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Expert Opinion on Pharmacotherapy Volume 10, 2009 - Issue 7
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Reviews

Treatment strategies for cystic fibrosis: what's in the pipeline?

Carley Frerichs Academic Clinical Fellow, Queens Medical Centre, Division of Human Development, Nottingham NG7 2UH, UK
, MBChB, BSc, Med Sci &
Alan Smyth Reader in Child Health and Honorary Consultant in Paediatric Respiratory Medicine, Division of Child Health, E Floor East Block, Queens Medical Centre, Nottingham NG7 2UH, UK +44 115 823 1703; +44 115 823 1946; [email protected]
, MA, MBBS, MRCP, MD, FRCPCH
Pages 1191-1202 | Published online: 30 Apr 2009

Bibliography

  • Dodge JA, Lewis PA, Stanton M, Wilsher J. Cystic fibrosis mortality and survival in the UK: 1947-2003. Eur Respir J 2007;29:522-6
  • Bell SC, Robinson PJ. Exacerbations in cystic fibrosis: 2. Prevention. Thorax 2007;62:723-32
  • Proesmans M, Vermeulen F, De Boeck K. What's new in cystic fibrosis? From treating symptoms to correction of the basic defect. Eur J Pediatr 2008;167:839-49
  • Davies JC, Alton EW, Bush A. Cystic fibrosis. BMJ 2007;335:1255-9
  • Doull IJM. Recent advances in cystic fibrosis. Arch Dis Child 2001;85:62-6
  • Goss CH, Burns JL. Exacerbations in cystic fibrosis. 1: epidemiology and pathogenesis. Thorax 2007;62:360-7
  • Boucher RC. New concepts of the pathogenesis of cystic fibrosis lung disease. Eur Respir J 2004;23:146-58
  • Armstrong DS, Grimwood K, Carzino R, et al. Lower respiratory infection and inflammation in infants with newly diagnosed cystic fibrosis. BMJ 1995;310:1571-2
  • Gibson RL, Burns JL, Ramsey BW. Pathophysiology and management of pulmonary infections in cystic fibrosis. Am J Respir Crit Care Med 2003;168:918-51
  • Emerson J, Rosenfeld M, McNamara S, et al. Pseudomonas aeruginosa and other predictors of mortality and morbidity in young children with cystic fibrosis. Pediatr Pulmonol 2002;34:91-100
  • Al Aloul M, Crawley J, Winstanley C, et al. Increased morbidity associated with chronic infection by an epidemic Pseudomonas aeruginosa strain in CF patients. Thorax 2004;59:334-6
  • Littlewood JM, Wolfe SP, Conway SP. State of the art. Diagnosis and treatment of intestinal malabsorption in cystic fibrosis. Pediatr Pulmonol 2009;41:35-49
  • US CF Foundation. Patient registry. Annual Data Report 2007. Bethesda: Cystic Fibrosis Foundation, 2008
  • Lindblad A, Glaumann H, Strandvik B, et al. Natural history of liver disease in cystic fibrosis. Hepatology 1999;30:1151-8
  • Akata D, Akhan O. Liver manifestations of cystic fibrosis. Eur J Radiol 2007;61:11-7
  • Brennan AL, Geddes DM, Gyi KM, Baker EH. Clinical importance of cystic fibrosis-related diabetes. J Cyst Fibros 2004;3:209-22
  • UK Cystic Fibrosis Trust Bone Mineralisation Working Group. Bone Mineralisation in Cystic Fibrosis. London: UK Cystic Fibrosis Trust, 2007
  • Aris RM, Merkel PA, Bachrach LK, et al. Guide to bone health and disease in cystic fibrosis. J Clin Endocrinol Metab 2005;90:1888-96
  • Frederiksen B, Lanng S, Koch C, Hoiby N. Improved survival in the Danish center-treated cystic fibrosis patients: results of aggressive treatment. Pediatr Pulmonol 1996;21:153-8
  • Smyth A, Walters S. Prophylactic antibiotics for cystic fibrosis. Cochrane Database Syst Rev 2003; Issue 3. Art. No.:CD001912. DOI: 10.1002/14651858.CD001912
  • UK Cystic Fibrosis Trust Antibiotic Group. Antibiotic Treatment for Cystic Fibrosis (3rd edition). London: UK Cystic Fibrosis Trust, 2009
  • Rayner RJ, Hiller EJ, Ispahani P, Baker M. Haemophilus infection in cystic fibrosis. Arch Dis Child 1997;65:255-8
  • Lam J, Chan R, Lam K, Costerton JW. Production of mucoid microcolonies by Pseudomonas aeruginosa within infected lungs in cystic fibrosis. Infect Immun 1980;28:546-56
  • Hansen CR, Pressler T, Hoiby N. Early aggressive eradication therapy for intermittent Pseudomonas aeruginosa airway colonization in cystic fibrosis patients: 15 years experience. J Cyst Fibros 2008;7:523-30
  • Wood DM, Smyth AR. Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis. Cochrane Database Syst Rev 2006; Issue 1. Art. No.:CD004197.pub2. DOI: 10.1002/14651858.CD004197.pub2
  • Steinkamp G, Tummler B, Malottke R, von der Hardt H. Treatment of pseudomonas aeruginosa colonisation in cystic fibrosis. Arch Dis Child 1989;64:1022-8
  • Flume PA, O'Sullivan BP, Robinson KA, et al. Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med 2007;176:957-69
  • Ryan G, Mukhopadhyay S, Singh M. Nebulised anti-pseudomonal antibiotics for cystic fibrosis. Cochrane Database Syst Rev 2003; Issue 3. Art. No.:CD001021. DOI: 10.1002/14651858.CD001021
  • Saiman L, Marshall BC, Mayer-Hamblett N, et al. Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA 2003;290:1749-56
  • Jaffe A, Bush A. Anti-inflammatory effects of macrolides in lung disease. Pediatr Pulmonol 2001;31:464-73
  • Southern KW, Barker PM, Solis-Moya A. Macrolide antibiotics for cystic fibrosis. Cochrane Database Syst Rev 2004; Issue 2. Art. No.:CD002203. DOI: 10.1002/14651858.CD002203.pub2
  • Smyth A, Tan KH, Hyman-Taylor P, et al. Once versus three-times daily regimens of tobramycin treatment for pulmonary exacerbations of cystic fibrosis–the TOPIC study: a randomised controlled trial. Lancet 2005;365:573-8
  • Chan GLC. Alternative dosing strategy for aminoglycosides: impact on efficacy, nephrotoxicity, and ototoxicity. DICP 1989;23:788-94
  • Ramsey BW, Pepe MS, Quan JM, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. N Engl J Med 1999;340:23-30
  • Geller DE, Konstan MW, Smith J, et al. Novel tobramycin inhalation powder in cystic fibrosis subjects: pharmacokinetics and safety. Pediatr Pulmonol 2007;42:307-13
  • 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;178:921-8
  • 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;43:47-58
  • Oermann C, McCoy K, Retsch-Bogart G, et al. Effect of multiple aztreonam lysine for inhalation (AZLI) cycles on disease-related endpoints and safety in patients with cystic fibrosis (CF) and Pseudomonas aeruginosa (PA): Interim analysis of 12 month data. J Cyst Fibros 2008;7(Suppl 2):S25
  • Goldman MH, Pitt T. Lack of emergence of antimicrobial resistance of Pseudommonas aeruginosa after six months of inhalation of dry powder colistimethate. Pediatr Pulmonol 2008;43:331
  • Lee TWR, Matthews DA, Blair GE. Novel molecular approaches to cystic fibrosis gene therapy. Biochem J 2005;387:1-15
  • Available from: http://www.cfgenetherapy.org.uk/
  • Amaral MD, Kunzelmann K. Molecular targeting of CFTR as a therapeutic approach to cystic fibrosis. Trends Pharmacol Sci 2007;28:334-41
  • Kerem E, Hirawat S, Armoni S, et al. Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. Lancet 2008;372:719-27
  • Clancy JP, Konstan MW, Rowe SM, et al. A phase 2 study of PTC124 in CF patients harboring premature stop mutations. Pediatr Pulmonol 2006;41(Suppl 29):291
  • Storey S, Wald G. Novel agents in cystic fibrosis. Nat Rev Drug Discov 2008;7:555-6
  • Dormer RL, Harris CM, Clark Z, et al. Sildenafil (Viagra) corrects DeltaF508-CFTR location in nasal epithelial cells from patients with cystic fibrosis. Thorax 2005;60:55-9
  • Norez C, Antigny F, Noel S, et al. A CF respiratory epithelial cell chronically treated by miglustat acquires a non-CF like phenotype. Am J Respir Cell Mol Biol 2009;2008-0285OC
  • Bye PTP, Elkins MR. Other mucoactive agents for cystic fibrosis. Paediatr Respir Rev 2007;8:30-9
  • Burrows EF, Southern KW. Sodium channel blockers for cystic fibrosis. Cochrane Database Syst Rev 2006; Issue 3. Art. No.:CD005087. DOI: 10.1002/14651858.CD005087.pub2
  • Zhou Z, Treis D, Schubert SC, et al. Preventive but not late amiloride therapy reduces morbidity and mortality of lung disease in beta ENaC-overexpressing mice. Am J Respir Crit Care Med 2008;178:1245-56
  • Oliynyk I, Varelo G, Roomans GM, Johannesson M. Moli1901 (duramycin) increases chloride transport in cystic fibrosis airway epithelial and pancreatic cell lines. J Cyst Fibros 2008;7(Suppl 2):S23
  • Grasemann H, Stehling F, Brunar H, et al. Inhalation of Moli1901 in patients with cystic fibrosis. Chest 2007;131:1461-6
  • Kellerman D, Rossi Mospan A, Engels J, et al. Denufosol: a review of studies with inhaled P2Y2 agonists that led to Phase 3. Pulm Pharmacol Ther 2008;21:600-7
  • Jaques A, Daviskas E, Turton JA, et al. Inhaled mannitol improves lung function in cystic fibrosis. Chest 2008;133:1388-96
  • Pharmaxis. Press release 04 April 2008. Available from: http://www.pharmaxis.com.au/library/2008_04_04_CF203.pdf. Pharmaxis web site [online] 2008
  • Lands LC, Stanojevic S. Oral non-steroidal anti-inflammatory drug therapy for cystic fibrosis. Cochrane Database Syst Rev 2007; Issue 4. Art. No.:CD001505. DOI: 10.1002/14651858.CD001505.pub2
  • Cheng K, Ashby D, Smyth R. Oral steroids for cystic fibrosis. Cochrane Database Syst Rev 1999; Issue 4. Art. No.:CD000407. DOI: 10.1002/14651858.CD000407
  • Balfour-lynn I, Welch K. Inhaled corticosteroids for cystic fibrosis. Cochrane Database Syst Rev 2009; Issue 1. Art. No.:CD001915. DOI: 10.1002/14651858.CD001915.pub2
  • Balfour-Lynn IM, Lees B, Hall P, et al. Multicenter randomized controlled trial of withdrawal of inhaled corticosteroids in cystic fibrosis. Am J Respir Crit Care Med 2006;173:1356-62
  • Graff GR, Weber A, Wessler-Starman D, Smith AL. Montelukast pharmacokinetics in cystic fibrosis. J Pediatr 2003;142:53-6
  • Schmitt-Grohe S, Eickmeier O, Schubert R, et al. Anti-inflammatory effects of montelukast in mild cystic fibrosis. Ann Allergy Asthma Immunol 2002;89:599-605
  • Stelmach I, Korzeniewska A, Smejda K, et al. Effect of montelukast on lung function and clinical symptoms in patients with cystic fibrosis. Pneumonol Alergol Pol 2004;72:85-9
  • Jones AP, Wallis C, Kearney CE. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev 2003; Issue 3. Art. No.:CD001127. DOI: 10.1002/14651858.CD001127. (Updated 2006)
  • Johnson CA, Butler SM, Konstan MW, et al. Estimating effectiveness in an observational study: a case study of dornase alfa in cystic fibrosis. The Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. J Pediatr 1999;134:734-9
  • Quan J, Tiddens HAWM, Sy JP, et al. A two-year randomised placebo controlled trial of dornase alfa in young patients with cystic fibrosis with mild lung function abnormalities. J Pediatr 2001;139:813-20
  • Berge MT, Wiel E, Tiddens HA, et al. DNase in stable cystic fibrosis infants: a pilot study. J Cyst Fibros 2003;2:183-8
  • Wark P, McDonald VM, Jones AP. Nebulised hypertonic saline for cystic fibrosis. Cochrane Database Syst Rev 2005; Issue 3. Art. No.:CD001506. DOI: 10.1002/14651858.CD001506.pub2
  • 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;354:229-40
  • Donaldson SH, Bennett WD, Zeman KL, et al. Mucus clearance and lung function in cystic fibrosis with hypertonic saline. N Engl J Med 2006;354:241-50
  • Smyth RL, Ashby D, O'Hea U, et al. Fibrosing colonopathy in cystic fibrosis: results of a case-control study. Lancet 1995;346:1247-51
  • Borowitz D, Goss CH, Limauro S, et al. Study of a novel pancreatic enzyme replacement therapy in pancreatic insufficient subjects with cystic fibrosis. J Pediatr 2006;149:658-62
  • Lenoir G, Dubray C, Hubert D, et al. Phase II study in young CF adults with the recombinant acid lipase MERISPASE®. J Cyst Fibros 2008;7(Suppl 2):S29
  • Cheng K, Ashby D, Smyth R. Ursodeoxycholic acid for cystic fibrosis-related liver disease. Cochrane Database Syst Rev 1999; Issue 3. Art. No.:CD000222. DOI: 10.1002/14651858.CD000222
  • UK Cystic Fibrosis Trust Diabetes Working Group. Management of Cystic Fibrosis related diabetes mellitus. London: UK Cystic Fibrosis Trust, 2004
  • Onady GM, Stolfi A. Insulin and oral agents for managing cystic fibrosis-related diabetes. Cochrane Database Syst Rev 2005; Issue 3. Art. No.:CD004730. DOI: 10.1002/14651858.CD004730.pub2
  • Moran A, Phillips J, Milla C. Insulin and glucose excursion following premeal insulin lispro or repaglinide in cystic fibrosis-related diabetes. Diabetes Care 2001;24:1706-10
  • Aris RM, Lester GE, Caminiti M, et al. Efficacy of alendronate in adults with cystic fibrosis with low bone density. Am J Respir Crit Care Med 2004;169:77-82
  • Hyde SC, Gill DR. Ignoring the nonsense: a phase II trial in cystic fibrosis. Lancet 2008;372:691-2

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