Thank you for participating!

Thank you to all who contributed to the National Heart, Lung, and Blood Institute (NHLBI) Strategic Visioning Forum. Ultimately, over 1,000 ideas were submitted, with more than 42,000 votes. This remarkable response exceeded expectations and provided a wealth of ideas to draw upon as NHLBI moves forward. Please visit the NHLBI Strategic Visioning page to find out more about the NHLBI Strategic Visioning process.


Welcome to the National Heart, Lung, and Blood Institute (NHLBI) Strategic Visioning Forum. The Institute is gathering ideas for the most compelling scientific priorities in the four NHLBI Strategic Goals to address over the next decade.

Goal 2: Reduce Human Disease

Optimal hemoglobin threshold for transfusion in children with ARDS?

Do different hemoglobin transfusion thresholds alter outcomes in children with ARDS? What is the optimal *minimum* transfusion threshold for children with ARDS? What patient-centered outcomes can be affected by transfusion strategies: ventilator free days, time to organ function recovery, duration of intensive care stay, survival?

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 Society of Critical Care Medicine Executive Committee/Council

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8 net votes
9 up votes
1 down votes
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Goal 1: Promote Human Health

How do Circulating Precursor Endothelial Cells contribute to newly formed vessels

Endothelial cells derive from cells in the bone marrow. Circulating precursor endothelial cells contribute to newly forming vessels.

Do Alk 1 and/or Endogln mutations affect the functions of these cells once they incorporate into growing vessels. These vessels then go on to form arteriovenous malformations

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 Marianne Clancy MPA, Chris Hughes PhD

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2 net votes
2 up votes
0 down votes
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Goal 3: Advance Translational Research

Improving Community-Based Care for Sickle Cell Disease

Sickle cell treatment centers are located throughout the United States, primarily in urban areas, and play an invaluable role. However, there is a critical need to identify and educate primary care providers who can provide routine and preventive care, but will also know when to consult with/refer to hematologists and other appropriate providers when necessary.

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

Details on the impact of addressing this CQ or CC

For the first time, comprehensive guidelines addressing the management of sickle cell disease were issued in 2014 by the NHLBI. (Previous guidelines were not comprehensive.) The 2014 guidelines, which address issues such as health maintenance and the treatment of acute and chronic complications, are based on a systematic review of available evidence; consensus of an expert panel; and published, vetted guidelines by other organizations when evidence was unavailable or insufficient. These guidelines can provide a solid overview of the knowledge essential for the care of people with sickle cell disease.

Identifying primary care providers who can provide routine and preventive care but at the same time are knowledgeable about sickle cell disease, should be a more efficient, less costly method of provide important health services to people with sickle cell disease and should ultimately improve the health and well being of this population, particularly for people who do not live near a sickle cell center.

Feasibility and challenges of addressing this CQ or CC

Addressing this question is very feasible. However, for a variety of reasons, including misconceptions about patients with the condition, it may be challenging to recruit large numbers of primary care providers.

Name of idea submitter and other team members who worked on this idea The Sickle Cell Association of New Jersey

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

Funding Limitations Block Intervention Research

The cap on R01 research grants at $500,000 per year has not changed in over 20 years. In the current fiscal crisis for research it has become an immovable block to submitting intervention studies (randomized clinical trials on treatment). Routine advice from NIH staff is to not even try for a larger study. The cap applies to every year, so one can design a trial that costs less than $2.5 million but exceeds $500,000 in... more »

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

Details on the impact of addressing this CQ or CC

While it is difficult to argue for increasing funding for anything these days, the $500,000 cap on R01 funding is exerting a perverse bias on research. Very few intervention trials can be accomplished without exceeding this limit in at least one year. This means that trials are limited to those of interest to the NIH staff and to the pharmaceutical industry. Investigators interested in solving therapeutic problems are being force to abandon trials and rely on natural experiments and observational studies, which cannot address all important questions. The natural creativity of the scientific community is being artificially suppressed, distorting the field. The notion that pragmatic trials can substitute for explanatory trials is misplaced. Many unsettled questions cannot be pursued in pragmatic trials, which generally reduce informed consent to a degree to call into question their ethics. It is also unclear that many pragmatic trials can be done under the cap, since they often require extensive work with providers and the healthcare delivery system hierarchy.

Feasibility and challenges of addressing this CQ or CC

Even a modest change in the cap would be very helpful. Perhaps allowing 1-2 years to be as high as $600,000 without triggering the permission process. Alternatively, the review process for studies exceeding $500,000 could be streamlined if the total does not exceed $2.5 million. Other ways to introduce flexibility are possible. This would allow more ideas to go to review and the staff, who are increasingly deciding which grants get funded, will have more to choose from.

Name of idea submitter and other team members who worked on this idea Stephen P. Fortmann

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

Improving Outcomes for Lung Transplantation

How can the relatively poor outcomes of lung transplantation be improved through better understanding of basic biology and/or clinical care?

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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-1 net votes
3 up votes
4 down votes
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Goal 4: Develop Workforce and Resources

Accounting for indirect costs

Strengthen institutional accountability to the investigator for appropriate use of indirect funds

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

Details on the impact of addressing this CQ or CC

If the PI had some way of affirming institutional accountability for use of indirect funds, it would greatly strengthen PI support and respect. This would result in better space, admin support, support for resubmission,and overall prestige within the institution. The end result would be more effective research and attractiveness of a research career. Clinician scientists would benefit greatly.

Feasibility and challenges of addressing this CQ or CC

Institutions rarely are interested in accounting to the investigator for use of IDC. Quite possibly due to fear that the investigator would be unreasonable demanding. Some creativity could get around this. A list of Investigators "rights" or "expectations" in terms of institutional support in the effective use of public funds. A committee of PIs to report to NIH on Institutional support. Perhaps with a response for the Institution. The value of this might be judged by the intensity of the pushback from institutions. Nonetheless, hospitals and schools are accountable to JCAH, etc. Having PIs participate in an evaluation by the NIH would in essence be no different.

Name of idea submitter and other team members who worked on this idea Frank W LoGerfo

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17 net votes
24 up votes
7 down votes
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Goal 3: Advance Translational Research

Increased testing of new ideas

As indicated in two influential papers we have published, there are many potential contributing factors to obesity beyond those commonly discussed and studied and some of these are potentially modifiable. Increased research to understand whether these putative factors are really influential and whether they can be modified to produce benefits with respect to obesity are in order.

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

Feasibility and challenges of addressing this CQ or CC

The two papers we referenced in this Critical Challenge were: 1) Keith SW, Redden DT, Katzmarzyk PT, Boggiano MM, Hanlon EC, Benca RM, Ruden D, Pietrobelli A, Barger JL, Fontaine KR, Wang C, Aronne LJ, Wright SM, Baskin M, Dhurandhar NV, Lijoi MC, Grilo CM, DeLuca M, Westfall AO, Allison DB. Putative contributors to the secular increase in obesity: exploring the roads less traveled. Int J Obes (Lond). 2006;30(11):1585-94. doi: 10.1038/sj.ijo.0803326. PMID: 16801930; and 2) McAllister EJ, Dhurandhar NV, Keith SW, Aronne LJ, Barger J, Baskin M, Benca RM, Biggio J, Boggiano MM, Eisenmann JC, Elobeid M, Fontaine KR, Gluckman P, Hanlon EC, Katzmarzyk P, Pietrobelli A, Redden DT, Ruden DM, Wang C, Waterland RA, Wright SM, Allison DB. Ten putative contributors to the obesity epidemic. Crit Rev Food Sci Nutr. 2009;49(10):868-913. doi: 10.1080/10408390903372599. PMID: 19960394; PMCID: PMC2932668.

Name of idea submitter and other team members who worked on this idea David B. Allison, Ph.D.; Kevin Fontaine, Ph.D.; Kathryn A. Kaiser, Ph.D.; Andrew W. Brown, Ph.D.; Edward C. Archer, Ph.D.

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0 net votes
1 up votes
1 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?

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

Voting

83 net votes
129 up votes
46 down votes
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Goal 1: Promote Human Health

Missing upper teeth & sleep apnea treatment: Problems?

I am a 73 year old female with Hypersensitivity Pneumonitis, Complex Sleep Neap, using oxygen @ 4-5 L/min 24/7 who just had my upper teeth extracted. I notice this has a negative effect, or appears to, on the effectiveness of apnea treatment. I wake several times during the night with lips flapping! This did not happen before the extraction. Because there are still some lower teeth, I am unable to close my mouth... more »

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 this question has an answer, it would help an unknown number of persons by once again providing effective treatment for sleep apnea. I find fatigue is building again, as it did before I was diagnosed and treated.

It may be simply a matter of a different style of mask, but it appears ideas on what kind are a bit sparse on the ground.

Feasibility and challenges of addressing this CQ or CC

One would need a sampling of persons with lower teeth (some, or all) who also have sleep apnea. One would have to determine whether indeed, there is a deterioration in quality of treatment, and if the number of lower teeth are a factor. Does the form of apnea make a difference? Does age or body weight play a part?

This is not exactly couched in academic, medical terms, but it is still a valid question. Its solution, or if a solution already exists, it's distribution among sleep physicians, would help those who experience this combination of circumstances.

Name of idea submitter and other team members who worked on this idea Leslie H. Smyth

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-42 net votes
5 up votes
47 down votes
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Goal 2: Reduce Human Disease

Innovations in Red Cell Transfusion in Sickle Cell Disease

Challenges that need to be overcome in blood transfusion, especially in SCD, include:
a. Adopting molecular genotyping as the standard in blood transfusion therapy.
b. Advancing new generation, anti-oxidant hemoglobin-based oxygen carriers (HBOCs) for use in emergencies such as trauma, stroke, acute hemolysis, and in transfusion in SCD and related disorders. In SCD, HBOCs have the capacity to not only serve as substitutes... more »

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

Details on the impact of addressing this CQ or CC

Transfusion of RBC is major adjunct in the management of trauma, acute and chronic illness. Issues in blood transfusion include availability of donors, RBC typing and crossmatching, cold storage of donor cells, and limited viability of stored RBC. Globally, in many situations where blood is critically needed, these systems are not available.
An increasing percentage of people with SCD require regular RBC transfusion to prevent stroke and other major complications. In addition, RBC transfusion is employed repeatedly in the management of serious acute complications of SCD. Transfusion of normal RBC to replace or supplement the patient’s defective RBC is the most effective intervention in the management of SCD.

Impacts:
• Molecular genotyping of RBC will reduce alloimmunization.
• Use of new generation HBOCs that do not require blood typing, crossmatching, refrigeration, and that do not transmit infection, would save lives in conditions of severe hemorrhage, stroke, possibly heart attack, especially where there is no immediate access to adequate medical facilities.
• In SCD, HBOCs could prevent pain or reduce its severity and duration, prevent stroke, reduce severity of acute chest syndrome, and other vasoocclusive complications. Finally, HBOCs have the potential to alter the pathogenesis of SCD.

Feasibility and challenges of addressing this CQ or CC

The problems in managing chronic RBC transfusion in SCD remain the same as they have been for decades: all immunization, iron overload, and infection transmission. It is clear that traditional serological RBC phenotyping is unable to detect several variants of RBC antigens, especially those in the Rh system, in populations of African descent. This leads to erroneous phenotyping and the appearance of “auto antibodies” that are truly alloantibodies resulting from transfusion of mismatched blood. As a result, people with SCD are the most frequent users of the American Rare Donor Program.

Name of idea submitter and other team members who worked on this idea Kwaku Ohene-Frempong, MD

Voting

29 net votes
47 up votes
18 down votes
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Goal 1: Promote Human Health

Extreme makeover: harnessing adaptation mechanisms for therapy

What extreme adaptive physiological mechanisms in heart, lung, and blood systems might have the greatest potential to be targeted or employed in therapeutic strategies? Human physiology, including the heart, lung and blood systems, is known to possess extreme adaptive mechanisms to counter extreme conditions or unusual situations. Although some studies are being done, many of these mechanisms have not been fully explored... more »

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

Details on the impact of addressing this CQ or CC

May lead to new therapeutic strategies for HLB diseases. May improve our understanding of HLB physiology and adaptation.

Feasibility and challenges of addressing this CQ or CC

It is feasible to ask specific questions about adaptation mechanisms under extreme conditions and develop experiments to test them. This can be done in various experimental models as well as in humans.

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

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-4 net votes
8 up votes
12 down votes
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Goal 1: Promote Human Health

Stem cell niche in the lung

How do lung progenitors recognize stem cell niches, and what cell-cell interactions mediate normal repair?

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

Details on the impact of addressing this CQ or CC

Research on the stem cell-niche interaction will enhance our understanding of stem cell behavior, enable manipulation of stem cell activity and differentiation potential, and facilitate the development of stem cell-based therapy.

Feasibility and challenges of addressing this CQ or CC

Developing novel models for in vitro 3D culture and in vivo transplantation assays will facilitate the progress.

Recent advances have identified and characterized several lung progenitor cell types. However research gaps remain on understanding the interaction of stem cells with the niche, and how the microenvironment impacts on the stem cell activity during injury/repair.

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

Voting

8 net votes
23 up votes
15 down votes
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Goal 2: Reduce Human Disease

Reduce the immunological burden of Heart Transplantation.

The research in the field of heart transplantation is importantly targeted to minimization of the undue toxicity of immunosuppressive regimens. This practice is limited by the natural tendency to develop chronic and humoral rejection if immunosuppression is too low.
Allocation of organs in Heart Transplantation (HTx) is traditionally limited by the logistic of donation (Cold Ischemic Time) and by the urgency of recipients.... more »

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

Details on the impact of addressing this CQ or CC

Costs of HTx is related to the commonly observed complication of immunosuppression. The attempt of reduce immunosuppression to reduce such Adverse Events may be risky. Humoral rejection is becoming a frequently observed complication requiring treatments with very high costs and long hospital stay.
Reducing the immunologic burden, immunosuppression may be more safely minimized to reduce costs and risks of an excess of immunosuppression afte HTx.
The systems of Machine perfusion extend organ preservation time and have the potential to modify the actual allocation policies.

Feasibility and challenges of addressing this CQ or CC

Machine perfusion has been considered as a way to reduce the hazard of graft failure.
In a first phase of clinical experience, data coming out from registries of Machine perfusion preservation could be analyzed with a particular eye to the different outcomes of organs with different mismatch and/or HLA compatibility.

Voting

0 net votes
1 up votes
1 down votes
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