Goal 2: Reduce Human Disease

Data from regulatory studies a barrier to evidence-based medicine

Alignment of regulatory, healthcare, and research arms of the government is poor. There is a need to improve the design, quality and usefulness of data from regulatory studies to address major clinical questions and also to facilitate scientific inquiry. This is a barrier to evidence based medicine and improved treatments.

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 Society for Vascular Surgery

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Goal 2: Reduce Human Disease

Basic Research & Precision Medicine

How can NHLBI best encourage basic research areas that are critical to the development of precision medicine approaches for lung disease?

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

A toxic relationship? AF and subclinical and clinical thyroid dis

There is a need to create opportunities to explore clinically relevant basic and clinical research related to myocardial effects of thyroid hormone excess and deficiency, especially as it is related to atrial fibrillation.

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

Details on the impact of addressing this CQ or CC

Decrease development AF

Feasibility and challenges of addressing this CQ or CC

AF - top causes in HF, stroke.

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

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3 up votes
14 down votes
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Goal 2: Reduce Human Disease

How to maximize the opportunities and promise of emerging omics research? Develop scientific commons

In the late 20th century, the NHLBI cohorts were created as separate entities with specific research goals. The NHLBI’s funding of GWAS served as a powerful incentive for collaboration among the NHLBI-funded cohort studies. The creation of a scientific commons would provide a major national resource comprising the participants, their deeply phenotyped data, their biological samples, and the investigator expertise to... 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

Advance discovery and treatment

Feasibility and challenges of addressing this CQ or CC

Feasible with few challenges. The system would need infrastructure support.

Name of idea submitter and other team members who worked on this idea Psaty & Tracy

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6 net votes
20 up votes
14 down votes
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Goal 2: Reduce Human Disease

Development of non-contrast alternatives in cardiac magnetic resonance imaging

Critical Challenge

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

Details on the impact of addressing this CQ or CC

Late gadolinium-enhancement cardiac magnetic resonance imaging (MR) plays a crucial role in the evaluation of patients with suspected myocardial scar tissue. Alternative methods to contrast-enhanced MR however are in need, given the number of patients who have concomitant compromised renal function and concern for nephrogenic systemic fibrosis. Noncontrast MR techniques such as diffusion-weighted imaging would complement and eventually replace gadolinium administration thus impacting the evaluation of those with suspected and confirmed infiltrative cardiac processes and systemic diseases.

Feasibility and challenges of addressing this CQ or CC

Late gadolinium enhancement technique characterizes enhancement patterns of heart disease, identifies areas amenable to ablation, and aids in decisions pertaining to workup and therapy. The underlying mechanism of Brownian motion/diffusion in the expanded extracellular space makes diffusion weighted imaging a potential gadolinium-saving modality. Diffusion MR, applied primarily in the brain and abdominal imaging, is underutilized in the heart given respiratory and cardiac motion. A need exists to further develop and apply noncontrast MR techniques towards cardiovascular applications. Such methods are promising noncontrast alternatives to characterize patients with myocardial disease, determine those with differing prognoses, and direct appropriate therapies to subgroups.

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

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

Preventing hospitalization of asthma patients through agressive acute asthma management

Develop simplified agressive acute asthma treatment protocol and promote the implementation for primary care, ED, Urgent care, Internists, Pediatricians, Allergists, Pulmonologists, and Families. Goal is to prevent hospitalization of children and adults with asthma.
For example: 3 cycles of Duoneb and Albuterol, monitoring pulsoximetry, with oral / IM Dexamethasone dosage. Also initiation of controller medication at... more »

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 D M Meier MD

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7 up votes
7 down votes
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Goal 2: Reduce Human Disease

New Clinical Research Methodologies for Rare Diseases

What innovative methodologies applicable to small cohorts and rare outcomes can better ensure the success of clinical and implementation studies in the rare diseases affecting heart, lung, blood, and sleep?

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

Details on the impact of addressing this CQ or CC

Efficient translation of clinical observation into discovery science, and, particularly, the timely translation of potential therapeutic target identification into the treatment of rare diseases has been impeded by the reality that rare disease populations are often too small to be studied using “classical” epidemiology, clinical trial, and implementation science methodology. Overcoming these barriers will require both the adaptation of current clinical research methods and the development of novel methodologies. The requirement for better methods in rare disease clinical science will become even more urgent with time as systems biology more specifically defines and sub-characterizes ‘common’ heart, lung, blood, and sleep disease populations

Feasibility and challenges of addressing this CQ or CC

This problem has been recently addressed through special initiatives in the application of small trial methodology into the planning and design of clinical trials in rare hemostatic disorders and sickle cell disease. Furthermore, small clinical trial methodologists have begun to populate the CTSAs and Regulatory Agencies. Their expertise in clinical trial design and biostatistical methods, and their creative ideas can be brought to bear in the clinical trials required to advance NHLBI scientific priorities.

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

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24 net votes
37 up votes
13 down votes
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Goal 2: Reduce Human Disease

Systems Approaches to "Phenosimilar" Diseases

There are diverse diseases that share similar features. For example, many chronic airways diseases (chronic aspiration, ciliary dyskinesia, some COPD) "phenocopy" cystic fibrosis in terms of infectious agents, mucus clearance problems, progressive loss of lung function, etc. Use multiplatform "omics" approaches and Big Data bioinformatics to identify common nodes for therapeutic targeting. Other examples: emphysema and... 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 be able to target multiple diseases with new or repurposed therapies. Might narrow down the broad array of potential "high value" targets to study.

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12 up 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.

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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Goal 2: Reduce Human Disease

Clinical Trials in Pediatric Sleep Disorders - Effect of adenotonsillectomy

Effect of adenotonsillectomy on behavioral and cardiovascular outcomes in children with primary snoring

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 recent NHLBI Childhood Adenotonsillectomy (CHAT) study showed highly significant behavioral improvements in children with obstructive sleep apnea syndrome . This study included children with an apnea hypopnea index as low as 2/hr. Many small or suboptimally controlled studies suggest that even primary snoring can affect behavior. If large randomized controlled trials confirm this finding, it will radically affect the treatment of the estimated 10% of children who snore.

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

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Goal 2: Reduce Human Disease

Genetic engineering in lung progenitor cells

Can genome engineering be used to correct or alter lung stem/progenitor cells to ameliorate lung disease and promote regeneration?

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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-15 net votes
3 up votes
18 down votes
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Goal 2: Reduce Human Disease

Clinical Trials in Pediatric Sleep Disorders

Effect of anti-inflammatory medications (including nasal steroids and leukotriene antagonists) in children with obstructive sleep apnea syndrome, stratified for severity of OSAS as well as presence of atopy.

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

Details on the impact of addressing this CQ or CC

Small studies suggest a therapeutic effect of anti-inflammatory medication in childhood OSAS. This may be especially useful in children with residual OSAS following adenotonsillectomy (as CPAP adherence tends to be low) or children who are poor candidates for surgery. Current studies have been limited to children with extremely mild OSAS, have not determined whether atopy plays a role in the response to therapy, and have been limited to very short-term trials.

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

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1 up votes
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Goal 2: Reduce Human Disease

Pulmonary Complications of Sickle Cell Disease

Do SCD patients with hemodynamics consistent with pulmonary arterial hypertension (PAH) respond to medications designed to treat PAH?

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

Details on the impact of addressing this CQ or CC

What is known about this topic:

  1. Case series have demonstrated potential therapeutic benefits for endothelin receptor antagonists, phosphodiesterase 5 inhibitors and prostacyclins in PH of SCD patients
  2. Three attempted randomized placebo controlled trials of these agents in SCD patients have not gone to completion and, as a result, were under-powered to demonstrate efficacy.
  3. Sildenafil produced an increase in hospitalization for pain crises in this population.
  4. Anecdotally, select SCD patients with PAH have hemodynamic and clinical benefits from PAH medications.
  5. Approximately ½ of PH in SCD patients have some degree of pulmonary venous hypertension and these medications may not be helpful here.
Feasibility and challenges of addressing this CQ or CC

Areas of controversy:

  1. Only one of the three randomized controlled trials required a PAH diagnosis prior to randomization, so the actual question hasn’t been properly addressed.
  2. SCD patients with PAH are different than idiopathic PAH patients in terms of their underlying disease, so possibly the treatment response is different.
  3. What are the right clinical trial endpoints for this population?
  4. What is the role of SCD specific therapy (hydroxyurea, transfusions) in treating PAH of SCD?
  5. How can investigators design a clinical trial which allows for enough patient accrual to achieve its endpoints?
  6. What novel therapies can be developed to treat this population?
  7. What unrecognized medication toxicities are SCD patients at increased risk for?

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

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Goal 2: Reduce Human Disease

Lung Transplantation

Although the majority of lung recipients experience significant health improvement, they also frequently face serious symptom distress, impaired physical functioning and poor quality of life due to post-transplant morbidity, such as chronic rejection, infection and multiple side-effects of immunosuppression.

a) Conduct clinical trials of interventions designed to maximize clinicians' support of patients' self-management... more »

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

Feasibility and challenges of addressing this CQ or CC

a) Conduct clinical trials of interventions designed to maximize clinicians' support of patients' self-management behaviors so that patients and clinicians working together can achieve optimal control of disease, reduce symptom distress and complications, and promote quality of life.

b) Evaluate the impact of integrating palliative care and transplant care for symptom management, goal setting and advanced care planning along the entire lung transplant illness trajectory (pre, post and end of life).

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

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