Stein Gold Linda, Bissonnette Robert, Forman Seth, Zaenglein Andrea, Kuo YuTzu, Angel Brett, Chen Xuejun, Kallender Howard, Paller Amy S
Henry Ford Health System, 1 Ford Pl, Detroit, MI, 48202, USA.
Innovaderm Research, Montreal, QC, Canada.
Am J Clin Dermatol. 2025 Mar;26(2):275-289. doi: 10.1007/s40257-024-00909-5. Epub 2025 Jan 6.
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.
芦可替尼乳膏已显示出抗炎和止痒活性，并且在一项针对2至11岁轻度至中度特应性皮炎（AD）患者的3期研究中耐受性良好。
本研究在最大使用条件和长期使用情况下，考察了芦可替尼乳膏的安全性、耐受性、药代动力学、疗效和生活质量（QoL）。
符合条件的患者年龄为2至11岁，患有中度至重度AD[研究者整体评估（IGA）评分为3 - 4]，且体表面积（BSA）受累≥35%。在为期4周的最大使用期内，患者每天两次将1.5%芦可替尼乳膏涂抹于所有基线确定的皮损上，然后仅在第52周前（从第8周起BSA受累≤20%的患者）涂抹于活动性皮损上。通过不良事件的频率和严重程度评估安全性。药代动力学参数作为次要终点进行评估，疗效和QoL作为探索性终点。
总体而言，共纳入29例患者（中位年龄5岁）。9/29例患者（31.0%）报告了治疗中出现的不良事件；研究期间未出现特别关注的不良事件（即无严重感染、恶性肿瘤、重大心血管不良事件或血栓形成）。最大使用期内的平均稳态血浆浓度低于已知的成人Janus激酶介导的骨髓抑制的半数最大抑制浓度。在第4周观察到的受累BSA和IGA的降低在按需使用至52周期间得以维持。患者报告结局和QoL指标的改善与疗效结果一致。
这些结果支持芦可替尼乳膏在患有AD的儿童（2至11岁）中的安全性，包括那些患有广泛性疾病的儿童，并且与先前的疗效研究结果一致。
NCT05034822，首次注册于2021年8月30日。
