Introduction Nontuberculous mycobacteria (NTM) cause chronic, debilitating pulmonary disease. Patient-reported outcomes provide measures of symptoms, functioning and treatment response. Here we describe the preliminary validation of the recently developed NTM Module. Methods The study population included Northwest NTM Biobank patients in whom Mycobacterium avium complex (MAC) was isolated and who had ever met the 2007 American Thoracic Society/Infectious Diseases Society of America pulmonary disease criteria. The NTM Module was administered at enrolment and 12 months; a subset also completed the Quality of Life Questionnaire–Bronchiectasis (QOL-B). The NTM Module generates four domain scores (0–100; higher scores indicate better functioning) reflecting NTM-specific symptoms (NTM Symptoms, Body Image, Digestive Symptoms and Eating Problems). We described patient characteristics and mean scores, and evaluated psychometric properties, including response to treatment at 12 months, for each domain. Results Overall, 203 patients with pulmonary MAC disease were included. Average enrolment scores ranged from 76 (NTM Symptoms) to 84 (Eating Problems). Ceiling effects were observed for Body Image (26% of participants) and Eating Problems (52%). Internal consistency (Cronbach's alpha) ranged from 0.67 (Digestive Symptoms) to 0.89 (Eating Problems). The intraclass correlation for test–retest reproducibility (n=27) ranged from 0.72 (Body Image) to 0.94 (Eating Problems). Patients starting treatment (n=35) had statistically significant increases in scores for NTM Symptoms (+5, p=0.04), Digestive Symptoms (+7, p=0.002), Body Image (+7, p=0.03) and QOL-B Respiratory Symptoms (n=25, +10, p=0.006). NTM Symptoms scores increased by 15 points (p=0.002) in the 16 patients with scores ≤80 at enrolment. Conclusion The NTM Module generally performs well as a valid patient-reported outcome for pulmonary MAC disease and was responsive to MAC treatment. The NTM Symptom Module is a valid patient-reported outcome tool that can facilitate patient-centred care and may be used as an outcome in clinical trials to support labelling claims for regulatory bodies. http://bit.ly/2nwlPgi

中文翻译：

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NTM 模块的初步验证：肺非结核分枝杆菌疾病患者报告的结果测量

简介 非结核分枝杆菌 (NTM) 会导致慢性、虚弱的肺部疾病。患者报告的结果提供了症状、功能和治疗反应的措施。在这里，我们描述了最近开发的 NTM 模块的初步验证。方法 研究人群包括 Northwest NTM Biobank 患者，其中分离出鸟分​​枝杆菌复合体 (MAC)，并且曾符合 2007 年美国胸科学会/美国传染病学会肺病标准。NTM 模块在注册时和 12 个月时进行管理；一个子集还完成了生活质量问卷 - 支气管扩张 (QOL-B)。NTM 模块生成反映 NTM 特定症状（NTM 症状、身体形象、消化症状和饮食问题）的四个领域分数（0-100；分数越高表示功能越好）。我们描述了每个领域的患者特征和平均评分，并评估了心理测量特性，包括 12 个月时对治疗的反应。结果 总共包括 203 名患有肺 MAC 疾病的患者。平均入学分数范围从 76（NTM 症状）到 84（饮食问题）。观察到身体形象（26% 的参与者）和饮食问题（52%）的天花板效应。内部一致性（Cronbach's alpha）范围从 0.67（消化系统症状）到 0.89（饮食问题）。测试-再测试可重复性的组内相关性 (n=27) 范围从 0.72（身体形象）到 0.94（饮食问题）。开始治疗的患者 (n=35) 在 NTM 症状 (+5, p=0.04)、消化症状 (+7, p=0.002)、身体形象 (+7, p=0.03) 和 QOL- B 呼吸道症状（n=25，+10，p=0。006）。在 16 名评分≤80 的患者中，NTM 症状评分增加了 15 分 (p=0.002)。结论 NTM 模块作为肺 MAC 疾病的有效患者报告结果通常表现良好，并且对 MAC 治疗有反应。NTM 症状模块是一种有效的患者报告结果工具，可以促进以患者为中心的护理，并可用作临床试验的结果，以支持监管机构的标签声明。http://bit.ly/2nwlPgi NTM 症状模块是一种有效的患者报告结果工具，可以促进以患者为中心的护理，并可用作临床试验的结果，以支持监管机构的标签声明。http://bit.ly/2nwlPgi NTM 症状模块是一种有效的患者报告结果工具，可以促进以患者为中心的护理，并可用作临床试验的结果，以支持监管机构的标签声明。http://bit.ly/2nwlPgi