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Abstract Number: 1713

Developing an Index for Disease Activity and Therapeutic Response in Connective Tissue Disease Related Interstitial Lung Disease: Results From A Delphi Exercise: Delivering A Consensus On Domains

Lesley Ann Saketkoo1, Dörte Huscher2, Dinesh Khanna3, Paul F. Dellaripa4, Kevin Flaherty5, Chester V. Oddis6, Kristine Phillips7, Athol U. Wells8, Christopher P. Denton9, Oliver Distler10, Otylia M. Kowal-Bielecka11, Romy Christmann12, Nora Sandorfi13, David Pittrow14, Vibeke Strand15, James R. Seibold16, Kevin K. Brown17 and Eric L. Matteson18, 1LSU Scleroderma and Sarcoidosis Patient Care and Research Center, New Orleans, LA, 2German Rheumatism Research Centre and Charité University Medicine, Berlin, Germany, 3Division of Rheumatology, University of Michigan Medical Center, Ann Arbor, MI, 4Division of Rheumatology, Immunology, and Allergy, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, 5Department of Critical Care and Pulmonary Medicine, University of Michigan, Ann Arbor, MI, 6Rheum/Clinical Immunology, University of Pittsburgh, Pittsburgh, PA, 7Rheumatology, University of Michigan, Ann Arbor, MI, 8Royal Brompton and Harefield NHS Foundation Trust, Department of Radiology, London, United Kingdom, 9Centre for Rheumatology and Connective Tissue Diseases, UCL Medical School, London, United Kingdom, 10Department of Rheumatology and Center of Experimental Rheumatology, University Hospital Zurich, Zurich, Switzerland, 11Department of Rheumatology and Internal Medicine, Medical University in Bialystok, Bialystok, Poland, 12Rheumatology, Boston University School of Medicine, Boston, MA, 13Division of Rheumatology, Thomas Jefferson Univ Med Coll, Philadelphia, PA, 14Institute of Clinical Pharmacology - University of Dresden, Dresden, Germany, 15Adjunct, Division of Immunology / Rheumatology, Stanford University, Portola Valley, CA, 16Scleroderma Research Consultants LLC, Avon, CT, 17Autoimmune Lung Center, National Jewish Hospital, Denver, CO, 18Rheumatology, Mayo Clinic, Rochester, MN

Meeting: 2012 ACR/ARHP Annual Meeting

Keywords: clinical trials, Connective tissue diseases, Lung, outcome measures and pulmonary complications

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Session Information

Title: Systemic Sclerosis, Fibrosing Syndromes, and Raynaud’s – Clinical Aspects and Therapeutics II

Session Type: Abstract Submissions (ACR)

Background/Purpose: Lack of reliable and valid measures of disease activity and clinical response in patients with connective tissue disease (CTD)-related interstitial lung disease (ILD) makes clinical trial design difficult.  From a multi-tiered investigation to develop consensus on criteria in both CTD-ILD and idiopathic pulmonary fibrosis (IPF), we report results of expert voting from a 3-tiered Delphi exercise to identify domains ‘important’ to measure in a 1 year randomized controlled trial (RCT) in IPF and CTD-ILD.

 
Methods:  
Using OMERACT methodology, 270 experts nominated 23 “domains” and 616 “instruments” that were assembled into an initial voting survey for a 3-tiered Delphi exercise with survey items anchored by degree of importance on a 9-point Likert scale with as a voting option.  All stages of data collection used a custom-designed secure web-site that included related articles and opportunities for participants to upload commentary supporting or refuting importance of each item.

Tier 1 Analysis:  A cut-off median <4 was applied to the results.  Final review demanded 100% consensus agreement for dismissal of an item based on lack of: 1. Face validity, 2. Content validity (more suited to diagnostic, demographic, or inclusion criteria) and 3. Feasibility in a multicenter trial.   

Tiers 2 and 3 Analysis:  To protect against bias introduced by using an arbitrary cut-off, cluster analysis was implemented to identify patterns of consensus within the data.
 
Results:
90% of invited experts: 137 pulmonary, 102 rheumatology and 4 cardiology specialists from 32 countries/6 continents participated. 74% and 69% of participants considered ILD and rheumatologic lung disease respectively as their primary field of research or clinical interest.  Recidivism after Tier 1 was <1% with each subsequent Tier.  Five common domains with their candidate instruments were identified for CTD-ILD and IPF (Table 1).  Three domains identified for CTD-ILD: biomarkers, cough and medications await nominal group decisions.

 

Conclusion: Development of valid, discriminatory and feasible outcome measures to assess disease progression and therapeutic responses is essential for performing RCTs in CTD-ILD.  This is the first comprehensive, multi-disciplinary, international effort to assess domains for study of ILD.  Experts identified a core set of domains including radiographic, physiologic and patient-reported outcomes culled from a large number of candidate items.  A research agenda focusing on candidate biomarkers and domains requiring instrument development has emerged. Broad participation from a multidisciplinary ILD research community reflects the high perceived need in this area.

 

Table 1.  Results of Tier 3

 

DOMAINS

 

(median /mean ratings)

 

Candidate Instruments

 

Dyspnea

CTD-ILD             IPF

(8.0/7.8)            (8.0/8.1)

Borg Dyspnea Index

Dyspnea 12

Medical Research Council (MRC) Breathlessness (Chronic Dyspnea) Scale                                 

Modified MRC Dyspnea Scale

Borg Dyspnea Index – Pre and Post Exercise

 

Health Related Quality of Life (HRQoL)

CTD-ILD             IPF

(8.0/7.7)            (8.0/7.8)

Medical Outcomes Trust Short Form-36 Health Survey

St. George’s Dyspnea Respiratory Questionnaire

Visual Analogue Scale of Patient Assessment Disease Activity

Ability to Carry Out Activities of Daily Living (ADLs)

Health Assessment Questionnaire Disability Index (HAQ-DI)

 

Lung Imaging

CTD-ILD             IPF

(9.0/8.3)           (9.0/8.3)

Extent of Honeycombing on HRCT

Extent of Reticulation on HRCT

Extent of Ground Glass Opacities on HRCT

Overall Extent of Interstitial Lung Disease on HRCT

 

Lung Physiology / Function

CTD-ILD             IPF 

(9.0/8.7)            (9.0/8.7)

Supplemental Oxygen Requirement

Forced Vital Capacity on Spirometry

Diffusion Capacity of Lung for Carbon Dioxide

6 MWT with Maximal Desaturation on Pulse Oximetry

6 MWT for Distance

 

Survival

CTD-ILD             IPF

(8.0/8.2)            (9.0/8.4)

 

Time to Decline in Forced Vital Capacity

Progression Free Survival

Time to Death

 


Disclosure:

L. A. Saketkoo,

United Therapeutics,

2,

Actelion Pharmaceuticals US,

2;

D. Huscher,
None;

D. Khanna,

Actelion, BMS, Gilead, Genentech, ISDIN, and United Therapeutics,

2,

Actelion, BMS, Gilead, Genentech, ISDIN, and United Therapeutics,

5,

Actelion, BMS, Gilead, Genentech, ISDIN, and United Therapeutics,

8;

P. F. Dellaripa,

Novartis Pharmaceutical Corporation,

2,

Stomedix, Inc.,

2,

Intermune, Inc.,

2,

Genentech and Biogen IDEC Inc.,

2;

K. Flaherty,
None;

C. V. Oddis,

NIAMS, NIH,

2;

K. Phillips,
None;

A. U. Wells,
None;

C. P. Denton,

Actelion Pharmaceuticals US,

5,

GlaxoSmithKline,

5,

Pfizer Inc,

5,

United Therapeutics,

5;

O. Distler,

Actelion, Pfizer, Boehringer-Ingelheim, Bayer, Roche, Ergonex, BMS, Sanofi-Aventis, United BioSource Corporation, medac, Biovitrium, Novartis and Active Biotec,

2,

Actelion, Pfizer, Boehringer-Ingelheim, Bayer, Roche, Ergonex, BMS, Sanofi-Aventis, United BioSource Corporation, medac, Biovitrium, Novartis and Active Biotec,

5,

Actelion, Pfizer and Ergonex,

8;

O. M. Kowal-Bielecka,
None;

R. Christmann,
None;

N. Sandorfi,
None;

D. Pittrow,

Actelion Pharmaceuticals US,

8,

Pfizer Inc,

5,

Baxter, Inc,

5,

Mbiotec,

1,

Bayer,

5,

Sanofi-Aventis Pharmaceutical,

5,

MSD Germany,

5,

Novartis Pharmaceutical Corporation,

5;

V. Strand,
None;

J. R. Seibold,

Actelion Pharmaceuticals EU,

5,

United Therapeutics,

5,

Bayer Pharmaceuticals,

5;

K. K. Brown,

Actelion Pharmaceuticals US,

2,

Amgen,

2,

Fibrogen,

2,

gilead,

2,

Genentech and Biogen IDEC Inc.,

2,

Celgene,

2;

E. L. Matteson,

American College of Rheumatology and EULAR grant to develop classification criteria for rheumatoid arthritis.,

2,

Novartis Pharmaceutical Corporation,

2,

Horizon,

5.

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