Abstract: Nontuberculous mycobacteria (NTM) can cause and perpetuate chronic inflammation and lung infection. Despite having the diagnostic criteria, as defined by the American Thoracic Society (ATS) and Infectious Diseases Society of America (IDSA), clinicians find it challenging to diagnose and treat NTM-induced lung disease. Inhaled antibiotics are suitable for patients with lung infection caused by Pseudomonas aeruginosa and other organisms, but until recently, their utility in NTM-induced infection was not established. The most common NTM pathogens identified are the slow-growing Mycobacterium avium complex (MAC) and the rapid-growing M. abscessus complex (MABSC), both of which include several subspecies. Other less commonly isolated species include M. kansasii, M. simiae, and M. fortuitum. NTM strains are frequently more resistant than what is found in bacterial sputum cultures. Until recently, there was no approved inhaled antibiotic therapy for patients who were culture positive for pulmonary NTM infection. Of late, inhaled amikacin has been under investigation for the treatment of NTM-induced pulmonary infection. The FDA approved Arikayce (amikacin liposome inhalation suspension or ALIS) based on results from the ongoing Phase 3 CONVERT trial. In this study, the use of Arikayce met its primary endpoint of sputum culture conversion by the sixth month of treatment. The addition of Arikayce to guideline-based therapy led to negative sputum cultures for NTM by month 6 in 29% of patients compared to 8.9% of patients treated with guideline-based therapy alone. The effectiveness of Arikayce holds promise. However, due to limited data on Arikayce’s safety, it is currently useful only for a specific population, particularly patients with refractory NTM-induced lung disease. Future trials must verify the target group and endorse the clinical benefits of Arikayce.

Keywords: Arikayce, amikacin liposome inhalation suspension, nontuberculous mycobacterium, Mycobacterium avium complex, Mycobacterium abscessus complex, aminoglycosides


中文翻译：

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阿米卡星脂质体吸入混悬液（Arikayce）在治疗成人难治性非结核分枝杆菌肺病中的应用。


摘要：非结核分枝杆菌（NTM）可引起并使慢性炎症和肺部感染持续存在。尽管有美国胸科学会 (ATS) 和美国传染病学会 (IDSA) 定义的诊断标准，但临床医生发现诊断和治疗 NTM 引起的肺部疾病具有挑战性。吸入性抗生素适用于铜绿假单胞菌和其他微生物引起的肺部感染患者，但直到最近，其在 NTM 引起的感染中的效用尚未确定。最常见的 NTM 病原体是生长缓慢的鸟分枝杆菌复合体 (MAC) 和快速生长的脓肿分枝杆菌复合体 (MABSC)，这两种病原体都包括多个亚种。其他不太常见的分离物种包括M. kansasii、M. simiae和M. fortuitum 。 NTM 菌株通常比细菌痰培养物中发现的菌株具有更强的抵抗力。直到最近，还没有批准用于肺部 NTM 感染培养阳性患者的吸入抗生素治疗。最近，人们正在研究吸入阿米卡星用于治疗 NTM 引起的肺部感染。 FDA 根据正在进行的 3 期 CONVERT 试验的结果批准了 Arikayce（阿米卡星脂质体吸入混悬液或 ALIS）。在这项研究中，使用 Arikayce 在治疗第六个月时达到了痰培养转化的主要终点。将 Arikayce 添加到基于指南的治疗中，到第 6 个月时，29% 的患者的 NTM 痰培养结果为阴性，而仅接受基于指南的治疗的患者为 8.9%。 Arikayce 的有效性值得期待。 然而，由于 Arikayce 安全性的数据有限，目前它仅对特定人群有用，特别是患有难治性 NTM 引起的肺部疾病的患者。未来的试验必须验证目标群体并认可 Arikayce 的临床益处。


关键词： Arikayce、阿米卡星脂质体吸入混悬液、非结核分枝杆菌、鸟分枝杆菌复合体、脓肿分枝杆菌复合体、氨基糖苷类