Impact of acute antibiotic therapy on the pulmonary exacerbation endpoint in cystic fibrosis clinical trials

Contemporary Clinical Trials

Volumn 36, Issue 1, 2013, Pages 99-105

  Mayer Hamblett, Nicole ^a,b   Saiman, Lisa ^c   Lands, Larry C ^d   Anstead, Michael ^e   Rosenfeld, Margaret ^a,b   Kloster, Margaret ^b   Fisher, Leigh ^b   Ratjen, Felix ^f  

^a UNIVERSITY OF WASHINGTON   (United States)

^b SEATTLE CHILDREN S HOSPITAL   (United States)

^c Och Spine at New York Presbyterian Hospitals   (United States)

^d MCGILL UNIVERSITY   (Canada)

^e UNIVERSITY OF KENTUCKY   (United States)

^f HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

Antibiotic therapy; Cystic fibrosis; Pulmonary exacerbation definitions; Sample size; Study design

Indexed keywords

AZITHROMYCIN; PLACEBO;

ADOLESCENT; ADULT; ANTIBIOTIC THERAPY; ARTICLE; CHILD; CONFIDENCE INTERVAL; CONTROLLED STUDY; CYSTIC FIBROSIS; DISEASE EXACERBATION; DRUG EFFICACY; FEMALE; HUMAN; LUNG DISEASE; MAJOR CLINICAL STUDY; MALE; PHYSICIAN; PRESCHOOL CHILD; PRESCRIPTION; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD; TREATMENT RESPONSE;

ANTIBIOTIC THERAPY; AZ; AZITHROMYCIN; CF; CI; CONFIDENCE INTERVAL; CYSTIC FIBROSIS; HAZARD RATIO; HR; INTRAVENOUS; IV; P; P-VALUE; PATIENT REPORTED OUTCOMES; PE; PRO; PULMONARY EXACERBATION; PULMONARY EXACERBATION DEFINITIONS; SAMPLE SIZE; STUDY DESIGN;

ADOLESCENT; ANTI-BACTERIAL AGENTS; AZITHROMYCIN; CHILD; CLINICAL PROTOCOLS; CYSTIC FIBROSIS; FEMALE; HUMANS; LUNG DISEASES; MALE; RESEARCH DESIGN; SAMPLE SIZE;

EID: 84880296779     PISSN: 15517144     EISSN: 15592030     Source Type: Journal    
DOI: 10.1016/j.cct.2013.06.004     Document Type: Article

References (34)

1. Flume P.A., Mogayzel P.J., Robinson K.A., Rosenblatt R.L., Quittell L., Marshall B.C., et al. Cystic fibrosis pulmonary guidelines: pulmonary complications: treatment of pulmonary exacerbations. Am J Respir Crit Care Med 2009, 180(9):802-808.
2. Sanders D.B., Hoffman L.R., Emerson J., Gibson R.L., Rosenfeld M., Redding G.J., et al. Return of FEV1 after pulmonary exacerbation in children with cystic fibrosis. Pediatr Pulmonol 2010, 45:127-134.
3. Waters V., Stanojevic S., Atenafu E.G., Lu A., Yau Y., Tullis E., et al. Effect of pulmonary exacerbations on long-term lung function decline in cystic fibrosis. Eur Respir J 2012, 40:61-66.
4. Sanders D.B., Bittner R.C., Rosenfeld M., Redding G.J., Goss C.H. Pulmonary exacerbations are associated with subsequent FEV1 decline in both adults and children with cystic fibrosis. Pediatr Pulmonol 2011, 46:393-400.
5. de Boer K., Vandemheen K.L., Tullis E., Doucette S., Fergusson D., Freitag A., et al. Exacerbation frequency and clinical outcomes in adult patients with cystic fibrosis. Thorax 2011, 66:680-685.
6. Konstan M.W., Morgan W.J., Butler S.M., Pasta D.J., Craib M.L., Silva S.J., et al. Risk factors for rate of decline in forced expiratory volume in one second in children and adolescents with cystic fibrosis. J Pediatr 2007, 151:134-139. [39 e1].
7. Orenstein D.M., Pattishall E.N., Nixon P.A., Ross E.A., Kaplan R.M. Quality of well-being before and after antibiotic treatment of pulmonary exacerbation in patients with cystic fibrosis. Chest 1990, 98:1081-1084.
8. Britto M.T., Kotagal U.R., Hornung R.W., Atherton H.D., Tsevat J., Wilmott R.W. Impact of recent pulmonary exacerbations on quality of life in patients with cystic fibrosis. Chest 2002, 121:64-72.
9. Goss C.H., Edwards T.C., Ramsey B.W., Aitken M.L., Patrick D.L. Patient-reported respiratory symptoms in cystic fibrosis. J Cyst Fibros 2009, 8:245-252.
10. Liou T.G., Adler F.R., Fitzsimmons S.C., Cahill B.C., Hibbs J.R., Marshall B.C. Predictive 5-year survivorship model of cystic fibrosis. Am J Epidemiol 2001, 153:345-352.
11. Mayer-Hamblett N., Rosenfeld M., Emerson J., Goss C.H., Aitken M.L. Developing cystic fibrosis lung transplant referral criteria using predictors of 2-year mortality. Am J Respir Crit Care Med 2002, 166:1550-1555.
12. Emerson J., Rosenfeld M., McNamara S., Ramsey B., Gibson R.L. Pseudomonas aeruginosa and other predictors of mortality and morbidity in young children with cystic fibrosis. Pediatr Pulmonol Aug 2002, 34(2):91-100.
13. Montgomery A.B., Abuan T., Yeager M.A. Regulatory aspects of phase 3 endpoints for new inhaled antibiotics for cystic fibrosis patients with chronic Pseudomonas aeruginosa infections. J Aerosol Med Pulm Drug Deliv 2012, 25:198-203.
14. Mayer-Hamblett N., Ramsey B.W., Kronmal R.A. Advancing outcome measures for the new era of drug development in cystic fibrosis. Proc Am Thorac Soc 2007, 4:370-377. [PMCID: 2647602].
15. Rosenfeld M., Emerson J., Williams-Warren J., Pepe M., Smith A., Montgomery A.B., et al. Defining a pulmonary exacerbation in cystic fibrosis. J Pediatr 2001, 139:359-365.
16. Bilton D., Canny G., Conway S., Dumcius S., Hjelte L., Proesmans M., et al. Pulmonary exacerbation: towards a definition for use in clinical trials. Report from the EuroCareCF Working Group on outcome parameters in clinical trials. J Cyst Fibros Jun;10 2011, (Suppl. 2):S79-S81.
17. Goss C.H., Burns J.L. Exacerbations in cystic fibrosis. 1: Epidemiology and pathogenesis. Thorax 2007, 62:360-367.
18. Ramsey B.W., Pepe M.S., Quan J.M., Otto K.L., Montgomery A.B., Williams-Warren J., et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. N Engl J Med 1999, 340:23-30.
19. Fuchs H.J., Borowitz D.S., Christiansen D.H., Morris E.M., Nash M.L., Ramsey B.W., et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl J Med 1994, 331:637-642.
20. Saiman L., Marshall B.C., Mayer-Hamblett N., Burns J.L., Quittner A.L., Cibene D.A., et al. Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA 2003, 290:1749-1756.
21. Saiman L., Anstead M., Mayer-Hamblett N., Lands L.C., Kloster M., Hocevar-Trnka J., et al. Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA 2010, 303:1707-1715.
22. Elkins M.R., Robinson M., Rose B.R., Harbour C., Moriarty C.P., Marks G.B., et al. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med 2006, 354:229-240.
23. McCoy K.S., Quittner A.L., Oermann C.M., Gibson R.L., Retsch-Bogart G.Z., Montgomery A.B. Inhaled aztreonam lysine for chronic airway Pseudomonas aeruginosa in cystic fibrosis. Am J Respir Crit Care Med 2008, 178:921-928.
24. Treggiari M.M., Retsch-Bogart G., Mayer-Hamblett N., Khan U., Kulich M., Kronmal R., et al. Comparative efficacy and safety of 4 randomized regimens to treat early Pseudomonas aeruginosa infection in children with cystic fibrosis. Arch Pediatr Adolesc Med 2011, 165:847-856.
25. Ramsey B.W., Davies J., McElvaney N.G., Tullis E., Bell S.C., Dřevínek P., et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011, 365:1663-1672. [PMCID: 3230303].
26. Rosenfeld M., Ratjen F., Brumback L., Daniel S., Rowbotham R., McNamara S., et al. ISIS Study Group. Inhaled hypertonic saline in infants and children younger than 6years with cystic fibrosis: the ISIS randomized controlled trial. JAMA Jun 6 2012, 307(21):2269-2277.
27. Mayer-Hamblett N., Ratjen F. "Background" antibiotic usage in cystic fibrosis clinical trials: does it impact our assessment of clinical efficacy?. Lancet Respir Apr 2013, 1(2):91-174.
28. Treggiari M.M., Rosenfeld M., Mayer-Hamblett N., Retsch-Bogart G., Gibson R.L., Williams J., et al. Early anti-pseudomonal acquisition in young patients with cystic fibrosis: rationale and design of the EPIC clinical trial and observational study. Contemp Clin Trials 2009, 30:256-268. [PMCID: 2783320].
29. Food and Drug Administration [20 Mar 2013]. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/Anti-InfectiveDrugsAdvisoryCommittee/UCM193023.pdf.
30. O'Connor A.B. The need for improved access to FDA reviews. JAMA 2009, 302(2):191-193.
31. Regelmann W.E., Schechter M.S., Wagener J.S., Morgan W.J., Pasta D.J., Elkin E.P., et al. For the investigators of the epidemiologic study of cystic fibrosis. Pulmonary exacerbations in cystic fibrosis: young children with characteristic signs and symptoms. Pediatr Pulmonol Sep 4 2012, [Epub ahead of print]. 10.1002/ppul.22658.
32. Byrnes C.A., Vidmar S., Cheney J.L., Carlin J.B., Armstrong D.S., Cooper P.J., et al. For the ACFBAL Study investigators. Prospective evaluation of respiratory exacerbations in children with cystic fibrosis from newborn screening to 5years of age. Thorax Jan 23 2013, [Epub ahead of print]. 10.1136/thoraxjnl-2012-202342.
33. Quittner A.L., Modi A.C., Wainwright C., Otto K., Kirihara J., Montgomery A.B. Determination of the minimal clinically important difference scores for the Cystic Fibrosis Questionnaire - Revised respiratory symptom scale in two populations of patients with cystic fibrosis and chronic Pseudomonas aeruginosa airway infection. Chest Jun 2009, 135(6):1610-1618. (Epub 2009 May 15. Review). 10.1378/chest.08-1190.
34. Food and Drug Administration [02 Jan 2013]. http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/ucm148011.htm.