Liu Norrice M, Pijnenburg Mariëlle W, Deschildre Antoine, de Mir-Messa Ines, Adalen Sigve, Amat Flore, Antonino Luna, Biermé Priscille, Bravo-Lopez Maynor, Carlsen Karin C L, Carraro Silvia, Cros Pierrick, Delestrain Celine, Diaz Garcia Carolina, Epaud Ralph, Fenu Grazia, Ferraro Valentina A, Fleming Louise, Hanssens Laurence, Heine Anouk, Labouret Géraldine, Leoni Maria-Chiara, Licari Amelia, Lombardi Enrico, Lopez Neyra Alejandro, Marguet Christophe, Mazenq Julie, Pérez Tarazona Santiago, Díaz Juan Carlos Ramos, Schweitzer Cyril, Spada Elena, Valverde Molina José, Verhulst Stijn, Wanin Stéphanie, Rusconi Franca
Centre for Genomics and Child Health, Blizard Institute, Queen Mary University of London, UK.
Erasmus University Medical Centre, Rotterdam, The Netherlands.
ERJ Open Res. 2025 Jun 23;11(3). doi: 10.1183/23120541.00709-2024. eCollection 2025 May.
Real-world data on children with severe asthma is scarce. We report characteristics of children with severe asthma already on biologics, enrolled in the Severe Paediatric Asthma Collaborative in Europe, a clinical research collaboration of the European Respiratory Society.
We describe patient's characteristics including asthma control assessed with Global Initiative for Asthma (GINA) criteria, composite asthma severity index (CASI), exacerbations, unscheduled medical attendances, lung function and quality of life in children on biologic treatment because of severe asthma. We also assessed previous biologics use. Forced expiratory volume in 1 s, CASI, GINA, Paediatric Asthma Quality of Life Questionnaire score, exacerbations, unscheduled medical attendance and hospital admission comparisons in patients treated with different biologics were adjusted by age, sex and biologic therapy duration.
Among the 250 children (median age 13.2 years) recruited, 56.8% used omalizumab, 21.6% mepolizumab and 21.6% dupilumab. At enrolment, the dupilumab group was older (median 15.0 years), while the omalizumab group had been on biologic treatment the longest (median 622 days). Overall, 27% and 8% had partly controlled and uncontrolled asthma respectively, according to GINA. In the last 12 months, 52% and 29% had at least one and two exacerbations, respectively; airflow obstruction was found in 33%. 10% were admitted to hospital due to exacerbation. A previous switch from another biologic was recorded in 16%, predominantly due to nonresponse.
Most children on biologics obtained good symptom control, but many still experienced asthma attacks. Switching between biologics was substantial. There is still an unmet need in severe paediatric asthma.
关于重度哮喘儿童的真实世界数据稀缺。我们报告了已使用生物制剂的重度哮喘儿童的特征，这些儿童参与了欧洲重度儿科哮喘协作组，这是欧洲呼吸学会的一项临床研究合作项目。
我们描述了患者的特征，包括根据全球哮喘防治创议（GINA）标准评估的哮喘控制情况、综合哮喘严重指数（CASI）、急性加重、非计划就诊、肺功能以及因重度哮喘接受生物治疗的儿童的生活质量。我们还评估了既往生物制剂的使用情况。对接受不同生物制剂治疗的患者，在1秒用力呼气量、CASI、GINA、儿科哮喘生活质量问卷评分、急性加重、非计划就诊和住院情况进行比较时，根据年龄、性别和生物治疗持续时间进行了调整。
在招募的250名儿童（中位年龄13.2岁）中，56.8%使用奥马珠单抗，21.6%使用美泊利单抗，21.6%使用度普利尤单抗。入组时，度普利尤单抗组年龄较大（中位年龄15.0岁），而奥马珠单抗组接受生物治疗的时间最长（中位时间622天）。总体而言，根据GINA标准，分别有27%和8%的儿童哮喘部分控制和未控制。在过去12个月中，分别有52%和29%的儿童至少发生1次和2次急性加重；33%存在气流受限。10%因急性加重入院。记录到16%的患者曾从另一种生物制剂换药，主要原因是无反应。
大多数接受生物制剂治疗的儿童症状得到了良好控制，但仍有许多儿童经历哮喘发作。生物制剂之间的换药情况较为普遍。重度儿科哮喘仍存在未满足的需求。
