Scottish Centre for Respiratory Research, University of Dundee, Ninewells Hospital and Medical School, Dundee, UK.
Lee Kong Chian School of Medicine, Nanyang Technological University, Singapore.
Lancet Respir Med. 2018 Sep;6(9):715-726. doi: 10.1016/S2213-2600(18)30053-5. Epub 2018 Feb 23.
European Respiratory Society guidelines for the management of adult bronchiectasis highlight the paucity of treatment options available for patients with this disorder. No treatments have been licensed by regulatory agencies worldwide, and most therapies used in clinical practice are based on very little evidence. Development of new treatments is needed urgently. We did a systematic review of scientific literature and clinical trial registries to identify agents in early-to-late clinical development for bronchiectasis in adults. In this Review, we discuss the mechanisms and potential roles of emerging therapies, including drugs that target airway and systemic inflammation, mucociliary clearance, and epithelial dysfunction. To ensure these treatments achieve success in randomised clinical trials-and therefore reach patients-we propose a reassessment of the current approach to bronchiectasis. Although understanding of the pathophysiology of bronchiectasis is at an early stage, we argue that bronchiectasis is a heterogeneous disease with many different biological mechanisms that drive disease progression (endotypes), and therefore the so-called treatable traits approach used in asthma and chronic obstructive pulmonary disease could be applied to bronchiectasis, with future trials targeted at the specific disease subgroups most likely to benefit.
欧洲呼吸学会成人支气管扩张症管理指南强调了该疾病患者可用的治疗选择有限。全球监管机构尚未批准任何治疗方法，而临床实践中使用的大多数疗法几乎没有任何证据支持。迫切需要开发新的治疗方法。我们对科学文献和临床试验登记处进行了系统回顾，以确定处于早期至晚期临床开发阶段的用于成人支气管扩张症的药物。在本综述中，我们讨论了新兴疗法的机制和潜在作用，包括靶向气道和全身炎症、黏液清除和上皮功能障碍的药物。为了确保这些治疗方法在随机临床试验中取得成功——并因此惠及患者——我们提出重新评估目前的支气管扩张症治疗方法。尽管对支气管扩张症的病理生理学的理解还处于早期阶段，但我们认为支气管扩张症是一种具有许多不同生物学机制的异质性疾病，这些机制驱动着疾病的进展（表型），因此，在哮喘和慢性阻塞性肺疾病中使用的所谓可治疗特征方法可能适用于支气管扩张症，未来的试验针对最有可能受益的特定疾病亚组。
