Allergy Unit, Department of Pediatrics, Anna Meyer Children's University Hospital, Viale Pieraccini 24, 50139, Florence, Italy.
Post-Graduate School of Pediatrics, Department of Health Sciences, Anna Meyer Children's University Hospital, Viale Pieraccini 24, 50139, Florence, Italy.
Ital J Pediatr. 2019 Nov 28;45(1):151. doi: 10.1186/s13052-019-0737-4.
Severe asthma has a substantial epidemiological impact on children and biological treatments can be an option to take into account, as they target specific molecules and pathways involved in its pathogenesis. Modern medicine is continuously and progressively oriented towards tailored treatments designed specifically for the pathology patterns observed in individual patients and identified as endotypes with associated biomarkers. In this regard, biologic treatments in asthma are one of the best examples. Among the biological drugs currently available, omalizumab is the one with the greatest amount of data on efficacy and safety, and the one we have more real-life clinical experience with. However, mepolizumab will likely be accessible soon globally for clinical use. Moreover, research on biological drugs for the treatment of severe asthma is expanding rapidly, with some molecules currently used in adult patients that could be registered also for pediatric use and new molecules that could be available in the future. On the other hand, due to this potential abundance of therapeutic options, new criteria could become necessary to guide clinicians through an evidence-based choice between omalizumab and these new drugs. For the same reason, more data collected specifically from pediatric clinical trials are necessary. In this review we aim to analyze the factors that could help clinicians make their choice and to highlight the unmet need for a more evidence-based choice.
严重哮喘对儿童有重大的流行病学影响,生物治疗可以作为一种考虑方案,因为它们针对的是其发病机制中涉及的特定分子和途径。现代医学不断地朝着针对个体患者观察到的病理模式和确定为相关生物标志物的特定内型的个体化治疗方向发展。在这方面,哮喘的生物治疗是最好的例子之一。在目前可用的生物药物中,奥马珠单抗是在疗效和安全性方面数据最多的药物,也是我们在现实临床中拥有更多经验的药物。然而,美泊利珠单抗可能很快在全球范围内可用于临床使用。此外,用于治疗严重哮喘的生物药物的研究正在迅速扩展,一些目前用于成年患者的分子可能也会被注册用于儿科使用,未来可能会有新的分子问世。另一方面,由于治疗选择的可能性如此丰富,可能需要新的标准来指导临床医生在奥马珠单抗和这些新药之间进行基于证据的选择。出于同样的原因,有必要从儿科临床试验中收集更多专门的数据。在这篇综述中,我们旨在分析有助于临床医生做出选择的因素,并强调需要更基于证据的选择来满足未满足的需求。