Goal 2: Reduce Human Disease

Fibrosis Care Center Network and Patient Registry

Complex diseases such as interstitial lung disease and pulmonary fibrosis requires a collaborative effort to effectively characterize, appropriately diagnose, and efficient evaluate novel therapies. Similarly, basic, translational and clinical research in this field requires the integration of clinical phenotypes with biologic specimens. We propose the expanded development of the Care Center Network and Patient Registry ...more »

Submitted by (@gcosgrove)

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

Details on the impact of addressing this CQ or CC :

The envisioned impact of an integrated Care Center Network and Patient Registry is to create a resource that:

 

• Informs the understanding of interstitial lung disease (ILD), its epidemiology and natural history;

• Assists to understand treatment patterns associated with optimal outcomes that will inform an emerging standard of care and development of treatment guidelines;

• Facilitates patient and clinician engagement in support of future prospective studies;

• Furthers study of biomarkers and predictors of disease and severity;

• Documents patient experience of living with ILD as described through patient reported outcomes (PRO) including quality of life, functioning, and symptoms;

• Generates new hypotheses and new endpoints in support of future studies;

• Increases awareness of relevant issues and needs among the immediate ILD community;

• Provides the opportunity to promote and inform policies in the larger health care community in support of those with ILD

Feasibility and challenges of addressing this CQ or CC :

With the establishment of collaborations between several partners, we initiated the PFF Care Center Network and Patient Registry in 2014. The Care Center Network and Patient Registry has since expanded to 21 centers regionally dispersed throughout the United States. The challenges of effectively and efficiently investigating the cause, care and treatment of pulmonary fibrosis are predominantly those of organization and integration of effort. Expertise is present throughout the United States. We suggest that with the continued expansion of the Care Center Network and Patient Registry, those challenges will be overcome and the focus of the fibrosis community efforts can be on diligently investigating the diseases that devastatingly affect patients. An integrated repository of well-phenotyped patients and biologic specimens is the first step in Precision Medicine for patients with interstitial lung disease and pulmonary fibrosis.

Name of idea submitter and other team members who worked on this idea : Gregory P. Cosgrove, MD, The Pulmonary Fibrosis Foundation

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Goal 3: Advance Translational Research

Pulmonary Rehabilitation

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 (@fzaidi1)

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 : Farhan Zaidi, Jerry Krishnan

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5 net votes
6 up votes
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Goal 3: Advance Translational Research

A comparison of community- and hospital-based pulmonary rehabilitation programs

Compelling Question Text:

 

What is the comparative effectiveness and cost-effectiveness of community-based vs. hospital-based pulmonary rehabilitation programs?

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 :

The ability to reach more patients in need of recommended treatment for the COPD patient.

The availability is not always easy with transportation, cost, and coverage issues being the importatnt factors.

Feasibility and challenges of addressing this CQ or CC :

Easily feasible to address for home or community availability, like YMCAs, Senior Centers.

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11 up votes
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Goal 3: Advance Translational Research

Definitive Evidence of the Effectiveness of Pulmonary Rehabilitation

What is the clinical effectiveness of pulmonary rehabilitation in reducing hospital admissions and readmissions, improving health outcomes such as exercise tolerance and dyspnea, and positively impacting patient centered outcomes. Does this effectiveness vary based on the types of settings rehab is conducted in, urban vs rural environments, the components to the program, the timing of the program and the overall support ...more »

Submitted by (@gacdk0)

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

Details on the impact of addressing this CQ or CC :

Pulmonary rehabilitation is a critical component in the treatment of COPD patients but several barriers persist that have resulted in very limited access to rehab, low referral rates for eligible patients and limited standardization of best practices within the rehab facilities that do exist. Large, definitive studies accounting for patient subgroups, site characteristics and program components can generate the level of evidence needed to expand access, educate providers and improve referral systems and create quality programs. This level of evidence is necessary to change policy to properly value the role of pulmonary rehabilitation and to convince integrated health systems in a value based market that pulmonary rehabilitation is beyond a doubt, a requirement of providing quality COPD care.

Name of idea submitter and other team members who worked on this idea : Grace Anne Dorney Koppel, COPD Foundation Board of Directors, COPD Patient Advocate

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9 net votes
12 up 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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Goal 2: Reduce Human Disease

Identification and validation of surrogate endpoints for long-term morbidity in Sickle Cell Disease

Research in sickle cell disease (SCD) has mostly focused on preventing or treating acute medical events, such as vaso-occlusive pain, acute chest syndrome, and, in pediatric patients, acute strokes. Chronic SCD complications such as chronic kidney disease or pulmonary hypertension, develop over decades, thus are poor choices for clinical trial endpoints. There is a great need to develop surrogate endpoints that predict ...more »

Submitted by (@hulbertm)

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

Details on the impact of addressing this CQ or CC :

Longitudinal cohorts of SCD patients, spanning childhood and adulthood, with biobanking DNA, plasma, and serum, and standardized clinical and imaging assessments will allow identification predictors of negative clinical outcomes. An NHLBI-funded national SCD clinical registry with biobanking will be necessary to validate any surrogate endpoints.

Name of idea submitter and other team members who worked on this idea : Monica Hulbert

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13 net votes
16 up votes
3 down votes
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Goal 3: Advance Translational Research

The impact of a COPD patient education program

What is the impact of an organized, comprehensive, COPD patient education program, on medication delivery effectiveness, care plan adherence, appropriate use of LTOT and Pulmonary Rehabilitation? Metrics could include incidence and severity of exacerbations, and health care resource consumption.

Submitted by (@dprieto)

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

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11 net votes
15 up votes
4 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 2: Reduce Human Disease

Is there a Biomarker for the Pulmonary Fibrosis of HPS?

Hermansky-Pudlak Syndrome is characterized by a bleeding disorder as well as pulmonary fibrosis. Invasive procedures such as a lung biopsy are contraindicated due to bleeding and bronchoscopy is not without risk. Finding a biomarker would reduce the necessity for more invasive data collection while improving outcomes.

Submitted by (@dappell)

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

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

Does the genetic cause of pulmonary fibrosis have implications for treatment response?

Is pulmonary fibrosis due to genetic/heritable causes different than 'sporadic' 'idiopathic' pulmonary fibrosis? With findings in genetics and the emphasis on precision medicine the issue of pulmonary fibrosis being idiopathic will diminish with time. Treatment for this fatal disease should be accessible to those who have pulmonary fibrosis with no other alternatives.

Submitted by (@dappell)

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 : Donna Appell

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

Pulmonary rehabilitation

Can pulmonary rehabilitation change the course of COPD? Is pulmonary rehabilitation a disease-modifying therapy? Does pulmonary rehabilitation improve survival? Does pulmonary rehabilitation reduce readmissions?

Submitted by (@makeb0)

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

Details on the impact of addressing this CQ or CC :

Pulmonary rehabilitation is the most effective therapy for COPD, leading to reduced shortness of breath and improved exercise capacity. It is under-utilized because of the relative paucity of studies evaluating its efficacy on mortality, hospitalization and hospital readmission.

Feasibility and challenges of addressing this CQ or CC :

Feasibility is very high. In the NIH National Emphysema Treatment Trial, a standardized pulmonary rehabilitation program was developed and implemented at multiple sites, many of which did not have existing pulmonary rehabilitation programs. The effective of this pulmonary rehabilitation program was documented (Chest 2005; 128:3799). Challenges include maintenance of the benefits of pulmonary rehabilitation using modern approaches to increase adherence.

Name of idea submitter and other team members who worked on this idea : Barry Make

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20 net votes
26 up votes
6 down votes
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Goal 3: Advance Translational Research

Regenerative Medicine 2.0 in Heart and Lung Research - Back to the Drawing Board

Stem cell therapies have been quite successful in hematologic disease but the outcomes of clinical studies using stem cells for cardiopulmonary disease have been rather modest. Explanations for this discrepancy such as the fact that our blood has a high rate of physiologic, endogenous turnover and regeneration whereas these processes occur at far lower rates in the heart and lung. Furthermore, hematopoietic stem cells ...more »

Submitted by (@jalees)

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

Details on the impact of addressing this CQ or CC :

Some barriers to successfully implementing cardiopulmonary regeneration include the complex heterogeneous nature of the heart and lung.

 

Hematopoietic stem cells can give rise to all hematopoietic cells but the heart and lung appear to contain numerous pools of distinct regenerative stem and progenitor cells, many of which only regenerate a limited cell type in the respective organ. The approach of injecting one stem cell type that worked so well for hematopoietic stem cells is unlikely to work in the heart and lung.

 

We therefore need new approaches which combine multiple regenerative cell types and pathways in order to successfully repair and regenerate heart and lung tissues. These cell types will likely also require specific matrix cues since there are numerous, heterogeneous microenvironments in the heart and lung.

 

If we rethink our current approaches to regenerating the heart and lung and we use combined approaches in which multiple cell types and microevironments are concomitantly regenerated (ideally by large scale collaborations between laboratories), we are much more likely to achieve success.

 

This will represent a departure from the often practiced "Hey, let us inject our favorite cell" approach that worked so well in hematologic disease but these novel, combined approaches targeting multiple endogenous and/or exogenous regenerative cells could fundamentally change our ability to treat heart and lung disease.

Name of idea submitter and other team members who worked on this idea : Jalees Rehman

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7 net votes
11 up votes
4 down votes
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