Iznardo Helena, Roé Esther, Vicente Asunción, Prat Carolina, Casals Miquel, Martín-Santiago Ana, Esteve Altea, Viñas Miguel, Munera-Campos Mónica, Corella Francesca, Mollet Jordi, Figueras-Nart Ignasi, Vila Aina, Soria Xavier, Azón-Masoliver Antoni, Marqués-Martín Laura, Nadal-Lladó Cristina, Bel Susana, Pujol-Montcusí Josep, Bertolín-Colilla Marta, Curto-Barredo Laia, Melé-Ninot Gemma, Evole Montserrat, Berbegal Laura, Puig Lluís, Baselga Eulàlia
Department of Dermatology, Hospital de la Santa Creu i Sant Pau, IR SANT PAU, Universitat Autònoma de Barcelona, Barcelona, Spain.
Department of Dermatology, Hospital Sant Joan de Déu, Esplugues de Llobregat, Spain.
Clin Exp Dermatol. 2024 Dec 23;50(1):104-112. doi: 10.1093/ced/llae300.
Moderate-to-severe atopic dermatitis (AD) can be difficult to manage in paediatric patients, and there are few licensed treatments available for this age group. Dupilumab is approved for the treatment of AD in children older than 6 months.
To assess the effectiveness and safety of dupilumab in a real-world cohort of paediatric patients with AD in Spain.
A multicentre, retrospective real-world study on the effectiveness and safety of dupilumab in patients aged 2-18 years with moderate-to-severe AD was conducted. Demographic and clinical characteristics were analysed, and effectiveness (Eczema Area and Severity Index, Investigator's Global Assessment, Dermatology Life Quality Index, Numerical Rating Scale itch), safety and drug survival measures were assessed. A comparison of our results with other real-world outcomes and with clinical trials was made.
Data from 243 patients from 19 centres were collected, with a mean follow-up of 85 weeks. Dupilumab exhibited significant effectiveness, with marked reductions in severity scores from week 4. By week 16, 79.4% of patients achieved ≥ 75% improvement in Eczema Area and Severity Index (EASI) score (EASI 75) and 40.5% achieved ≥ 90% improvement in EASI score (EASI 90). Mean percentage reduction in EASI was 79.7%. Increasing improvements were observed until week 52, with 85.8% and 49.6% of patients achieving EASI 75 and EASI 90, respectively. Forty-three patients developed adverse events (AEs) (43 of 243, 17.7%). The most frequent AEs were ocular surface diseases (20 of 243, 8.2%), injection site reactions (8 of 243, 3.3%) and facial redness (7 of 243, 2.9%). Drug survival was high (96.9% and 93.1% after 1 and 2 years of follow-up, respectively), with only 19 patients (7.8%) interrupting treatment: 7 (2.9%) owing to AEs; 2 (0.82%) owing to secondary failure; 5 (2.1%) were lost to follow-up; and 5 (2.1%) entered remission and stopped treatment.
Real-world use of dupilumab in paediatric AD demonstrated sustained effectiveness, high drug survival and acceptable safety profiles. Longer-term studies are crucial for AE surveillance and understanding how to manage disease remission.
中重度特应性皮炎（AD）在儿科患者中难以管理，且该年龄组可用的获批治疗方法很少。度普利尤单抗被批准用于治疗6个月以上儿童的AD。
评估度普利尤单抗在西班牙儿科AD患者真实队列中的有效性和安全性。
开展了一项关于度普利尤单抗在2至18岁中重度AD患者中的有效性和安全性的多中心、回顾性真实世界研究。分析了人口统计学和临床特征，并评估了有效性（湿疹面积和严重程度指数、研究者整体评估、皮肤病生活质量指数、数字评定量表瘙痒程度）、安全性和药物留存率指标。将我们的结果与其他真实世界结果及临床试验结果进行了比较。
收集了来自19个中心的243例患者的数据，平均随访85周。度普利尤单抗显示出显著疗效，从第4周起严重程度评分显著降低。到第16周时，79.4%的患者湿疹面积和严重程度指数（EASI）评分改善≥75%（EASI 75），40.5%的患者EASI评分改善≥90%（EASI 90）。EASI平均降低百分比为79.7%。直到第52周改善情况持续增加，分别有85.8%和49.6%的患者达到EASI 75和EASI 90。43例患者发生了不良事件（AE）（243例中的43例，17.7%）。最常见的AE是眼表疾病（243例中的20例，8.2%）、注射部位反应（243例中的8例，3.3%）和面部发红（243例中的7例，2.9%）。药物留存率较高（随访1年和2年后分别为96.9%和93.1%），只有19例患者（7.8%）中断治疗：7例（2.9%）因AE；2例（0.82%）因继发治疗失败；5例（2.1%）失访；5例（2.1%）进入缓解期并停止治疗。
度普利尤单抗在儿科AD中的真实世界应用显示出持续的有效性、高药物留存率和可接受的安全性。长期研究对于AE监测以及了解如何管理疾病缓解至关重要。
