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

Kinetic analysis of transcriptional regulation by aberrant transcription factors in vivo

Aberrant transcription factors, such as leukemia fusion proteins, promote diseases by deregulating transcription of target genes. Recent genome-wide studies have provided new insight into transcriptional deregulation by aberrant transcription factors. Most of these studies have not used cells with synchronized transcriptional activities at specific repression/activation steps and, therefore, may have limited value in ...more »

Submitted by (@jinsongzhang8012)

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

Feasibility and challenges of addressing this CQ or CC :

These studies are feasible. Transcriptional regulation of a population of cells may be synchronized by epigenetic drugs, or by using inducible expression/knockdown approaches. Alternatively, it may be possible to overcome the heterogeneity issue by single-cell-based analysis. Next, time-dependent genome-wide studies may be applied to the cells to reconstruct dynamic transcriptional pathways associated with aberrant transcription factors. These approaches may be generally applicable to the study of other disease-related transcriptional and signaling molecules.

Name of idea submitter and other team members who worked on this idea : Jinsong Zhang

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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

Interstitial Lung Disease

Do the non-pharmacological interventions of pulmonary rehabilitation and supplemental oxygen for exertional hypoxemia improve quality of life and functional status in patients with chronic fibrotic ILD?

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 :

There is good evidence from small controlled trials that pulmonary rehabilitation improves walk distance and shortness of breath in patients with chronic ILD. Both pulmonary rehabilitation and supplemental oxygen are commonly recommended to these patients at great cost. A randomized clinical trial using factorial design could address the impact of these two therapies individually as well as together on clinically meaningful outcomes.

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

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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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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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Goal 2: Reduce Human Disease

Integrating palliative care into the care of patients with heart, lung, and blood disease

How can we best integrate primary and specialty palliative care into the care of patients with serious heart, lung, and blood diseases?

Submitted by (@jrc000)

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 is increasing awareness of the importance of palliative care in the care of patients with serious illness. Palliative care focuses on improving quality of life and reducing the stress of a serious illness for patients and their families and can be provided together with curative or life-prolonging treatments. It includes both primary palliative care (delivered by all cliniicans who care for patients with serious illness) and specialty palliative care (delivered by physicians, nurses, social workers, chaplains and others with special training in palliative care.) Patients with serious heart, lung, and blood diseases have profound unmet palliative care needs and it is not clear how these needs can best be met.

Feasibility and challenges of addressing this CQ or CC :

Studies show dramatic benefit of integrating palliative care into the care of patients with cancer, but there is little data on the best way to improve palliative care for patients with serious heart, lung, and blood disease. The opportunity exists to support research that develops and evaluates interventions to improve primary and specialty palliative care for patients with heart, lung, and blood diseases.

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Goal 2: Reduce Human Disease

Reducing Atrial Fibrillation by treating modifiable risk factors

Would better management of modifiable risk factors, including obesity, sleep apnea, hypertension, hyperglycemia, and metabolic syndrome, reduce atrial fibrillation recurrence? Furthermore, what are the best methods to reduce the onset, hospitalization, and death due to atrial fibrillation, especially that associated with aging

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 :

Identify strategies to prevent or reduce recurrence of atrial fibrillation using available lifestyle and medical therapies.

Feasibility and challenges of addressing this CQ or CC :

There is a large population of patients with atrial fibrillation available to test this hypothesis along with strategies for treatment of modifiable risk factors. A challenge is to identify the good strategies to ensure adherence.

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

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Goal 2: Reduce Human Disease

Moonshot: Turning the BMT EMR into a Research Record

The critical challenge is to develop a standards-based BMT electronic medical record (EMR) and integrate research capacity into the architecture of EMR systems. The ultimate goal would be to build de-identified complete data-sets which can be used to support observational studies and clinical trials, improve transplant outcomes and inform public policy.

Submitted by (@rakeshgoyal)

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

Details on the impact of addressing this CQ or CC :

Clinical research is constrained by a clumsy method of acquiring biomedical data, generally relying on manual capture of information from EMR back-to-paper which is then transcribed into registry or specific clinical trial databases. This method is labor intensive, fraught with opportunities for error, and increasingly difficult to defend in light of the high costs associated with clinical trials. Adoption of standards-based clinical documentation and creating access to source clinical data would reduce or do-away with resource-intensive, very expensive and time-consuming data abstraction, enhance data quality and depth, and accelerate translational research.

Feasibility and challenges of addressing this CQ or CC :

As BMT centers increasingly adopt EMR systems in the United States, a vast and potentially very useful data resource is being created. However, most EMR systems offer very generic formats for clinical documentation and the medical information is inconsistently expressed in vocabulary, structure, and format. One challenge is the development of common standards-based clinical documentation format and its adoption by EMR system vendors and BMT institutions to support structured data sharing.

 

Large transplant centers can build their own integration engines to link EMR with stem cell lab, HLA, donor care, workflow etc. However, a broad implementation of integration IT solutions would be needed amongst centers conducting BMT clinical trials.

 

While CIBMTR's FormsNet application and Clinical Trials Network allow electronic data submission, data professionals still need to manually enter the data. Another challenge in creating a centralized data resource would be to build interoperability between transplant centers and research entities. An alternative to a large centralized database could be a distributed research network which allows data holders to maintain logical and physical control over their data and mitigate security, proprietary, legal, and privacy concerns.

Name of idea submitter and other team members who worked on this idea : Rakesh Goyal

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Goal 2: Reduce Human Disease

Hypertension in the Pediatric Population

We also wish to draw attention to the rise in the prevalence of hypertension in the pediatric population, mostly as a consequence of the childhood obesity epidemic.

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 :

Additionally, hospitalizations related to pediatric hypertension have doubled over the past decade. These phenomena have clear and profound implications for the future cardiovascular health of the American population. The NHLBI has been instrumental in supporting studies in pediatric hypertension in the past, and we encourage a continued focus on pediatric populations for future hypertension research.

Name of idea submitter and other team members who worked on this idea : American Society of Pediatric Nephrology (ASPN)

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Goal 2: Reduce Human Disease

Pulmonary Vascular Diseases

Does pulmonary rehabilitation or regular exercise improve outcomes in patients with PVD?

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 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

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Goal 2: Reduce Human Disease

Airway remodeling

What are the features of airway remodeling about which we consistently agree and in whom are they most likely to occur?

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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