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

Quantitative imaging biomarkers for chronic lung disease

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 :

Methods for stratifying patients with diffuse lung disease are crucial for predicting their clinical course and directing appropriate therapies accordingly. Currently imaging markers for prognostic stratification are limited, due to observer variability in characterizing the type and degree of computed tomography (CT) abnormalities. A reproducible method for categorizing varying diffuse lung diseases on CT imaging is needed, particularly in combination with other biomarkers in a multidisciplinary approach. With lung cancer screening, the characterization and stratification of patients with varying COPD phenotypes and interstitial lung disease are essential to aid in management of the large number of patients who currently satisfy criteria for CT lung cancer screening.

Feasibility and challenges of addressing this CQ or CC :

Currently the classification of diffuse lung disease on CT is based upon visual evaluation and qualitative or semi-quantitative evaluation of CT data. Diffuse lung disease manifests with varying CT findings and distribution within the lung. Computer-assisted tools for quantifying airways and parenchymal disease have been developed. More-sophisticated quantitative computer image-analysis methods, such as those that address three-dimensional spatial orientation, are possible given advances in computer capabilities yet remain in need of further development. Advances in magnetic resonance imaging (MR) technology, positron emission tomography (PET), and PET/MR will increase the ability to characterize diffuse lung disease quantitatively. The ability of such technology to differentiate subtypes within more frequently occurring and clinically-significant diffuse lung disease is feasible. Such tools would impact a large population, particularly given the potential need to phenotype emphysema and smoking-related interstitial pneumonias in those undergoing CT screening.

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

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Goal 2: Reduce Human Disease

Novel technologies to save minutes and lives

There is a need to develop hand-held portable imaging or other technologies that can help paramedics to collect and transmit data when patients are undergoing cardiovascular events.

Submitted by (@nhlbiforumadministrator1)

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

Details on the impact of addressing this CQ or CC :

Save time and lives for patients destined to Emergency

Feasibility and challenges of addressing this CQ or CC :

The component technologies are developed enough to integrate them in the next 5-10 years

Time spent waiting for an ambulance, driving to a hospital and enduring diagnostic tests before medical intervention can quickly add up, especially with patients undergoing cardiovascular events. In the era of internet, smartphones, and portable imaging and handheld technologies, saving valuable time during which paramedics can be assisting the emergency physicians in diagnosing heart attacks and help to identify and start the needed intervention at once when the patient arrive at the hospital.

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

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6 net votes
13 up votes
7 down votes
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Goal 2: Reduce Human Disease

Non-obstructive Coronary Disease

It is increasingly apparent that ischemic heart disease does not equal obstructive coronary disease. There is a large, heterogeneous population of individuals who present to the ED with chest pain syndrome with or without a troponin elevation, who on diagnostic evaluation have non-obstructive disease and who on prospective studies have increased risk for ACS and early mortality; other literature shows the same for coronary ...more »

Submitted by (@matthew.burg)

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

Details on the impact of addressing this CQ or CC :

Patients with non-obstructive coronary disease represent a large proportion of patients who report to the ED with chest pain of cardiac origin. This hetergeneous group of patients have elevated ACS/mortality risk, yet little is known of the underlying pathophysiology or of the precipitants of chest pain and events. Research focused on characterizing distinct patient groups and associated pathways by which environmental factors precipitate chest pain and events would provide enormous benefit in the development and targeting of interventions to improve patient outcomes and reduce risk of catastrophic cardiac events.

Feasibility and challenges of addressing this CQ or CC :

The challenges include assembling a sufficiently large patient sample to characterize distinct phenotypes. The technologies for this characterization are sufficiently disseminated to accomplish this. Furthermore, the technologies for ascertaining key elements of pathophysiology - e.g., vascular, autonomic - are also widely available. The recent expansion of technologies for ecological measurement of environmental factors and of physiological phenomena make this a feasible undertaking at this time

Name of idea submitter and other team members who worked on this idea : Matthew Burg, PhD

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52 up votes
18 down votes
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Goal 2: Reduce Human Disease

Behavior change labs: an interdisciplinary team approach

Will integration of behavior science in clinical research improve effectiveness of interventions for HLBS diseases associated with behavioral risk factors?

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 :

Currently, there is no industry support for T1 (basic to clinical) behavioral research and therefore little incentive for basic and clinical behavioral scientists to work together to develop and test new, innovative strategies for changing HLBS-related behaviors based on basic behavioral science findings on motivation, perception, cognition and social relationships. Bringing together collaborative, interdisciplinary teams of basic behavioral scientists and clinically-oriented behavioral researchers could spur development and testing of innovative new approaches to difficult HLBS-related behavioral problems such as obesity, unhealthy diets, sedentary lifestyles, smoking and non-adherence to preventive and therapeutic HLBS regimens.

Feasibility and challenges of addressing this CQ or CC :

New research in the behavioral sciences is elucidating the basic psychological, cognitive, social and behavioral processes underlying behavior and behavior change. Findings in this area could be developed into new strategies targeting problematic HLBS-related behaviors, but a mechanism for developing and testing novel ideas is needed. Networks designed to bring together basic and clinically-oriented behavioral researchers can enable better understanding of the bases of HLBS-related behaviors and accelerate the translation of findings into new approaches.

Adopting and maintaining healthy habits and lifestyles – such as eating healthy diets, engaging in regular physical activity, stopping smoking, and regularly taking prescribed medications – are crucial to heart, lung, blood and sleep (HLBS) health (Akesson et al, 2014; Mozaffarian, 2014). However, for most people, engaging in and maintaining a healthy lifestyle is challenging. Interventions designed to promote behavior change have had limited success, often influencing individuals over the short-term but failing to alter behaviors over longer periods of time, which is necessary to realizing the full benefits of a healthy lifestyle. Underlying the problematic behaviors associated with HLBS-related behavioral risk factors are fundamental psychological, motivational, cognitive and social processes that represent promising targets for the development of new, more effective behavioral interventions. For example, basic behavioral scientists are investigating the role of poor executive function in unhealthy eating behavior and exploring new ways to address the "self-control" failures that lead to impulsive eating.

 

However, unlike the biomedical arena where the translational pathway from basic science to clinical application is supported by both NIH and industry, there is no industry support and relatively little NIH funding devoted to T1 behavioral research -- i.e., research translating basic behavioral science findings into clinically significant behavioral interventions. As a result, basic behavioral science researchers have little incentive to collaborate with clinical researchers to develop and test novel behavioral treatments. Bringing together collaborative, interdisciplinary teams of basic behavioral scientists and clinically-oriented behavioral researchers could spur development and testing of innovative new approaches to difficult HLBS-related behavioral problems.

 

A compelling question is how to bring together these disparate researchers over a long enough time frame to enable them to identify, develop and testing new strategies for tackling resistant behavioral problems. One way to address this question is to fund a network of "behavior change labs," each of which brings together teams of basic behavioral scientists who are investigating the bases of behavior and behavior change with clinical researchers interested in designing, optimizing and testing novel ideas for tackling the difficult behavioral problems represented by obesity, unhealthy diets, sedentary lifestyles, smoking and non-adherence to medications used to prevent or treat HLBS diseases and disorders.

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

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83 net votes
129 up votes
46 down votes
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Goal 2: Reduce Human Disease

Research on in vitro and in vivo Biomarker Predictors of Clinical Response in Lung Disease

How can research on in vitro and in vivo biomarker predictors of clinical response in lung disease be encouraged?

Submitted by (@skrenrich)

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 : Cystic Fibrosis Foundation

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

Blood Pressure Recommendation

What should be the systolic blood pressure goal for pharmacological treatment, and should it vary by age or by cardiovascular disease (CVD) risk category?

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 :

Despite fifty years of clinical trial research and forty years of national guideline activity, important clinical questions remain under intense scientific debate. The importance of these questions are underline by the scientific consensus that hypertension is most important cardiovascular risk factor globally, in fact, more important than even tobacco use.

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

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-1 net votes
13 up votes
14 down votes
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Goal 2: Reduce Human Disease

Does epinephrine improve outcomes in OHCA

Epinephrine is the primary drug that is used in resuscitation but observational studies and a few small RCT suggest that it improves short term but not long term outcomes. Factors such as timing, dose, quality fo CPR and post-resuscitation care all confound the issue. Large RCTs conducted at multiple centers are desperately needed to address this question.

Submitted by (@dayam0)

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

Details on the impact of addressing this CQ or CC :

If short terms outcomes are improved but not long term outcomes, we are only adding costs and not improving population health

Feasibility and challenges of addressing this CQ or CC :

Will require a large prehospital clinical trials network and ideally also a current national registry of OHCA to address secular changes in other confounding variables

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

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1 net vote
3 up votes
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Goal 2: Reduce Human Disease

Immunologic Treatment of Hematologic Malignancies

How can the use of CAR T-cell and checkpoint blockade strategies be optimized in order to cure hematologic diseases?

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

Details on the impact of addressing this CQ or CC :

As the body of evidence continues to grow on the potential applications for advanced immunotherapies, next-generation research must focus on addressing the possible curative effects that checkpoint blockades or adoptive CAR T-cell strategies can have for blood diseases including hematologic cancers. This will require specific research programs to fully understand the optimal role for these therapies within the continuum of care. To optimize these strategies for treatment of hematologic diseases, studies are needed to decipher specific hematologic diseases and circumstances under which these checkpoint blockers and CAR T-cell therapies may be employed as frontline approaches. Furthermore, while the optimal approach for these therapies is unclear, advanced studies are needed to elucidate the potential benefit in combining these promising approaches and whether patients can be better identified a priori for these therapies.

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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13 net votes
28 up votes
15 down votes
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Goal 2: Reduce Human Disease

Interstitial Lung Disease

Does prednisone therapy improve outcomes in acute exacerbation of IPF?

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 :

It is common practice to give high-dose prednisone to patients experiencing acute exacerbation of IPF, a relatively common (10-15% annual risk) and deadly event, but there are no good data to support its use. There is a significant risk of morbidity from this therapy. A well-designed multicenter trial could answer this question definitively.

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
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Goal 2: Reduce Human Disease

Prevent cytopenia in septic patients

Sepsis is the leading cause of death in critically ill patients in the USA, affecting particularly young children and the elderly. A hallmark of septic shock patients upon diagnosis is peripheral blood cytopenia. This persistent cytopenia commonly affects myeloid, lymphoid and erythroid lineages resulting in immunosuppression and is a well-established predictor of fatal outcome. Clinical trials targeting the production ...more »

Submitted by (@ben.croker)

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 current standard-of-care for sepsis patients involves supportive therapy and the early administration of antibiotics, which has been essentially unchanged in 40 years. Therapies that prevent the loss of immune cells are likely to be beneficial to avoid immune suppression and prevent the development of life-threatening systemic infection.

Feasibility and challenges of addressing this CQ or CC :

One of the challenges of addressing this question are the large number of biochemical pathways that influence hematopoiesis. Most have not been studied in the context of infection in animal models or in clinical samples. But many animal models and reagents exist that would enable researchers to study this problem. The study of hematopoiesis during infection is feasible and a number of broad questions could be addressed:

a. Do hematopoietic cells and their precursors undergo cell death in response to intracellular pathogens leading to immune suppression?

b. Are hematopoietic cells and their precursors affected by extracellular pathogen-derived products or host-derived molecules resulting from severe injury? Does this lead to cell death and/or prevent the proliferation and differentiation of hematopoietic stem and progenitor cells?

c. How do genetic factors, chronic infection and comorbidities increase the activity of cell death pathways and/or impair the proliferation and differentiation of hematopoietic stem and progenitor cells?

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

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11 up votes
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Goal 2: Reduce Human Disease

Randomized trial of low carbohydrate high fat dietary pattern

There is a need for a large, simple, hard outcome trial of dietary advice to measure the effects of lowering simple carbohydrates versus lowering unhealthy fats. The main challenge will be to overcome bias favoring the more conventional dietary approach.

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 :

Substantial numbers are moving towards low carbohydrate/high fat diets to the extent that since 2000 the macronutrient composition of the US diet has changed towards lower carbohydrate and higher fat intakes; however, CV mortality rates continue to decline. The outcome of the trial will help frame future guidelines for healthy diets.

Feasibility and challenges of addressing this CQ or CC :

Yes, since computer or smartphone based intervention materials and passive outcome collections (CMS, NDI, health care organizations) are increasingly being utilized.

Low carbohydrate high fat (LCHF) diets first popularized for weight reduction are increasingly being promoted to prevent diabetes and cardiovascular disease. All trials to date have been small and short term. Compared to more traditional high carbohydrate low fat diets, LCHF diets may be somewhat more beneficial for weight control, glucose homeostasis, and dyslipidemia, but less beneficial for LDL reduction.

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

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-17 net votes
5 up votes
22 down votes
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Goal 2: Reduce Human Disease

Sleep Apnea

Does treatment of sleep apnea prevent cardiovascular disease?

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 : ATS Member

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1 net vote
1 up votes
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