Goal 2: Reduce Human Disease

Submitted by (@nhlbiforumadministrator)

Clinical trial of AAT augmentation therapy in PI MZ subjects

Although severely alpha-1 antitrypsin deficiency (PI ZZ) individuals are rare, PI MZ subjects comprise approximately 3% of the US population. Mounting evidence suggests that PI MZ is a significant risk factor for COPD. A clinical trial of COPD exacerbation frequency using AAT augmentation therapy in PI MZ subjects with moderate-to-severe COPD could provide a rationale for AAT augmentation therapy (or small molecule neutrophil ...more »

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Goal 3: Advance Translational Research

Submitted by (@k.willard)

Predicting COPD exacerbations and relapse

What measures other than PFT data can be used to predict risk of 1) COPD exacerbations (e.g., hospitalization, urgent care visit, or ED visit for COPD exacerbation) or 2) relapse (e.g., re-hospitalization, urgent care visit, or ED visit) following hospital discharge after treatment of COPD exacerbations?

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Goal 3: Advance Translational Research

Submitted by (@lungmatbio1)

Next Stage of COPD Discovery

1) Refinement of COPD subphenotypes for therapeutics, diagnostics and mechanistic interrogation. The NIH should encourage a strong focus on a) rigorous, mechanistically-reinforced definitions (chronic bronchitis, emphysema (with and without obstruction), frequent exacerbators, combined pulmonary fibrosis and emphysema) and 2) the development and optimization of animal model systems that replicate the different subphenotypes. ...more »

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Goal 2: Reduce Human Disease

Submitted by (@nhlbiforumadministrator)

Understanding Pathobiology of Exacerbations

COPD exacerbations contribute to poor quality of life, lung function and functional decline. In addition, they are costly to the patient and to health care cost. However, they remain defined exclusively on symptoms and constitute a “soft” endpoint. Actually, the number of exacerbations has become the GOLD standard to establish the risk of outcome (exacerbations). However, a mild episode that only requires outpatient ...more »

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Goal 3: Advance Translational Research

Submitted by (@gacdk0)

Definitive Evidence of the Effectiveness of Pulmonary Rehabilitation

What is the clinical effectiveness of pulmonary rehabilitation in reducing hospital admissions and readmissions, improving health outcomes such as exercise tolerance and dyspnea, and positively impacting patient centered outcomes. Does this effectiveness vary based on the types of settings rehab is conducted in, urban vs rural environments, the components to the program, the timing of the program and the overall support ...more »

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Goal 3: Advance Translational Research

Submitted by (@richard.mularski)

What strategies improve implementation of COPD therapy, palliation, and dyspnea management

Chronic obstructive pulmonary disease (COPD) affects over 12-24 million individuals in the U.S. where it is responsible for ~ 800,000 hospitalizations per year, and recently became America’s 3rd leading cause of death. The lag between clinical practice and treatment options described by efficacy studies to improve the quality of life, functional status, and survival in patients with COPD make it ideal for efforts in patient-centered ...more »

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