Radboudumc Center for Infectious Diseases, Department of Medical Microbiology, Radboud University Medical Center, Nijmegen, The Netherlands.
Insmed Incorporated, Bridgewater, NJ, US.
Expert Rev Respir Med. 2021 Nov;15(11):1387-1401. doi: 10.1080/17476348.2021.1987891. Epub 2021 Oct 18.
Nontuberculous mycobacterial (NTM) lung disease (LD) is the most common clinical manifestation of NTM infection and is a growing health concern. Up to 85% of NTM-LD cases are caused by complex (MAC). Increased awareness of NTM-LD caused by MAC is needed as patients with this disease experience substantial burden and unmet treatment needs.
This review provides clinicians and regulatory and healthcare decision makers an overview of the clinical, economic, and humanistic burden of NTM-LD and the unmet treatment needs faced by patients and clinicians. The review focuses on NTM-LD caused by MAC. A summary of the 2020 NTM guidelines specifically for MAC-LD and an overview of novel treatment options, including amikacin liposome inhalation suspension (ALIS) as the first approved therapy for refractory MAC-LD, and investigational drugs in testing phase are provided.
Key advancements in NTM-LD management include recent updates to clinical practice guidelines, approval of ALIS for the treatment of refractory MAC-LD, and ongoing clinical trials of investigational treatments. Yet opportunities still exist to improve patient outcomes, including development of better screening tools, such as reliable and responsive biomarkers to help identify high-risk patients, and addressing unmet treatment needs.
非结核分枝杆菌(NTM)肺部疾病(LD)是 NTM 感染最常见的临床表现,也是一个日益严重的健康问题。高达 85%的 NTM-LD 病例是由复杂(MAC)引起的。由于患有这种疾病的患者会承受巨大的负担和未满足的治疗需求,因此需要提高对 MAC 引起的 NTM-LD 的认识。
这篇综述为临床医生以及监管和医疗保健决策者提供了 NTM-LD 的临床、经济和人文负担以及患者和临床医生面临的未满足治疗需求的概述。该综述重点介绍了 MAC 引起的 NTM-LD。概述了 2020 年 NTM 指南中专门针对 MAC-LD 的内容,并概述了新型治疗选择,包括阿米卡星脂质体吸入混悬液(ALIS)作为治疗难治性 MAC-LD 的首个获批疗法,以及处于测试阶段的研究性药物。
NTM-LD 管理方面的主要进展包括临床实践指南的最新更新、ALIS 获批用于治疗难治性 MAC-LD 以及正在进行的研究性治疗临床试验。然而,仍有机会改善患者的预后,包括开发更好的筛选工具,如可靠和敏感的生物标志物,以帮助识别高危患者,并解决未满足的治疗需求。