Goal 2: Reduce Human Disease

Research priorities in Pediatric Cardiomyopathy and Heart Failure

If NHLBI set research priorities for pediatric cardiomyopathy and heart failure, it would help investigators better align their application with institute goals.

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The most promising areas are genotype-deep phenotype studies, advances in cardiac imaging to quantify extracellular volume as a measure of fibrosis; working with industry to develop better VADS/pre-transplant devices for small children, QOL studies in heart transplant children and their families, personalized medicine, pharmacogenomics, RCTs for new heart failure interventions and also cardiac rehabilitation, leverage and expand registries to support RCTs, molecular mechanism studies including studies of restrictive physiology, using genomic findings to directly inform clinical practice.

Name of idea submitter and other team members who worked on this idea : Jwilkins1953

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Goal 2: Reduce Human Disease

International collaboration for genetic and metabolic research on specific human population

During recent years, clinical research including well-organized randomized clinical trials in developed countries generated large database and human biological sample banks. These are valuable resources for human disease research. Mechanisms to encourage and facilitate international collaboration for genetic and metabolic research using database and human biological samples from specific human disease population of international ...more »

Submitted by (@hongw0)

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Name of idea submitter and other team members who worked on this idea : Hong Wang, Xiaofeng Yang

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Goal 3: Advance Translational Research

Inhaled antibiotics for patients with bronchiectasis

Many pharma companies are developing inhaled antibiotics for patients with bronchiectasis, recognizing the increasing market (over 110,000 patients in the US). These drugs cost thousands of dollars per year and will be heavily marketed and heavily used, as there are limited options for these patients. It is unknown if rotating oral antibiotics (cost is a fraction of the inhaled ABx) would yield similar results.  A Cochrane ...more »

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 2: Reduce Human Disease

Phase III efficacy trials of tuberculosis drugs

1) Phase III efficacy trials of new tuberculosis drugs (e.g., bedaquiline, delamanid, PA-824) that have shown promise in early phase studies for multidrug-resistant tuberculosis. 2) Phase III efficacy trials of new and existing tuberculosis drugs to development very short course regimens (3-4 months). 3) Phase III efficacy trials of new and existing drugs for treatment of latent tuberculosis infection in contacts of ...more »

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 2: Reduce Human Disease

Best antibiotic regimen to treat community-acquired pneumonia

Community-acquired pneumonia (CAP) is responsible for over 1 million hospital admissions and about 100,000 deaths per year. We still do not know the best antibiotic regimen to treat CAP. Retrospective studies and cohort studies support giving macrolides, while randomized controlled studies (essentially all done by pharma) have not shown benefit of macrolides.  Guidelines allow either a macrolide or a quinolone.

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

This is an area that needs to be studied, because if the macrolides provide the benefit suggested by many studies (about 20% mortality benefit), much morbidity and mortality would be prevented by universal macrolide use for CAP.  I am not aware of any studies being performed that would answer this question.

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 2: Reduce Human Disease

Nontuberculous mycobacterial (NTM) lung infections

The true prevalence of Nontuberculous mycobacterial (NTM) lung infections remains incompletely understood, however several aspects of NTM lung disease prevalence are becoming more clear. NTM lung disease is currently more common in the U.S. than TB (by a factor of 3) and has consistently been shown to be increasing in prevalence. When viewed in the context of likely universal environmental NTM exposure this increasing ...more »

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

NTM lung disease is most common among older and female patients with estimated prevalences as high as 50-60 cases per 100,000 in this group and can be seen in this context as a women’s health issue.  While it may appear paradoxical, NTM lung disease studies would both profoundly change outcomes for a relatively rare condition overall, but would also benefit, in a potentially profound way, a large number of patients in a selected population (older female patients) in which the disease is not so rare.   To date there has been no significant funding for any aspect of NTM lung disease outside of limited support from the pharmaceutical industry.  The studies that have been done to date are largely single center/institution studies with relatively small numbers of patients.  Again, the few collaborative studies that have been done were accomplished with support from the pharmaceutical industry.  NTM lung diseases, and associated lung diseases such as bronchiectasis have not attracted the interest or support of major non-industry funding sources.

Feasibility and challenges of addressing this CQ or CC :

Essentially every aspect of Nontuberculous Mycobacterial (NTM) lung disease is associated with some level of controversy. The diagnostic guidelines were adopted primarily as a result of experience with common NTM pathogens such as Mycobacterium avium complex (MAC), M. kansasii , and M. abscessus. The relevance of the diagnostic guidelines for other potential NTM pathogens, especially rarely encountered organisms (in the U.S.) such as M. xenopi and organisms usually associated with specimen contamination such as M. fortuitum , is untested and unknown. The treatment of NTM lung disease is almost entirely based on case series, both retrospective and prospective, with consensus on basic treatment concepts. Treatment outcomes remain unsatisfactory for many pathogens including the most common, MAC, and prospective treatment trials are needed to establish not just optimal treatment regimens but the roles of agents in common use, such as clofazimine, the fluoroquinolones and inhaled amikacin, of unproven value.  Other basic studies including radiographic and pulmonary function evolution for treated and untreated disease and long term natural history of treated and untreated NTM lung disease patients are lacking.

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 2: Reduce Human Disease

Therapy for lung infections

1. Monotherapy with a quinolone vs combination therapy with a 3rd generation cephalosporin. The issue of the best antibiotic treatment for severe CAP has been a major area of contention now for a decade and it is the most common cause of infectious death in the United States. 2. Combination therapy vs monotherapy for pneumonia due to Pseudomonas. This is another major area of contention – for nearly 2 decades, and generates ...more »

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 1: Promote Human Health

Study wellness instead of diseases by longitudinal follow-up of frequent and long term blood donors

Blood donors (especially young donors) in general represent healthy populations. Longitudinal follow-up of frequent and long term blood donors can be useful to establish data and sample sources for the study of wellness, instead of disease (especially for blood diseases). Not only it can be used as healthy controls, it can also be used to predict the wellness factors such as genetic variation, life style, exercise patterns, ...more »

Submitted by (@yanyunw)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

Once such study is established, it can be used as a wellness model/control for many diseases and conditions. The longer we have the study, more useful it will become. This can help us to change or optimize our health care model, from treating diseases to maintain wellness.

Feasibility and challenges of addressing this CQ or CC :

This can be quite feasible as there are many frequent and long term donors. They care about others’ health as well as their own health. They are also very willing to support research. Follow-up can start from early adulthood.

 

With funding support this can be achieved.

Name of idea submitter and other team members who worked on this idea : Yanyun Wu

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Goal 4: Develop Workforce and Resources

Number of clinical scientists

How can we insure that there are sufficient numbers of clinical scientists over the next 20 years?

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 4: Develop Workforce and Resources

NIH trans-IC office of critical care research

Would an NIH trans-IC office of critical care research improve coordination and strategic planning across?

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 3: Advance Translational Research

Novel therapeutics for patients with severe acute lung injury

Can novel therapeutics including cell-based therapy be tested in patients with severe acute lung injury (P/F <200) and shock (need for vasopressors) since these are the patients with the highest mortality (> 30%) based on NHLBI ARDS Network data?

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 3: Advance Translational Research

Novel extracorporeal devices that remove carbon dioxide

Can novel extracorporeal devices that remove carbon dioxide be tested to limit or avoid positive pressure ventilation in patients with acute respiratory failure from COPD?

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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