Frank J. Accurso, Fredrick Van Goor, Jiuhong Zha, Anne J

Slides:

Advertisements

Similar presentations

Comparing age of cystic fibrosis diagnosis and treatment initiation after newborn screening with two common strategies Don B. Sanders, Huichuan J. Lai,

Advertisements

Efficacy and safety of Creon�� 24,000 in subjects with exocrine pancreatic insufficiency due to cystic fibrosis Bruce C. Trapnell, Karen Maguiness, Gavin.

Cheryl Cameron, Mark W. Lodes, William M. Gershan

Effect of dornase alfa on inflammation and lung function: Potential role in the early treatment of cystic fibrosis Michael W. Konstan, Felix Ratjen

Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation Kris De Boeck, Anne Munck, Seth Walker, Albert Faro,

CFTR mutation in an Arab patient: Clinical and functional features of 875+1G→A/875+1G→A genotype Elide Spinelli, Manuela Seia, Paola Melotti, Eleonora.

The immediate effect of musculoskeletal physiotherapy techniques and massage on pain and ease of breathing in adults with cystic fibrosis Annemarie Lee,

Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function Fredrick Van Goor, Haihui Yu, Bill.

Use of high-dose ibuprofen in a pediatric cystic fibrosis center

R. Roberts, L. Speight, J. Lee, L. George, R. I. Ketchell, D. Lau, J

Risk factors for age at initial Pseudomonas acquisition in the cystic fibrosis epic observational cohort Margaret Rosenfeld, Julia Emerson, Sharon McNamara,

How does heart rate recovery after sub-maximal exercise correlate with maximal exercise testing in children with CF? Sarah P. Cohen, David M. Orenstein

Avani C. Modi, Crystal S. Lim, Nami Yu, David Geller, Mary H

A randomized double blind, placebo controlled phase 2 trial of BIIL 284 BS (an LTB4 receptor antagonist) for the treatment of lung disease in children.

Single high-dose oral vitamin D3 (stoss) therapy — A solution to vitamin D deficiency in children with cystic fibrosis? Darren Shepherd, Yvonne Belessis,

Measuring and improving respiratory outcomes in cystic fibrosis lung disease: Opportunities and challenges to therapy Edith T. Zemanick, J. Kirk Harris,

Patrick A. Flume, John P. Clancy, George Z. Retsch-Bogart, D

Megan R. Nelson, Craig R. Adamski, Audrey Tluczek

Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation Kris De Boeck, Anne Munck, Seth Walker, Albert Faro,

Early attained weight and length predict growth faltering better than velocity measures in infants with CF Sonya L. Heltshe, Drucy S. Borowitz, Daniel.

Cirrhosis and other liver disease in cystic fibrosis

Delayed publication of clinical trials in cystic fibrosis

Safety and early treatment effects of the CXCR2 antagonist SB in patients with cystic fibrosis Richard B. Moss, Sunil J. Mistry, Michael W. Konstan,

Controlled clinical trials in cystic fibrosis — are we doing better?

Long-term azitromycin treatment of cystic fibrosis patients with chronic Pseudomonas aeruginosa infection; an observational cohort study Christine Rønne.

Cynthia M. Ward, Tara Brinkman, Keith J. Slifer, Shruti M. Paranjape

Anju Anand, Elizabeth Tullis, Anne Stephenson, Preyanka Abhyankar

D. Stevens, P.J. Oades, N. Armstrong, C.A. Williams

Claire J. Tipping, Rebecca L. Scholes, Narelle S. Cox

Influence of digital clubbing on oxygen saturation measurements by pulse-oximetry in cystic fibrosis patients Filip Van Ginderdeuren, Karlien Van Cauwelaert,

Long-term daily high and low doses of azithromycin in children with cystic fibrosis: A randomized controlled trial S.K. Kabra, R. Pawaiya, Rakesh Lodha,

The predictive potential of the sweat chloride test in cystic fibrosis patients with the G551D mutation Verena I. Seliger, David Rodman, Fredrick Van.

Efficacy and safety of Creon�� 24,000 in subjects with exocrine pancreatic insufficiency due to cystic fibrosis Bruce C. Trapnell, Karen Maguiness, Gavin.

A.H. Gifford Journal of Cystic Fibrosis

Demographics of glucose metabolism in cystic fibrosis

Economic effects of an eradication protocol for first appearance of Pseudomonas aeruginosa in cystic fibrosis patients: 1995 vs. 2009 Yolanda P. Lillquist,

Inhalation solutions — Which ones may be mixed

Physiologic endpoints for clinical studies for cystic fibrosis

Antonia V. Bennett, Donald L. Patrick, James F. Lymp, Todd C

Characterisation of mutations and genotype–phenotype correlation in cystic fibrosis: Experience from India Shivaram S. Shastri, Madhulika Kabra, Sushil.

Maximal daily dose of pancreatic enzyme replacement therapy in infants with cystic fibrosis: A reconsideration Drucy Borowitz, Daniel Gelfond, Karen.

Correlation of sweat chloride and percent predicted FEV1 in cystic fibrosis patients treated with ivacaftor Meredith C. Fidler, Jack Beusmans, Paul Panorchan,

E. Tschiedel, H. Grasemann, F. Ratjen Journal of Cystic Fibrosis

Elliott C. Dasenbrook, Gregory S. Sawicki Journal of Cystic Fibrosis

Chee Y. Ooi, Peter R. Durie Journal of Cystic Fibrosis

Concentration of fractional excretion of nitric oxide (FENO): A potential airway biomarker of restored CFTR function Kavitha Kotha, Rhonda D. Szczesniak,

Novel concepts in evaluating antimicrobial therapy for bacterial lung infections in patients with cystic fibrosis Geraint B. Rogers, Lucas R. Hoffman,

Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United.

Australian epidemic strain pseudomonas (AES-1) declines further in a cohort segregated cystic fibrosis clinic Amanda L. Griffiths, Danielle F. Wurzel,

Gender differences in treatment adherence among youth with cystic fibrosis: Development of a new questionnaire Joän M. Patterson, Melanie Wall, Jerica.

Michael D. Parkins, R. Andres Floto Journal of Cystic Fibrosis

Higher risk of hospitalization among females with cystic fibrosis

Trends in pathogens colonising the respiratory tract of adult patients with cystic fibrosis, 1985–2005 F.A. Millar, N.J. Simmonds, M.E. Hodson Journal.

F. Vermeulen, P. Lebecque, K. De Boeck, T. Leal

A new method of sweat testing: the CF Quantum®sweat test

V. Thompson, N. Mayer-Hamblett, M. Kloster, D. Bilton, P.A. Flume

Comparing age of cystic fibrosis diagnosis and treatment initiation after newborn screening with two common strategies Don B. Sanders, Huichuan J. Lai,

J.E. Spahr, R.B. Love, M. Francois, K. Radford, K.C. Meyer

Michael W. Konstan, David J. Pasta, Jeffrey S. Wagener, Donald R

Randomized trial of efficacy and safety of dornase alfa delivered by eRapid nebulizer in cystic fibrosis patients Gregory S. Sawicki, Will Chou, Karina.

C. L. Vreede, M. C. Berkhout, A. J. Sprij, W. J. Fokkens, H. G. M

CFTR modulators and pregnancy: Our work has only just begun

Cheryl Cameron, Mark W. Lodes, William M. Gershan

Michael W. Konstan, Jeffrey S. Wagener, Donald R. VanDevanter, David J

Lutz Goldbeck, Sven Zerrer, Tim G. Schmitz Journal of Cystic Fibrosis

David Adeboyeku, Sandra Scott, Margaret E. Hodson

Theresa A. Laguna, Scott D. Sagel, Marci K. Sontag, Frank J. Accurso

Absence of a gender gap in survival

R. D. Gray, M. Imrie, A. C. Boyd, D. Porteous, J. A. Innes, A. P

Respiratory physicians and clinic coordinators' attitudes to population-based cystic fibrosis carrier screening Fiona Cunningham, Sharon Lewis, Lisette.

Presentation transcript:

Sweat chloride as a biomarker of CFTR activity: Proof of concept and ivacaftor clinical trial data Frank J. Accurso, Fredrick Van Goor, Jiuhong Zha, Anne J. Stone, Qunming Dong, Claudia L. Ordonez, Steven M. Rowe, John Paul Clancy, Michael W. Konstan, Heather E. Hoch, Sonya L. Heltshe, Bonnie W. Ramsey, Preston W. Campbell, Melissa A. Ashlock Journal of Cystic Fibrosis Volume 13, Issue 2, Pages 139-147 (March 2014) DOI: 10.1016/j.jcf.2013.09.007 Copyright © 2013 European Cystic Fibrosis Society. Terms and Conditions

Fig. 1 Within- and between-patient variance in sweat chloride over repeated measures by dose of ivacaftor and placebo. Data are shown for Parts 1 and 2 of the clinical trial. Within-patient variances were similar across treatment groups, indicating a consistent treatment effect in given individuals. Between-patient variances were high in the treatment groups, indicating variable effects of ivacaftor in different individuals. Journal of Cystic Fibrosis 2014 13, 139-147DOI: (10.1016/j.jcf.2013.09.007) Copyright © 2013 European Cystic Fibrosis Society. Terms and Conditions

Fig. 2 Sweat chloride levels vs. CFTR activity in patients with varying CF genotypes and patients treated with ivacaftor at varying dose levels.*% CFTR activity with ivacaftor was estimated using NPD measurements taken at the same time as the sweat chloride. Data are shown for 22 cohorts reported in 10 published studies [15–24], described and tabulated in Table 3. Journal of Cystic Fibrosis 2014 13, 139-147DOI: (10.1016/j.jcf.2013.09.007) Copyright © 2013 European Cystic Fibrosis Society. Terms and Conditions