Goal 2: Reduce Human Disease

Exercise in congenital heart disease and arrhythmia syndromes

What are the true risks of physical activity and competitive sports for children/young adults with congenital heart disease and arrhythmia syndromes?

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 :

Improve the quality of life of patients with congenital heart disease or arrhythmia syndromes who are currently restricted from competitive sports.

Feasibility and challenges of addressing this CQ or CC :

While we previously restricted many of these patients, the pendulum seems to be swinging back in terms of our thinking about this. While some preliminary data has suggested safety, more data is needed.

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

Voting

4 net votes
15 up votes
11 down votes
Active

Goal 2: Reduce Human Disease

Pulmonary Vascular Diseases

Does "goal-targeted" therapy (with adjustments/additional therapy, if certain "goals" are not achieved) improve quality of life, functional status, and survival in patients with pulmonary arterial hypertension? Trials of therapies for hepatopulmonary syndrome.

Submitted by (@nhlbiforumadministrator)

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

Feasibility and challenges of addressing this CQ or CC :

This is a view of problems in the field.

Pulmonary Hypertension Clinical Research: Current Problems and Possibilities

Current studies limited to the short term, with soft outcomes.

No mechanistic studies embedded in trials.

Control of phenotype is weak.

Small n: lumping of cohorts.

No factorial of advanced design.

No biological samples obtained for study.

Failure to study basic management issues.

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

Voting

2 net votes
2 up votes
0 down votes
Active

Goal 3: Advance Translational Research

Sickle cell disease and fatigue

What factors, other than hemoglobin count, are involved in the extreme fatigue experienced by individuals 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 :

Many in the sickle cell community report continuous fatigue that interrupts their daily activities in spite of normal, or above baseline, hemoglobin levels

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

Voting

17 net votes
29 up votes
12 down votes
Active

Goal 2: Reduce Human Disease

Defining the developmental abnormalities leading to birth defects

Can we define the developmental abnormalities leading to birth defects and extrapolate that knowledge to define strategies for regeneration?

 

How can we recognize initiation of disease earlier?

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

Voting

-1 net votes
8 up votes
9 down votes
Active

Goal 3: Advance Translational Research

Financial and psycosocial impact of transition in sickle cell disease

What are the financial and social implications for individuals living with SCD from adolescence transitioning into adult care? What impact does this transition have on the caregiver and family as a whole?

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 :

It is very difficult for many living with SCD to maintain employment (many people have to rely on disability benefits to survive) finish school, even maintain relationships. Greater understanding of the financial and social impact would enable advocacy and direct patient service organizations to better prepare the transitioning population, reducing the financial strain on the individual and the growing medical debt incurred by hospitals and government social service programs.

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

Voting

30 net votes
38 up votes
8 down votes
Active

Goal 2: Reduce Human Disease

Enhanced Pain Research in Sickle Cell Disease

There is a need for more enhanced pain research in order to help improve sickle cell disease patient outcomes and quality of life.

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

Details on the impact of addressing this CQ or CC :

Pain is the most common clinical manifestation of sickle cell disease (SCD) and accounts for a large proportion of emergency department visits and hospitalizations. Due to its impact on the patients’ quality of life, there is a need for more basic and clinical research studies focused on understanding the mechanisms of different pain syndromes as well as the role of neurotransmitters and inflammation in acute and chronic SCD pain. Also, comparative effectiveness studies in the management of chronic pain will be crucial in helping to improve the patients’ overall quality of life.

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

Voting

39 net votes
58 up votes
19 down votes
Active

Goal 2: Reduce Human Disease

Asymptomatic carotid artery disease

What is the most effective approach to asymptomatic carotid artery disease to reduce the burden of stroke?

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 :

­Develop improved understanding of factors associated with carotid plaque progression and the reliable identification of higher risk asymptomatic lesions. This will lead to improved selection of asymptomatic patients for carotid intervention, a critical area of controversy and a highly prevalent condition.

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

Voting

2 net votes
3 up votes
1 down votes
Active

Goal 2: Reduce Human Disease

The Use of Therapeutic Apheresis to Reduce Circulating Levels of Galectin-3 and other Cancer and Inflammation Promoting Factors

Inflammation plays roles in cancer initiation, promotion, and progression. Elevated circulating galectin-3 (Gal-3) protein and other cancer and inflammation promoting factors (CIPFs) such as C-reactive protein and VEGF are associated with tumorigenesis and may play causative roles. Plasma Gal-3 is a biomarker, prognosticator, and pathogenic mediator of diverse cancers and is emerging as a therapeutic target. Preliminary ...more »

Submitted by (@elaine)

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

Details on the impact of addressing this CQ or CC :

Apheresis therapy in a clinical setting, both alone and in combination with conventional protocols, shows great potential to enhance treatment regimens, reduce dosage and side effects, improve drug deliver to target tissues, reduce long term treatment related morbidity and improve outcomes with significant benefits for patients with a broad range of cancer types and stages.

Feasibility and challenges of addressing this CQ or CC :

The need for well designed, randomized clinical trials would be readily feasible with the appropriate IND. Grant support will be needed for further development of this concept, as well as to develop columns with more optimized and specific capabilities, in addition to clinical trials demonstrating efficacy.

 

Apheresis is highly underutilized and underfunded in the US, while Apheresis research and development is much more advanced and widely utilized in Europe and Asia.

Name of idea submitter and other team members who worked on this idea : Isaac Eliaz, MD

Voting

33 net votes
40 up votes
7 down votes
Active

Goal 1: Promote Human Health

Enhancing Understanding of Determinants of Health in Rural Areas & Developing Solutions

What are the biological, environmental, social and economic determinants of health in rural areas related to COPD and other lung disease.

Submitted by (@gacdk0)

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

Details on the impact of addressing this CQ or CC :

There are extreme disparities in the impact of COPD in rural areas, especially in rural Appalachia. These areas experience much larger prevalence rates and higher rates of hospitalization, readmissions and other health indicators that contribute to increased cost and decreased quality of life. These are also areas with the least ability to make improvements. Research that can inform both the causes of these disparities and identify proven methods for systematically confronting these issues has the potential to dramatically improve overall health status in rural America

Name of idea submitter and other team members who worked on this idea : Grace Anne Dorney Koppel

Voting

7 net votes
8 up votes
1 down votes
Active

Goal 2: Reduce Human Disease

Identify microvascular angina in emergency department patients

Assess better screening measures (phsyiological tests, biomarkers or novel technology) for diagnosis of microvascular angina in emergency department patients with chest pain.

Submitted by (@basmah.safdar)

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

Details on the impact of addressing this CQ or CC :

Microvascular dysfunction affects up to 40% of emergency department patients presenting with chest pain and without obstructive coronary artery disease. These patients are often undetected with traditional tests including ECG, troponin, stress tests or even conventional angiograms. Microvascular dysfunction can occur in presence or absence of non-obstructive or obstructive coronary artery disease and needs to be investigated as a discrete entity. In the absence of clear diagnosis, these patient often return to emergency departments for recurrent symptoms. These presentations are associated with functional impairment, increased health care costs and resource utilization. By improved testing and detection, we could identify the source of patient's recurrent symptoms and presentation, address the underlying disease and improve resource utilization and costs.

Feasibility and challenges of addressing this CQ or CC :

Currently available tests for microvascular angina are either invasive or sophisticated with limited generalizability, are expensive and may be associated with radiation.

 

The challenge is to find practical, non invasive screening measures that could help differentiate patients with microvascular angina from patients with normal coronary blood flow or those with obstructive coronary artery disease.

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

Voting

11 net votes
26 up votes
15 down votes
Active

Goal 2: Reduce Human Disease

Does lowering circulating lipoprotein(a) levels influence cardiovascular outcomes?

A comprehensive research strategy and plan is needed to determine the most efficient, safe, cost-effective and widely applicable strategy to decrease circulating levels of lipoprotein(a) and to determine whether lowering circulating lipoprotein(a) levels will reduce the risk of developing cardiovascular disease such as a heart attack or a stroke as well as the progression of atherosclerosis or aortic stenosis.

Submitted by (@serevill)

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

Details on the impact of addressing this CQ or CC :

Approximately 20% of the population are characterized by elevated circulating levels of lipoprotein(a), regardless of age, gender or blood cholesterol levels. Estimates suggest that up to 90% of the variation in plasma lipoprotein(a) levels could be due to genetic factors, which makes lipoprotein(a) the most prevalent inherited risk factor for cardiovascular diseases (CVD). Large-scale genetic studies have shown that Lipoprotein(a) was the strongest genetic determinant of CVD such as atherosclerosis and aortic stenosis. Lipoprotein(a) is one of the strongest predictors of residual CVD risk and has been shown to improve CVD risk prediction in several population-based studies. Lipoprotein(a) is also one of the strongest known risk factors for spontaneous ischemic stroke in childhood.

A comprehensive research strategy aiming at identifying, evaluating interaction with other risk factors, treating and educating patients with elevated lipoprotein(a) levels would result in substantial reductions of health care costs in the US and around the globe by reducing the burden of CVD while simultaneously improving the quality of life of these patients.

Feasibility and challenges of addressing this CQ or CC :

The list of pharmaceutical agents that reduce lipoprotein(a) levels is steadily increasing. There are approximately half a dozen strategies that have been shown to significantly and safely lower lipoprotein(a) levels. One of the challenges of this research strategy will be to determine which of these strategies represent the most efficient, safe, cost-effective and widely applicable approach to lower lipoprotein(a) levels and CVD outcomes.

Increasing awareness on lipoprotein(a) and CVD will also be of utmost importance for this effort as relatively few physicians perform lipoprotein(a) testing and even fewer patients are aware of their lipoprotein(a) level. The first sign of high lipoprotein(a) is often a heart attack or stroke. Our challenge will be to identify patients with high lipoprotein(a) that could be enrolled in trials of risk characterization and lipoprotein(a)-lowering.

Name of idea submitter and other team members who worked on this idea : Sandra Revill Tremulis on behalf of the Lipoprotein(a) Foundation Scientific Advisory Board

Voting

235 net votes
297 up votes
62 down votes
Active

Goal 2: Reduce Human Disease

Childhood Interstitial Lung Disease

What is the natural history of the best characterized ChILD disorders (surfactantrelated sequence variants, neuroendocrine cell hyperplasia of infancy (NEHI),pulmonary interstitial glycogenosis (PIG),idiopathic pulmonary hemosiderosis)?

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 :

We know little about the natural history of many of the child entities, and their relative rarity makes it difficult for any one center to answer the major questions they pose. The children has begun a patient registry that will begin to address the issue of natural history and disease tracking.

 

a. To improve the power of such a registry, we suggest that support be provided to find novel methods to link this data base to available electronic medical records of participating centers in order to assess physiologic and other clinical associations with specific diseases.

 

b. Support for a biomarker repository holding serum, frozen and fixed lung tissue, patient DNA, RNA, and proteomic and metabolomic materials, and bronchoalveolar lavage effluent and cell pellets, will allow for genome wide analysis as well as proteomic and metabolomic analysis.

Feasibility and challenges of addressing this CQ or CC :

This question is best addressed in the context of a multicenter data registry, ideally linked to a clinical sample.

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

Voting

2 net votes
2 up votes
0 down votes
Active