Clinical Trial Simulation To Assist In COPD Trial Planning And Design With A Biomarker-Based Diagnostic: When To Pull The Trigger?

References

• Barnes PJ, Celli BR. Systemic manifestations and comorbidities of COPD. Eur Respir J 2009; 33(5):1165–1185.
   PubMed Web of Science ®Google Scholar
• Allen-Ramey FC, Gupta S, DiBonaventura MD, Patient characteristics, treatment patterns, and health outcomes among COPD phenotypes. Int J Chron Obstruct Pulmon Dis 2012; 7:779–787.
   PubMed Web of Science ®Google Scholar
• Toy EL, Gallagher KF, Stanley EL, Swensen AR, Duh MS. The economic impact of exacerbations of chronic obstructive pulmonary disease and exacerbation definition: a review. COPD 2010; 7(3):214–228.
   PubMedGoogle Scholar
• Mannino DM, Buist AS. Global burden of COPD: risk factors, prevalence, and future trends. Lancet 2007; 370(9589):765–773.
   PubMed Web of Science ®Google Scholar
• Lopez AD, Shibuya K, Rao C, Mathers CD, Hansell AL, Held LS, Schmid V, Buist S. Chronic obstructive pulmonary disease: current burden and future projections. Eur Respir J 2006; 27:397–412.
   PubMed Web of Science ®Google Scholar
• Richard Russell R, Anzueto A, Weisman I. Optimizing management of chronic obstructive pulmonary disease in the upcoming decade. Int J Chron Obstruct Pulmon Dis 2011; 6:47–61.
   PubMedGoogle Scholar
• Vestbo J, Hurd SS, Agusti AG, Jones PW, Vogelmeier C, Anzueto A, Global Strategy for the Diagnosis, Management and Prevention of Chronic Obstructive Pulmonary Disease, GOLD Executive Summary. Am J Respir Crit Care Med 2012; Epub ahead of print.
   Web of Science ®Google Scholar
• Gómez FP, Rodriguez-Roisin R. Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines for chronic obstructive pulmonary disease. Curr Opin Pulm Med 2002; 8(2):81–86.
   PubMed Web of Science ®Google Scholar
• Silverman EK, Vestbo J, Agusti A, Anderson W, Bakke PS, Barnes KC, Opportunities and challenges in the genetics of COPD 2010: an International COPD Genetics Conference report. COPD 2011; 8(2):121–135.
   PubMedGoogle Scholar
• Simpson JL, Scott R, Boyle MJ, Gibson PG. Inflammatory subtypes in asthma: assessment and identification using induced sputum. Respirology 2006; 11(1):54–61.
   PubMed Web of Science ®Google Scholar
• Saha S, Brightling CE. Eosinophilic airway inflammation in COPD. Int J Chron Obstruct Pulmon Dis 2006; 1(1):39–47.
   PubMedGoogle Scholar
• Siva R, Green RH, Brightling CE, Shelley M, Hargadon B, McKenna S, Eosinophilic airway inflammation and exacerbations of COPD: a randomised controlled trial. Eur Respir J 2007; 29(5):906–913.
   PubMed Web of Science ®Google Scholar
• Bafadhel M, McCormick M, Saha S, McKenna S, Shelley M, Hargadon B, Profiling of sputum inflammatory mediators in asthma and chronic obstructive pulmonary disease. Respiration 2012; 83(1):36–44.
   PubMed Web of Science ®Google Scholar
• Bafadhel M, McKenna S, Terry S, Mistry V, Pancholi M, Venge P, Blood eosinophils to direct corticosteroid treatment of exacerbations of chronic obstructive pulmonary disease: a randomized placebo-controlled trial. Am J Respir Crit Care Med 2012; 186(1):48–55.
   PubMed Web of Science ®Google Scholar
• Bafadhel M, McKenna S, Terry S, Mistry V, Reid C, Haldar P, Acute exacerbations of chronic obstructive pulmonary disease: identification of biologic clusters and their biomarkers. Am J Respir Crit Care Med 2011: 184(6):662–671.
   PubMed Web of Science ®Google Scholar
• Brightling CE, Monteiro W, Ward R, Sputum eosinophilia and short-term response to prednisolone in chronic obstructive pulmonary disease: a randomized controlled trial. Lancet 2000; 356:1480–1485.
   PubMed Web of Science ®Google Scholar
• Leigh R, Pizzichini MMM, Morris MM, Maltais F, Hargreave FE, Pizzichini E. Stable COPD: predicting benefit from high-dose inhaled corticosteroid treatment. Euro Resp Journ 2006; 27(5):946–971.
   Google Scholar
• Kitaguchi Y, Komatsu Y, Fujimoto K, Hanaoka M, Kubo K. Sputum eosinophilia can predict responsiveness to inhaled corticosteroid treatment in patients with overlap syndrome of COPD and asthma. Int Journ of Chron Obstr Pul Dis 2012; 7:283–289.
   PubMed Web of Science ®Google Scholar
• Soter S, Barta I, Antus B. Predicting sputum eosinophilia in exacerbations of COPD using exhaled nitric oxide. Inflammation 2013; Epub: 10.1007/s10753–013–9653–8.
   PubMed Web of Science ®Google Scholar
• Saha S, Doe C, Mistry V, Siddiqui S, Parker D, Sleeman M, Cohen ES, Brightling CE. Granulocyte–macrophage colony-stimulating factor expression in induced sputum and bronchial mucosa in asthma and COPD. Thorax 2009; 64(8):671–676.
   PubMed Web of Science ®Google Scholar
• Pavord ID, Korn S, Howarth P, Bleecker ER, Buhl R, Keene ON, Ortega H, Chanez P. Mepolizumab for severe eosinophilic asthma (DREAM): a multicentre, double-blind, placebo-controlled trial. Lancet 2012; 380(9842):651–659.
   PubMed Web of Science ®Google Scholar
• Castro M, Mathur S, Hargreave F, Boulet LP, Xie F, Young J, Wilkins HJ, Henkel T, Nair P. Reslizumab for poorly controlled, eosinophilic asthma: a randomized, placebo-controlled study. Am J Respir Crit Care Med 2011; 184(10):1125–1132.
   PubMed Web of Science ®Google Scholar
• Holford N, Ma SC, Ploeger BA. Clinical trial simulation: a review. Clin Pharmacol Ther 2010; 88(2):166–182.
   PubMed Web of Science ®Google Scholar
• Girard P. Clinical trial simulation: a tool for understanding study failures and preventing them. Basic Clin Pharmacol Toxicol 2005; 96(3):228–234.
   PubMed Web of Science ®Google Scholar
• Eddy DM, Schlessinger L.Validation of the Archimedes diabetes model. Diabetes Care 2003;26(11):3102–3110.
   PubMed Web of Science ®Google Scholar
• Weinstein MC, O'Brien B, Hornberger J, Jackson J, Johannesson M, McCabe C, Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR Task Force on Good Research Practices–Modeling Studies. Value Health 2003; 6(1):9–17.
   PubMed Web of Science ®Google Scholar
• Hurst JR, Vestbo J, Anzueto A, Locantore N, Müllerova H, Tal-Singer R, Susceptibility to exacerbation in chronic obstructive pulmonary disease. Evaluation of COPD Longitudinally to Identify Predictive Surrogate Endpoints (ECLIPSE) Investigators. N Engl J Med 2010; 363(12):1128–1138.
   PubMed Web of Science ®Google Scholar
• Magda S. Susceptibility to exacerbation in chronic obstructive pulmonary disease - Data from the ECLIPSE study. Maedica 2010; 5(3):223–224.
   PubMedGoogle Scholar
• Westfall PH, Tsai K, Ogenstad S, Tomoiaga A, Moseley S, Lu Y. Clinical Trials Simulation: A Statistical Approach. J Biopharm Stat 2008; 18:611–630.
   PubMed Web of Science ®Google Scholar
• Casella G, Berger RL. Statistical Inference.. Chapter 5 Properties of a Random Sample. Belmont (CA): Duxbury Press; 1990: 201–235.
   Google Scholar
• Vogelmeier C, Ramos-Barbon D, Jack D, Piggott S, Owen R, Higgins M, Indacaterol provides 24-hour bronchodilation in COPD: a placebo-controlled blinded comparison with tiotropium. Respir Res 2010; 11:135.
   PubMed Web of Science ®Google Scholar
• Calverley PMA, Rabe KF, Goehring UM, Kristiansen S, Fabbri LM, Martinez FJ. Roflumilast in symptomatic chronic obstructive pulmonary disease: two randomised clinical trials. Lancet 2009; 374(9691):685–694.
   PubMed Web of Science ®Google Scholar
• Pepe S. The Statistical Evaluation of Medical Tests For Classification and Prediction: Oxford Statistical Science Series 28. New York: Oxford University Press; c2003. Ch 2, Measures of accuracy for binary tests; p. 14–34.
   Google Scholar
• Trusheim MR, Burgess B, Hu SX, Long T, Averbuch SD, Flynn AA, Quantifying factors for the success of stratified medicine. Nat Rev Drug Discov 2011; 10(11):817–833.
   PubMed Web of Science ®Google Scholar
• Lalonde RL, Kowalski KG, Hutmacher MM, Ewy W, Nichols DJ, Milligan PA, Model-based drug development. Clin Pharmacol Ther 2007; 82(1):21–32.
   PubMed Web of Science ®Google Scholar