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.
(@nhlbiforumadministrator)

Goal 2: Reduce Human Disease

Pulmonary Vascular Diseases

Does anticoagulation with warfarin improve outcomes (time to clinical worsening, qualtiy of life, exercise capacity) in patients with pulmonary arterial hypertension treated with current oral/inhaled therapies? There are substantial "unknowns" and practice variation in anticoagulation in PAH. Resource utilization is also a factor here. We may either be helping patients (or hurting them with side effects) by using anticoagulation.... more »

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2 up votes
0 down votes
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(@nhlbiforumadministrator1)

Goal 2: Reduce Human Disease

A Program of Research in the Prevention of Chronic Heart Failure

There is a need to address chronic heart failure (HF) through improved identification of patients at risk for HF and of patients with pathological ventricular remodeling who have minimal evidence of clinical HF, and more focused and individualized pharmacologic and lifestyle treatments and monitoring of patients with HF risk. Approaches would include big data collection, omics, statistical modeling, and focused clinical... more »

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17 net votes
28 up votes
11 down votes
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(@abrams)

Goal 2: Reduce Human Disease

Funding for Hemostasis & Thrombosis Research

Thrombotic disorders, a result of the inappropriate activation of the hemostatic system, remain major causes of morbidity and mortality in the United States. Cancer, cardiovascular disease, trauma, and many of the other causes of death in the U.S. frequently culminate in a fatal thrombotic event. Notably, thromboembolic disease affects 500,000 people annually and leads to 100,000 deaths in the United States alone. Current... more »

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128 net votes
136 up votes
8 down votes
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(@nhlbiforumadministrator)

Goal 2: Reduce Human Disease

Outcomes of heterogeneous responses to tiotropium

Tiotropium is widely used for COPD, but patients report highly variable responses to this medication. A clinical trial of severe COPD subjects could address both short-term (bronchodilator effect) and intermediate-term (COPD exacerbations) outcomes of this heterogeneous response by examining radiologic (chest CT), clinical (Six Minute Walk, Spirometry), and pharmacogenetic (GWAS) influences on this heterogeneous response.... more »

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2 net votes
2 up votes
0 down votes
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(@jhagood)

Goal 2: Reduce Human Disease

Systems Approaches to "Phenosimilar" Diseases

There are diverse diseases that share similar features. For example, many chronic airways diseases (chronic aspiration, ciliary dyskinesia, some COPD) "phenocopy" cystic fibrosis in terms of infectious agents, mucus clearance problems, progressive loss of lung function, etc. Use multiplatform "omics" approaches and Big Data bioinformatics to identify common nodes for therapeutic targeting. Other examples: emphysema and... more »

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4 net votes
12 up votes
8 down votes
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(@daniel.perez)

Goal 2: Reduce Human Disease

Developing Standards of Care for adult muscular dystrophy (FSHD, DM) patients affected by hypercarbic respiratory insufficiency

There is an unmet need for the NHLBI to foster basic, preclinical and clinical research on the pulmonary consequences of respiratory insufficiency, and specifically with hypercarbic (high CO2) respiratory insufficiency, in facioscapulohumeral muscular dystrophy (FSHD) and other adult muscular dystrophies. The adult muscular dystrophies have received insufficient attention, both from research and clinical practice perspectives.... more »

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-11 net votes
4 up votes
15 down votes
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(@lvanhorn)

Goal 2: Reduce Human Disease

Transforming Clinical Practice through Patient-Centered Medical Nutrition and Lifestyle Education

The fact that diet contributes significantly to prevention and treatment of disease is now a foregone conclusion. National and international guidelines offer evidence based recommendations advocating nutrients, foods and eating patterns that are most closely associated with reduced risk. Patients assume that physicians are knowledgeable regarding the role of diet in health and that they are trained to counsel patients... more »

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3 net votes
7 up votes
4 down votes
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(@lholmber)

Goal 2: Reduce Human Disease

Can we safely move CAR therapy i beyond limited academic centers?

CAR therapy has resurrected the role of immunotherapy in treating cancer. But it has significant toxicity. Can we train , standardize the care of and adequately oversee the care of patients getting CAR therapy , especially as company begin to approach FDA for approval of this therapy and up scale for mass treatment.

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11 net votes
26 up votes
15 down votes
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(@bavtad)

Goal 2: Reduce Human Disease

Bicuspid aortic valve is not a “one size fits all” condition. Variations exist within families and the population generally.

Clinical disease manifestations in individuals and families where bicuspid aortic valve is present may best be described as a spectrum, with considerable variability in presentation. Patients and families would benefit from an individualized approach, which does include familial medical history, but couples that information with current best understanding of BAV, considering both variations and risks.

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-4 net votes
10 up votes
14 down votes
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