Introdução à Medicina Turma 12

1. Outcome measures in randomized controlled trials of inhaled asthma medications: how often they are being put in practice Authors: Melo AR, Pinto C, Alves FC, Pereira JP, Garcia M, Gouveia P, Pinto RJ, Cavaco TC, Valente TAuthor’scontact: intromed1011turma12@gmail.comAdviser: Professor Tiago Jacinto Introdução à Medicina Turma 12

2. Introduction Turma 12

3. There is still no clear definition of asthma control: the criteria used in its evaluation varies from study to study (Reddel H et al. American Thoracic Society Documents 2008) American Thoracic Society – 2006 • List of outcome measures: • Baseline • During the study Turma 12

4. Both on baseline characteristics and on the assessment of the treatment effect on current clinical control: • Symptom free days • Relievers use • Composite scores (He Q. Ofqual 2009) • Quality of life (LJ Bilingham et al, Health Technology Assessment 1999) • Baseline characteristics: • Pre-BD Fev 1 (Spirometry for health care providers) • Pos-BD Fev 1 (Spirometry for health care providers) • Assessment of the treatment effect on current clinical control: • Exacerbation within last 1-4 weeks Turma 12

5. Research question Turma 12

6. MAIN RESEARCH QUESTION - Are randomized controlled trials that assess inhaled asthma therapy drugs using the minimum outcome measures recommended by the American Thoracic Society both on baseline and during the trial? • Is there any relation between pharmaceutical industry sponsorship and outcome measures taken? Turma 12

7. Aims Turma 12

8. Determine if RCTs are using the minimum set of outcome measures recommended. • Analyze which measures are used the most. • Understand the evolution of measures taken: • Through time; • Before and after the ATS’s guideline (2006). • Determine if there is a relation between pharmaceutical industry sponsorship and outcome measures taken. Turma 12

9. MethodsandParticipants Turma 12

10. Data collection • "Asthma” - clinicaltrials.gov database • Interventional studies phase III • Closed studies with results • 12 years and older participants • 61 articles • "Asthma” - clinicaltrialsregister.eu database • Interventional studies phase III • Completed studies • 12 years and older participants • 81 articles • Total of 142 articles found Turma 12

11. Eligibility criteria: • Randomized controlled trial phase III; • Asthma; • English language; • Pos 1980 (included); • 12 years and older; • Medication: beta-2-agonists and inhaled corticosteroids. Turma 12

12. Flowchart n=81 n=61 Turma 12

13. Inclusions • 17 trials included • 5 from clinicaltrials.gov • 12 from clinicaltrialsregister.eu Turma 12

14. Variables to exam • Measures taken • Sponsoring by pharmaceutical industry Turma 12

15. Results Turma 12

16. Articles’ Graphs

17. -Number of Recommended Outcome Measures used by each article according to time and database it was searched The line represent the Cut-off n =17 American Database European Database Turma 12

18. -Recommended Outcome Measures’ Frequency in articles included n=11 Turma 12

19. Pharmaceutical Industry Sponsorship • There is not enough data to analyse the influence of pharmaceutical industry. • Only 2 in 17 articles were not sponsored Turma 12

20. Statistical Analysis • Variable examined: number of recommended outcome measures used • T-test to compare databases • p= 0,002 (µ=5 , µ=2) • T-test to compare before/after 2007 • p= 0,018 (µ=5 , µ=2) Turma 12

21. Discussion and Conclusion Turma 12

22. Discussion and Conclusion • There is no evidence that the number of recommended outcome measures taken by articles has increased after 2007. • It seems that the ATS guideline is not being followed • No conclusions concerning the influence of pharmaceutical industry sponsoring Turma 12

23. References Turma 12

24. [1] - Bateman E, B.L., Cruz A, FitzGerald M, Haahtela T, Levy M, O'Byrne P, Ohta K, Paggario P, Pedersen S, Soto-Quiroz M, Wong G, executive committee. Becker A, Lemanske R, Pedersen S, Sly P, Soto-Quiroz M, Wong G, Zar H, pediatricwritting group. Pocket Guide for Asthma Management and Prevention in Children 5 years and younger. GINA - Global Initiative for Asthma . 2009. • [2] - Masoli M, Fabian D, Holt S, Beasley R. Global Burden of Asthma. GINA - Global Initiative for Asthma . 2006. • [3] - Frampton GK, Shepherd J. Patient-reported outcomes in clinical trials of inhaled asthma medications: systematic review and research needs. Qual Life Res. 2010 Oct 14. • [4] - Fromer L. Managing asthma: an evidence-based approach to optimizing inhaled corticosteroid treatment. Department of Family Medicine, UCLA School of Medicine (Los Angeles, USA). Souther Medical Journal. 2010 Oct; 103(10): 2-3 • [5] – The Diagnosis and Treatment of Adult Asthma. Best Practice Evidence-based Guideline (Wellington, New Zealand). New Zealand Guidelines Group (NZGG).2002 Sep. 1-3. Turma 12

25. [6] - Asthma Management Handbook. National Asthma Council Australia(South Melbourne). 2006 • [7] - Reddel HK, Taylor DR, Bateman ED, Boulet LP, Boushey HA, Busse WW, Casale TB, Chanez P, Enright PL, Gibson PG, de Jongste JC, Kerstjens HA, Lazarus SC, Levy ML, O'Byrne PM, Partridge MR, Pavord ID, Sears MR, Sterk PJ, Stoloff SW, Sullivan SD, Szefler SJ, Thomas MD, Wenzel SE. An official American Thoracic Society/European Respiratory Society statement: asthma control and exacerbations: standardizing endpoints for clinical asthma trials and clinical practice. American Journal of Respiratory and Critical Care Medicine. 2009 Jul 1;180(1): 59-99 • [8] - He Q. Estimating the Reliability of Composite Scores. Ofqual. 2009 Dec. 10: 1 • [9] - Billingham LJ, Abrams KR, Jones DR. Methods for the analysis of quality-of-life and survival data in health technology assessment. Health Technology Assessment (Winchester, England). 1999;3(10):1-152. • [10] – Spirometry for health care providers. Global Initiative for Chronic Obstructive Lung Disease (GOLD). 1-2 Turma 12