Analysis of an ordinal endpoint for use in evaluating treatments for severe influenza requiring hospitalization

Clinical Trials

Volumn 14, Issue 3, 2017, Pages 264-276

  Peterson, Ross L ^a   Vock, David M ^a   Powers, John H ^b   Emery, Sean ^c   Cruz, Eduardo Fernandez ^d,e   Hunsberger, Sally ^f   Jain, Mamta K ^g   Pett, Sarah ^c,h   Neaton, James D ^a  

^a UNIVERSITY OF MINNESOTA   (United States)

^b GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

^c UNIVERSITY OF NEW SOUTH WALES   (Australia)

^d HOSPITAL GENERAL UNIVERSITARIO GREGORIO MARAÑÓN   (Spain)

^e UNIVERSIDAD COMPLUTENSE DE MADRID   (Spain)

^f NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^g UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^h UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Clinical trials; endpoints; misspecified model; proportional odds model; statistical power

Indexed keywords

IMMUNOGLOBULIN; PLACEBO;

ARTICLE; CLASSIFICATION; CONTROLLED STUDY; DEATH; DOUBLE BLIND PROCEDURE; HOSPITAL DISCHARGE; HOSPITALIZATION; HUMAN; INFLUENZA; INTENSIVE CARE UNIT; MONTE CARLO METHOD; MULTICENTER STUDY; OXYGEN SUPPLY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; STUDY DESIGN; TREATMENT OUTCOME; ALGORITHM; PROPORTIONAL HAZARDS MODEL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SEVERITY OF ILLNESS INDEX; STATISTICAL ANALYSIS; STATISTICS AND NUMERICAL DATA;

ALGORITHMS; DATA INTERPRETATION, STATISTICAL; HOSPITALIZATION; HUMANS; IMMUNOGLOBULINS, INTRAVENOUS; INFLUENZA, HUMAN; MONTE CARLO METHOD; PROPORTIONAL HAZARDS MODELS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 85019719832     PISSN: 17407745     EISSN: 17407753     Source Type: Journal    
DOI: 10.1177/1740774517697919     Document Type: Article

References (24)

1. World Health Organization. Influenza., http://www.who.int/topics/influenza/en (2016,. accessed 30 June 2016).
2. Food and Drug Administration. Guidance for industry influenza: developing drugs for treatment and/or prophylaxis. Report, Food and Drug Administration, 2011.
3. Ison MG, Fraiz J, Heller B, et al. Intravenous peramivir for treatment of influenza in hospitalized patients. Antivir Ther 2014; 19: 349-361.
4. Ison MG, Hui DS, Clezy K, et al. A clinical trial of intravenous peramivir compared with oral oseltamivir for the treatment of seasonal influenza in hospitalized adults. Antivir Ther 2013; 18: 651-661.
5. Ison MG, De Jong MD, Gilligan KJ, et al. End points for testing influenza antiviral treatments for patients at high risk of severe and life-threatening disease. J Infect Dis 2010; 201: 1654-1662.
6. De Jong MD, Ison MG, Monto AS, et al. Evaluation of intravenous peramivir for treatment of influenza in hospitalized patients. Clin Infect Dis 2014; 59: 172-185.
7. South East Asia Infectious Disease Clinical Research Network. Effect of double dose oseltamivir on clinical and virological outcomes in children and adults admitted to hospital with severe influenza: double blind randomised controlled trial. BMJ 2013; 346: 1-16.
8. INSIGHT FLU005 IVIG Pilot Study Group. INSIGHT FLU005: an anti-influenza virus hyperimmune intravenous immunoglobulin pilot study. J Infect Dis 2016; 213: 574-578.
9. National Institute of Allergy and Infectious Diseases. Evaluating the safety and efficacy of anti-influenza intravenous hyperimmune immunoglobulin (IVIG) in adults hospitalized with influenza., https://clinicaltrials.gov/ct2/show/NCT02287467 (2016,. accessed 30 June 2016).
10. Royal College of Physicians of London. National Early Warning Score (NEWS)., https://www.rcplondon.ac.uk/projects/outputs/national-early-warning-score-news (2015,. accessed 30 June 2016).
11. Lynfield R, Davey R, Dwyer DE, et al. Outcomes of influenza A(H1N1)pdm09 virus infection: results from two international cohort studies. PLoS ONE 2014; 9: 1-15.
12. Dwyer DE., Surveillance of illness associated with pandemic (H1N1) 2009 virus infection among adults using a global clinical site network approach: the INSIGHT FLU 002 and FLU 003 studies. Vaccine 2011; 29: 56-62.
13. McCullagh P., Regression models for ordinal data. J R Stat Soc B 1980; 42: 109-142.
14. Whitehead J., Sample size calculations for ordered categorical data. Stat Med 1993; 12: 2257-2271.
15. Jeong JH., A note on asymptotic efficiency of a regression coefficient parameter under ordinal logistic regression model. Commun Stat Theory Methods 2001; 30: 1257-1269.
16. Strömberg U., Collapsing ordered outcome categories: a note of concern. Am J Epidemiol 1996; 144: 421-424.
17. Powers JH, Guerrero ML, Leidy NK, et al. Development of the Flu-PRO: a patient-reported outcome (PRO) instrument to evaluate symptoms of influenza. BMC Infect Dis 2015; 16: 1-11.
18. Roozenbeek B, Lingsma HF, Perel P, et al. The added value of ordinal analysis in clinical trials: an example in traumatic brain injury. Crit Care 2011; 15: 1-7.
19. Pédrono G, Thiébaut R, Alioum A, et al. A new endpoint definition improved clinical relevance and statistical power in a vaccine trial. J Clin Epidemiol 2009; 62: 1054-1061.
20. Maas AIR, Steyerberg EW, Marmarou A, et al. IMPACT recommendations for improving the design and analysis of clinical trials in moderate to severe traumatic brain injury. Neurotherapeutics 2010; 7: 127-134.
21. Bath PMW, Geeganage C, Gray LJ, et al. Use of ordinal endpoints in vascular prevention trials: comparison with binary outcomes in published trials. Stroke 2008; 39: 2817-2823.
22. Kromrey JD, Hogarty KY., Analysis options for testing group differences on ordered categorical variables: an empirical investigation of type I error control and statistical power. Mult Linear Regres Viewpoints 1998; 25: 70-82.
23. McHugh GS, Butcher I, Steyerberg EW, et al. A simulation study evaluating approaches to the analysis of ordinal endpoint data in randomized controlled trials in traumatic brain injury: results from the IMPACT Project. Clin Trials 2010; 7: 44-57.
24. Pocock SJ, Ariti CA, Collier TJ, et al. The win ratio: a new approach to the analysis of composite endpoints in clinical trials based on clinical priorities. Eur Heart J 2012; 33: 176-182.