Goal 2: Reduce Human Disease

To extend our knowledge of the pathobiology of heart, lung, blood, and sleep disorders and enable clinical investigations that advance the prediction, prevention, preemption, treatment, and cures of human disease.

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.

Submitted by (@nhlbiforumadministrator)

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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)

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 : Hong Wang, Xiaofeng Yang

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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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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.

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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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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

Addition of albumin to fluid conservative management of AL

Does the addition of albumin to fluid conservative management of ALI (ARDSnet FACTT trial protocol, Wiedemann et al) further shorten ventilator time and/or improve survival?

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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

Embrace and fund RCTs that enroll heterogeneous samples of patients

Critical care medicine comprises a diffuse array of diseases, syndromes, illnesses and symptoms arising from those sources requiring advanced care by highly trained teams of interdisciplinary professionals. Research is sorely needed on generating evidence that is broadly applicable to a heterogeneous group of patients. This is a major challenge for researchers who enroll critically ill patients into their clinical trials. ...more »

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Feasibility and challenges of addressing this CQ or CC :

This will require a different mind-set by grant reviewers.

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

Palliation of symptoms associated with pulmonary conditions; promotion of patient participation in symptom self-management

Palliation of symptoms associated with a number of pulmonary conditions; promotion of patient participation in symptom self-management across the spectrum of illness, from ICU admission to rehabilitation to home; requires a multi-disciplinary perspective and team. There are a plethora of distressing symptoms (anxiety, shortness of breath, cough, fatigue, weakness) associated with a number of chronic pulmonary conditions, ...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 :

Innovative symptom management strategies are needed that empower patients and their family members to engage in self-management strategies across the spectrum of illness from hospitalization through recovery.

Feasibility and challenges of addressing this CQ or CC :

Longitudinal studies with a multi-disciplinary team of investigators are needed to address the varying needs of these patients and their family members over the course of recovery or transition to death in these patients. Interventions need to be cost-effective and prevent readmission to the critical care unit.

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

Vasopressin layered on to norepinephrine treatment for septic shock

We know that vasopressin layered on to norepinephrine treatment for septic shock tends to produce better outcomes (VASST trial, Russell et al) than norepinephrine alone. We still need to know if norepinephrine should be first line or if vasopressin should be first line (and perhaps monotherapy) for septic shock.

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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

Relation of environmental factors to lung injury

What is the relation of environmental factors such as cigarette smoke exposure to the risk of developing acute lung injury as well as the outcome from acute lung injury and sepsis?

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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 2: Reduce Human Disease

“Change of culture” studies

Large scale implementation of “change of culture” studies by which to revamp the approach to early removal of sedation and mechanical ventilation, coupled with monitoring of the brain and early mobility.

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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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