Caminiti Lucia, Galletta Francesca, Foti Randazzese Simone, Barraco Paolo, Passanisi Stefano, Gambadauro Antonella, Crisafulli Giuseppe, Valenzise Mariella, Manti Sara
Pediatric Unit, Department of Human Pathology in Adult and Developmental Age "Gaetano Barresi", University of Messina, Street Consolare Valeria 1, 98124 Messina, Italy.
Children (Basel). 2024 Jan 28;11(2):170. doi: 10.3390/children11020170.
Despite the increasing interest in biologics for the management of allergic diseases, sparse real-world data are still available in the pediatric population. This study aimed to evaluate the early real-life efficacy and safety of omalizumab for patients with moderate-to-severe asthma and chronic spontaneous urticaria (CSU), and Dupilumab for patients with moderate-to-severe atopic dermatitis (AD).
A prospective study enrolling children aged 6-18 years was designed to assess the efficacy and safety of biologic drugs at 16 weeks of treatment (T1). The effectiveness was measured using validated questionnaires (ACQ-5 for asthma, UAS7 for CSU, and EASI score for AD). Secondary outcome measures included reductions in inhaled corticosteroid (ICS) dosages, asthma-related hospitalizations/exacerbations, and quality of life (QoL) indicators (iNRS, sNRS, DLQI/cDLQI) for CSU and AD. Safety was expressed according to the descriptions of adverse events provided by EMA and FDA.
The study cohort consisted of eighteen children (mean age 12.9 ± 3.4 years). The omalizumab treatment significantly reduced ACQ-5 and UAS7 scores ( = 0.002 and < 0.001, respectively). In patients with asthma, decreased ICS dosage and hospitalization/exacerbation rates were observed. QoL parameters significantly improved in CSU and AD patients. No severe adverse events were reported for either treatment.
Our findings validate omalizumab and dupilumab as effective and safe therapeutic options for managing moderate-to-severe allergic diseases in children and adolescents.
尽管生物制剂在过敏性疾病管理中的应用越来越受到关注，但儿科人群的真实世界数据仍然稀少。本研究旨在评估奥马珠单抗治疗中重度哮喘和慢性自发性荨麻疹（CSU）患者，以及度普利尤单抗治疗中重度特应性皮炎（AD）患者的早期实际疗效和安全性。
设计了一项纳入6至18岁儿童的前瞻性研究，以评估生物药物在治疗16周（T1）时的疗效和安全性。使用经过验证的问卷（哮喘用ACQ-5、CSU用UAS7、AD用EASI评分）来衡量有效性。次要结局指标包括CSU和AD患者吸入性糖皮质激素（ICS）剂量的减少、哮喘相关住院/加重情况以及生活质量（QoL）指标（CSU和AD用iNRS、sNRS、DLQI/cDLQI）。根据欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）提供的不良事件描述来表达安全性。
研究队列由18名儿童组成（平均年龄12.9±3.4岁）。奥马珠单抗治疗显著降低了ACQ-5和UAS7评分（分别为 = 0.002和 < 0.001）。在哮喘患者中，观察到ICS剂量减少以及住院/加重率降低。CSU和AD患者的QoL参数显著改善。两种治疗均未报告严重不良事件。
我们的研究结果证实奥马珠单抗和度普利尤单抗是治疗儿童和青少年中重度过敏性疾病的有效且安全的治疗选择。
