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

Improve hemostatic outcomes in patients on extracorporeal life support

As patients who suffer a hemostatic complication (hemorrhage or thrombosis) while on extracorporeal membrane oxygenation (ECMO) have worse outcomes, how do optimize anticoagulation in this population to minimize complications and improve outcomes?

Submitted by (@wong00)

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

Details on the impact of addressing this CQ or CC :

Extracorporeal membrane oxygenation (ECMO) is being used earlier in adult and pediatric patients disease course to support them through reversible causes of heart and/or lung failure. Therefore, it's use has increased and, I speculate, will continue to increase.

 

Hemostatic complications, such as thrombosis and intracranial hemorrhages, limit the duration and intensity of ECMO support. Not surprisingly then, multiple studies show that ECMO patients who suffered a hemostatic complication have worse outcomes.

 

Every ECMO center builds their circuit a little different and has their own anticoagulation protocol, but yet, no one center treats enough patients on ECMO to power any robust study. To date, anticoagulation in ECMO is driven by some case series, retrospective single-center studies, and experience. We need multi-institutional studies to study this so that we can harness this technology to its fullest so that we can save lives and minimize morbidity.

Feasibility and challenges of addressing this CQ or CC :

This CC is feasible in that the community of ECMO providers is small and cooperative and anticoagulation is a top concern amongst ECMO physicians. Multi-center study proposals have been submitted but, to date, not funded.

 

Obstacles include:

- The urgency: ECMO is usually initiated emergently so accruing and consenting patients (or their power of attorney) under the duress of ECMO is difficult, but as we have proven, is possible

- Vulnerable population: Many (if not most) patients who go on ECMO are neonates.

- Patient sample size. A huge ECMO center does 40-60 ECMO cases/year so many centers would need to be involved to accrue an adequate sample size

- Patient heterogeneity: Patients who are initiated on ECMO are very different. Differences in age, degree of heart failure versus lung failure, mechanism of lung and/or heart failure, whether they are post-operative and even who their surgeon is can all affect rates of hemostatic complications

- Variable clinical practice: Every ECMO center runs their ECMO different than the next so we would need to standardize some procedures before we could do a multi-center study.

Name of idea submitter and other team members who worked on this idea : Trish Wong

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Goal 2: Reduce Human Disease

Bariatric surgery and CV outcome

Does bariatric surgery reduce the risk of MI, stroke, heart failure, and premature death in moderately obese adults?

Does the type of surgery matter?

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 :

Should bariatric surgery be offered to moderately obese adults as a way to prevent CV events.

Feasibility and challenges of addressing this CQ or CC :

A number of smaller trials have been done and have shown that bariatric surgery can induce remission of diabetes. The bariatric surgery community is primed to conduct trials. Through the HCS collaboratory and PCORI, we believe it is possible to conduct pragmatic trials at reasonable cost by leveraging existing resources.

Bring in parties to aid in data collection and procedure payments (insurance companies, CMS, professional society registries)

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

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-9 net votes
21 up votes
30 down votes
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Goal 2: Reduce Human Disease

Interstitial Lung Disease

Is FVC a valid surrogate for mortality in patients with idiopathic pulmonary disease?

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 :

This question is central to all future clinical research in the field and MUST be answered in the context of an interventional clinical trial. Longitudinal cohort studies correlating FVC change with survival are insufficient. The intervention that would make the most sense to test currently is pirfenidone. It is approved for use in many countries and many experts (including the submitter of this compelling question) believe it is likely effective. A mortality-driven clinical trial of pirfenidone vs. placebo would address this question directly, as well as determine the true impact of pirfenidone on clinically meaningful endpoints.

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

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Goal 2: Reduce Human Disease

Identifying Chronic Pain and exploring the onset and severity among patients with thalassemia

While the primary pathophysiology of thalassemia is related to globin gene mutations and unbalanced globin chain expression, the downstream consequences are manifold. Chronic pain turns out to be one of the most important factors identified by patients with transfusion dependent thalassemia major in health-related quality of life surveys and patient-reported outcome measures. Even as therapies aimed and gene editing, ...more »

Submitted by (@gcioffi)

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

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37 net votes
47 up votes
10 down votes
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Goal 2: Reduce Human Disease

Heterogeneity in Asthma Phenotypes

As the current chair of the Research and Training Division, I would like to convey that the AAAAI membership would like the NHLBI to consider the following in the development of its strategic plan: Asthma appears to be due to heterogenous etiologies.  To better characterize the various phenotypes and potential etiologies, it would be important to create more epidemiologic and biomarker focused databases, which could ...more »

Submitted by (@wheeze)

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 : Mitchell Grayson on behalf of the American Academy of Allergy, Asthma, and Immunology

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-12 net votes
6 up votes
18 down votes
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Goal 2: Reduce Human Disease

Predicting and monitoring atherosclerosis progression and vulnerable plaque

How to develop novel methods for predicting and monitoring atherosclerosis progression and vulnerable plaque including biomarkers and imaging?

Submitted by (@societyforvascularsurgery)

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 for Vascular Surgery

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

Optimizing therapeutic hypothermia

What is the optimal dose and duration of post-cardiac arrest hypothermic targeted temperature management?

Submitted by (@rebecca.lehotzky)

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 : AHA Staff & Volunteers

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-3 net votes
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Goal 2: Reduce Human Disease

Neuroendocrine system in heart and lung disease

What is the role of the non-neuronal neuroendocrine system in lung and heart pathophysiological processes, and can it be targeted for lung diseases therapies?

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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3 net votes
13 up votes
10 down votes
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Goal 2: Reduce Human Disease

Cure COPD

How can the structure and function lost in COPD be restored?

Submitted by (@jsullivan)

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 tissue alterations in COPD and their physiologic consequences of those changes are reasonable well described. It is now clear that, like all organs, the lung can repair damaged tissue and that repair processed can be modulated. Strategies for assessing restoration of lost tissue structure and function should be developed, together with the development of clinical measures that can gauge progress of treatment.

Feasibility and challenges of addressing this CQ or CC :

Animal studies demonstrate that emphysema can be repaired, at least in some species. Several forms of airways disease in humans are also reversible. Studies of interventions to augment tissue repair are needed with the goal of applying them to clinical interventions.

Name of idea submitter and other team members who worked on this idea : COPD Foundation, COPDF MASAC

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18 net votes
20 up votes
2 down votes
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Goal 2: Reduce Human Disease

Novel medical management of COPD

Is there a role for telemedicine in the prevention of acute exacerbations of COPD?

Submitted by (@nhlbiforumadministrator1)

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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Goal 2: Reduce Human Disease

Contribution of non-lipid factors to atherosclerotic vascular disease

Despite the generally accepted thesis linking lipids to atherosclerosis, a large number of patients fail to benefit from statins, the current lipid-lowering drugs of choice. Moreover statins are reported to cause diabetes and severe muscle weakness in some patients thus adding to the general conundrum associated with statin therapy. This prompts an important, yet unresolved question related to non-lipid factors contributing ...more »

Submitted by (@priyaraman)

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 : Priya Raman

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

Rare Disease Biorepository

NHLBI should establish a rare disease biorepository that can be used by PhD and MD scientists, and propose new therapies based on the insights gained.

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 :

If you think about improving rare disease treatment, any clinical trial is better than no clinical trial. Although I make this point in jest, the important aspect is that the control group is actually as important as the treatment group in establishing the natural history of the disease, establishing a biorepository that can be used by PhD and MD scientists, and proposing new therapies based on the insights gained. I would use as an example the current GRADS program in sarcoidosis and Alpha-1 antitrypsin deficiency. My prediction is that we will find rather minor microbiome elements of disease pathogenesis (it is worth looking). However, the integrated genomics aspects of the protocols will be useful for a decade to stimulate discovery.

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

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