Goal 3: Advance Translational Research

Non-invasive vs Invasive Positive Pressure Ventilation in Managing Acute Respiratory Failure

What is the comparative effectiveness of a Non-invasive vs. Invasive Positive Pressure

 

Ventilation Protocol for managing acute respiratory failure due to acute exacerbations of COPD

Submitted by (@joem00)

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

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Goal 2: Reduce Human Disease

Long-term pulmonary function in survivors of critical illness

Pulmonary function is known to suffer during the early recovery phases from critical illness, but the long-term patterns of recovery and associated consequences are uncertain. In addition, the clinical and molecular determinants of progressive deterioration or recovery of pulmonary function remain unknown.

Submitted by (@greg.martin)

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

Effect of obesity on recovery of lung function in pediatric survivors of critical illness

What are the determinants of persistent respiratory failure in children? Are obese children at greater risk for prolonged mechanical ventilation than non-obese children? Does BMI affect the time to recovery of lung function in obese children with ARDS? What is the pathogenesis and molecular contributors of obesity on respiratory failure in critical illness?

Submitted by (@greg.martin)

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

Sex and Race Differences in Replacement Lung Surfactant Efficacy

Baby boys and girls can respond quite differently to replacement lung surfactants given in response to neonatal respiratory distress syndrome. There are also variations in efficacy between babies of different races, in addition to the generally increased risk of NRDS in minority populations overall to lack of prenatal care or access to neonatal intensive care units. Increasing the availability of surfactant treatment ...more »

Submitted by (@josephzasadzinski)

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

Details on the impact of addressing this CQ or CC :

Infant mortality varies dramatically across racial and economic status, and prematurity and its accompanying diseases is one important aspect of this discrepancy. Replacement surfactant therapy dramatically decreased the infant mortality rate when it was originally initiated, but certain populations are less served, and variations in efficacy of treatment are poorly understood. New methods of delivering surfactant, such as aerosols combined with CPAP and a better understanding of the properties of surfactant might be two ways to address this issue.

Feasibility and challenges of addressing this CQ or CC :

This challenge requires a broad, multidisciplinary approach including engineers, neonatologists and biophysicists to produce the necessary materials and tools to properly synthesize and deliver surfactant. A larger challenge is to simplify the process enough that surfactant replacement therapy could be introduced into less developed countries where prematurity is of epidemic proportion.

Name of idea submitter and other team members who worked on this idea : Joe Zasadzinski

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-7 net votes
3 up votes
10 down votes
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Goal 2: Reduce Human Disease

Lung cell stimuli responses

What alterations in respiratory epithelia in response to environmental / external insults are irreversible and lead to disease onset or progression?

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

Predict the needs for inter and intra-hospital transfer for acute care surgery patients with respiratory failure

Density mapping of the need and flow of patients requiring acute care surgery vis-a-vis inter-facility transfer, care hand-off failures, post-acute care resource mismatch to articulate a funding plan resource allocation and development akin to what has been done for trauma care.

Submitted by (@greg.martin)

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 of Critical Care Medicine Executive Committee/Council

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0 net votes
2 up votes
2 down votes
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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?

Submitted by (@greg.martin)

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 2: Reduce Human Disease

Developing Standards of Care for adult muscular dystrophy (FSHD, DM) patients affected by hypercarbic respiratory insufficiency

There is an unmet need for the NHLBI to foster basic, preclinical and clinical research on the pulmonary consequences of respiratory insufficiency, and specifically with hypercarbic (high CO2) respiratory insufficiency, in facioscapulohumeral muscular dystrophy (FSHD) and other adult muscular dystrophies. The adult muscular dystrophies have received insufficient attention, both from research and clinical practice perspectives. ...more »

Submitted by (@daniel.perez)

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

Feasibility and challenges of addressing this CQ or CC :

As with cardiomyopathy and arrhythmia, currently little data is available as to how to best measure/monitor, and when and how to intervene in, the respiratory complications of the adult muscular dystrophies (FSHD, DM, LGMD) using respiratory therapy, non-invasive ventilation (bi-Pap, Trilogy) or ventilation. The absence of home-based sleep studies and technologies to easily assess hypercapnia are identified as a significant gap in knowledge. Additionally, since the cardiac and pulmonary hypercarbic respiratory insufficiency complications are inextricably linked, studies of the interrelationship between cardiac and pulmonary consequences of the muscular dystrophies (congestive heart failure, pulmonary hypertension) are needed, and interdisciplinary teams of researchers may be best equipped to conduct them.

Name of idea submitter and other team members who worked on this idea : FSH Society

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-11 net votes
4 up votes
15 down votes
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Goal 2: Reduce Human Disease

Non-Adherence of Patients with Chronic Respiratory Diseases

There are various reasons responsible for patients’ non-adherence. One of them is insufficient or lack of education about medications and equipment required for their treatment.

Submitted by (@vlady.rozenbaum)

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

Details on the impact of addressing this CQ or CC :

There is a critical need to develop uniform guidelines and handouts addressing the confusion over the proper use of medications (particularly inhalers) and equipment (i.e. oxygen). Improper use leads to diminished or no benefit, frustration, and, ultimately, even to a patient's decision to stop the treatment.

Feasibility and challenges of addressing this CQ or CC :

This is an issue that has been universally acknowledged for a number of years. With the help of patient focus groups, convened at the NHLBI, national pulmonological conferences, or at local venues around the country, appropriate materials can be created to benefit patients and reduce a huge burden on nation's economy due to decreased productivity and increase in hospital admissions.

Name of idea submitter and other team members who worked on this idea : COPD-ALERT

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

National network to study the pathobiology of sepsis

Sepsis is the leading cause of death in hospitalized patients, the 3rd leading cause of death in all people in the US, the most common condition leading to widespread vascular collapse, among the most common causes of respiratory failure, and a frequent cause of acute cardiac dysfunction.

Submitted by (@greg.martin)

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

Details on the impact of addressing this CQ or CC :

Developing a national network to address important aspects of sepsis (causes and consequences of cardiac dysfunction, molecular determinants of respiratory failure) and serve as a trials group for testing novel interventions for new discoveries.

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

Develop guidelines, standard of care, new technologies for respiratory care for adult facioscapulohumeral muscular dystrophy

There is a need for NHLBI to develop guidelines, standard of care, new technologies for respiratory care for adult muscular dystrophy (facioscapulohumeral, myotonic and limb girdle) patients with undiagnosed or unforeseen hypercarbia CO2 retention in the acute setting who end up in trouble to help the families, doctors and patients navigate their way back to stable condition e.g. perhaps going forward with non-invasive ...more »

Submitted by (@daniel.perez)

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

Details on the impact of addressing this CQ or CC :

Prolonged and healthier lives. Lower costs and disease burden.

Name of idea submitter and other team members who worked on this idea : FSH Society

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

What causes variation in severity of skeletal muscle, lung, pulmonary and heart system symptoms in FSHD muscular dystrophy

Loss of diaphragm function and impaired respiration is a leading driver of morbidity and mortality in the adult muscular dystrophies such as facioscapulohumeral muscular dystrophy, and therefore requires additional study

Submitted by (@daniel.perez)

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

Details on the impact of addressing this CQ or CC :

Muscular dystrophies comprise a heterogeneous group of genetic diseases often characterized by multi-systemic effects and distinct patterns of muscle involvement. While a characteristic pattern of muscle weakness has traditionally been used to define the different subtypes of muscular dystrophy, the cause for the regional distribution of muscle weakness, often with sparing of specific muscle groups, has largely remained unresolved. Moreover, many muscular dystrophies such as the most common form facioscapulohumeral muscular dystrophy, show noticeable variation in disease onset and progression, both between as well as within families. Involvement of the diaphragm and muscles of respiration often proceeds at a rate different from other striated muscles. Loss of diaphragm function and impaired respiration is a leading driver of morbidity and mortality in the muscle diseases, and therefore requires additional study in adult muscular dystrophy.

Feasibility and challenges of addressing this CQ or CC :

Studies in mice and humans have provided some evidence for genetic modifiers of disease onset, presentation and progression, but a comprehensive explanation for the observed differences in skeletal muscle, lung, pulmonary and heart system involvement and disease progression is currently lacking. Disease penetrance may be affected by genetic background or gene-environment interactions. Future studies on the identification and validation of such factors, both genetic and non-genetic (off target effects of drugs, diet, exercise), may provide insight into strategies that delay disease onset, prevent off-target effects of drugs and improve quality of life.

Name of idea submitter and other team members who worked on this idea : FSH Society

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-10 net votes
6 up votes
16 down votes
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