Outcome measures for clinical trials in interstitial lung diseases

Matthew R. Lammi, Robert P. Baughman, Surinder S. Birring, Anne Marie Russell, Jay H Ryu, Mary Beth Scholand, Oliver Distler, Daphne Lesage, Catherine Sarver, Katerina Antoniou, Kristin B. Highland, Otylia Kowal-Bielecka, Joseph A. Lasky, Athol U. Wells, Lesley Ann Saketkoo

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

The chronic fibrosing idiopathic interstitial pneumonias (IIPs) are a group of heterogeneous pulmonary parenchymal disorders described by radiologic and histological patterns termed usual interstitial pneumonia (UIP) and non-specific interstitial pneumonia (NSIP). These include idiopathic pulmonary fibrosis (IPF) and those related to connective tissue disease (CTD) and are associated with substantial morbidity and mortality. Beyond the importance of establishing an appropriate diagnosis, designing optimal clinical trials for IIPs has been fraught with difficulties in consistency of clinical endpoints making power analyses, and the establishment of efficacy and interpretation of results across trials challenging. Preliminary recommendations, developed by rigorous consensus methods, proposed a minimum set of outcome measures, a ‘core set’, to be incorporated into future clinical trials (Saketkoo et al, THORAX. 2014.). This paper sets out to examine the candidate instruments for each domain (Dyspnea, Cough, Health Related Quality of Life, Imaging, Lung Physiology and Function, Mortality). Candidate measures that were not selected as well as measures that were not available for examination at the time of the consensus process will also be discussed.

Original languageEnglish (US)
Pages (from-to)163-174
Number of pages12
JournalCurrent Respiratory Medicine Reviews
Volume11
Issue number2
StatePublished - Sep 1 2015

Fingerprint

Idiopathic Interstitial Pneumonias
Idiopathic Pulmonary Fibrosis
Interstitial Lung Diseases
Outcome Assessment (Health Care)
Clinical Trials
Lung
Connective Tissue Diseases
Mortality
Cough
Dyspnea
Quality of Life
Morbidity

Keywords

  • Chronic fibrosing
  • Connective tissue disease related interstitial lung disease
  • Idiopathic interstitial pneumonia
  • Idiopathic pulmonary fibrosis
  • Rheumatoid arthritis
  • Scleroderma
  • Systemic sclerosis

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

Cite this

Lammi, M. R., Baughman, R. P., Birring, S. S., Russell, A. M., Ryu, J. H., Scholand, M. B., ... Saketkoo, L. A. (2015). Outcome measures for clinical trials in interstitial lung diseases. Current Respiratory Medicine Reviews, 11(2), 163-174.

Outcome measures for clinical trials in interstitial lung diseases. / Lammi, Matthew R.; Baughman, Robert P.; Birring, Surinder S.; Russell, Anne Marie; Ryu, Jay H; Scholand, Mary Beth; Distler, Oliver; Lesage, Daphne; Sarver, Catherine; Antoniou, Katerina; Highland, Kristin B.; Kowal-Bielecka, Otylia; Lasky, Joseph A.; Wells, Athol U.; Saketkoo, Lesley Ann.

In: Current Respiratory Medicine Reviews, Vol. 11, No. 2, 01.09.2015, p. 163-174.

Research output: Contribution to journalArticle

Lammi, MR, Baughman, RP, Birring, SS, Russell, AM, Ryu, JH, Scholand, MB, Distler, O, Lesage, D, Sarver, C, Antoniou, K, Highland, KB, Kowal-Bielecka, O, Lasky, JA, Wells, AU & Saketkoo, LA 2015, 'Outcome measures for clinical trials in interstitial lung diseases', Current Respiratory Medicine Reviews, vol. 11, no. 2, pp. 163-174.
Lammi MR, Baughman RP, Birring SS, Russell AM, Ryu JH, Scholand MB et al. Outcome measures for clinical trials in interstitial lung diseases. Current Respiratory Medicine Reviews. 2015 Sep 1;11(2):163-174.
Lammi, Matthew R. ; Baughman, Robert P. ; Birring, Surinder S. ; Russell, Anne Marie ; Ryu, Jay H ; Scholand, Mary Beth ; Distler, Oliver ; Lesage, Daphne ; Sarver, Catherine ; Antoniou, Katerina ; Highland, Kristin B. ; Kowal-Bielecka, Otylia ; Lasky, Joseph A. ; Wells, Athol U. ; Saketkoo, Lesley Ann. / Outcome measures for clinical trials in interstitial lung diseases. In: Current Respiratory Medicine Reviews. 2015 ; Vol. 11, No. 2. pp. 163-174.
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