Division of Pediatric Immunology, Stanford University School of Medicine, Stanford, California, USA.
Curr Opin Pediatr. 2018 Jun;30(3):366-371. doi: 10.1097/MOP.0000000000000614.
Although biologic therapies can provide outstanding efficacy in the management of lung disease, especially asthma, most of these agents have been approved only for adults. Recent findings provide new strategies for using these agents in children.
Extensive evidence has consistently demonstrated the efficacy and safety of biologic therapy for asthma. In addition, some studies have documented potentially important secondary effects, such as improving response to respiratory virus infection in asthmatic patients. Additional strategies for improving asthma control using biologic therapy, such as seasonal administration, have been suggested, and may limit cost while still providing a high degree of efficacy.
Many of the current biologics are able to readily establish control even in asthmatic patients for whom inhaled steroid and long-acting β agonist have failed. However, biologics currently have limited regulatory approval and availability in the pediatric age range, despite this age being disproportionately affected by asthma. In addition, successful biologics for asthma to date have largely been limited to the Th2-high endotype of asthma, and there is great need for similar medications to target the Th2-low endotype. Other pediatric lung disease might well benefit from the specificity allowed by biologic therapy.
尽管生物疗法在肺部疾病(尤其是哮喘)的治疗中具有显著疗效,但这些药物大多仅获得成人适应证。最近的研究结果为儿童使用这些药物提供了新的策略。
大量证据一致证实了生物疗法治疗哮喘的疗效和安全性。此外,一些研究记录了潜在的重要次要作用,例如改善哮喘患者对呼吸道病毒感染的反应。还提出了使用生物疗法改善哮喘控制的其他策略,例如季节性给药,这可能在保持高疗效的同时限制成本。
许多目前的生物制剂能够在吸入性类固醇和长效β激动剂治疗失败的哮喘患者中迅速控制病情。然而,尽管儿童哮喘的发病率不成比例地较高,但生物制剂目前在儿科年龄范围内的监管批准和可及性有限。此外,迄今为止,成功的哮喘生物制剂主要局限于 Th2 高表型哮喘,迫切需要针对 Th2 低表型哮喘的类似药物。其他儿科肺部疾病可能会受益于生物治疗的特异性。