Goal 2: Reduce Human Disease

Biologic mechanisms of prolonged morbidity in survivors of ARDS and sepsis

What are the biologic mechanisms and risk factors that lead to prolonged morbidity in survivors of ARDS and sepsis? What factors during the acute disease phase distinguish patients that recover from those that develop long-term physical, psychological, or cognitive deficits?

Submitted by (@lar000)

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

Details on the impact of addressing this CQ or CC :

Reductions in mortality rates for ARDS and sepsis have led to an increase in the number of survivors. Many of these survivors develop new or worsened physical, mental, or cognitive morbidities that persist months or years after hospital discharge. Identifying the biologic mechanisms and risk factors during the acute disease phase that lead to prolonged morbidity could help develop therapies to prevent/treat the long-term morbidities and determine the subgroup of patients that interventions should target.

Feasibility and challenges of addressing this CQ or CC :

Research over the past several years has highlighted the increasing number of survivors of ARDS and sepsis that are at risk for long-term physical, psychological, and cognitive impairments. One challenge to answering this question is the limited availability of data and biospecimens of patients with ARDS or sepsis linked to long-term outcomes. Developing a robust resource for this work would require facilitation by NHLBI (potentially in collaboration with other NIH institutes).

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-4 net votes
7 up votes
11 down votes
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Goal 3: Advance Translational Research

Effect of short-term vs. chronic pulmonary rehabilitation on patient-reported outcomes

What is the comparative effectiveness of short-term vs. chronic (indefinite) pulmonary rehabilitation on patient-reported outcomes (symptom frequency, activities of daily living, quality of life, sleep quality, exacerbations)?

Submitted by (@scerreta)

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

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14 net votes
19 up votes
5 down votes
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Goal 3: Advance Translational Research

The effectiveness of a protocol-based screening in treating common COPD comorbidities

Does a protocol-based screening for commonly occurring comorbid conditions in patients with COPD (eg. CAD, CHF, depression, sleep apnea) improve management and outcomes for patients with COPD?

Submitted by (@dmcgowan)

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

Details on the impact of addressing this CQ or CC :

Many times co- morbidities are not address appropriately in patients with COPD- a protocol- based screening would support better identification and adherence to guidelines and would improve management and outcomes of individuals with COPD>

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13 net votes
15 up votes
2 down votes
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Goal 3: Advance Translational Research

Can we leverage exisiting registries to perform prospective trials and advance reduce the cost of doing research?

Current costs for multicenter randomized or non randomized trials are astronomical, and a major obstacle to rapid implementation of potential lifesaving discoveries. In the field of hematopoietic cell transplantation (HCT) their is a federal mandate to have a treatment outcome registry. Funds should be made available to leverage that registry to perform prospective trials either randomized or not since HCT programs need ...more »

Submitted by (@giralts)

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

Details on the impact of addressing this CQ or CC :

This would allow for rapid exploration of novel concepts. Would reduce the time and cost of doing research in the HCT field. Would allow to explore questions related to best supportive care practices in HCT

Feasibility and challenges of addressing this CQ or CC :

Very feasible. Last year we prepared a proposal in response to an RFA demonstrating that all elements are in place.

The CIBMTR has already shown that prospective observational studies are feasible and useful

Name of idea submitter and other team members who worked on this idea : sergio giralt

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123 net votes
154 up votes
31 down votes
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Goal 3: Advance Translational Research

Developing Methods and Metrics for T4 Outcomes and Impact

How can methods and metrics capable of conducting high quality T4 research be developed to accurately capture outcomes and the overall impact new T4 knowledge has on population health for heart, lung, blood, sleep diseases and disorders?

Submitted by (@nhlbiforumadministrator1)

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

Details on the impact of addressing this CQ or CC :

High quality T4 research methods and metrics are needed to move the field of T4 translation research forward while linking large data sets from different sources.

Feasibility and challenges of addressing this CQ or CC :

Demand for high quality methods, metric and evaluation of T4 translation research interest is growing and needs to be addressed immediately to move the field forward.

Recent IOM/NRC studies recommended that the NIH and other research funding agencies support the development of more refined analytic methods and study designs for cross-national health research. These methods should include innovative study designs, creative uses of existing data, and novel analytical approaches to better elucidate the complex causal pathways. The T4 field has some specific metrics including acceptability, reach, adoption, appropriateness, feasibility, fidelity, cost, penetration, and sustainability, each with its standard measurement approach. In addition to a rigorous study design, including these metrics along with population level impact direct measures (e.g., morbidity, mortality) and intermediate measures (e.g. blood pressure reduction) will be critical to assess what has been accomplished and to define success. Finally, measuring the overall impact of new knowledge generated from T4 research is challenging because publication bibliometrics of high impact scholarly journals may not fully capture it.

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

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-1 net votes
7 up votes
8 down votes
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Goal 3: Advance Translational Research

Palliative and hospice care for COPD patients

Does palliative care and/or hospice care as practiced across communities improve end-of-life care for COPD – specifically, does it reduce the burden of symptoms, improve HRQoL and satisfaction, reduce utilization in last 6 months of life (i.e. hospital visits, cost, invasive ventilation use, etc), improve the end-of-life experience, and increase the concordance of place of death to expressed patient preferences?

Submitted by (@k.willard)

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

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12 net votes
16 up votes
4 down votes
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Goal 3: Advance Translational Research

The impact of team consultation on COPD outcomes

Does use of periodic and automatic multidisciplinary team consultation improve care and health outcomes of patients with COPD?

Submitted by (@freeborn1956)

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 : John Linnell, COPD Foundation Minnesota State Captain

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

Lack of large-scale data sources to track outcomes

The lack of large-scale data sources that provide a) detailed clinical phenotyping; b) longitudinal assessment of independent variables (incident events, medications, testing); c) collection of a broad range of outcomes, including patient-reported outcomes including health status.

Submitted by (@rebecca.lehotzky)

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 : AHA Staff & Volunteers

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7 net votes
10 up votes
3 down votes
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Goal 1: Promote Human Health

Improving longterm outcomes after surgery for congenital heart disease

Survival has improved but neurobehavioral disabilty remains a common complication with adverse impacts on quality of life, educational and occupational attainments, and resource utilization. There is increasing evidence that brain development is abnormal, and leads to a rrisk of peri-operative brain injury. Studies are needed to; 1. Further define the prevalence and spectrum of neurobehavioral disability. 2, Understand ...more »

Submitted by (@gaynor)

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

Details on the impact of addressing this CQ or CC :

Survival has improved following surgery for complex congenital heart disease. There is an ever increasing population of adolescents and adults with repaired congenital heart defects. Neurobehvaioral disability can be identified in over 50% of survivors, including ADHD, autism spectrum disorders, learning disabilities and impaired motor skills. These deficits adversly affect their schools and job performance, as well as interactions with their peers and families. The need for special education and other rehabilitative services leads to significant resurce utilization and costs to society. Development of novel neuroprotective therapies will significantly improve the long-term outcomes for these fragile children.

Feasibility and challenges of addressing this CQ or CC :

Because of the small numbers of patients treated at single instituions, this project will require multi-institutional collaboration with long-term follow-up assessments. There is need for collabortaive databases, standardized neurodevelopment evaluations, and acquistion of genomic data. In particular, there is a need to development methodolgy to track outcomes from fetal life to adulthood.

Name of idea submitter and other team members who worked on this idea : J William Gaynor

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18 net votes
23 up votes
5 down votes
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Goal 3: Advance Translational Research

Brief vs. teach-to-goal interventions in teaching patients with COPD to use inhalers

What is the comparative effectiveness of brief interventions to teach patients respiratory inhaler use (e.g., verbal and written instructions) vs. teach-to-goal interventions (brief interventions plus demonstration of correct technique, patient teach-back, feedback, and repeat instruction if needed) on respiratory inhaler technique and patient-reported outcomes (symptom frequency, activities of daily living, quality of ...more »

Submitted by (@jimandmarynelson)

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

Details on the impact of addressing this CQ or CC :

Respiratory inhalers come in a staggering array of types, contents, and methods of use. It is not uncommon for the COPD patient to use two or more types of inhalers each day. The misuse of the application of these devices is rampant, due to confusion, forgetfulness, or lack of proper education in their use. If multiple inhalers are used by the patient, many of them must be used in a particular order, and the inhalation methods may will be vastly different.

Understanding on the part of the patient and/or caregiver begins with the initial instruction in the use of inhalers by medical personnel. They must find, and use, methods of instruction that are understandable and retainable by the patient.

Feasibility and challenges of addressing this CQ or CC :

The study of comparing the two type of instruction is entirely feasible, while the challenges lie with studying a large enough sample of patients to encompass the ranges of COPD stages, mental capacity, and degree of compliance of the patients.

Name of idea submitter and other team members who worked on this idea : Jim Nelson - Patient, Arizona State Advocacy Captain

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

what are the molecular pheontypic differences in IPF/ILD

What are the molecular phenotypic differences in blood and tissue of IPF ILD and how do they relate to disease course and potential response to therapy. There is a need to gain understanding in humans of the differences and similarities in iPF and iLD in general to eliminate the idiopathic nature and establish human targets. The challenge is coupling such research to longer term studies/outcomes and potentially clinical ...more »

Submitted by (@inoth0)

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

Details on the impact of addressing this CQ or CC :

Establishing molecular definitions for this idiopathic disease would a) provide greater clarity and definition to a what is otherwise a syndrome b) establish targets for intervention both for IPF and ILD in general c) refocus translational efforts on human setting for this purely human disease d) establish molecular relationships between IPF and outcomes e) establish intermediate biomarkers for more rapid evaluation for treatment development f) allow potential crossover development with acute lung injury fibrosis g) establish molecular relationships for crossover with human immunology studies and other autoimmune diseases with fibrotic tissue development (CAD, Glomerulonephritis, etc).

Feasibility and challenges of addressing this CQ or CC :

Challenges surround lack of a larger more comprehensive and integrative approach to studying human disease. In an uncommon disease such as IPF, mutlicenter patient enrollment and biologics acquisition must occur in conjunction with both long term longitudinal outcomes and the influence of both new standard of care therapies and novel clinical trials. The scope of studies must be larger, more pragmatic and longer than previously designed NIH clinical studies to allow for integration of translational research. The challenge surrounds failure to allow these elements to coexist. The potential very large for true ILD program with a vision for a long term integrative plan with vision rather than just individual RO1 efforts. An example would an overriding longitudinal study in which patients could enroll and participate in other projects/studies/treatment but where the patient is never lost to follow up. This as cornerstone would then allow other programatic efforts to coexist.

Name of idea submitter and other team members who worked on this idea : Imre Noth

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

The effect of continuous LTOT in COPD targeting fixed oxygen flow rates vs. oxygen saturation on patient-reported outcomes

What is the comparative effectiveness of prescribing continuous LTOT in COPD that targets fixed oxygen flow rates vs. oxygen saturation on patient-reported outcomes (symptom frequency, activities of daily living, quality of life, sleep quality, exacerbations)?

Submitted by (@amutso)

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 : Amelia Mutso, PhD, collaborator with COPD Foundation

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13 net votes
16 up votes
3 down votes
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