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.

项目摘要肺部无菌分枝杆菌（NTM）疾病由于分枝杆菌复合物（MAC）是一种越来越普遍的稀有疾病。 Mac是土壤和水中存在的环境病原体引起慢性，使人衰弱的肺部疾病，主要是老年患者的肺部疾病。有两种不同的表型，更常见的是与支气管扩张有关的结节疾病，较少常见的疾病常见于潜在的慢性阻塞性肺部疾病。治疗包括3-5 标签外抗生素18-24个月。肺Mac难以根除，并从环境很常见。尽管经常达到微生物响应，但这种结果的能力对反映患者的感觉，功能或生存方式的措施知之甚少。在此应用程序中，我们将与NTM患者和患者倡导者利益相关者进行以前的工作确定了使用与健康相关的有意义的Pro措施的发展和测量治疗反应生活质量（HRQOL）作为主要研究重点。我们建议的工作使用混合方法方法，利用我们的区域NTM推荐诊所，并最近开始了2-Clug VS的前瞻性随机临床试验。 3-药物治疗肺MAC（MAC2V3试验）。凯文·温特罗普（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，我们将首先进行定性评估NTM模块是否错过了非特征NTM症状支气管检查患者（AIM 1A）。我们将在MAC2V3试验中进一步验证治疗患者中的NTM模块。 MAC2V3患者具有结节性支气管切开症MAC（AIM 1B）。优点是在5个时间点收集的（基线，1， 3、6和12个月），包括HRQOL和症状（Qol-chronchiectasis和NTM模块），以及症状/毒性（亲CTCAE，选定的40个项目）。我们将描述HRQOL和随着时间的推移，治疗患者的毒性进一步评估毒性与HRQOL之间的关系（AIMS 2A 和2b）。最后，为了评估将优点纳入临床实践的可行性（AIM 3），我们将进行重点小组与患者和调查临床医生一起选择最相关的临床护理专业人士。在飞行员中实施目标我们将在Winthrop博士的NTM诊所中纳入简短的Visit Electronic Pro调查，评估专业人士在临床实践中的可行性和可接受性，并描述护理/管理的变化试点实施后。我们将在第1年完成AIM 1A，在第2年的AIM 1B，AIMS 2A和2B中的AIM 1B和3年度的AIMS 2A和2B 4，AIM 3在3年级。我们预计我们的工作将产生经过验证的NTM-Disease特定专业支持整合到临床实践中的证据。这项工作将为将来的实施奠定基础以及针对NTM患者专业人士的结果研究，从而促进了改善患者护理的复杂，慢性疾病。