메뉴 건너뛰기




Volumn 14, Issue 3, 2013, Pages 323-329

From pipeline to patient: New developments in cystic fibrosis therapeutics

Author keywords

Aerosolised antibiotics; CFTR modulation; Cystic fibrosis; New therapies

Indexed keywords

AMIKACIN; ATALUREN; AZITHROMYCIN; AZTREONAM; CHLORIDE CHANNEL STIMULATING AGENT; CIPROFLOXACIN; COLISTIMETHATE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DENUSOFOL; FOSFOMYCIN; GROWTH HORMONE; INSULIN; ISOPRENALINE; IVACAFTOR; LEVOFLOXACIN; LIPROTAMASE; LUMACAFTOR; MANNITOL; MIGLUSTAT; PLACEBO; RISEDRONIC ACID; TOBRAMYCIN; UNCLASSIFIED DRUG;

EID: 84873398616     PISSN: 14656566     EISSN: 17447666     Source Type: Journal    
DOI: 10.1517/14656566.2013.769958     Document Type: Review
Times cited : (3)

References (37)
  • 1
    • 65849522497 scopus 로고    scopus 로고
    • Cystic fibrosis
    • O'Sullivan BP, Freedman SD. Cystic fibrosis. Lancet 2009;373(9678):1891- 904
    • (2009) Lancet , vol.373 , Issue.9678 , pp. 1891-1904
    • O'Sullivan, B.P.1    Freedman, S.D.2
  • 3
    • 33847711906 scopus 로고    scopus 로고
    • Cystic fibrosis mortality and survival in the UK: 1947-2003
    • DOI 10.1183/09031936.00099506
    • 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 (Pubitemid 46372100)
    • (2007) European Respiratory Journal , vol.29 , Issue.3 , pp. 522-526
    • Dodge, J.A.1    Lewis, P.A.2    Stanton, M.3    Wilsher, J.4
  • 4
    • 67649550403 scopus 로고    scopus 로고
    • Treatment strategies for cystic fibrosis: What's in the pipelinë
    • Frerichs C, Smyth A. Treatment strategies for cystic fibrosis: What's in the pipelinë Expert Opin Pharmacother 2009;10(7):1191-202
    • (2009) Expert Opin Pharmacother , vol.10 , Issue.7 , pp. 1191-1202
    • Frerichs, C.1    Smyth, A.2
  • 5
    • 84863476402 scopus 로고    scopus 로고
    • Reduced airway surface ph impairs bacterial killing in the porcine cystic fibrosis lung
    • Pezzulo AA, Tang XX, Hoegger MJ, et al. Reduced airway surface pH impairs bacterial killing in the porcine cystic fibrosis lung. Nature 2012;487(7405):109-13
    • (2012) Nature , vol.487 , Issue.7405 , pp. 109-113
    • Pezzulo, A.A.1    Tang, X.X.2    Hoegger, M.J.3
  • 6
    • 34548278908 scopus 로고    scopus 로고
    • Power considerations for studies of lung function in cystic fibrosis
    • DOI 10.1513/pats.200611-176HT
    • Corey M. Power considerations for studies of lung function in cystic fibrosis. Proc Am Thorac Soc 2007;4(4):334-7 (Pubitemid 47330866)
    • (2007) Proceedings of the American Thoracic Society , vol.4 , Issue.4 , pp. 334-337
    • Corey, M.1
  • 8
    • 84871981271 scopus 로고    scopus 로고
    • Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: The ivacaftor experience
    • 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
    • (2013) Chest , vol.143 , Issue.1 , pp. 14-18
    • Durmowicz, A.G.1    Witzmann, K.A.2    Rosebraugh, C.J.3
  • 9
    • 84862776813 scopus 로고    scopus 로고
    • Progression of early structural lung disease in young children with cystic fibrosis assessed using ct
    • Mott LS, Park J, Murray CP, et al. Progression of early structural lung disease in young children with cystic fibrosis assessed using CT. Thorax 2012;67(6):509-16
    • (2012) Thorax , vol.67 , Issue.6 , pp. 509-516
    • Mott, L.S.1    Park, J.2    Murray, C.P.3
  • 11
    • 84862023311 scopus 로고    scopus 로고
    • Radiologic imaging in cystic fibrosis: Cumulative effective dose and changing trends over 2 decades
    • O'Connell OJ, McWilliams S, McGarrigle A, et al. Radiologic imaging in cystic fibrosis: Cumulative effective dose and changing trends over 2 decades. Chest 2012;141(6):1575-83
    • (2012) Chest , vol.141 , Issue.6 , pp. 1575-1583
    • O'Connell, O.J.1    McWilliams, S.2    McGarrigle, A.3
  • 12
    • 80955131827 scopus 로고    scopus 로고
    • Lung clearance index: Clinical and research applications in children
    • Fuchs SI, GappaM. Lung clearance index: Clinical and research applications in children. Paediatr Respir Rev 2011;12(4):264-70
    • (2011) Paediatr Respir Rev , vol.12 , Issue.4 , pp. 264-270
    • Fuchs, S.I.1    Gappa, M.2
  • 13
    • 79952504576 scopus 로고    scopus 로고
    • Lung clearance index at 4 years predicts subsequent lung function in children with cystic fibrosis
    • Aurora P, Stanojevic S, Wade A, et al. Lung clearance index at 4 years predicts subsequent lung function in children with cystic fibrosis. Am J Respir Crit Care Med 2011;183(6):752-8
    • (2011) Am J Respir Crit Care Med , vol.183 , Issue.6 , pp. 752-758
    • Aurora, P.1    Stanojevic, S.2    Wade, A.3
  • 14
    • 78651412529 scopus 로고    scopus 로고
    • Safety, efficacy and convenience of tobramycin inhalation powder in cystic fibrosis patients: The EAGER trial
    • 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
    • (2011) J Cyst Fibros , vol.10 , Issue.1 , pp. 54-61
    • Konstan, M.W.1    Flume, P.A.2    Kappler, M.3
  • 15
    • 84875215401 scopus 로고    scopus 로고
    • Safety efficacy and convenience of colistimethate sodium dry powder for inhalation (colobreathe dpi) in patients with cystic fibrosis: A randomised study
    • doi:10.1136/thoraxjnl-2012-202059
    • 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 2012; doi:10.1136/thoraxjnl-2012-202059
    • (2012) Thorax
    • Schuster, A.1    Haliburn, C.2    Doring, G.3
  • 16
    • 66949178892 scopus 로고    scopus 로고
    • A randomized placebo-Controlled study of nebulized liposomal amikacin (Arikace (TM)) in the treatment of cystic fibrosis patients with chronic Pseudomonas aeruginosa lung infection
    • Dupont L, Minic P, Fustic S, et al. A randomized placebo-Controlled study of nebulized liposomal amikacin (Arikace (TM)) in the treatment of cystic fibrosis patients with chronic Pseudomonas aeruginosa lung infection. J Cystic Fibrosis 2008;7(SUPPL2):S26-6
    • (2008) J Cystic Fibrosis , vol.7 , Issue.SUPPL. 2
    • Dupont, L.1    Minic, P.2    Fustic, S.3
  • 17
    • 70349084570 scopus 로고    scopus 로고
    • Pharmacokinetic and pharmacodynamic evaluation of liposomal amikacin for inhalation in cystic fibrosis patients with chronic pseudomonal infection
    • Okusanya OO, Bhavnani SM, Hammel J, et al. Pharmacokinetic and pharmacodynamic evaluation of liposomal amikacin for inhalation in cystic fibrosis patients with chronic pseudomonal infection. Antimicrob Agents Chemother 2009;53(9):3847-54
    • (2009) Antimicrob Agents Chemother , vol.53 , Issue.9 , pp. 3847-3854
    • Okusanya, O.O.1    Bhavnani, S.M.2    Hammel, J.3
  • 18
    • 79957942626 scopus 로고    scopus 로고
    • Levofloxacin inhalation solution (MP-376) in patients with cystic fibrosis with Pseudomonas aeruginosa
    • Geller DE, Flume PA, Staab D, et al. Levofloxacin inhalation solution (MP-376) in patients with cystic fibrosis with Pseudomonas aeruginosa. Am J Respir Crit Care Med 2011;183(11):1510-16
    • (2011) Am J Respir Crit Care Med , vol.183 , Issue.11 , pp. 1510-1516
    • Geller, D.E.1    Flume, P.A.2    Staab, D.3
  • 19
    • 84856010859 scopus 로고    scopus 로고
    • Fosfomycin/tobramycin for inhalation in patients with cystic fibrosis with pseudomonas airway infection
    • Trapnell BC, McColley SA, Kissner DG, et al. Fosfomycin/tobramycin for inhalation in patients with cystic fibrosis with pseudomonas airway infection. Am J Respir Crit Care Med 2012;185(2):171-8
    • (2012) Am J Respir Crit Care Med , vol.185 , Issue.2 , pp. 171-178
    • Trapnell, B.C.1    McColley, S.A.2    Kissner, D.G.3
  • 20
    • 80051566309 scopus 로고    scopus 로고
    • Exciting new clinical trials in cystic fibrosis: Infants need not apply
    • Stick SM, Sly PD. Exciting new clinical trials in cystic fibrosis: Infants need not apply. Am J Respir Crit Care Med 2011;183(12):1577-8
    • (2011) Am J Respir Crit Care Med , vol.183 , Issue.12 , pp. 1577-1578
    • Stick, S.M.1    Sly, P.D.2
  • 22
    • 80355127141 scopus 로고    scopus 로고
    • Inhaled dry powder mannitol in cystic fibrosis: An efficacy and safety study
    • 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
    • (2011) Eur Respir J , vol.38 , Issue.5 , pp. 1071-1080
    • Bilton, D.1    Robinson, P.2    Cooper, P.3
  • 23
    • 84858234304 scopus 로고    scopus 로고
    • Long-Term inhaled dry powder mannitol in cystic fibrosis: An international randomized study
    • 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
    • (2012) Am J Respir Crit Care Med , vol.185 , Issue.6 , pp. 645-652
    • Aitken, M.L.1    Bellon, G.2    De Boeck, K.3
  • 24
    • 79952211206 scopus 로고    scopus 로고
    • Denufosol tetrasodium in patients with cystic fibrosis and normal to mildly impaired lung function
    • Accurso FJ, Moss RB, Wilmott RW, et al. Denufosol tetrasodium in patients with cystic fibrosis and normal to mildly impaired lung function. Am J Respir Crit Care Med 2011;183(5):627-34
    • (2011) Am J Respir Crit Care Med , vol.183 , Issue.5 , pp. 627-634
    • Accurso, F.J.1    Moss, R.B.2    Wilmott, R.W.3
  • 25
    • 84869090967 scopus 로고    scopus 로고
    • Long term effects of denufosol tetrasodium in patients with cystic fibrosis
    • Ratjen F, Durham T, Navratil T, et al. Long term effects of denufosol tetrasodium in patients with cystic fibrosis. J Cyst Fibros 2012;11(6):539-49
    • (2012) J Cyst Fibros , vol.11 , Issue.6 , pp. 539-549
    • Ratjen, F.1    Durham, T.2    Navratil, T.3
  • 26
    • 79960187095 scopus 로고    scopus 로고
    • Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis
    • Wilschanski M, Miller LL, Shoseyov D, et al. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur Respir J 2011;38(1):59-69
    • (2011) Eur Respir J , vol.38 , Issue.1 , pp. 59-69
    • Wilschanski, M.1    Miller, L.L.2    Shoseyov, D.3
  • 27
    • 84873345288 scopus 로고    scopus 로고
    • Results of the phase 3 study of ataluren in nonsense mutation cystic fibrosis (NMCF) [abstract
    • Rowe S, Sermet-Gaudelus I, Konstan MW, et al. Results of the phase 3 study of ataluren in nonsense mutation cystic fibrosis (NMCF) [abstract]. Pediatric Pulmonology 2012;47(SUPPL35):290
    • (2012) Pediatric Pulmonology , vol.47 , Issue.SUPPL. 35 , pp. 290
    • Rowe, S.1    Sermet-Gaudelus, I.2    Konstan, M.W.3
  • 28
    • 80455162465 scopus 로고    scopus 로고
    • A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
    • 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
    • (2011) N Engl J Med , vol.365 , Issue.18 , pp. 1663-1672
    • Ramsey, B.W.1    Davies, J.2    McElvaney, N.G.3
  • 29
    • 84865853894 scopus 로고    scopus 로고
    • Ivacaftor in subjects with cystic fibrosis who are homozygous for the f508del-Cftr mutation
    • Flume PA, Liou TG, Borowitz DS, et al. Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. Chest 2012;142(3):718-24
    • (2012) Chest , vol.142 , Issue.3 , pp. 718-724
    • Flume, P.A.1    Liou, T.G.2    Borowitz, D.S.3
  • 30
    • 84873362529 scopus 로고    scopus 로고
    • The investigational cftr corrector vx809 (lumacaftor) co-Administered with the oral potentiator ivacaftor improved cftr and lung function in f508del homozygous patients: Phase ii study results
    • Boyle MP, Bell S, Konstan M, et al. The investigational CFTR corrector, VX809 (lumacaftor) co-Administered with the oral potentiator ivacaftor improved CFTR and lung function in F508DEL homozygous patients: Phase II study results. Pediatric Pulmonology 2012 ;47(Supplt 35):315
    • (2012) Pediatric Pulmonology , vol.47 , Issue.SUPPL. 35 , pp. 315
    • Boyle, M.P.1    Bell, S.2    Konstan, M.3
  • 31
    • 33645211759 scopus 로고    scopus 로고
    • Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat
    • Norez C, Noel S, Wilke M, et al. Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat. FEBS Lett 2006;580(8):2081-6
    • (2006) FEBS Lett , vol.580 , Issue.8 , pp. 2081-2086
    • Norez, C.1    Noel, S.2    Wilke, M.3
  • 32
    • 84860605804 scopus 로고    scopus 로고
    • A randomized placebo-Controlled trial of miglustat in cystic fibrosis based on nasal potential difference
    • Leonard A, Lebecque P, Dingemanse J, et al. A randomized placebo-Controlled trial of miglustat in cystic fibrosis based on nasal potential difference. J Cyst Fibros 2012;11(3):231-6
    • (2012) J Cyst Fibros , vol.11 , Issue.3 , pp. 231-236
    • Leonard, A.1    Lebecque, P.2    Dingemanse, J.3
  • 33
    • 84857500402 scopus 로고    scopus 로고
    • Slow-release insulin in cystic fibrosis patients with glucose intolerance: A randomized clinical trial
    • Minicucci L, HauPTM, Casciaro R, et al. Slow-release insulin in cystic fibrosis patients with glucose intolerance: A randomized clinical trial. Pediatr Diabetes 2012;13(2):197-202
    • (2012) Pediatr Diabetes , vol.13 , Issue.2 , pp. 197-202
    • Minicucci, L.1    Hau, P.T.M.2    Casciaro, R.3
  • 34
    • 84857370463 scopus 로고    scopus 로고
    • A multi-Center controlled trial of growth hormone treatment in children with cystic fibrosis
    • Stalvey MS, Anbar RD, Konstan MW, et al. A multi-Center controlled trial of growth hormone treatment in children with cystic fibrosis. Pediatr Pulmonol 2012;47(3):252-63
    • (2012) Pediatr Pulmonol , vol.47 , Issue.3 , pp. 252-263
    • Stalvey, M.S.1    Anbar, R.D.2    Konstan, M.W.3
  • 35
    • 81455158920 scopus 로고    scopus 로고
    • Multicentre trial of weekly risedronate on bone density in adults with cystic fibrosis
    • Haworth CS, Sharples L, Hughes V, et al. Multicentre trial of weekly risedronate on bone density in adults with cystic fibrosis. J Cyst Fibros 2011;10(6):470-6
    • (2011) J Cyst Fibros , vol.10 , Issue.6 , pp. 470-476
    • Haworth, C.S.1    Sharples, L.2    Hughes, V.3
  • 36
    • 84864033253 scopus 로고    scopus 로고
    • Bisphosphonates for osteoporosis in people with cystic fibrosis
    • Conwell LS, Chang AB. Bisphosphonates for osteoporosis in people with cystic fibrosis. Cochrane Database Syst Rev 2012;4:CD002010
    • (2012) Cochrane Database Syst Rev , vol.4
    • Conwell, L.S.1    Chang, A.B.2
  • 37
    • 81455141412 scopus 로고    scopus 로고
    • International phase III trial of liprotamase efficacy and safety in pancreatic-insufficient cystic fibrosis patients
    • Borowitz D, Stevens C, Brettman LR, et al. International phase III trial of liprotamase efficacy and safety in pancreatic-insufficient cystic fibrosis patients. J Cyst Fibros 2011;10(6):443-52
    • (2011) J Cyst Fibros , vol.10 , Issue.6 , pp. 443-452
    • Borowitz, D.1    Stevens, C.2    Brettman, L.R.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.