187
Views
2
CrossRef citations to date
0
Altmetric
Reviews

Improvements in symptomatic treatment strategies for cystic fibrosis: delivering CF care in the 21st century

, &

Bibliography

  • Prayle A, Smyth A. From pipeline to patient: new developments in CF therapeutics. Expert Opin Pharmacother. 2013;14(3):323–329.
  • Conway S, Balfour-Lynn I, De Rijke K, et al. European Cystic Fibrosis Society standards of care: framework for the Cystic Fibrosis Centre. J Cyst Fibros. 2014;13(Suppl 1):S3–S22.
  • Burgel P, Bellis G, Oleson H, et al. Future trends in cystic fibrosis demography in 34 European countries. ERJ. 2015: DOI:10.1183/09031936.00196314.
  • Bell S, Boeck K, Amaral M. New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls. Pharmacol Ther. 2015;145:19–34.
  • The Clinical and Functional Translation of CFTR (CFTR2); 2015. [cited 2015 Apr 20]. Available from: www.CFTR2.com.
  • Schecter MS. Non-genetic influences on cystic fibrosis outcomes. Curr Opin Pulm Med. 2011;17:448–454.
  • Huang Y, Charlson E, Collman R, et al. The role of the lung microbiome in health and disease. A National Heart, Lung, and Blood Institute workshop report. Am J Respir Crit Care Med. 2013;187:1382–1387.
  • Dickson R, Erb-Downward J, Huffnagle G. The role of the bacterial microbiome in chest disease. Expert Rev Respir Med. 2013;7:245–257.
  • Dickson R, Martinez F, Huffnagle G. The role of the microbiome in exacerbations of chronic lung diseases. Lancet. 2014;384:691–702.
  • Kramer R, Sauer-Heilborn A, Welte T, et al. High individuality of respiratory bacterial communities in a large cohort of adult cystic fibrosis patients under continuous antibiotic treatment. PLOS One. 2015;10(2):e0117436.
  • Lynch S, Bruce K. The cystic fibrosis airway microbiome. Cold Spring Harb Perspect Med. 2013;3:a009738.
  • Cox M, Allgaier M, Taylor B, et al. Airway microbiota and pathogen abundance in age-stratified cystic fibrosis patients. Plos One. 2010;5(6):e11044.
  • Carmody L, Zhao J, Schloss P, et al. Changes in cystic fibrosis airway microbiota at pulmonary exacerbation. Ann Am Thorac Soc. 2013;10:179–187.
  • Zemanick E, Harris J, Wagner B, et al. Inflammation and airway microbiota during cystic fibrosis pulmonary exacerbations. Plos One. 2013;8(4):e62917.
  • 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;303:1707–1715.
  • Hurley M, Ariff A, Bertenshaw C, et al. Results of antibiotic sensitivity testing do not influence clinical outcomes in children with cystic fibrosis. J Cyst Fibros. 2012;11:288–292.
  • Cystic fibrosis microbiome-determined antimicrobial therapy trial in exacerbations: results stratified (CF-MATTERS). [Internet]; 2015 [cited 2015 Apr 20]. Available from: http://www.cfmatters.eu/.
  • Schenchang S, Hasset D. Anaerobic Pseudomonas aeruginosa and other obligately anaerobic bacterial biofilms growing in the thick airway mucus of chronically infected cystic fibrosis patients: an emerging paradigm or “old hat”? Expert Opin Ther Targets. 2012;16(9):859–873.
  • Trapnell B, McColley S, Kissner D, et al. Fosfomycin/tobramycin for inhalation in patients with cystic fibrosis with pseudomonas airway infection. Am J Respir Crit Care Med. 2012;185(2):171–178.
  • Waters V, Smyth A. Cystic fibrosis microbiology: advances in antimicrobial therapy. J Cyst Fibros. 2015;14(5):551–560.
  • Montuschi P, Paris D, Melck D, et al. NMR spectroscopy metabolomic profiling of exhaled breath condensate in patients with stable and unstable cystic fibrosis. Thorax. 2012;67:222–228.
  • Tyrrell J, Tarran R. Gaining the upper hand on pulmonary drug delivery. J Pharmacolvigil. 2014;2(1):118–125.
  • Newhoues M, Hirst P, Duddu S, et al. Inhalation of dry powder tobramycin PulmoSphere formulation in healthy volunteers. Chest. 2003;124:360–366.
  • 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.
  • 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):344–350.
  • Clancy J, Dupont L, Konstan M, et al. Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection. Thorax. 2013;68(9):818–825.
  • Hodson ME, Gallagher CG, Govan JR. A randomised clinical trial of nebulised tobramycin or colistin in cystic fibrosis. Eur Respir J. 2002;20(3):658–664.
  • Assael BM, Pressler T, Bilton D, et al. Inhaled aztreonam lysine vs inhaled tobramycin in cystic fibrosis: a comparative efficacy trial. J Cyst Fibros. 2013;12:130–140.
  • Maiz L, Giron R, Olveira C, et al. Inhaled antibiotics for the treatment of chronic bronchopulmonary Pseudomonas aeruginosa infection in cystic fibrosis: systematic review of randomised controlled trials. Expert Opin Pharmacother. 2013;14(9):1135–1149.
  • Fuchs H, Borowitz D, Christiansen D, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N Engl J Med. 1994;331(10):637–642.
  • Wark P, McDonald V. Nebulised hypertonic saline for cystic fibrosis. Cochrane Database Syst Rev. 2009;2:CD001506.
  • Cystic Fibrosis Trust Place. Cystic fibrosis trust UK standards for the clinical care of children and adults with cystic fibrosis in the UK: Second Edition. 2011 [cited 2015 May 14]. Available from: http://cysticfibrosis.org.uk/media/448939/cd-standards-of-care-dec-2011.pdf
  • Castellani C, Conway S, Smyth A, et al. Standards of care for cystic fibrosis ten years later. J Cyst Fibros. 2014;13(Suppl 1):S1–S2.
  • Sawicki G, Tiddens H. Managing treatment complexity in cystic fibrosis: challenges and opportunities. Pediatr Pulmonol. 2012;47:523–533.
  • National Institute for Health and Care Excellence. National Institute for Health and Care Excellence Health Technology appraisal: mannitol dry powder for inhalation for treating cystic fibrosis [Internet]. 2012 [cited 2015 Apr 7]. Available from: https://www.nice.org.uk/guidance/ta266/resources/guidance-mannitol-dry-powder-for-inhalation-for-treating-cystic-fibrosis-pdf
  • Tam J, Tullis E. Inhaled dry powder mannitol in cystic fibrosis. Expert Opin Orphan Drugs. 2014;2(7):751–757.
  • Syed B, Hamad B. The cystic fibrosis drug market. Nat Drug Discov. 2014;14:721–722.
  • Warnock L, Gates A, van der Schans C. Chest physiotherapy compared to no chest physiotherapy for cystic fibrosis. Cochrane Database Syst Rev. 2013;(9). Art. No.: CD001401. DOI:10.1002/14651858.CD001401.pub2.
  • Morrison L, Agnew J. Oscillating devices for airway clearance in people with cystic fibrosis. Cochrane Database Syst Rev. 2014;(7). Art. No.: CD006842. DOI:10.1002/14651858.CD006842.pub3.
  • Sagel S. The challenges of developing effective anti-inflammatory agents in cystic fibrosis. J Cyst Fibros. 2015;14:164–166.
  • Lands L, Stanojevic S. Oral non-steroidal anti-inflammatory drug therapy for lung disease in cystic fibrosis. Cochrane Database Syst Rev. 2013;(6). Art. No.: CD001505. DOI:10.1002/14651858.CD001505.pub3.
  • Sinaasappel M, Stern M, Littlewood J, et al. Nutrition in patients with cystic fibrosis: a European consensus. J Cyst Fibros. 2002;1:67–91.
  • Cystic Fibrosis Australia. Cystic fibrosis in Australia, 13th annual report from the Australian Cystic Fibrosis Data Registry. Cystic Fibrosis Australia [Internet]. 2010. Available from: http://www.cysticfibrosis.org.au/media/wysiwyg/CF-Australia/medical-documents/ACFDR_2010/ACFDR_2010_-_Report_CF_in_Australia.pdf
  • Abbott J, Morton A, Musson H, et al. Nutritional status, perceived body image and eating behaviours in adults with cystic fibrosis. Clin Nutr. 2007;26(1):91–99.
  • White H, Morton A, Conway S, et al. Enteral feeding in adults with cystic fibrosis: patient choice and impact on long term outcomes. J Cyst Fibros. 2013;12(6):616–622.
  • 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;365:1663–1672.
  • Rowe S, Heltshe S, Gonska T, et al. Clinical mechanism of the cystic fibrosis transmembrane regulator potentiator ivacaftor in G551D potentiated cystic fibrosis. AMJCCM. 2014;190(2):175–184.
  • Wainwright C, Elborn J, Ramsay B, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. NEJM. 2015;373:220–231.
  • Li L, Somerset S. The clinical significance of the gut microbiota in cystic fibrosis and the potential for dietary therapies. Clin Nutr. 2014;33:571–580.
  • Del Campo R, Garriga M, Perez-Aragon A, et al. Improvement of digestive health and reduction in proteobacterial populations in the gut microbiota of cystic fibrosis patients using a Lactobacillus reuteri probiotic preparation: a double blind prospective study. J Cyst Fibros. 2014;13:716–722.
  • Bruzzese E, Raia V, Spagnuolo M, et al. Effect of Lactobacillus GG supplementation on pulmonary exacerbations in patients with cystic fibrosis: a pilot study. Clin Nutr. 2007;26:322–3288.
  • Bron PA, Van Baarlen P, Kleerebezem M. Emerging molecular insights into the interaction between probiotics and the host intestinal mucosa. Nat Rev Micro. 2012;10:66–78.
  • Lanng S, Thorsteinsson B, Nerup J, et al. Influence of the development of diabetes mellitus on clinical status in patients with cystic fibrosis. Eur J Pediatr. 1992;151:684–687.
  • Rolon MA, Benali K, Munck A, et al. Cystic fibrosis related diabetes mellitus: clinical impact of prediabetes and effects of insulin therapy. Acta Paediatr. 2001;90:860–867.
  • Waugh N, Royle P, Craigie I, et al. Screening for cystic fibrosis related diabetes: a systematic review. Health Technol Assess. 2012;16:24.
  • Minicuccia L, Haupta M, Casciaroa R, et al. Slow-release insulin in cystic fibrosis patients with glucose intolerance: a randomized clinical trial. Pediatric Diabetes. 2012;13:197–202.
  • Kalra S, Gupta Y. Clinical use of insulin degludec: practical experience and pragmatic suggestions. N Am J Med Sci. 2015;7(3):81–85.
  • Nazareth D, Walshaw M. A review of renal disease in cystic fibrosis. JCF. 2013;12:309–317.
  • Al-Aloul M, Jackson M, Bell G, et al. Comparison of methods of assessment of renal function in cystic fibrosis patients. J Cyst Fibros. 2007;6:41–47.
  • Dobson L, Stride E, Bingham C, et al. Microalbuminuria as a screening tool in cystic fibrosis related diabetes (CFRD). J Cyst Fibros. 2008;7:515–519.
  • Van Den Berg J, Morton A, Kok S, et al. Microvascular complications in patients with cystic fibrosis related diabetes (CFRD). J Cyst Fibros. 2008;7:515–519.
  • Stalvey M, Clines G. Cystic fibrosis related bone disease: insights into a growing problem. Curr Opin Endocrinol Diabetes Obes. 2013;20:547–552.
  • Rovner AJ, Zemel BS, Leonard MB, et al. Mild to moderate cystic fibrosis is not associated with increased fracture risk in children and adolescents. J Pediatr. 2005;147:327–331.
  • Haworth S, Webb A, Egan J, et al. Bone histomorphometry in adult patients with cystic fibrosis. Chest. 2000;118:434–439.
  • Ferguson J, Chang A, Vitamin D Supplementation for cystic fibrosis. Cochrane Database Syst Rev. 2014;5:CD007298. DOI:10.1002/14651858.CD007298.pub4.
  • Conwell L, Chang A. Bisphosphonates for osteoporosis in people with cystic fibrosis. Cochrane Database Syst Rev. 2014;3:CD002010. DOI:10.1002/14651858.CD002010.pub4.
  • Siwamogsatham O, Stephens K, Tangpricha K. Evaluation of teriparatide for treatment of osteoporosis in four patients with cystic fibrosis: a case series. Case Rep Endocrinol. 2014;2014:893589.
  • Törring O. Effects of denosumab on bone density, mass and strength in women with postmenopausal osteoporosis. Ther Adv Musculoskelet Dis. 2015;7(3):88–102.
  • Mueller-Abt P, Frawley K, Greer R, et al. Comparison of ultrasound and biopsy findings in children with cystic fibrosis related liver disease. J Cyst Fibros. 2008;7:215–221.
  • Cheng K, Ashby D, Smyth RL. Ursodeoxycholic acid for cystic fibrosis-related liver disease. Cochrane Database Syst Rev. 2012;(10). Art. No.: CD000222. DOI:10.1002/14651858.CD000222.pub2.
  • Rath T, Hage L, Kugler M, et al. Serum proteome profiling identifies novel and powerful markers of cystic fibrosis liver disease. PLOS One. 2013;8(3):e58955. DOI:10.1371/journal.pone.0058955.
  • Bourke S, Doe S, Gascoigne A, et al. An integrated model of provision of palliative care to patients with cystic fibrosis. Palliative Med. 2009;23:512–517.
  • Goldbeck L, Besier T, Hinz A, et al. Prevalence of symptoms of anxiety and depression in German patients with cystic fibrosis. Chest. 2010;138:929–936.
  • Zemanick ET, Harris JK, Conway S, et al. Measuring and improving respiratory outcomes in cystic fibrosis lung disease: opportunities and challenges to therapy. J Cyst Fibros. 2010;9:1–16.
  • Kraynack N, Gothard M, Falletta L, et al. Approach to treating cystic fibrosis pulmonary exacerbations varies widely across US CF care centres. Paediatr Pulmol. 2011;46:870–881.
  • Collins F, Varmus H. A new initiative on precision medicine. NEJM. 2015;372:793–795.
  • Boyle M, Bell S, Konstan M, et al. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014;2(7):527–538.
  • Wilschanski M, Miller L, Shoseyov D, et al. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. ERJ. 2011;38:59–69.
  • Rowe S, Sermet-Gaudelus I, Konstan M, et al. Results of the phase 3 study of ataluren in nonsense mutation cystic fibrosis (NMCF) [abstract]. Pediat Pulmonol. 2012;47(Suppl 35):290.
  • Berge M, Guillemain R, Tre´gouet DA, et al. Effect of cytochrome P450 2C19 genotype on voriconazole exposure in cystic fibrosis lung transplant patients. Eur J Clin Pharmacol. 2011;67:253–260.
  • Rahman S, Ecob R, Costello H, et al. Hearing in 44-45 year olds with m.1555A>G, a genetic mutation predisposing to aminoglycoside-induced deafness: a population based cohort study. BMJ Open. 2012 Jan 5;2:e000411.
  • Balfour-Lynn I. Personalised medicine in CF is unaffordable. Paed Resp Rev. 2014;15S:2–5.
  • Cohen D, Raftery J. Paying twice: questions over high cost of cystic fibrosis drug developed with charitable funding. BMJ. 2014;348:g1445.
  • O’Sullivan B, Orenstein D, Milla C. Pricing for orphan drugs: will the market bear what society cannot? JAMA. 2013;310(13):1343–1344.
  • Elborn S. Personalised medicine for cystic fibrosis: treating the basic defect. Eur Respir Rev. 2013;22(127):3–5.
  • Wildman M, Hoo Z. Moving cystic fibrosis care from rescue to prevention by embedding adherence measurement in routine care. Paed Resp Rev. 2014;155:16–18.
  • De Boeck K, Bulteel V, Tiddens H, et al. Guideline on the design and conduct of cystis fibrosis clinical trials: the European Cystic Fibrosis Society – Clinical Trials Network (ECFS-CTN). J Cyst Fibros. 2011;10(Suppl 2):S67–S74.
  • Tiddens H, Brody A. Monitoring cystic fibrosis lung disease in clinical trials: is it time for a change? Proc Am Thorac Soc. 2007;4:297–298.
  • Tiddens H, Puderbach M, Venegas J, et al. Novel outcome measures for clinical trials in cystic fibrosis. Pediatr Pulmonol. 2015;50:302–315.
  • Stick M, Sly P. Exciting new clinic trials in CF: infants need not apply. Am J Respir Crit Care Med. 2011;183:1577–1586.
  • O’Connell O, McWilliams S, McGarigle A, et al. Radiologic imaging in cystic fibrosis: cumulative effective dose and changing trends over 2 decades. Chest. 2012;141(6):1575–1583.
  • O’Connor O, Vandeleur M, McGarrigle A, et al. Development of low dose protocols for thin section CT assessment of Cystic Fibrosis in paediatric patients. Radiology. 2010;257(3):820–829.
  • Durmowicz A, Witzmann K, Rosebraugh C, 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.
  • Knowles M, Gatzy J, Boucher R. Increased bioelectric potential difference across respiratory epithelia in cystis fibrosis. NEJM. 1981;305:1489–1495.
  • Leal T, Fajac I, Wallace H, et al. Airway ion transport impacts on disease presentation and severity in cystic fibrosis. Clin Biochem. 2008;41:764–772.
  • Naehrlich L, Ballmann M, Davies J, et al. Nasal potential difference measurements in diagnosis of cystic fibrosis: an international survey. J Cyst Fibros. 2014;13:24–28.
  • Sands D. Transepithelial nasal potential difference(NPD) measurements in cystic fibrosis (CF). Dev Period Med. 2013;XVII(1):13–17.
  • European Medicines Agency. Report of the Workshop on Endpoints for Cystic Fibrosis Clinical Trials. London: European Medicines Agency; 2012.
  • De Boeck K, Derichs N, Fajac I, et al. New clinical diagnostic procedures for cystic fibrosis in Europe. J Cyst Fibros. 2011;10:S53–S66.
  • Bodewes F, Verkade H, Taminiau JA, et al. Cystic fibrosis and the role of gastrointestinal outcome measures in the new era of therapeutic CFTR modulation. J Cyst Fibros. 2015;14:169–177.
  • Quan J, Tiddens H, Sy J, et al. A two-year randomized, placebo-controlled trial of dornase alfa in young patients with cystic fibrosis with mild lung function abnormalities. J Pediatr. 2001;139(6):813–820.
  • Bakker EM, Volpi S, Salonini E, et al. Improved treatment response to dornase alfa in cystic fibrosis patients using controlled inhalation. Eur Respir J. 2011;38:1328–1335.
  • Aurora P, Bush A, Gustafsson P, et al. Multiple-breath washout as a marker of lung disease in preschool children with cystic fibrosis. Am J Respir Crit Care Med. 2005;171:249–256.
  • 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;183:752–758.
  • Robinson P, Latzin P, Verbanck S, et al. Consensus statement for inert gas washout measurement using multiple- and single breath tests. Eur Respir J. 2013;41:507–522.
  • Kent L, Reix P, Innes JA, et al. Lung clearance index: evidence for use in clinical trials in cystic fibrosis: on behalf of the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN) standardisation committee. J Cyst Fibros. 2014;13(2):123–138.
  • Abbott J. Health-related quality of life measurement in cystic fibrosis: advances and limitations. Chron Respir Dis. 2009;6:31–41.
  • Collins S, Reynolds F. How do adults with cystic fibrosis cope following a diagnosis of diabetes? J Adv Nurs. 2008;64(5):478–487.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.