PDF(Pubmed)
Abstract：
The increasing prevalence of Mycobacterium avium complex (MAC) pulmonary disease poses a significant therapeutic challenge, particularly due to the limited efficacy and systemic toxicity associated with conventional guideline-based therapy. Amikacin liposome inhalation suspension (ALIS) has been developed, yet its real-world application remains underreported. This retrospective analysis, conducted from March 2021 to February 2024, examined ALIS\'s clinical use in patients aged 20 years or older with refractory MAC pulmonary disease at our institution. The primary objective of this study is to describe the patient characteristics and clinical trajectories associated with the initiation of ALIS therapy in real-world settings for individuals diagnosed with MAC pulmonary disease. Of 11 patients initiated on ALIS, one was excluded due to financial constraints impacting continuation. The analysis proceeded with the remaining 10 subjects. The mean age of participants was 70.2 years, with a predominance of female patients (n = 7, 70%) and a higher incidence of M. avium infections (n = 6, 60%). Forty percent of the cohort (n = 4) had a history of ethambutol-induced optic neuritis leading to the cessation of the drug. The average interval from the initiation of guideline-based therapy to the start of ALIS was 8.5 ± 6.9 years (mean ± standard deviation). The majority (80%) presented with positive Gaffky scores at ALIS initiation, and a significant proportion exhibited resistance to clarithromycin and ethambutol. Comorbid conditions, including diabetes and previous cancer, were noted. The study also observed elevated anti-MAC antibody levels. Treatment duration varied, with fatigue leading to discontinuation in two cases. Treatment-emergent adverse events were documented in individual patients, each presenting with grade 1 severity: hemoptysis (n = 1, 10%), elevated creatinine levels (n = 1, 10%), and dysphonia (n = 2, 20%) were observed, respectively. Correlation analysis revealed a significant inverse relationship between body mass index (BMI) and ALIS discontinuation due to fatigue, and a positive correlation between Gaffky scores and C-reactive protein (CRP) levels. These results underscore the potential benefits and limitations of ALIS, suggesting that timely intervention and comprehensive healthcare support are crucial for optimal outcomes in the treatment of advanced MAC pulmonary disease.
摘要：
鸟分枝杆菌复杂（MAC）肺部疾病的患病率不断增加，构成了重大的治疗挑战。特别是由于与基于常规指南的治疗相关的有限疗效和全身毒性.阿米卡星脂质体吸入混悬液(ALIS)的研制,然而，它的实际应用仍然被低估。这项回顾性分析，从2021年3月至2024年2月进行，在我们机构检查了ALIS在20岁或20岁以上难治性MAC肺部疾病患者中的临床使用情况。这项研究的主要目的是描述与ALIS治疗在现实世界中启动相关的患者特征和临床轨迹诊断为MAC肺病的个体。在ALIS上开始的11名患者中，由于财务限制影响延续，其中一个被排除在外。对其余10名受试者进行分析。参与者的平均年龄为70.2岁,以女性患者为主（n=7，70％），鸟分枝杆菌感染的发生率较高（n=6，60％）。40％的队列（n=4）有乙胺丁醇诱导的视神经炎病史，导致药物停止。从开始基于指南的治疗到开始ALIS的平均间隔为8.5±6.9年（平均值±标准偏差）。大多数人(80%)在ALIS开始时给出了正的Gaffky分数，相当比例的人对克拉霉素和乙胺丁醇表现出耐药性。共病条件,包括糖尿病和以前的癌症，被注意到。该研究还观察到抗MAC抗体水平升高。治疗持续时间不同，疲劳导致两种情况下停药。在个别患者中记录治疗引起的不良事件，每个表现为1级严重程度：咯血（n=1，10%），肌酐水平升高（n=1，10%），并观察到发音障碍（n=2，20%），分别。相关分析显示，体重指数（BMI）与由于疲劳导致的ALIS停药之间存在显着的负相关关系，Gaffky评分与C反应蛋白（CRP）水平呈正相关。这些结果强调了ALIS的潜在好处和局限性，提示及时的干预和全面的医疗支持对于治疗晚期MAC肺病的最佳结果至关重要.
