A Maximum-Use Trial of Ruxolitinib Cream in Children Aged 2–11 Years with Moderate to Severe Atopic Dermatitis

Ruxolitinib cream has demonstrated anti-inflammatory and antipruritic activity and was well tolerated in a phase 3 study in patients aged 2–11 years with mild to moderate atopic dermatitis (AD). This study examined the safety, tolerability, pharmacokinetics, efficacy, and quality of life (QoL) with ruxolitinib cream under maximum-use conditions and with longer-term use. Eligible patients were aged 2–11 years with moderate to severe AD [Investigator’s Global Assessment (IGA) score 3–4], and ≥ 35% affected body surface area (BSA). Patients applied 1.5% ruxolitinib cream twice daily to all baseline-identified lesions during the 4-week maximum-use period, then to active lesions only up to week 52 (patients with ≤ 20% affected BSA from week 8). Safety was assessed by frequency and severity of adverse events. Pharmacokinetic parameters were assessed as secondary endpoints, and efficacy and QoL were exploratory endpoints. Overall, 29 patients (median age 5 years) were enrolled. Treatment-emergent adverse events were reported in 9/29 patients (31.0%); there were no adverse events of special interest (i.e., no serious infections, malignancies, major adverse cardiovascular events, or thromboses) during the study period. Mean steady-state plasma concentration during the maximum-use period was below the known half-maximal inhibitory concentration of Janus kinase–mediated myelosuppression in adults. Reductions in affected BSA and IGA observed at week 4 were sustained with as-needed use through 52 weeks. Improvements in patient-reported outcomes and QoL measures were consistent with efficacy results. These results support the safety of ruxolitinib cream in children (2–11 years) with AD, including those with extensive disease, and are consistent with previous efficacy findings. NCT05034822, first registered 30 August 2021. Ruxolitinib cream 1.5% is approved in the USA for patients aged ≥ 12 years for the treatment of mild to moderate atopic dermatitis (AD) involving ≤ 20% of the body, with recent studies supporting the safety and efficacy of ruxolitinib cream in younger children with mild to moderate AD. Maximum-use trials look at the safety of treatments applied to more extensive areas of skin, assessing potential for side effects. This maximum-use trial assessed safety, absorption, and effectiveness of 1.5% ruxolitinib cream when applied twice daily for 4 weeks in children aged 2–11 years with AD involving ≥ 35% of the body. Patients then applied ruxolitinib cream as needed for ≤ 52 weeks, and safety and disease control were assessed. During the first 4 weeks, 31% of patients reported side effects. Only one patient experienced treatment-related side effects at the application site. As-needed ruxolitinib cream did not cause any other side effects of concern. The average ruxolitinib blood level during the first 4 weeks was low. As expected with low ruxolitinib blood levels, no side effects associated with oral drugs of the same class (e.g., low levels of white blood cells, serious infections, cancers, major cardiovascular events, or blood clots) were seen. AD lesions decreased in size and there was relief of itching during the first 4 weeks of treatment, and the amount of ruxolitinib cream applied decreased thereafter. Disease control was maintained for ≤ 52 weeks with as-needed ruxolitinib cream. These findings help to confirm the safety and effectiveness of ruxolitinib cream in children.

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