Patient reported outcomes in patients with nontuberculous mycobacterial pulmonary disease

非结核分枝杆菌肺病患者报告的结果

• 批准号: 10720789
• 负责人: Emily M. Henkle
• 金额: $ 67.28万
• 依托单位: OREGON HEALTH & SCIENCE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-08-01 至 2028-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10720789
• 关键词: Acid Fast Bacillae Staining Method; Adult; Adverse event; Affect; Aftercare; Age; Airway Disease; American; Antibiotic Therapy; Antibiotics; Bacillus; Belief; Biological; Bronchiectasis; Caring; Characteristics; Chest; Chronic; Chronic Disease; Chronic Obstructive Pulmonary Disease; Clinical; Clinical Data; Clinical Trials; Clinical Trials Network; Collaborations; Common Terminology Criteria for Adverse Events; Communicable Diseases; Coughing; Data; Diagnosis; Disease; Disease Outcome; Elderly; Electronics; Enrollment; Evaluation; Fatigue; Frequencies; Friends; Fright; Future; Incidence; Inflammatory; Intervention; Judgment; Knowledge; Left; Longitudinal cohort; Lung; Lung diseases; Measures; Mental Health; Monitor; Mycobacterium abscessus; Mycobacterium avium Complex; Outcome; Outcome Measure; Outcomes Research; Patient Monitoring; Patient Outcomes Assessments; Patient Preferences; Patient-Focused Outcomes; Patients; Pattern; Pharmaceutical Preparations; Pharmacotherapy; Prospective cohort; Prospective, cohort study; Quality of life; Rare Diseases; Recurrence; Regimen; Reporting; Research; Respiratory Signs and Symptoms; Risk; Role; Shortness of Breath; Site; Societies; Soil; Sputum; Standardization; Symptoms; Testing; Time; Toxic effect; Tuberculosis; United States Food and Drug Administration; Water; biobank; clinical care; clinical outcome measures; clinical practice; cohort; data repository; disability; disease natural history; experience; health related quality of life; improved; interest; longitudinal analysis; meetings; non-tuberculosis mycobacteria; novel; older patient; pathogen; patient oriented; primary outcome; radiological imaging; respiratory; response; success; tool; treatment comparison; treatment response

PROJECT SUMMARY/ABSTRACT Nontuberculous mycobacteria (NTM) are environmental pathogens present in the soil and water that can cause chronic, debilitating disease in predominately older patients with underlying bronchiectasis or chronic obstructive pulmonary disease. The most common causes of NTM pulmonary disease (NTM-PD) are Mycobacterium avium complex (MAC) and M. abscessus (MAB). Key symptoms of NTM-PD include cough, extreme fatigue, and shortness of breath. NTM-PD is heterogeneous in presentation: nodular-bronchiectatic disease (~60-70%) tends to be slowly progressing and if MAC may not require immediate treatment, while those with cavitary disease (~25%) or MAB require immediate treatment. Multi-drug therapy, when initiated, consists of 3-5 antibiotics taken for 18+ months. NTM treatment carries risks of significant toxicity and is poorly tolerated by some patients. Although most patients have microbiologic response to treatment, many are unable to produce sputum for testing, and treatment success is evaluated as subjective clinical improvement. Changes in cough rates and patterns are presumed to be important but have not been objectively measured. Further, after completing treatment, around half will re-develop NTM-PD within 3 years. Unlike its “cousin” tuberculosis where culture conversion is a surrogate for cure, the meaningfulness of culture conversion to patients in pulmonary NTM is less clear. Patient-reported outcomes (PROs) which measure symptoms, functioning, and health-related quality of life are key components of patient-centered research and care. Improvements in daily symptoms and functioning are critical outcomes from the patient’s perspective, but significant evidence gaps exist in the role of PRO data to inform decisions about when to treat among those who do not require immediate treatment, how to measure treatment response and tolerability, and how to monitor patients post-treatment. We propose to fill critical gaps in knowledge regarding the trajectory of PRO measures for NTM-PD and incorporate an objective cough monitor to measure rates and patterns of cough in patients. This prospective cohort study leverages our experience establishing an NTM Clinical Trials Network and Northwest NTM Biobank data repository. We will continue longstanding collaborations with Drs. Winthrop, Daley, Flume, McShane, and Aksamit for all proposed aims. During Years 1-4 we will enroll 3 well-characterized cohorts of patients representing key stages of NTM-PD – initial diagnosis (NTM-WATCH), treatment start (NTM-TREAT), and at end of treatment (NTM-TRACK) from participating sites and collect PROs and clinical outcome data for up to 24 months for each cohort. During Years 3-5 we will conduct our descriptive and longitudinal analysis of PROs in these prospective cohorts, evaluating PRO measures’ ability to monitor disease activity and tolerability of treatment. Our findings will lay the groundwork for future evaluation of combined outcome measures for clinical trials endpoints and, most importantly, incorporation of PRO measures in clinical practice.

项目摘要/摘要 非结核分枝杆菌（NTM）是土壤和水中存在的环境病原体 慢性疾病的慢性疾病，主要是老年患者的基本支气管张或慢性阻塞性的患者 肺病。 NTM肺部疾病（NTM-PD）的最常见原因是分枝杆菌 复合物（MAC）和M. abcessus（mAb）。 NTM-PD的关键症状包括咳嗽，极度疲劳和 气促。 NTM-PD在介绍中是异质的：结节性结核病（〜60-70％）趋于 要缓慢进展，如果MAC可能不需要立即治疗，而患有空洞的患者 （〜25％）或mAb需要立即治疗。多药疗法启动时，由3-5种抗生素组成 18个月以上。 NTM治疗具有明显的毒性风险，某些患者的耐受性不佳。 尽管大多数患者对治疗有微生物的反应，但许多患者无法为 测试和治疗成功被评估为主观临床改善。咳嗽率的变化和 模式被提出很重要，但尚未客观地衡量。此外，完成后 治疗，大约一半将在3年内重新开发NTM-PD。与培养的“表弟”结核病不同 转化是治愈的替代物，培养物转化为肺NTM中患者的意义是 不太清楚。衡量症状，功能和与健康相关的质量的患者报告结果（PRO） 生活是以患者为中心的研究和护理的关键组成部分。日常符号的改进和 从患者的角度来看，功能是关键的结果，但是在 专业数据以告知关于何时不需要立即治疗的人何时治疗的决定，如何 测量治疗反应和耐受性，以及如何监测治疗后患者。 我们建议在有关NTM-PD和 将客观的咳嗽监测器纳入患者的咳嗽率和咳嗽模式。这个前瞻性 队列研究利用我们建立NTM临床试验网络和西北NTM生物库的经验 数据存储库。我们将继续与Drs长期合作。 Winthrop，Daley，Flume，McShane和 AKSAMIT适用于所有拟议的目标。在1 - 4年期间 代表NTM-PD的关键阶段 - 初始诊断（NTM-WATCH），治疗开始（NTM-TREAT）和AT 治疗终结（NTM-track）来自参与地点，并收集高达24的优点和临床结果数据 每个队列的月份。在3 - 5年中，我们将对专业人士的描述性和纵向分析 这些前瞻性队列，评估Pro措施监测疾病活动的能力和可耐受性 治疗。我们的发现将为将来评估临床结果综合评估奠定基础 试验终点，最重要的是，在临床实践中纳入了Pro测量中。