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

Optimal treatment of patients with PAD

What is the optimal treatment of patients with PAD?

Submitted by (@societyforvascularsurgery)

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

Details on the impact of addressing this CQ or CC :

­PAD is a global epidemic that has a large and growing public health burden.

­

Conduct pragmatic trials to define the comparative effectiveness of treatments (medical, surgical, interventional, and combination) for all stages of peripheral arterial disease - claudication and limb-threatening ischemia

Name of idea submitter and other team members who worked on this idea : Society for Vascular Surgery

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0 net votes
1 up votes
1 down votes
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Goal 2: Reduce Human Disease

Further development and clinical usefulness of COPD phenotypes

Further development and clinical usefulness of COPD phenotypes

a. Linking phenotypes to clinically meaningful outcomes

b. Establishing treatment algorithms for different phenotypes

Submitted by (@nhlbiforumadministrator)

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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3 net votes
3 up votes
0 down votes
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Goal 2: Reduce Human Disease

Clinical Trials in Pediatric Sleep Disorders - Effect of adenotonsillectomy

Effect of adenotonsillectomy on behavioral and cardiovascular outcomes in children with primary snoring

Submitted by (@nhlbiforumadministrator)

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

Details on the impact of addressing this CQ or CC :

The recent NHLBI Childhood Adenotonsillectomy (CHAT) study showed highly significant behavioral improvements in children with obstructive sleep apnea syndrome . This study included children with an apnea hypopnea index as low as 2/hr. Many small or suboptimally controlled studies suggest that even primary snoring can affect behavior. If large randomized controlled trials confirm this finding, it will radically affect the treatment of the estimated 10% of children who snore.

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

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0 net votes
1 up votes
1 down votes
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Goal 2: Reduce Human Disease

Characterizing tissue response to injury

Can we characterize in detail the nature of tissue response to injury, resulting in either aberrant repair or regeneration, to generate predictive algorithms and targetable nodes where manipulation can alter the outcome?

 

Can we characterize tissue remodeling, repair and injury? What processes are involved that lead to outcome X and dependent on various perturbegens, genetics? How do you translate that?

Submitted by (@nhlbiforumadministrator)

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 : NHLBI Staff

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13 net votes
22 up votes
9 down votes
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Goal 2: Reduce Human Disease

Single nucleotide polymorphisms, microarray and sickle cell disease

Do SNPs account for any of the variability seen in the phenotypic expression of sickle cell disease? Can microarray analysis be used to map these SNPs, promoting refined care plans for those living with sickle cell disease?

Submitted by (@sicklecellwarrior)

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

Details on the impact of addressing this CQ or CC :

Multiple Single Nucleotide Polymorphisms (SNPs) have previously been identified in the mu opioid receptor and it is estimated that millions might exist throughout the genome. Drastic phenotypic variability exists among patients in the sickle cell community. Do SNPs account for any of this variability and can this information be used to more effectively treat sickle cell disease.

Name of idea submitter and other team members who worked on this idea : Sickle Cell Warriors, Inc. community members

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18 net votes
32 up votes
14 down votes
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Goal 2: Reduce Human Disease

Interaction with Office of Rare Diseases

NHLBI should engage more interactively with the Office of Rare Diseases (ORD).

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 :

There is a synergy that comes with collaboration with rare disease clinical science from other institutes. Some of this synergy is scientific, much of it falls into the support engendered to the patient support groups that are partners in filling the funding gap that many NHLBI initiatives have.

Feasibility and challenges of addressing this CQ or CC :

To go it alone in rare disease dissociates the lung disease clinicians from the huge movements in registry science that will impact the field for years to come.

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

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2 net votes
2 up votes
0 down votes
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Goal 2: Reduce Human Disease

Stem Cell Biology

There is a need to develop an artificial and functional hematopoietic stem cell (HSC) niche that allows for the expansion of repopulating HSCs.

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

Details on the impact of addressing this CQ or CC :

Methods to expand hematopoietic stem cells have continued to be examined extensively because stem cell numbers in the graft are important for clinical outcomes following transplantation. These numbers are particularly relevant in umbilical cord blood (UCB) transplantation, where low numbers of stem cells are directly related to delayed hematopoietic and immune reconstitution. Improved HSC expansion strategies may significantly impact transplantation outcome, enabling broader applications beyond UCB transplantation. Furthermore, these strategies are also needed to realize the full therapeutic potential of genome editing technologies to correct hematopoietic stem cells derived from patients with hematologic disorders. Since efforts to expand HSCs in cytokine-supported liquid cultures have been largely unsuccessful, efficient expansion will require an appropriate context that is provided by the hematopoietic stem cell niche. Future studies must also evaluate how niche signals regulate stem cell function to optimize cell expansion, and proper humanized mouse models must be developed to help predict stem cell function and regulation by the niche.

Name of idea submitter and other team members who worked on this idea : Alice Kuaban on behalf of the American Society of Hematology (ASH)

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28 net votes
46 up votes
18 down votes
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Goal 2: Reduce Human Disease

Adult cardiovascular risk in patients with congenital heart disease

Do patients with congenital heart disease have the same, higher, or lower risk for coronary artery disease as they age into adulthood?

Submitted by (@nhlbiforumadministrator)

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

Details on the impact of addressing this CQ or CC :

By improving our understanding of cardiovascular risk in patients with congenital heart disease we may be able to improve our surveillance for disease and intervene earlier to address this risk factors. The adult population with congenital heart disease is undergoing rapid growth.

Feasibility and challenges of addressing this CQ or CC :

The adult population with congenital heart disease is undergoing rapid growth. Novel uses of electronic health records (EHRs) and registries may enable us to answer these questions in a cost-efficient manner.

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

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3 net votes
15 up votes
12 down votes
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Goal 2: Reduce Human Disease

Mechanism and target identification for cardiovascular disease using database mining and experimental strategies

The big database of -omics and noncoding RNAs provide a valuable search engine to establish the link of various disease conditions with pathways and molecules. During recent year, data mining of available biomedical data and information has merged for target discovery and mechanism identification. However, current database mining research remains limited and largely hypothetical. Comprehensive research using the combinatory ...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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19 net votes
26 up votes
7 down votes
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Goal 2: Reduce Human Disease

Development of non-contrast alternatives in cardiac magnetic resonance imaging

Critical Challenge

Submitted by (@str0001)

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

Details on the impact of addressing this CQ or CC :

Late gadolinium-enhancement cardiac magnetic resonance imaging (MR) plays a crucial role in the evaluation of patients with suspected myocardial scar tissue. Alternative methods to contrast-enhanced MR however are in need, given the number of patients who have concomitant compromised renal function and concern for nephrogenic systemic fibrosis. Noncontrast MR techniques such as diffusion-weighted imaging would complement and eventually replace gadolinium administration thus impacting the evaluation of those with suspected and confirmed infiltrative cardiac processes and systemic diseases.

Feasibility and challenges of addressing this CQ or CC :

Late gadolinium enhancement technique characterizes enhancement patterns of heart disease, identifies areas amenable to ablation, and aids in decisions pertaining to workup and therapy. The underlying mechanism of Brownian motion/diffusion in the expanded extracellular space makes diffusion weighted imaging a potential gadolinium-saving modality. Diffusion MR, applied primarily in the brain and abdominal imaging, is underutilized in the heart given respiratory and cardiac motion. A need exists to further develop and apply noncontrast MR techniques towards cardiovascular applications. Such methods are promising noncontrast alternatives to characterize patients with myocardial disease, determine those with differing prognoses, and direct appropriate therapies to subgroups.

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

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3 net votes
6 up votes
3 down votes
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Goal 2: Reduce Human Disease

mechansisms of post thrombotic syndrome

No good medical therapy exists to prevent and treat post thrombotic syndrome, the most common sequlae from a deep vein thrombosis. Recent trial data suggests that compression stockings do not prevent PTS, and thrombolysis is expensive and risky. The basic mechanisms related to fibrosis of the vein wall are not well understood.

Submitted by (@henke0)

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

Details on the impact of addressing this CQ or CC :

Further research into this area, both at the human and animal model level would allow potential translation of novel agents without the risks of anticoagulatnts, as well as shine light on basic fibrotic vascular disease processes.

Feasibility and challenges of addressing this CQ or CC :

Doable with both well defined human patients, and animal models of the disease that are similar to humans

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

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1 net vote
5 up votes
4 down votes
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Goal 2: Reduce Human Disease

Sleep effects on lung health

How does insufficient sleep impair hormonal, cellular, and tissues barriers mediating lung immunity and risk of lung infection?

Submitted by (@nhlbiforumadministrator)

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 : NHLBI Staff

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-7 net votes
13 up votes
20 down votes
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