Efficacy and adverse effects of drugs used to treat adult cystic fibrosis

Bibliography

• MacKenzie T, Gifford AH, Sabadosa KA, et al. Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the cystic fibrosis foundation patient registry. Ann Intern Med 2014;161(4):233-41
   PubMed Web of Science ®Google Scholar
• Becq F, Mall MA, Sheppard DN, et al. Pharmacological therapy for cystic fibrosis: from bench to bedside. J Cyst Fibros 2011;10(Suppl 2):S129-45
   PubMedGoogle Scholar
• Ratjen F. What’s new in CF airway inflammation: an update. Paediatr Respir Rev 2006;7(Suppl 1):S70-2
   Google Scholar
• Hoffman LR, Ramsey BW. Cystic fibrosis therapeutics: the road ahead. Chest 2013;143(1):207-13
   PubMed Web of Science ®Google Scholar
• Rowe SM, Miller S, Sorscher EJ. Cystic fibrosis. N Engl J Med 2005;352(19):1992-2001
   PubMed Web of Science ®Google Scholar
• Mogayzel PJJr, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med 2013;187(7):680-9
   PubMed Web of Science ®Google Scholar
• Geller DE. Aerosol antibiotics in cystic fibrosis. Respir Care 2009;54(5):658-70
   PubMed Web of Science ®Google Scholar
• Ramsey BW, Dorkin HL, Eisenberg JD, et al. Efficacy of aerosolized tobramycin in patients with cystic fibrosis. N Engl J Med 1993;328(24):1740-6
   PubMed Web of Science ®Google Scholar
• Harrison MJ, McCarthy M, Fleming C, et al. Inhaled versus nebulised tobramycin: A real world comparison in adult cystic fibrosis (CF). J Cyst Fibros 2014;13(6):692-8
   PubMed Web of Science ®Google Scholar
• Galeva I, Konstan MW, Higgins M, et al. Tobramycin inhalation powder manufactured by improved process in cystic fibrosis: the randomized EDIT trial. Curr Med Res Opin 2013;29(8):947-56
   PubMed Web of Science ®Google Scholar
• Konstan MW, Geller DE, Minic P, et al. Tobramycin inhalation powder for P. aeruginosa infection in cystic fibrosis: the EVOLVE trial. Pediatr Pulmonol 2011;46(3):230-8
   PubMed Web of Science ®Google Scholar
• Konstan MW, Flume PA, Kappler M, et al. Safety, efficacy and convenience of tobramycin inhalation powder in cystic fibrosis patients: the EAGER trial. J Cyst Fibros 2011;10(1):54-61
   PubMed Web of Science ®Google Scholar
• Retsch-Bogart GZ, Burns JL, Otto KL, et al. A phase 2 study of aztreonam lysine for inhalation to treat patients with cystic fibrosis and Pseudomonas aeruginosa infection. Pediatr Pulmonol 2008;43(1):47-58
   PubMed Web of Science ®Google Scholar
• Retsch-Bogart GZ, Quittner AL, Gibson RL, et al. Efficacy and safety of inhaled aztreonam lysine for airway pseudomonas in cystic fibrosis. Chest 2009;135(5):1223-32
   PubMed Web of Science ®Google Scholar
• McCoy KS, Quittner AL, Oermann CM, et al. Inhaled aztreonam lysine for chronic airway Pseudomonas aeruginosa in cystic fibrosis. Am J Respir Crit Care Med 2008;178(9):921-8
   PubMed Web of Science ®Google Scholar
• Oermann CM, Retsch-Bogart GZ, Quittner AL, et al. An 18-month study of the safety and efficacy of repeated courses of inhaled aztreonam lysine in cystic fibrosis. Pediatr Pulmonol 2010;45(11):1121-34
   PubMed Web of Science ®Google Scholar
• Lim LM, Ly N, Anderson D, et al. Resurgence of colistin: a review of resistance, toxicity, pharmacodynamics, and dosing. Pharmacotherapy 2010;30(12):1279-91
   PubMed Web of Science ®Google Scholar
• Schuster A, Haliburn C, Doring G, et al. Safety, efficacy and convenience of colistimethate sodium dry powder for inhalation (Colobreathe DPI) in patients with cystic fibrosis: a randomised study. Thorax 2013;68(4):344-50
   PubMed Web of Science ®Google Scholar
• Westerman EM, Le Brun PP, Touw DJ, et al. Effect of nebulized colistin sulphate and colistin sulphomethate on lung function in patients with cystic fibrosis: a pilot study. J Cyst Fibros 2004;3(1):23-8
   PubMedGoogle Scholar
• Cigana C, Nicolis E, Pasetto M, et al. Anti-inflammatory effects of azithromycin in cystic fibrosis airway epithelial cells. Biochem Biophys Res Commun 2006;350(4):977-82
   PubMed Web of Science ®Google Scholar
• Jaffe A, Francis J, Rosenthal M, et al. Long-term azithromycin may improve lung function in children with cystic fibrosis. Lancet 1998;351(9100):420
   PubMed Web of Science ®Google Scholar
• Wolter J, Seeney S, Bell S, et al. Effect of long term treatment with azithromycin on disease parameters in cystic fibrosis: a randomised trial. Thorax 2002;57(3):212-16
   PubMed Web of Science ®Google Scholar
• Saiman L, Mayer-Hamblett N, Campbell P, et al. Heterogeneity of treatment response to azithromycin in patients with cystic fibrosis. Am J Respir Crit Care Med 2005;172(8):1008-12
   PubMed Web of Science ®Google Scholar
• Saiman L, Anstead M, Mayer-Hamblett N, et al. Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA 2010;303(17):1707-15
   PubMed Web of Science ®Google Scholar
• Southern KW, Barker PM, Solis-Moya A, et al. Macrolide antibiotics for cystic fibrosis. Cochrane Database Syst Rev 2011(12):CD002203
   Google Scholar
• McCormack J, Bell S, Senini S, et al. Daily versus weekly azithromycin in cystic fibrosis patients. Eur Respir J 2007;30(3):487-95
   PubMed Web of Science ®Google Scholar
• Wallace MR, Miller LK, Nguyen MT, et al. Ototoxicity with azithromycin. Lancet 1994;343(8891):241
   PubMed Web of Science ®Google Scholar
• Renna M, Schaffner C, Brown K, et al. Azithromycin blocks autophagy and may predispose cystic fibrosis patients to mycobacterial infection. J Clin Invest 2011;121(9):3554-63
   PubMed Web of Science ®Google Scholar
• Svanstrom H, Pasternak B, Hviid A. Use of azithromycin and death from cardiovascular causes. N Engl J Med 2013;368(18):1704-12
   PubMed Web of Science ®Google Scholar
• De Boeck K, De Baets F, Malfroot A, et al. Do inhaled corticosteroids impair long-term growth in prepubertal cystic fibrosis patients? Eur J Pediatr 2007;166(1):23-8
   PubMed Web of Science ®Google Scholar
• Balfour-Lynn IM, Welch K. Inhaled corticosteroids for cystic fibrosis. Cochrane Database Syst Rev 2012;11:CD001915
   PubMedGoogle Scholar
• Schiotz PO, Jorgensen M, Flensborg EW, et al. Chronic Pseudomonas aeruginosa lung infection in cystic fibrosis. A longitudinal study of immune complex activity and inflammatory response in sputum sol-phase of cystic fibrosis patients with chronic Pseudomonas aeruginosa lung infections: influence of local steroid treatment. Acta Paediatr Scand 1983;72(2):283-7
   PubMed Web of Science ®Google Scholar
• Koller DY, Nilsson M, Enander I, et al. Serum eosinophil cationic protein, eosinophil protein X and eosinophil peroxidase in relation to pulmonary function in cystic fibrosis. Clin Exp Allergy 1998;28(2):241-8
   PubMed Web of Science ®Google Scholar
• Stelmach I, Korzeniewska A, Stelmach W, et al. Effects of montelukast treatment on clinical and inflammatory variables in patients with cystic fibrosis. Ann Allergy Asthma Immunol 2005;95(4):372-80
   PubMed Web of Science ®Google Scholar
• Rose MC, Voynow JA. Respiratory tract mucin genes and mucin glycoproteins in health and disease. Physiol Rev 2006;86(1):245-78
   PubMed Web of Science ®Google Scholar
• Henke MO, Renner A, Huber RM, et al. MUC5AC and MUC5B Mucins Are Decreased in Cystic Fibrosis Airway Secretions. Am J Respir Cell Mol Biol 2004;31(1):86-91
   PubMed Web of Science ®Google Scholar
• Kaplan JB, LoVetri K, Cardona ST, et al. Recombinant human DNase I decreases biofilm and increases antimicrobial susceptibility in staphylococci. J Antibiot (Tokyo) 2012;65(2):73-7
   PubMed Web of Science ®Google Scholar
• Paul K, Rietschel E, Ballmann M, et al. Effect of treatment with dornase alpha on airway inflammation in patients with cystic fibrosis. Am J Respir Crit Care Med 2004;169(6):719-25
   PubMed Web of Science ®Google Scholar
• Jones AP, Wallis C. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev 2010(3):CD001127
   Google Scholar
• Konstan MW, Wagener JS, Pasta DJ, et al. Clinical use of dornase alpha is associated with a slower rate of FEV1 decline in cystic fibrosis. Pediatr Pulmonol 2011;46(6):545-53
   PubMed Web of Science ®Google Scholar
• Sawicki GS, Signorovitch JE, Zhang J, et al. Reduced mortality in cystic fibrosis patients treated with tobramycin inhalation solution. Pediatr Pulmonol 2012;47(1):44-52
   PubMed Web of Science ®Google Scholar
• Wagener JS, Kupfer O. Dornase alfa (Pulmozyme). Curr Opin Pulm Med 2012;18(6):609-14
   PubMed Web of Science ®Google Scholar
• Robinson M, Regnis JA, Bailey DL, et al. Effect of hypertonic saline, amiloride, and cough on mucociliary clearance in patients with cystic fibrosis. Am J Respir Crit Care Med 1996;153(5):1503-9
   PubMed Web of Science ®Google Scholar
• Reeves EP, Molloy K, Pohl K, et al. Hypertonic saline in treatment of pulmonary disease in cystic fibrosis. ScientificWorldJournal 2012;2012:465230
   PubMed Web of Science ®Google Scholar
• Elkins MR, Robinson M, Rose BR, et al. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med 2006;354(3):229-40
   PubMed Web of Science ®Google Scholar
• Dmello D, Nayak RP, Matuschak GM. Stratified assessment of the role of inhaled hypertonic saline in reducing cystic fibrosis pulmonary exacerbations: a retrospective analysis. BMJ Open 2011;1(1):e000019
   Web of Science ®Google Scholar
• Suri R, Metcalfe C, Lees B, et al. Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet 2001;358(9290):1316-21
   PubMed Web of Science ®Google Scholar
• Ratjen F. Restoring airway surface liquid in cystic fibrosis. N Engl J Med 2006;354(3):291-3
   PubMed Web of Science ®Google Scholar
• Furnari ML, Termini L, Traverso G, et al. Nebulized hypertonic saline containing hyaluronic acid improves tolerability in patients with cystic fibrosis and lung disease compared with nebulized hypertonic saline alone: a prospective, randomized, double-blind, controlled study. Ther Adv Respir Dis 2012;6(6):315-22
   PubMedGoogle Scholar
• Robinson M, Daviskas E, Eberl S, et al. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J 1999;14(3):678-85
   PubMed Web of Science ®Google Scholar
• Yaghi A, Zaman A, Dolovich MB. The direct effect of hyperosmolar agents on ciliary beating of human bronchial epithelial cells. J Aerosol Med Pulm Drug Deliv 2012;25(2):88-95
   PubMed Web of Science ®Google Scholar
• Bilton D, Robinson P, Cooper P, et al. Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study. Eur Respir J 2011;38(5):1071-80
   PubMed Web of Science ®Google Scholar
• Aitken ML, Bellon G, De Boeck K, et al. Long-term inhaled dry powder mannitol in cystic fibrosis: an international randomized study. Am J Respir Crit Care Med 2012;185(6):645-52
   PubMed Web of Science ®Google Scholar
• Burness CB and Keating GM. Mannitol dry powder for inhalation: in patients with cystic fibrosis. Drugs 2012;72(10):1411-21
   PubMed Web of Science ®Google Scholar
• Walkowiak J, Sands D, Nowakowska A, et al. Early decline of pancreatic function in cystic fibrosis patients with class 1 or 2 CFTR mutations. J Pediatr Gastroenterol Nutr 2005;40(2):199-201
   PubMed Web of Science ®Google Scholar
• Baker SS, Borowitz D, Baker RD. Pancreatic exocrine function in patients with cystic fibrosis. Curr Gastroenterol Rep 2005;7(3):227-33
   PubMedGoogle Scholar
• Stallings VA, Stark LJ, Robinson KA, et al. Evidence-based practice recommendations for nutrition-related management of children and adults with cystic fibrosis and pancreatic insufficiency: results of a systematic review. J Am Diet Assoc 2008;108(5):832-9
   PubMed Web of Science ®Google Scholar
• Trapnell BC, Strausbaugh SD, Woo MS, et al. Efficacy and safety of. PANCREAZE(R) for treatment of exocrine pancreatic insufficiency due to cystic fibrosis. J Cyst Fibros 2011;10(5):350-6
   PubMed Web of Science ®Google Scholar
• Halfhide C, Evans HJ, Couriel J. Inhaled bronchodilators for cystic fibrosis. Cochrane Database Syst Rev 2005(4):CD003428
   Google Scholar
• Deeks ED. Ivacaftor: a review of its use in patients with cystic fibrosis. Drugs 2013;73(14):1595-604
   PubMed Web of Science ®Google Scholar
• Pettit RS. Cystic fibrosis transmembrane conductance regulator-modifying medications: the future of cystic fibrosis treatment. Ann Pharmacother 2012;46(7-8):1065-75
   PubMed Web of Science ®Google Scholar
• Rogan MP, Stoltz DA, Hornick DB. Cystic fibrosis transmembrane conductance regulator intracellular processing, trafficking, and opportunities for mutation-specific treatment. Chest 2011;139(6):1480-90
   PubMed Web of Science ®Google Scholar
• Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011;365(18):1663-72
   PubMed Web of Science ®Google Scholar
• Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med 2013;187(11):1219-25
   PubMed Web of Science ®Google Scholar
• Durmowicz AG, Witzmann KA, Rosebraugh CJ, et al. Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: the ivacaftor experience. Chest 2013;143(1):14-18
   PubMed Web of Science ®Google Scholar
• Riordan JR, Rommens JM, Kerem B, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 1989;245(4922):1066-73
   PubMed Web of Science ®Google Scholar
• Griesenbach U, Inoue M, Meng C, et al. Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy. Am J Respir Crit Care Med 2012;186(9):846-56
   PubMed Web of Science ®Google Scholar
• Paracchini V, Carbone A, Colombo F, et al. Amniotic mesenchymal stem cells: a new source for hepatocyte-like cells and induction of CFTR expression by coculture with cystic fibrosis airway epithelial cells. J Biomed Biotechnol 2012;2012:575471
   PubMed Web of Science ®Google Scholar
• Ong T, Ramsey BW. Modifying disease in cystic fibrosis: current and future therapies on the horizon. Curr Opin Pulm Med 2013;19(6):645-51
   PubMed Web of Science ®Google Scholar
• Van Goor F, Hadida S, Grootenhuis PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci USA 2011;108(46):18843-8
   PubMed Web of Science ®Google Scholar
• Kerem E, Konstan MW, De Boeck K, et al. Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial. Lancet Respir Med 2014;2(7):539-47
   PubMed Web of Science ®Google Scholar