Biomarkers for cystic fibrosis drug development.

[1]  J. Clancy,et al.  Continuous alternating inhaled antibiotics for chronic pseudomonal infection in cystic fibrosis. , 2016, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[2]  J. Clancy,et al.  Cystic Fibrosis and Its Management Through Established and Emerging Therapies. , 2016, Annual review of genomics and human genetics.

[3]  S. Stanojevic,et al.  Physiologic endpoints for clinical studies for cystic fibrosis. , 2016, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[4]  W. T. Harris,et al.  Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. , 2016, The Lancet. Respiratory medicine.

[5]  F. Accurso,et al.  Use of ibuprofen to assess inflammatory biomarkers in induced sputum: Implications for clinical trials in cystic fibrosis. , 2015, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[6]  S. Stanojevic,et al.  Factors associated with response to treatment of pulmonary exacerbations in cystic fibrosis patients. , 2015, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[7]  Xiaohong Huang,et al.  Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. , 2015, The New England journal of medicine.

[8]  S. Molin,et al.  Diversity of metabolic profiles of cystic fibrosis Pseudomonas aeruginosa during the early stages of lung infection. , 2015, Microbiology.

[9]  R. Fischer,et al.  A phase 3, open-label, randomized trial to evaluate the safety and efficacy of levofloxacin inhalation solution (APT-1026) versus tobramycin inhalation solution in stable cystic fibrosis patients. , 2015, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[10]  S. McColley,et al.  Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. , 2015, The Lancet. Respiratory medicine.

[11]  S. Heltshe,et al.  Effect of treatment of cystic fibrosis pulmonary exacerbations on systemic inflammation. , 2015, Annals of the American Thoracic Society.

[12]  Zaid Abdo,et al.  The daily dynamics of cystic fibrosis airway microbiota during clinical stability and at exacerbation , 2015, Microbiome.

[13]  R. Ng,et al.  Discovery of novel plasma protein biomarkers to predict imminent cystic fibrosis pulmonary exacerbations using multiple reaction monitoring mass spectrometry , 2015, Thorax.

[14]  C. Goss,et al.  Long-term treatment with oral N-acetylcysteine: affects lung function but not sputum inflammation in cystic fibrosis subjects. A phase II randomized placebo-controlled trial. , 2015, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[15]  J. Harris,et al.  Assessment of airway microbiota and inflammation in cystic fibrosis using multiple sampling methods. , 2015, Annals of the American Thoracic Society.

[16]  D. Porteous,et al.  Measurement of serum calprotectin in stable patients predicts exacerbation and lung function decline in cystic fibrosis. , 2015, American journal of respiratory and critical care medicine.

[17]  P. Hiatt,et al.  Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. , 2014, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[18]  Umer Khan,et al.  Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis. , 2014, American journal of respiratory and critical care medicine.

[19]  S. Peltz,et al.  Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial. , 2014, The Lancet. Respiratory medicine.

[20]  S. McColley,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. , 2014, The Lancet. Respiratory medicine.

[21]  M. Milburn,et al.  Serum metabolomics indicate altered cellular energy metabolism in children with cystic fibrosis , 2014, Pediatric pulmonology.

[22]  K. Bambery,et al.  The Pathogen Candida albicans Hijacks Pyroptosis for Escape from Macrophages , 2014, mBio.

[23]  Forest Rohwer,et al.  Biogeochemical Forces Shape the Composition and Physiology of Polymicrobial Communities in the Cystic Fibrosis Lung , 2014, mBio.

[24]  Waldemar Swiercz,et al.  Automated CT scan scores of bronchiectasis and air trapping in cystic fibrosis. , 2014, Chest.

[25]  J. Clancy,et al.  Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data. , 2014, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[26]  Robert Schmieder,et al.  Breath gas metabolites and bacterial metagenomes from cystic fibrosis airways indicate active pH neutral 2,3-butanedione fermentation , 2014, The ISME Journal.

[27]  J. M. Dow,et al.  Microbiota and Metabolite Profiling Reveal Specific Alterations in Bacterial Community Structure and Environment in the Cystic Fibrosis Airway during Exacerbation , 2013, PloS one.

[28]  F. van Goor,et al.  The predictive potential of the sweat chloride test in cystic fibrosis patients with the G551D mutation. , 2013, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[29]  Barbara A. Bailey,et al.  Clinical Insights from Metagenomic Analysis of Sputum Samples from Patients with Cystic Fibrosis , 2013, Journal of Clinical Microbiology.

[30]  A. Webb,et al.  Sweat chloride is not a useful marker of clinical response to Ivacaftor , 2013, Thorax.

[31]  D. Sin,et al.  Systematic review of blood biomarkers in cystic fibrosis pulmonary exacerbations. , 2013, Chest.

[32]  R. Szczesniak,et al.  Multicenter Intestinal Current Measurements in Rectal Biopsies from CF and Non-CF Subjects to Monitor CFTR Function , 2013, PloS one.

[33]  J. Clancy,et al.  Optimizing Nasal Potential Difference Analysis for CFTR Modulator Development: Assessment of Ivacaftor in CF Subjects with the G551D-CFTR Mutation , 2013, PloS one.

[34]  L. Kent,et al.  Clinimetric properties of bronchoalveolar lavage inflammatory markers in cystic fibrosis , 2013, European Respiratory Journal.

[35]  M. Ballmann,et al.  Inhalation treatment with glutathione in patients with cystic fibrosis. A randomized clinical trial. , 2013, American journal of respiratory and critical care medicine.

[36]  P. Sterk,et al.  Exhaled breath analysis by electronic nose in airways disease. Established issues and key questions , 2013, Clinical and experimental allergy : journal of the British Society for Allergy and Clinical Immunology.

[37]  R. Le Berre,et al.  Proposal of a quantitative PCR-based protocol for an optimal Pseudomonas aeruginosa detection in patients with cystic fibrosis , 2013, BMC Microbiology.

[38]  M. Strand,et al.  Blood mRNA biomarkers for detection of treatment response in acute pulmonary exacerbations of cystic fibrosis , 2013, Thorax.

[39]  J. Mainz,et al.  Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. , 2013, American journal of respiratory and critical care medicine.

[40]  Peter D Sly,et al.  Risk factors for bronchiectasis in children with cystic fibrosis. , 2013, The New England journal of medicine.

[41]  D. Hansell,et al.  Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation , 2013, Thorax.

[42]  B. Chowdhury,et al.  Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: the ivacaftor experience. , 2013, Chest.

[43]  S. Sagel,et al.  Sputum biomarkers of inflammation and lung function decline in children with cystic fibrosis. , 2012, American journal of respiratory and critical care medicine.

[44]  G. Döring,et al.  Safety, efficacy and convenience of colistimethate sodium dry powder for inhalation (Colobreathe DPI) in patients with cystic fibrosis: a randomised study , 2012, Thorax.

[45]  L. Saiman,et al.  Effect of azithromycin on systemic markers of inflammation in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa. , 2012, Chest.

[46]  T. Liou,et al.  Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. , 2012, Chest.

[47]  R. Keogh,et al.  Sputum Biomarkers and the Prediction of Clinical Outcomes in Patients with Cystic Fibrosis , 2012, PloS one.

[48]  A. Cantin,et al.  Plasma biomarkers and cystic fibrosis lung disease. , 2012, Clinical and investigative medicine. Medecine clinique et experimentale.

[49]  B. Morrissey,et al.  Metabolomic profiling of regulatory lipid mediators in sputum from adult cystic fibrosis patients. , 2012, Free radical biology & medicine.

[50]  S. Plafker,et al.  Interaction with CREB binding protein modulates the activities of Nrf2 and NF-κB in cystic fibrosis airway epithelial cells. , 2012, American journal of physiology. Lung cellular and molecular physiology.

[51]  Susan Murray,et al.  Decade-long bacterial community dynamics in cystic fibrosis airways , 2012, Proceedings of the National Academy of Sciences.

[52]  J. Stockman Effect of Azithromycin on Pulmonary Function in Patients With Cystic Fibrosis Uninfected With Pseudomonas aeruginosa: A Randomized Controlled Trial , 2012 .

[53]  Matthias Griese,et al.  A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. , 2011, The New England journal of medicine.

[54]  D. Kerr,et al.  Predictive biomarkers: a paradigm shift towards personalized cancer medicine , 2011, Nature Reviews Clinical Oncology.

[55]  O. Eickelberg,et al.  The Chitinase-Like Protein YKL-40 Modulates Cystic Fibrosis Lung Disease , 2011, PloS one.

[56]  J. Clancy,et al.  Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation , 2011, Thorax.

[57]  M. Proesmans,et al.  Change in IgG and evolution of lung function in children with cystic fibrosis. , 2011, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[58]  Todd A. Durham,et al.  Denufosol tetrasodium in patients with cystic fibrosis and normal to mildly impaired lung function. , 2011, American journal of respiratory and critical care medicine.

[59]  J. Emerson,et al.  Randomized trial of biofilm testing to select antibiotics for cystic fibrosis airway infection , 2011, Pediatric pulmonology.

[60]  S. McColley,et al.  Clinical Significance of Microbial Infection and Adaptation in Cystic Fibrosis , 2011, Clinical Microbiology Reviews.

[61]  J. Clancy,et al.  Measurement of ion transport function in rectal biopsies. , 2011, Methods in molecular biology.

[62]  J. Harris,et al.  Reliability of Quantitative Real-Time PCR for Bacterial Detection in Cystic Fibrosis Airway Specimens , 2010, PloS one.

[63]  J. Clancy,et al.  Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. , 2010, The New England journal of medicine.

[64]  J. Carlin,et al.  Identifying peroxidases and their oxidants in the early pathology of cystic fibrosis. , 2010, Free radical biology & medicine.

[65]  R. Gibson,et al.  An 18‐month study of the safety and efficacy of repeated courses of inhaled aztreonam lysine in cystic fibrosis , 2010, Pediatric pulmonology.

[66]  J. Clancy,et al.  An international randomized multicenter comparison of nasal potential difference techniques. , 2010, Chest.

[67]  Antonia Zapf,et al.  Intestinal current measurement for diagnostic classification of patients with questionable cystic fibrosis: validation and reference data , 2010, Thorax.

[68]  Christine M. Micheel,et al.  Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease , 2010 .

[69]  Eoin L. Brodie,et al.  Airway Microbiota and Pathogen Abundance in Age-Stratified Cystic Fibrosis Patients , 2010, PloS one.

[70]  C. Merlo,et al.  Association between respiratory tract methicillin-resistant Staphylococcus aureus and survival in cystic fibrosis. , 2010, JAMA.

[71]  D. Porteous,et al.  Sputum and serum calprotectin are useful biomarkers during CF exacerbation. , 2010, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[72]  Boris Freidlin,et al.  Randomized clinical trials with biomarkers: design issues. , 2010, Journal of the National Cancer Institute.

[73]  S. Willsie Lung Disease at Diagnosis in Infants with Cystic Fibrosis Detected by Newborn Screening , 2010 .

[74]  J. Jais,et al.  A Novel Lipidomic Strategy Reveals Plasma Phospholipid Signatures Associated with Respiratory Disease Severity in Cystic Fibrosis Patients , 2009, PloS one.

[75]  P. Davis,et al.  The triterpenoid CDDO limits inflammation in preclinical models of cystic fibrosis lung disease. , 2009, American journal of physiology. Lung cellular and molecular physiology.

[76]  P. Sly,et al.  Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. , 2009, American journal of respiratory and critical care medicine.

[77]  R. Gibson,et al.  Efficacy and safety of inhaled aztreonam lysine for airway pseudomonas in cystic fibrosis. , 2009, Chest.

[78]  R. Gibson,et al.  Inhaled aztreonam lysine for chronic airway Pseudomonas aeruginosa in cystic fibrosis. , 2008, American journal of respiratory and critical care medicine.

[79]  J. Clancy,et al.  Potential role of high-mobility group box 1 in cystic fibrosis airway disease. , 2008, American journal of respiratory and critical care medicine.

[80]  C. Cotton,et al.  Dysfunction of Nrf-2 in CF Epithelia Leads to Excess Intracellular H2O2 and Inflammatory Cytokine Production , 2008, PloS one.

[81]  R. Gibson,et al.  A phase 2 study of aztreonam lysine for inhalation to treat patients with cystic fibrosis and Pseudomonas aeruginosa infection , 2008, Pediatric pulmonology.

[82]  M. Corey,et al.  High-dose ibuprofen in cystic fibrosis: Canadian safety and effectiveness trial. , 2007, The Journal of pediatrics.

[83]  M. Konstan,et al.  Sputum biomarkers of inflammation in cystic fibrosis lung disease. , 2007, Proceedings of the American Thoracic Society.

[84]  J. Elborn,et al.  The effect of treatment of cystic fibrosis pulmonary exacerbations on airways and systemic inflammation , 2007, Pediatric pulmonology.

[85]  R. Gibson,et al.  No detectable improvements in cystic fibrosis transmembrane conductance regulator by nasal aminoglycosides in patients with cystic fibrosis with stop mutations. , 2007, American journal of respiratory cell and molecular biology.

[86]  R. Kronmal,et al.  Association between pulmonary function and sputum biomarkers in cystic fibrosis. , 2007, American journal of respiratory and critical care medicine.

[87]  U. S. Department of Health and Human Services FDA Cen Research,et al.  Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance , 2006, Health and quality of life outcomes.

[88]  M. Corey,et al.  Mutations in the cystic fibrosis transmembrane regulator gene and in vivo transepithelial potentials. , 2006, American journal of respiratory and critical care medicine.

[89]  Michael P Boyle,et al.  Respiratory epithelial gene expression in patients with mild and severe cystic fibrosis lung disease. , 2006, American journal of respiratory cell and molecular biology.

[90]  T. Luider,et al.  Proteomic analysis of nasal cells from cystic fibrosis patients and non‐cystic fibrosis control individuals: Search for novel biomarkers of cystic fibrosis lung disease , 2006, Proteomics.

[91]  P. Bye,et al.  Proteomic analysis of sputum from adults and children with cystic fibrosis and from control subjects. , 2005, American journal of respiratory and critical care medicine.

[92]  M. Kosorok,et al.  Longitudinal development of mucoid Pseudomonas aeruginosa infection and lung disease progression in children with cystic fibrosis. , 2005, JAMA.

[93]  Thomas R Fleming,et al.  Surrogate endpoints and FDA's accelerated approval process. , 2005, Health affairs.

[94]  M. Amaral,et al.  CFTR Cl- channel function in native human colon correlates with the genotype and phenotype in cystic fibrosis. , 2004, Gastroenterology.

[95]  K. Kunzelmann,et al.  Assessment of CFTR function in rectal biopsies for the diagnosis of cystic fibrosis. , 2004, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[96]  R. Desnick Enzyme replacement therapy for Fabry disease: lessons from two α-galactosidase A orphan products and one FDA approval , 2004, Expert opinion on biological therapy.

[97]  J. Emerson,et al.  Standardized procedure for measurement of nasal potential difference: An outcome measure in multicenter cystic fibrosis clinical trials , 2004, Pediatric pulmonology.

[98]  J. Uddin,et al.  Defective lipoxin-mediated anti-inflammatory activity in the cystic fibrosis airway , 2004, Nature Immunology.

[99]  U. Wahn,et al.  Effect of treatment with dornase alpha on airway inflammation in patients with cystic fibrosis. , 2004, American journal of respiratory and critical care medicine.

[100]  M. Amaral,et al.  Assessment of CFTR function in native epithelia for the diagnosis of cystic fibrosis , 2004, Pediatric pulmonology. Supplement.

[101]  R. Gibson,et al.  Inflammatory and microbiologic markers in induced sputum after intravenous antibiotics in cystic fibrosis. , 2003, American journal of respiratory and critical care medicine.

[102]  R. Gibson,et al.  Pseudomonas acquisition in young patients with cystic fibrosis: pathophysiology, diagnosis, and management , 2003, Current opinion in pulmonary medicine.

[103]  B. Kerem,et al.  Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations. , 2003, The New England journal of medicine.

[104]  L. Saiman,et al.  Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial. , 2003, JAMA.

[105]  J. Emerson,et al.  Significant microbiological effect of inhaled tobramycin in young children with cystic fibrosis. , 2003, American journal of respiratory and critical care medicine.

[106]  P. Noone,et al.  Idiopathic Pancreatitis Related to CFTR: Complex Inheritance and Identification of a Modifier Gene , 2002, Journal of Investigative Medicine.

[107]  M. Knowles,et al.  Cystic fibrosis gene mutations and pancreatitis risk: relation to epithelial ion transport and trypsin inhibitor gene mutations. , 2001, Gastroenterology.

[108]  F. Accurso,et al.  Airway inflammation in children with cystic fibrosis and healthy children assessed by sputum induction. , 2001, American journal of respiratory and critical care medicine.

[109]  B. Strandvik,et al.  Factors associated with poor pulmonary function: cross-sectional analysis of data from the ERCF , 2001 .

[110]  J. Clancy,et al.  Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis. , 2001, American journal of respiratory and critical care medicine.

[111]  C. Cross,et al.  Myeloperoxidase and protein oxidation in cystic fibrosis. , 2000, American journal of physiology. Lung cellular and molecular physiology.

[112]  B. Tümmler,et al.  Residual chloride secretion in intestinal tissue of ΔF508 homozygous twins and siblings with cystic fibrosis , 2000 .

[113]  B. Tümmler,et al.  Residual chloride secretion in intestinal tissue of deltaF508 homozygous twins and siblings with cystic fibrosis. The European CF Twin and Sibling Study Consortium. , 2000, Gastroenterology.

[114]  D. Zurakowski,et al.  Pulmonary Outcome in Cystic Fibrosis Is Influenced Primarily by Mucoid Pseudomonas aeruginosa Infection and Immune Status and Only Modestly by Genotype , 1999, Infection and Immunity.

[115]  T. Hazinski Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. , 1999, The Journal of pediatrics.

[116]  M S Pepe,et al.  Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. , 1999, The New England journal of medicine.

[117]  P. Zeitlin,et al.  A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function. , 1998, American journal of respiratory and critical care medicine.

[118]  M. Knowles,et al.  In vivo nasal potential difference: techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis. , 1995, Human gene therapy.

[119]  P. J. Byard,et al.  Effect of high-dose ibuprofen in patients with cystic fibrosis. , 1995, The New England journal of medicine.

[120]  H. Fuchs,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. , 1994, The New England journal of medicine.

[121]  R. Prentice Surrogate endpoints in clinical trials: definition and operational criteria. , 1989, Statistics in medicine.