site reactions. Treatment-emergent adverse events (TEAEs) occurred in 42 participants (56.0%) aged 2-6 years receiving 0.75% cream and 52 of those (63.4%) receiving 1.5% cream. Among 7-11 year-olds, 38 participants (45.2%) experienced TEAEs with 0.75% cream and 40 (55.6%) with 1.5% cream.

The most common TEAEs included upper respiratory tract infection, affecting 12 participants (16.0%) aged 2-6 years and 10 participants (11.9%) aged 7-11 years receiving 0.75% cream, and 11 (13.4%) aged 2-6 years and 12 participants (16.7%) aged 7-11 years receiving 1.5% cream. Nasopharyngitis occurred in 2 participants (2.7%) and 7 participants (8.3%) in the respective age groups with 0.75% cream, and 12 (14.6%) and 8 (11.1%) with 1.5% cream.

There were few application site reactions, according to the poster, affecting 4 participants (5.3%) aged 2-6 years and 3 (3.6%) in the 7-11 year age group with 0.75% cream, and 5 (6.1%) and 2 participants (2.8%) respectively with 1.5% cream. TEAEs occurred in 9-10 patients (12.0-12.2%) aged 2-6 years and 4-7 patients (5.6-8.3%) aged 7-11 years across both concentrations.

Soong et al reported no serious TEAEs during the 52-week study and identified no new safety signals. Mean plasma concentrations of ruxolitinib remained well below levels associated with myelosuppression. The research team concluded that ruxolitinib cream's disease control and safety profiles in both pediatric age groups were similar to data previously reported in adults and adolescents with mild to moderate atopic dermatitis.