Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup:

Slides:

Advertisements

Similar presentations

Crohn's disease Prof Daniel C Baumgart, MD, Prof William J Sandborn, MD The Lancet Volume 380, Issue 9853, Pages (November 2012) DOI: /S (12)

Advertisements

Comparison of Inhaled Antibiotics for the Treatment of Chronic Pseudomonas aeruginosa Lung Infection in Patients With Cystic Fibrosis: Systematic Literature.

Volume 370, Issue 9586, Pages (August 2007)

Volume 11, Issue 5, Pages (May 2012)

Volume 378, Issue 9793, Pages (August 2011)

The effect of a reduction in alcohol consumption on blood pressure: a systematic review and meta-analysis Dr Michael Roerecke, PhD, Prof Janusz Kaczorowski,

Pharmacogenetics and future drug development and delivery

Volume 349, Issue 9057, Pages (April 1997)

Volume 11, Issue 4, Pages (April 2012)

Volume 128, Issue 3, Pages (September 2005)

Volume 100, Issue 6, Pages (June 2006)

Edward G. Seferian, Nancy K. Henry, Mark E. Wylam Respiratory Medicine

Thank God for Richard Dawkins?

Simple versus complex COPD: implications for health-care management

Tinnitus Dr David Baguley, PhD, Don McFerran, FRCS, Prof Deborah Hall, PhD The Lancet Volume 382, Issue 9904, Pages (November 2013) DOI: /S (13)

Intravenous or nebulised magnesium sulphate versus standard therapy for severe acute asthma (3Mg trial): a double-blind, randomised controlled trial

A phase 3, multi-center, multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of levofloxacin inhalation.

Volume 13, Issue 3, Pages (March 2012)

Volume 150, Issue 6, Pages (December 2016)

Mannose-binding lectin deficiency and disease severity in non-cystic fibrosis bronchiectasis: a prospective study Dr James D Chalmers, MBChB, Brian J.

Expert Patient Self-Management Program Versus Usual Care in Bronchiectasis: A Randomized Controlled Trial Katherine A. Lavery, BSc, Brenda O'Neill, PhD,

Volume 379, Issue 9816, Pages (February 2012)

Volume 2, Issue 12, Pages (December 2015)

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

Organisation of the care of patients with heart failure

The effect of explosive remnants of war on global public health: a systematic mixed- studies review using narrative synthesis Alexandra Frost, MSc, Prof.

Volume 379, Issue 9835, Pages (June 2012)

Volume 375, Issue 9732, Pages (June 2010)

Resuscitation at birth and cognition at 8 years of age: a cohort study

Safety and efficacy of prolonged levofloxacin inhalation solution (APT-1026) treatment for cystic fibrosis and chronic Pseudomonas aeruginosa airway infection

Minimum clinically important difference for the COPD Assessment Test: a prospective analysis Dr Samantha S C Kon, MBBS, Jane L Canavan, PhD, Sarah E.

Volume 390, Issue 10094, Pages (August 2017)

Expert Patient Self-Management Program Versus Usual Care in Bronchiectasis: A Randomized Controlled Trial Katherine A. Lavery, BSc, Brenda O'Neill, PhD,

Classification of CFTR mutation classes

PATHWAY-2: spironolactone for resistant hypertension – Authors' reply

Volume 143, Issue 1, Pages (January 2013)

Occupational asthma from a horticultural nematode, Steinernema feltiae

Volume 362, Pages s6-s7 (December 2003)

Cold water immersion: sudden death and prolonged survival

Tadalafil in patients with chronic obstructive pulmonary disease: a randomised, double- blind, parallel-group, placebo-controlled trial Dr Andrew R Goudie,

Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR Jennifer L. Taylor-Cousar, Manu Jain, Tara.

Volume 393, Issue 10171, Pages (February 2019)

A phase 3, open-label, randomized trial to evaluate the safety and efficacy of levofloxacin inhalation solution (APT-1026) versus tobramycin inhalation.

Volume 381, Issue 9868, Pages (March 2013)

Volume 388, Issue 10056, Pages (October 2016)

Serum VEGF-D concentration as a biomarker of lymphangioleiomyomatosis severity and treatment response: a prospective analysis of the Multicenter International.

Confessions of a journal junkie

Intravenous or nebulised magnesium sulphate versus standard therapy for severe acute asthma (3Mg trial): a double-blind, randomised controlled trial

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

Thank God for Richard Dawkins?

Department of Error The Lancet Volume 369, Issue 9564, (March 2007)

Low-technology approaches

Diagnosis of tuberculous meningitis with invasive pulmonary sampling

Epidemiology of stroke

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.

Association between the introduction of a new cystic fibrosis inhaled antibiotic class and change in prevalence of patients receiving multiple inhaled.

Pulmonary artery enlargement and cystic fibrosis pulmonary exacerbations: a cohort study Dr J Michael Wells, MD, Roopan F Farris, MD, Taylor A Gosdin,

Volume 373, Issue 9672, Pages (April 2009)

Volume 376, Issue 9737, Pages (July 2010)

Polygenic risk and the development and course of asthma: an analysis of data from a four-decade longitudinal study Dr Daniel W Belsky, PhD, Prof Malcolm.

The effect of social deprivation on clinical outcomes and the use of treatments in the UK cystic fibrosis population: a longitudinal study Dr David C.

Gautham Marigowda, Fang Liu, David Waltz Journal of Cystic Fibrosis

Sputum culture conversion in new TB regimens

Felix Ratjen, Todd Durham, Tomas Navratil, Amy Schaberg, Frank J

Partial restoration of pancreatic function in a child with cystic fibrosis Ciara Howlett, Nicola J Ronan, Muireann NiChroinin, David Mullane, Barry J.

Inhaled amiloride and tobramycin solutions fail to eradicate Burkholderia dolosa in patients with cystic fibrosis Ahmet Z. Uluer, David A. Waltz, Leslie.

Safety, efficacy and convenience of tobramycin inhalation powder in cystic fibrosis patients: The EAGER trial Michael W. Konstan, Patrick A. Flume, Matthias.

Journal of Cystic Fibrosis

The effect of a reduction in alcohol consumption on blood pressure: a systematic review and meta-analysis Dr Michael Roerecke, PhD, Prof Janusz Kaczorowski,

Fighting for health: meet the doctor-activists

Presentation transcript:

Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis Prof J Stuart Elborn, MD, Prof Bonnie W Ramsey, MD, Prof Michael P Boyle, MD, Michael W Konstan, MD, Xiaohong Huang, PhD, Gautham Marigowda, MD, David Waltz, MD, Prof Claire E Wainwright, MD The Lancet Respiratory Medicine Volume 4, Issue 8, Pages 617-626 (August 2016) DOI: 10.1016/S2213-2600(16)30121-7 Copyright © 2016 Elsevier Ltd Terms and Conditions

Figure 1 Absolute change from baseline in ppFEV1 at each study visit for patients with ppFEV1 levels lower than 40 (A) or 40 or higher (B) at baseline and lower than 70 (C) or 70 or higher (D) at screening Error bars show 95% CI. ppFEV1=percent predicted FEV1. The Lancet Respiratory Medicine 2016 4, 617-626DOI: (10.1016/S2213-2600(16)30121-7) Copyright © 2016 Elsevier Ltd Terms and Conditions

Figure 2 Percentage of patients with 5% or higher and 10% or higher average relative increases from baseline in ppFEV1 at weeks 16 and 24 in patients with ppFEV1 levels lower than 40 or 40 or higher at baseline (A) and ppFEV1 levels lower than 70 or 70 or higher at screening (B) ppFEV1=percent predicted FEV1. The Lancet Respiratory Medicine 2016 4, 617-626DOI: (10.1016/S2213-2600(16)30121-7) Copyright © 2016 Elsevier Ltd Terms and Conditions