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

基本信息

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
项目类别：
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

项目摘要

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.

项目概要/摘要非结核分枝杆菌 (NTM) 是存在于土壤和水中的环境病原体，可导致慢性、衰弱性疾病，主要发生于患有潜在支气管扩张或慢性阻塞性的老年患者 NTM 肺部疾病 (NTM-PD) 最常见的原因是鸟分枝杆菌。 NTM-PD 的主要症状包括咳嗽、极度疲劳和脓肿分枝杆菌 (MAB)。 NTM-PD 的表现具有异质性：结节性支气管扩张疾病 (~60-70%) 趋势。进展缓慢，如果 MAC 可能不需要立即治疗，而患有空洞病的人 (~25%) 或 MAB 需要立即治疗，开始时需要服用 3-5 种抗生素。超过 18 个月的 NTM 治疗存在显着毒性的风险，并且某些患者的耐受性较差。尽管大多数患者对治疗有微生物学反应，但许多患者无法产生痰液以进行治疗。测试和治疗成功根据咳嗽率和咳嗽率的主观临床改善进行评估。模式被认为很重要，但在完成后尚未进行客观测量。与它的“表亲”结核病不同，大约一半的人会在 3 年内重新患上 NTM-PD。转换是治愈的替代指标，文化转换对肺 NTM 患者的意义是衡量症状、功能和健康相关质量的患者报告结果 (PRO) 不太明确。生活的改善是以患者为中心的研究和护理的关键组成部分。从患者的角度来看，功能是关键的结果，但在其作用方面存在重大证据差距 PRO 数据可帮助决定何时对那些不需要立即治疗的人进行治疗、如何治疗衡量治疗反应和耐受性，以及如何监测患者治疗后。我们建议填补有关 NTM-PD 和 PRO 措施轨迹的关键知识空白这种前瞻性咳嗽监测仪采用客观咳嗽监测仪来测量患者咳嗽的频率和模式。队列研究利用我们建立 NTM 临床试验网络和西北 NTM 生物库的经验我们将继续与 Winthrop、Daley、Flume、McShane 博士和 Aksamit 旨在实现所有拟议目标，在第 1-4 年期间，我们将招募 3 组特征明确的患者。代表 NTM-PD 的关键阶段 – 初始诊断 (NTM-WATCH)、治疗开始 (NTM-TREAT) 和来自参与中心的治疗结束 (NTM-TRACK)，并收集最多 24 个项目的 PRO 和临床结果数据在第 3-5 年期间，我们将对每个队列的 PRO 进行描述性和纵向分析。这些前瞻性队列评估了 PRO 措施监测疾病活动性和耐受性的能力我们的研究结果将为未来评估临床综合结果指标奠定基础。试验终点，最重要的是，将 PRO 措施纳入临床实践。