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Evaluating patient-reported outcomes for pulmonary nontuberculous mycobacterial disease

评估患者报告的肺非结核分枝杆菌疾病的结果

基本信息

批准号：
10367984
负责人：
Emily M. Henkle
金额：
$ 14.87万
依托单位：
OREGON HEALTH & SCIENCE UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-03-15 至 2023-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10367984
关键词：
Advocate Antibiotics Belief Bronchiectasis Caring Characteristics Chronic Chronic Disease Chronic Obstructive Pulmonary Disease Chronic lung disease Clinic Clinical Clinical Trials Common Terminology Criteria for Adverse Events Complex Coughing Data Decision Making Development Disease Elderly Environment Fatigue Feasibility Studies Focus Groups Foundations Funding Goals Health Status Intervention Interview Label Lung Lung diseases Macrolides Measurement Measures Methods Microbiology Mycobacterium avium Complex National Cancer Institute Outcome Outcome Measure Outcomes Research Patient Care Patient Monitoring Patient Outcomes Assessments Patient Preferences Patient Self-Report Patient-Focused Outcomes Patients Pattern Pharmaceutical Preparations Pharmacotherapy Phenotype Physical Function Principal Investigator Provider Psychometrics Qualitative Evaluations Quality of life Randomized Clinical Trials Randomized Controlled Trials Rare Diseases Regimen Relapse Reporting Research Priority Respiratory Signs and Symptoms Risk Shortness of Breath Soil Surveys Symptoms Testing Therapeutic Time Toxic effect Treatment-related toxicity Validation Water Work acceptability and feasibility base cancer clinical trial cancer therapy clinical care clinical practice comparative effectiveness trial disability eVisit effective therapy experience future implementation health assessment health related quality of life implementation research improved insight interest longitudinal analysis non-tuberculosis mycobacteria older patient pathogen patient oriented prospective recruit response secondary outcome success therapy outcome tool treatment response trial comparing

项目摘要

PROJECT SUMMARY Pulmonary nontuberculous mycobacterial (NTM) disease due to Mycobacterium avium complex (MAC) is an increasingly common, rare disease. MAC are environmental pathogens present in the soil and water that can cause chronic, debilitating pulmonary disease in predominately older patients with underlying lung disease. There are two distinct phenotypes, more commonly nodular disease associated with bronchiectasis and less commonly cavitary disease with underlying chronic obstructive pulmonary disease. Treatment consists of 3-5 off-label antibiotics for 18-24 months. Pulmonary MAC is difficult to eradicate, and relapse or reinfection from the environment is common. While microbiologic response is frequently achieved, the ability of this outcome measure to reflect how the patient feels, functions, or survives is poorly understood. In this application, we will expand our prior work with NTM patients and patient advocate stakeholders that identified the development of meaningful PRO measures and measuring treatment response using health-related quality of life (HRQoL) as top research priorities. Our proposed work uses a mixed methods approach and leverages our regional NTM referral clinic and recently initiated prospective randomized clinical trial of 2-drug vs. 3-drug therapy for pulmonary MAC (MAC2v3 trial). Dr. Kevin Winthrop is the principal investigator and OHSU serves as the coordinating center for the MAC2v3 trial. Recruiting from the NTM clinic, we will first conduct qualitative evaluation of whether the NTM Module missed any characteristic NTM symptoms in non- bronchiectatic patients (Aim 1a). We will further validate the NTM Module in treated patients in the MAC2v3 trial. MAC2v3 patients have nodular-bronchiectatic MAC (Aim 1b). PROs are collected at 5 time points (baseline, 1, 3, 6, and 12 months), and include HRQoL and symptoms (QOL-Bronchiectasis and NTM Module), and symptoms/toxicity (PRO-CTCAE, selected 40 items). We will describe longitudinal patterns of HRQoL and toxicity in treated patients, and further evaluate the relationship between toxicity and HRQoL over time (Aims 2a and 2b). Last, to evaluate feasibility for incorporating PROs in clinical practice (Aim 3) we will conduct focus groups with patients and survey clinicians to select the most relevant PROs for clinical care. In the pilot implementation aim we will incorporate a brief pre-visit electronic PRO survey in Dr. Winthrop's NTM clinic, evaluating the feasibility and acceptability of PROs in clinical practice and describe changes in care/management after pilot implementation. We will complete Aim 1a during Year 1, Aim 1b in Year 2, Aims 2a and 2b in Years 3- 4, and Aim 3 in Year 3. We anticipate that our work will produce a validated, NTM-disease specific PRO and evidence to support integration into clinical practice. This work will lay the foundation for future implementation and outcomes research of PROs for NTM patients, thus facilitating improved patient care for a complex, chronic disease.

项目概要由鸟分枝杆菌复合体 (MAC) 引起的肺非结核分枝杆菌 (NTM) 疾病是一种越来越常见、罕见的疾病。 MAC 是存在于土壤和水中的环境病原体，导致慢性、衰弱性肺部疾病，主要是患有潜在肺部疾病的老年患者。有两种不同的表型，更常见的是与支气管扩张相关的结节性疾病，较少见的是与支气管扩张相关的结节性疾病。常见的空洞性疾病伴有潜在的慢性阻塞性肺病。治疗包括 3-5 标签外抗生素使用 18-24 个月。肺MAC很难根除，并且会复发或再感染环境一般。虽然经常实现微生物学反应，但这种结果的能力人们对反映患者感觉、功能或生存情况的衡量标准知之甚少。在此应用程序中，我们将扩展我们之前与 NTM 患者和患者倡导者利益相关者的合作，确定了有意义的 PRO 措施的制定，并使用与健康相关的指标来衡量治疗反应生活质量（HRQoL）作为首要研究重点。我们提出的工作采用混合方法利用我们的区域 NTM 转诊诊所和最近启动的 2 种药物与 2 种药物对比的前瞻性随机临床试验。肺 MAC 的 3 种药物治疗（MAC2v3 试验）。 Kevin Winthrop 博士是首席研究员和 OHSU 作为 MAC2v3 试验的协调中心。从NTM诊所招募，我们首先会进行定性评估 NTM 模块是否遗漏了非非患者中任何特征性 NTM 症状支气管扩张患者（目标 1a）。我们将在 MAC2v3 试验中在接受治疗的患者中进一步验证 NTM 模块。 MAC2v3 患者患有结节性支气管扩张型 MAC（目标 1b）。 PRO 在 5 个时间点收集（基线、1、 3、6 和 12 个月），并包括 HRQoL 和症状（QOL-支气管扩张和 NTM 模块），以及症状/毒性（PRO-CTCAE，选定 40 项）。我们将描述 HRQoL 的纵向模式治疗患者的毒性，并进一步评估随着时间的推移毒性与 HRQoL 之间的关系（目标 2a 和 2b)。最后，为了评估将 PRO 纳入临床实践的可行性（目标 3），我们将重点关注与患者分组并调查临床医生，以选择最相关的 PRO 进行临床护理。在试点中为了实现实施目标，我们将在 Winthrop 博士的 NTM 诊所进行简短的就诊前电子 PRO 调查，评估 PRO 在临床实践中的可行性和可接受性，并描述护理/管理方面的变化试点实施后。我们将在第 1 年完成目标 1a，在第 2 年完成目标 1b，在第 3 年完成目标 2a 和 2b - 4 和第 3 年的目标 3。我们预计我们的工作将产生经过验证的 NTM 疾病特异性 PRO 和支持融入临床实践的证据。这项工作将为今后的实施奠定基础 NTM 患者的 PRO 和结果研究，从而促进改善复杂、慢性疾病的患者护理疾病。