Ruxolitinib Cream Shows Strong Efficacy in Max-Use Pediatric Trial

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Atopic dermatitis (AD) affects approximately 10% of children, yet conventional topical treatments, including corticosteroids and calcineurin inhibitors, have limitations such as skin thinning and potential systemic adverse events.^1,2 Ruxolitinib cream, a selective Janus kinase (JAK) 1/2 inhibitor, has shown efficacy and safety in adult and adolescent populations with mild to moderate AD.³ This article discusses findings from a maximum-use trial (MUsT) evaluating the safety, tolerability, pharmacokinetics (PK), and efficacy of 1.5% ruxolitinib cream in children aged 2 to 11 years with extensive, moderate to severe AD.⁴

“The results from this MUsT support the role of ruxolitinib cream as a well-tolerated and effective topical treatment for children aged 2–11 years with AD, including for those with extensive, moderate to severe disease, consistent with phase 3 results in patients ≥ 2 years of age with AD,” researchers behind the study wrote.

Background

The study recognized AD’s chronic nature, and the limitations of existing treatments underscore the need for novel therapies. JAK signaling pathways are pivotal in mediating inflammation, itch, and skin barrier dysfunction, making them a viable therapeutic target.⁵ Ruxolitinib cream has demonstrated favorable outcomes in earlier studies, providing rapid symptom relief and maintaining disease control with long-term use.³

Study Design and Methods

The open-label study enrolled children aged 2 to 11 years with moderate to severe AD (≥35% body surface area [BSA] affected). Patients underwent a 4-week maximum-use period with continuous twice-daily (BID) application of 1.5% ruxolitinib cream, followed by a 4-week treatment-extension phase for active lesions only. Patients achieving ≤20% BSA involvement at week 8 entered a long-term safety (LTS) phase with as-needed application through week 52. Assessments included safety (primary endpoint), pharmacokinetics, efficacy, and quality-of-life (QoL) measures.

Results

A total of 29 patients participated, with a median age of 5 years. Mean baseline BSA involvement was reported to be 58%, with 82.8% of patients having prior topical treatment experience. Researchers stated most patients (96.6%) completed the 4-week maximum-use period, and 63.6% completed the LTS phase.

The study found ruxolitinib cream was well tolerated, with treatment-emergent adverse events (TEAEs) reported in 31% of patients, none of which were serious or led to treatment discontinuation. The most common TEAEs were upper respiratory infections, gastrointestinal symptoms, and application site reactions. Importantly, researchers stated no adverse events of special interest related to systemic JAK inhibition were observed.

According to the study results, plasma concentrations of ruxolitinib during the maximum-use period were below the threshold for JAK-mediated myelosuppression, even in patients with high BSA involvement (>50%). Mean daily cream usage decreased substantially during the LTS phase, correlating with reduced lesion burden.

Researchers reported significant reductions in affected BSA, with mean involvement decreasing from 58% at baseline to 2.2% by week 52. Rapid improvements in Investigator’s Global Assessment and Eczema Area and Severity Index (EASI) scores were achieved, with 84% of patients attaining EASI-75 at week 8. Itch relief, as measured by the numerical rating scale, was reported to be rapid and sustained, with a median time to significant improvement of 4 days. The study stated QoL measures, including sleep, depressive symptoms, and family impact, showed substantial improvement from baseline, sustained throughout the study.

Discussion

Researchers found ruxolitinib cream demonstrated consistent safety and efficacy in pediatric patients with extensive AD. The trial’s novel design, including long-term safety evaluation, supports its use as an effective nonsteroidal option for this challenging population. Minimal systemic absorption and the absence of significant safety signals enhance its appeal for broader application.

Conclusion

This study reinforces the potential of ruxolitinib cream as a safe, well-tolerated, and effective therapy for pediatric AD, addressing an unmet need in the management of this chronic disease. Researchers suggested future studies with larger cohorts may further validate these findings and explore extended indications.

References

1. Langan SM, Irvine AD, Weidinger S. Atopic dermatitis [published correction appears in Lancet. 2020 Sep 12;396(10253):758. doi: 10.1016/S0140-6736(20)31825-0]. Lancet. 2020;396(10247):345-360. doi:10.1016/S0140-6736(20)31286-1
2. Wollenberg A, Barbarot S, Bieber T, et al. Consensus-based European guidelines for treatment of atopic eczema (atopic dermatitis) in adults and children: part II. J EurAcad Dermatol Venereol. 2018;32(6):850-878. doi:10.1111/jdv.14888
3. Papp K, Szepietowski JC, Kircik L, et al. Efficacy and safety of ruxolitinib cream for the treatment of atopic dermatitis: Results from 2 phase 3, randomized, double-blind studies. J Am Acad Dermatol. 2021;85(4):863-872. doi:10.1016/j.jaad.2021.04.085
4. Stein Gold L, Bissonnette R, Forman S, et al. A maximum-use trial of ruxolitinib cream in children aged 2–11 years with moderate to severe atopic dermatitis. Am J Clin Dermatol. 2025. doi:10.1007/s40257-024-00909-5
5. Huang IH, Chung WH, Wu PC, Chen CB. JAK-STAT signaling pathway in the pathogenesis of atopic dermatitis: An updated review. Front Immunol. 2022;13:1068260. Published 2022 Dec 8. doi:10.3389/fimmu.2022.1068260