Emerging Treatments in Cystic Fibrosis (original) (raw)
Boucher RC. New concepts of the pathogenesis of cystic fibrosis lung disease. Eur Respir J 2004; 23(1): 146–58 ArticlePubMedCAS Google Scholar
Que C, Cullinan P, Geddes D. Improving rate of decline of FEV1 in young adults with cystic fibrosis. Thorax 2006; 61(2): 155–7 ArticlePubMedCAS Google Scholar
Farrell PM, Lai HJ, Li Z, et al. Evidence on improved outcomes with early diagnosis of cystic fibrosis through neonatal screening: enough is enough! J Pediatr 2005; 147 (3 Suppl.): S30–6 ArticlePubMed Google Scholar
Dodge JA, Lewis PA, Stanton M, et al. Cystic fibrosis mortality and survival in the UK: 1947-2003. Eur Respir J 2007; 29(3): 522–6 ArticlePubMedCAS Google Scholar
Doring G, Meisner C, Stern M. A double-blind randomized placebo-controlled phase III study of a Pseudomonas aeruginosa flagella vaccine in cystic fibrosis patients. Proc Natl Acad Sci U S A 2007; 104(26): 11020–5 ArticlePubMed Google Scholar
Johansen HK, Gotzsche PC. Vaccines for preventing infection with Pseudomonas aeruginosa in cystic fibrosis. Cochrane Database Syst Rev 2008; (4): CD001399
Zuercher AW, Horn MP, Que JU, et al. Antibody responses induced by long-term vaccination with an octovalent conjugate Pseudomonas aeruginosa vaccine in children with cystic fibrosis. FEMS Immunol Med Microbiol 2006; 47(2): 302–8 ArticlePubMedCAS Google Scholar
Doring G, Conway SP, Heijerman HG, et al. Antibiotic therapy against Pseudomonas aeruginosa in cystic fibrosis: a European consensus. Eur Respir J 2000; 16(4): 749–67 ArticlePubMedCAS Google Scholar
Lenoir G, Antypkin YG, Miano A, et al. Efficacy, safety, and local pharmacokinetics of highly concentrated nebulized tobramycin in patients with cystic fibrosis colonized with Pseudomonas aeruginosa. Paediatr Drugs 2007; 9 Suppl. 1: 11–20 ArticlePubMed Google Scholar
Ramsey BW, Pepe MS, Quan JM, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. N Engl J Med 1999; 340(1): 23–30 ArticlePubMedCAS Google Scholar
Dupont LJ, Minic P, Fustik S, et al. A randomized placebo-controlled study of nebulized liposomal amikacin (Arikace™) in the treatment of cystic fibrosis patients with chronic Pseudomonas aeruginosa lung infection [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23-25; Orlando (FL)
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 ArticlePubMedCAS Google Scholar
Oermann CM, McCoy KS, Retsch-Bogart GZ, et al. Effect of multiple aztreonam lysine for inhalation cycles on disease-related endpoints and safety in patients with cystic fibrosis and Pseudomonas aeruginosa: interim analysis of 12 month data [abstract]. 31st European Cystic Fibrosis Conference; 2008 Jun 11–14; Prague
Geller DE, Flume P, Schwab R, et al. A phase 1 safety, tolerability and pharmacokinetic (PK) study of MP-376 (levofloxacin solution for inhalation) in stable cystic fibrosis patients [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23-25; Orlando (FL)
Bruinenbeg P, Otulana B, Blanchard J, et al. The effect of once-a-day inhaled liposomal ciprofloxacin hydrochloride on sputum bacterial density in cystic fibrosis patients with chronic pulmonary P. aeruginosa colonization [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23-25; Orlando (FL)
Stass H, Ludwig M, Nagelschmitz J, et al. Safety and pharmacokinetics of inhaled dry powder ciprofloxacin after single and multiple inhalations in patients with cystic fibrosis [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23-25; Orlando (FL)
Wilson J, Moorehead L, Montgomery B. A phase 1 open label trial to assess the safety and tolerability of fosfomycin/tobramycin for inhalation (FTI) in subjects with cystic fibrosis or bronchiectasis [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23–25; Orlando (FL)
Milla C, Chmiel J, McCoy KS, et al. A phase 1/2 randomized, double-blind, single dose, dose escalation study of KB001 in cystic fibrosis patients infected with Pseudomonas aeruginosa [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23-25; Orlando (FL)
Equi A, Balfour-Lynn IM, Bush A, et al. Long term azithromycin in children with cystic fibrosis: a randomised, placebo-controlled crossover trial. Lancet 2002; 360(9338): 978–84 ArticlePubMedCAS Google Scholar
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(13): 1749–56 ArticlePubMedCAS 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): 212–6 ArticlePubMedCAS Google Scholar
Lai HC, FitzSimmons SC, Allen DB, et al. Risk of persistent growth impairment after alternate-day prednisone treatment in children with cystic fibrosis. N Engl J Med 2000; 342(12): 851–9 ArticlePubMedCAS Google Scholar
Balfour-Lynn IM, Lees B, Hall P, et al. Multicenter randomized controlled trial of withdrawal of inhaled cortico-steroids in cystic fibrosis. Am J Respir Crit Care Med 2006; 173(12): 1356–62 ArticlePubMedCAS Google Scholar
Konstan MW, Schluchter MD, Xue W, et al. Clinical use of ibuprofen is associated with slower FEV1 decline in children with cystic fibrosis. Am J Respir Crit Care Med 2007; 176(11): 1084–9 ArticlePubMed Google Scholar
Lands LC, Milner R, Cantin AM, et al. High-dose ibuprofen in cystic fibrosis: Canadian safety and effectiveness trial. J Pediatr 2007; 151(3): 249–54 ArticlePubMedCAS Google Scholar
Konstan MW, Hilliard KA, Bucur C, et al. Effect of proglitazone on sputum markers of inflammation in cystic fibrosis [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23-25; Orlando (FL)
Martin SL, Downey D, Bilton D, et al. Safety and efficacy of recombinant alpha(1)-antitrypsin therapy in cystic fibrosis. Pediatr Pulmonol 2006; 41(2): 177–83 ArticlePubMed Google Scholar
Williams B, Robinette M, Slovis B, et al. Hydroxy-chloroquine-pilot study of anti-inflammatory effects in cystic fibrosis [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23-25; Orlando (FL)
Konstan MW, Doring G, Lands LC, et al. Results of a phase II clinical trial of BIIL 284 BS (an LTB4 receptor antagonist) for the treatment of CF lung disease [abstract]. 19th North American Cystic Fibrosis Foundation Conference; 2005 Oct 15–17; Baltimore (MD)
Southern KW, Barker PM, Solis A. Macrolide antibiotics for cystic fibrosis. Cochrane Database Syst Rev 2004; (2): CD002203
Fuchs HJ, Borowitz DS, Christiansen DH, 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(10): 637–42 ArticlePubMedCAS Google Scholar
Hodson ME, McKenzie S, Harms HK, et al. Dornase alfa in the treatment of cystic fibrosis in Europe: a report from the Epidemiologic Registry of Cystic Fibrosis. Pediatr Pulmonol 2003; 36(5): 427–32 ArticlePubMedCAS Google Scholar
Davies J, Trindade MT, Wallis C, et al. Retrospective review of the effects of rhDNase in children with cystic fibrosis. Pediatr Pulmonol 1997; 23(4): 243–8 ArticlePubMedCAS Google Scholar
Nash EF, Stephenson A, Ratjen F, et al. Nebulized and oral thiol derivatives for pulmonary disease in cystic fibrosis. Cochrane Database Syst Rev 2009; (1): CD007168
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(3): 241–50 ArticlePubMedCAS 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 ArticlePubMedCAS Google Scholar
Jaques A, Daviskas E, Turton JA, et al. Inhaled mannitol improves lung function in cystic fibrosis. Chest 2008; 133(6): 1388–96 ArticlePubMedCAS Google Scholar
Minasian C, Wallis C, Metcalfe C, et al. Bronchial provocation testing with dry powder mannitol in children with cystic fibrosis. Pediatr Pulmonol 2008; 43(11): 1078–84 ArticlePubMed Google Scholar
Bilton D, Robinson P, Cooper P. Randomised, double blind, placebo-controlled study of Bronchitol (inhaled dry powder mannitol) in cystic fibrosis [abstract]. 32nd European Cystic Fibrosis Conference; 2009 Jun 10-13; Brest
Zeitlin PL, Boyle MP, Guggino WB, et al. A phase I trial of intranasal Moli1901 for cystic fibrosis. Chest 2004; 125(1): 143–9 ArticlePubMedCAS Google Scholar
Grasemann H, Stehling F, Brunar H, et al. Inhalation of Moli1901 in patients with cystic fibrosis. Chest 2007; 131(5): 1461–6 ArticlePubMedCAS Google Scholar
Deterding RR, Lavange LM, Engels JM, et al. Phase 2 randomized safety and efficacy trial of nebulized denufosol tetrasodium in cystic fibrosis. Am J Respir Crit Care Med 2007; 176(4): 362–9 ArticlePubMedCAS Google Scholar
Inspire Pharmaceuticals. Denufosol tetrasodium inhalation solution for cystic fibrosis: phase 3 press release; [online]. Available from URL: http://www.inspirepharm.com/r_and_d/denufosol.php [Accessed 2009 Aug 10]
Gilead Sciences. Trial to assess the safety, tolerability and pharmacokinetics of GS-9411 in healthy male volunteers [ClinicalTrials.gov identifier NCT00800579]. US National Institutes of Health, ClinicalTrials.gov [online]. Available from URL: http://www.clinicaltrials.gov [Accessed 2009 Aug 10]
Sucampo Pharmaceuticals Inc. Sucampo Pharmaceuticals initiates phase IIa safety and efficacy trial for proprietary agent SPI-8811 in treating cystic fibrosis [online]. Available from URL: http://www.sucampo.com/article_34.shtml [Accessed 2009 Aug 10]
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(9640): 719–27 ArticlePubMedCAS Google Scholar
Accurso FJ, Rowe SM, Durie PR, et al. Interim analysis of phase 2a study of VX-770 to evaluate safety, pharmacokinetics, and biomarkers of CFTR activity in cystic fibrosis subjects with G551D [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23–25; Orlando (FL)
Alton EW, Stern M, Farley R, et al. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet 1999; 353(9157): 947–54 ArticlePubMedCAS Google Scholar
Knowles MR, Hohneker KW, Zhou Z, et al. A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N Engl J Med 1995; 333(13): 823–31 ArticlePubMedCAS Google Scholar
Pringle IA, Hyde SC, Gill DR. Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects. Expert Opin Biol Ther 2009; 9(8): 991–1003 ArticlePubMedCAS Google Scholar
Lenoir G, Dubray C, Chiron R, et al. Recombinant gastric lipase for the treatment of fat malabsorption [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23–25; Orlando (FL)
Borowitz D, Falzone RP, Fratazzi C. A phase 3 study of the efficacy and safety of ALTU-135 (Trizytek) for treatment of pancreatic insufficiency in CF [abstract]. 22nd Annual North American Cystic Fibrosis Conference; 2008 Oct 23–25; Orlando (FL)
Kerem E, Reisman J, Corey M, et al. Prediction of mortality in patients with cystic fibrosis. N Engl J Med 1992; 326(18): 1187–91 ArticlePubMedCAS Google Scholar
Gustafsson PM, De Jong PA, Tiddens HA, et al. Multiple-breath inert gas washout and spirometry versus structural lung disease in cystic fibrosis. Thorax 2008; 63(2): 129–34 ArticlePubMedCAS Google Scholar
Lum S, Gustafsson P, Ljungberg H, et al. Early detection of cystic fibrosis lung disease: multiple-breath washout versus raised volume tests. Thorax 2007; 62(4): 341–7 ArticlePubMed Google Scholar
Brody AS, Tiddens HA, Castile RG, et al. Computed tomography in the evaluation of cystic fibrosis lung disease. Am J Respir Crit Care Med 2005; 172(10): 1246–52 ArticlePubMed Google Scholar
Doring G, Elborn JS, Johannesson M, et al. Clinical trials in cystic fibrosis. J Cyst Fibros 2007; 6(2): 85–99 ArticlePubMed Google Scholar
Bartholdson SJ, Brown AR, Mewburn BR, et al. Plant host and sugar alcohol induced exopolysaccharide biosynthesis in the Burkholderia cepacia complex. Microbiology 2008; 154(8): 2513–21 ArticlePubMedCAS Google Scholar