Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis: Provisional core sets of domains and instruments for use in clinical trials

Lesley Ann Saketkoo, Shikha Mittoo, Dörte Huscher, Dinesh Khanna, Paul F. Dellaripa, Oliver Distler, Kevin R. Flaherty, Sid Frankel, Chester V. Oddis, Christopher P. Denton, Aryeh Fischer, Otylia M. Kowal-Bielecka, Daphne Lesage, Peter A. Merkel, Kristine Phillips, David Pittrow, Jeffrey Swigris, Katerina Antoniou, Robert P. Baughman, Flavia V. CastelinoRomy B. Christmann, Lisa Christopher-Stine, Harold R. Collard, Vincent Cottin, Sonye Danoff, Kristin B. Highland, Laura Hummers, Ami A. Shah, Dong Soon Kim, David A. Lynch, Frederick W. Miller, Susanna M. Proudman, Luca Richeldi, Jay H Ryu, Nora Sandorfi, Catherine Sarver, Athol U. Wells, Vibeke Strand, Eric Lawrence Matteson, Kevin K. Brown, James R. Seibold

Research output: Contribution to journalArticle

58 Citations (Scopus)

Abstract

Rationale: Clinical trial design in interstitial lung diseases (ILDs) has been hampered by lack of consensus on appropriate outcome measures for reliably assessing treatment response. In the setting of connective tissue diseases (CTDs), some measures of ILD disease activity and severity may be confounded by non-pulmonary comorbidities. Methods: The Connective Tissue Disease associated Interstitial Lung Disease (CTD-ILD) working group of Outcome Measures in Rheumatology-a non-profit international organisation dedicated to consensus methodology in identification of outcome measures-conducted a series of investigations which included a Delphi process including >248 ILD medical experts as well as patient focus groups culminating in a nominal group panel of ILD experts and patients. The goal was to define and develop a consensus on the status of outcome measure candidates for use in randomised controlled trials in CTD-ILD and idiopathic pulmonary fibrosis (IPF). Results: A core set comprising specific measures in the domains of lung physiology, lung imaging, survival, dyspnoea, cough and health-related quality of life is proposed as appropriate for consideration for use in a hypothetical 1-year multicentre clinical trial for either CTD-ILD or IPF. As many widely used instruments were found to lack full validation, an agenda for future research is proposed. Conclusion: Identification of consensus preliminary domains and instruments to measure them was attained and is a major advance anticipated to facilitate multicentre RCTs in the field.

Original languageEnglish (US)
Pages (from-to)428-436
Number of pages9
JournalThorax
Volume69
Issue number5
DOIs
StatePublished - 2014

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Idiopathic Pulmonary Fibrosis
Connective Tissue Diseases
Interstitial Lung Diseases
Clinical Trials
Outcome Assessment (Health Care)
Nonprofit Organizations
Lung
Rheumatology
Focus Groups
Cough
Dyspnea
Multicenter Studies
Comorbidity
Randomized Controlled Trials
Quality of Life
Survival

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

Cite this

Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis : Provisional core sets of domains and instruments for use in clinical trials. / Saketkoo, Lesley Ann; Mittoo, Shikha; Huscher, Dörte; Khanna, Dinesh; Dellaripa, Paul F.; Distler, Oliver; Flaherty, Kevin R.; Frankel, Sid; Oddis, Chester V.; Denton, Christopher P.; Fischer, Aryeh; Kowal-Bielecka, Otylia M.; Lesage, Daphne; Merkel, Peter A.; Phillips, Kristine; Pittrow, David; Swigris, Jeffrey; Antoniou, Katerina; Baughman, Robert P.; Castelino, Flavia V.; Christmann, Romy B.; Christopher-Stine, Lisa; Collard, Harold R.; Cottin, Vincent; Danoff, Sonye; Highland, Kristin B.; Hummers, Laura; Shah, Ami A.; Kim, Dong Soon; Lynch, David A.; Miller, Frederick W.; Proudman, Susanna M.; Richeldi, Luca; Ryu, Jay H; Sandorfi, Nora; Sarver, Catherine; Wells, Athol U.; Strand, Vibeke; Matteson, Eric Lawrence; Brown, Kevin K.; Seibold, James R.

In: Thorax, Vol. 69, No. 5, 2014, p. 428-436.

Research output: Contribution to journalArticle

Saketkoo, LA, Mittoo, S, Huscher, D, Khanna, D, Dellaripa, PF, Distler, O, Flaherty, KR, Frankel, S, Oddis, CV, Denton, CP, Fischer, A, Kowal-Bielecka, OM, Lesage, D, Merkel, PA, Phillips, K, Pittrow, D, Swigris, J, Antoniou, K, Baughman, RP, Castelino, FV, Christmann, RB, Christopher-Stine, L, Collard, HR, Cottin, V, Danoff, S, Highland, KB, Hummers, L, Shah, AA, Kim, DS, Lynch, DA, Miller, FW, Proudman, SM, Richeldi, L, Ryu, JH, Sandorfi, N, Sarver, C, Wells, AU, Strand, V, Matteson, EL, Brown, KK & Seibold, JR 2014, 'Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis: Provisional core sets of domains and instruments for use in clinical trials', Thorax, vol. 69, no. 5, pp. 428-436. https://doi.org/10.1136/thoraxjnl-2013-204202
Saketkoo, Lesley Ann ; Mittoo, Shikha ; Huscher, Dörte ; Khanna, Dinesh ; Dellaripa, Paul F. ; Distler, Oliver ; Flaherty, Kevin R. ; Frankel, Sid ; Oddis, Chester V. ; Denton, Christopher P. ; Fischer, Aryeh ; Kowal-Bielecka, Otylia M. ; Lesage, Daphne ; Merkel, Peter A. ; Phillips, Kristine ; Pittrow, David ; Swigris, Jeffrey ; Antoniou, Katerina ; Baughman, Robert P. ; Castelino, Flavia V. ; Christmann, Romy B. ; Christopher-Stine, Lisa ; Collard, Harold R. ; Cottin, Vincent ; Danoff, Sonye ; Highland, Kristin B. ; Hummers, Laura ; Shah, Ami A. ; Kim, Dong Soon ; Lynch, David A. ; Miller, Frederick W. ; Proudman, Susanna M. ; Richeldi, Luca ; Ryu, Jay H ; Sandorfi, Nora ; Sarver, Catherine ; Wells, Athol U. ; Strand, Vibeke ; Matteson, Eric Lawrence ; Brown, Kevin K. ; Seibold, James R. / Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis : Provisional core sets of domains and instruments for use in clinical trials. In: Thorax. 2014 ; Vol. 69, No. 5. pp. 428-436.
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abstract = "Rationale: Clinical trial design in interstitial lung diseases (ILDs) has been hampered by lack of consensus on appropriate outcome measures for reliably assessing treatment response. In the setting of connective tissue diseases (CTDs), some measures of ILD disease activity and severity may be confounded by non-pulmonary comorbidities. Methods: The Connective Tissue Disease associated Interstitial Lung Disease (CTD-ILD) working group of Outcome Measures in Rheumatology-a non-profit international organisation dedicated to consensus methodology in identification of outcome measures-conducted a series of investigations which included a Delphi process including >248 ILD medical experts as well as patient focus groups culminating in a nominal group panel of ILD experts and patients. The goal was to define and develop a consensus on the status of outcome measure candidates for use in randomised controlled trials in CTD-ILD and idiopathic pulmonary fibrosis (IPF). Results: A core set comprising specific measures in the domains of lung physiology, lung imaging, survival, dyspnoea, cough and health-related quality of life is proposed as appropriate for consideration for use in a hypothetical 1-year multicentre clinical trial for either CTD-ILD or IPF. As many widely used instruments were found to lack full validation, an agenda for future research is proposed. Conclusion: Identification of consensus preliminary domains and instruments to measure them was attained and is a major advance anticipated to facilitate multicentre RCTs in the field.",
author = "Saketkoo, {Lesley Ann} and Shikha Mittoo and D{\"o}rte Huscher and Dinesh Khanna and Dellaripa, {Paul F.} and Oliver Distler and Flaherty, {Kevin R.} and Sid Frankel and Oddis, {Chester V.} and Denton, {Christopher P.} and Aryeh Fischer and Kowal-Bielecka, {Otylia M.} and Daphne Lesage and Merkel, {Peter A.} and Kristine Phillips and David Pittrow and Jeffrey Swigris and Katerina Antoniou and Baughman, {Robert P.} and Castelino, {Flavia V.} and Christmann, {Romy B.} and Lisa Christopher-Stine and Collard, {Harold R.} and Vincent Cottin and Sonye Danoff and Highland, {Kristin B.} and Laura Hummers and Shah, {Ami A.} and Kim, {Dong Soon} and Lynch, {David A.} and Miller, {Frederick W.} and Proudman, {Susanna M.} and Luca Richeldi and Ryu, {Jay H} and Nora Sandorfi and Catherine Sarver and Wells, {Athol U.} and Vibeke Strand and Matteson, {Eric Lawrence} and Brown, {Kevin K.} and Seibold, {James R.}",
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T1 - Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis

T2 - Provisional core sets of domains and instruments for use in clinical trials

AU - Saketkoo, Lesley Ann

AU - Mittoo, Shikha

AU - Huscher, Dörte

AU - Khanna, Dinesh

AU - Dellaripa, Paul F.

AU - Distler, Oliver

AU - Flaherty, Kevin R.

AU - Frankel, Sid

AU - Oddis, Chester V.

AU - Denton, Christopher P.

AU - Fischer, Aryeh

AU - Kowal-Bielecka, Otylia M.

AU - Lesage, Daphne

AU - Merkel, Peter A.

AU - Phillips, Kristine

AU - Pittrow, David

AU - Swigris, Jeffrey

AU - Antoniou, Katerina

AU - Baughman, Robert P.

AU - Castelino, Flavia V.

AU - Christmann, Romy B.

AU - Christopher-Stine, Lisa

AU - Collard, Harold R.

AU - Cottin, Vincent

AU - Danoff, Sonye

AU - Highland, Kristin B.

AU - Hummers, Laura

AU - Shah, Ami A.

AU - Kim, Dong Soon

AU - Lynch, David A.

AU - Miller, Frederick W.

AU - Proudman, Susanna M.

AU - Richeldi, Luca

AU - Ryu, Jay H

AU - Sandorfi, Nora

AU - Sarver, Catherine

AU - Wells, Athol U.

AU - Strand, Vibeke

AU - Matteson, Eric Lawrence

AU - Brown, Kevin K.

AU - Seibold, James R.

PY - 2014

Y1 - 2014

N2 - Rationale: Clinical trial design in interstitial lung diseases (ILDs) has been hampered by lack of consensus on appropriate outcome measures for reliably assessing treatment response. In the setting of connective tissue diseases (CTDs), some measures of ILD disease activity and severity may be confounded by non-pulmonary comorbidities. Methods: The Connective Tissue Disease associated Interstitial Lung Disease (CTD-ILD) working group of Outcome Measures in Rheumatology-a non-profit international organisation dedicated to consensus methodology in identification of outcome measures-conducted a series of investigations which included a Delphi process including >248 ILD medical experts as well as patient focus groups culminating in a nominal group panel of ILD experts and patients. The goal was to define and develop a consensus on the status of outcome measure candidates for use in randomised controlled trials in CTD-ILD and idiopathic pulmonary fibrosis (IPF). Results: A core set comprising specific measures in the domains of lung physiology, lung imaging, survival, dyspnoea, cough and health-related quality of life is proposed as appropriate for consideration for use in a hypothetical 1-year multicentre clinical trial for either CTD-ILD or IPF. As many widely used instruments were found to lack full validation, an agenda for future research is proposed. Conclusion: Identification of consensus preliminary domains and instruments to measure them was attained and is a major advance anticipated to facilitate multicentre RCTs in the field.

AB - Rationale: Clinical trial design in interstitial lung diseases (ILDs) has been hampered by lack of consensus on appropriate outcome measures for reliably assessing treatment response. In the setting of connective tissue diseases (CTDs), some measures of ILD disease activity and severity may be confounded by non-pulmonary comorbidities. Methods: The Connective Tissue Disease associated Interstitial Lung Disease (CTD-ILD) working group of Outcome Measures in Rheumatology-a non-profit international organisation dedicated to consensus methodology in identification of outcome measures-conducted a series of investigations which included a Delphi process including >248 ILD medical experts as well as patient focus groups culminating in a nominal group panel of ILD experts and patients. The goal was to define and develop a consensus on the status of outcome measure candidates for use in randomised controlled trials in CTD-ILD and idiopathic pulmonary fibrosis (IPF). Results: A core set comprising specific measures in the domains of lung physiology, lung imaging, survival, dyspnoea, cough and health-related quality of life is proposed as appropriate for consideration for use in a hypothetical 1-year multicentre clinical trial for either CTD-ILD or IPF. As many widely used instruments were found to lack full validation, an agenda for future research is proposed. Conclusion: Identification of consensus preliminary domains and instruments to measure them was attained and is a major advance anticipated to facilitate multicentre RCTs in the field.

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