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

Does prednisone therapy improve outcomes in acute exacerbation of IPF?

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 :

It is common practice to give high-dose prednisone to patients experiencing acute exacerbation of IPF, a relatively common (10-15% annual risk) and deadly event, but there are no good data to support its use. There is a significant risk of morbidity from this therapy. A well-designed multicenter trial could answer this question definitively.

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

Childhood Interstitial Lung Disease

What is the natural history of the best characterized ChILD disorders (surfactantrelated sequence variants, neuroendocrine cell hyperplasia of infancy (NEHI),pulmonary interstitial glycogenosis (PIG),idiopathic pulmonary hemosiderosis)?

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 :

We know little about the natural history of many of the child entities, and their relative rarity makes it difficult for any one center to answer the major questions they pose. The children has begun a patient registry that will begin to address the issue of natural history and disease tracking.

 

a. To improve the power of such a registry, we suggest that support be provided to find novel methods to link this data base to available electronic medical records of participating centers in order to assess physiologic and other clinical associations with specific diseases.

 

b. Support for a biomarker repository holding serum, frozen and fixed lung tissue, patient DNA, RNA, and proteomic and metabolomic materials, and bronchoalveolar lavage effluent and cell pellets, will allow for genome wide analysis as well as proteomic and metabolomic analysis.

Feasibility and challenges of addressing this CQ or CC :

This question is best addressed in the context of a multicenter data registry, ideally linked to a clinical sample.

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

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Goal 2: Reduce Human Disease

Childhood Interstitial Lung Disease

What is the relationship of ChILD disorders to adult diffuse lung disease?

Submitted by (@nhlbiforumadministrator)

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

Feasibility and challenges of addressing this CQ or CC :

This would need to be addressed in the context of databases such as those for familial idiopathic pulmonary fibrosis (F-IPF) or IPF clinical trials, as well as perhaps databases for COPD and pulmonary hypertension. What is the prevalence and spectrum of childhood respiratory disease in family members within these cohorts? What is the prevalence of adult lung disease in family members in ChILD registries? In disorders such as surfactant-related sequence variants, which can cause disease across the lifespan, what are likely “2nd hits”, genomic or environmental, that may lead to clinical disease at particular ages/developmental stages?

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

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Goal 2: Reduce Human Disease

Childhood Interstitial Lung Disease

What is the relationship of ChILD disorders to more common childhood respiratory diseases?

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 :

For example, what is the role of surfactant-related sequence variants in chronic lung disease of prematurity? Cystic fibrosis? Severe bronchiolitis? Refractory asthma?

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

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Goal 2: Reduce Human Disease

Childhood Interstitial Lung Disease

What is the relationship of ChILD disorders to other clinical populations that manifest 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 :

ILD is a prominent feature of systemic inflammatory diseases, such as lupus. ILD is also among the most common long-term complications of therapy for childhood cancer. What is the relationship of surfactant-related sequence variants to expression of clinical ILD in these cohorts?

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

Does the treatment of gastroesophageal reflux disease improve outcomes in patients with IPF?

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 are preliminary data to support the treatment of gastroesophageal

reflux (GER), in particular surgical treatment with laparoscopic fundoplication, as a disease modifying therapy for IPF. Many providers are prescribing medication or recommending surgery for GER in patients with IPF. Full disclosure, this compelling question submitter has submitted a UM1 grant application to study this question in a phase II clinical trial that is currently pending review.

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

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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 2: Reduce Human Disease

Reversing pulmonary fibrosis/interstitial lung disease

Can a multipronged approach to reversing/repairing scar tissue in pulmonary fibrosis be deployed as soon as possible?

Submitted by (@mmhand)

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

Details on the impact of addressing this CQ or CC :

Despite high mortality rates and frustrating disease management marked by exacerbations and uncertainty including chronic oxygen supplementation, pulmonary fibrosis patients lack evidence-based treatments to reverse and repair the fibrotic process. These patients comprise a comparatively smaller proportion of lung disease patients such as chronic obstructive pulmonary disease and asthma and owing to their smaller number, have not been studied in large-scale randomized controlled trials. Treatments that need to be critically studied include regenerating lung tissue through stem cell treatment, artificial/ mechanical lung (augmentation) technology, and new drugs to directly reverse scar tissue, not simply to arrest the fibrotic process as with the two new available drugs for IPF patients.

Feasibility and challenges of addressing this CQ or CC :

Challenges are smaller numbers of available patients available for clinical trials compared with other lung conditions such as COPD and asthma and lack of research funding. Also the etiology of the disease is multifactorial and the categories of fibrosis difficult to accurately diagnose and understand.

Name of idea submitter and other team members who worked on this idea : Mary Hand

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