HOW DISEASE ALTERING THERAPY IS CHANGING THE GOALS OF TREATMENT IN CF
|
|
- Jonathan Craig
- 5 years ago
- Views:
Transcription
1 HOW DISEASE ALTERING THERAPY IS CHANGING THE GOALS OF TREATMENT IN CF Peter D. Sly MBBS, MD, FRACP, DSc OUTLINE Goals of CF treatment Drivers of early disease neutrophilic inflammation oxidative stress infection Minimizing early lung disease Preserving lung function till new therapies available 1
2 IVACAFTOR Potentiates opening of CFTR channel with G551D normalizes sweat chloride improves lung function Improves nutrition Case study: 7y girl G551D/G551D ivacaftor: clinical and radiological improvement NOT reversal of structural damage 5m pre ivacaftor pre ivacaftor 7 months of ivacaftor 11 months of ivacaftor 2
3 GOALS OF CF TREATMENT Prescribing ivacaftor in Australia Cost >$300,000 pa per patient Supply available on PBS for patients who satisfy all: 6y with at least one G551D mutation FEV 1 40% predicted no upper limit Sweat chloride 60mMol/L Chronic sinopulmonary disease or GIT & nutritional abnormalities Continued supply depends on demonstrating sustained benefit (lung function, wt, days in hospital) Not available for primary prevention Thus preserving function is paramount GOALS OF CF TREATMENT 1. To diagnose CF as early as possible New-born screening 2. To preserve lung function CF lung disease begins early, treatment must begin early 3. To optimize growth Growth sub-optimal in early life Maintaining weight / BMI not sufficient 4. To prevent complications / co-morbidities No real evidence that this is possible Current trials with glucose management to prevent CFRD 3
4 DRIVERS OF EARLY DISEASE IN CF AREST CF early surveillance program 1998: BAL-based micro surveillance program Perth clinic 2002: Infant lung function 2003: Preschool FOT 2005: CFFT funding CT scans Melbourne clinic joined the program 2011: CFFT funding COMBAT CF RCT: Primary prevention of Bx with AZM / placebo from 3m to 3y THE AREST CF PROGRAM Comprehensive early surveillance program CF diagnosed following detection by NBS Initial assessment soon after diagnosis ( 3 months) Annual assessment close to birthday until 6 years Clinical and research components Separate consent for each component Assessments undertaken when clinically stable Very well accepted, >95% complete participation 4
5 AREST CF ASSESSMENTS CT/BAL GA; EI (25 cmh 2 O), EE (0 cmh 2 O) 3-slice scans; low-dose volumetric scans BAL after CT Infant lung function SF 6 MBW, LF-FOT, RVRTC Preschool lung function FOT, spirometry from 5 years Biomarker discovery and validation Matched BAL, serum, urine Infection, inflammation, metabolomics Epithelial cell biology (Stick) ONSET OF BRONCHIECTASIS Prevalence of Structural Changes 3m Age of life Bronchiectasis (years) Air Trapping Mott et al 2009, Journal of Cystic Fibrosis Stick et al 2009, Journal of Pediatrics 5
6 AREST CF Overall results 1285 BAL (305 children), 869 CT (258 children) Lung disease begins early in CF Bx, air trapping from 3 months Infection, including with P. aeruginosa from 3 months May occur in asymptomatic children Neutrophilic inflammation prominent Free NE activity from 3 months Associated with presence, extent and progression of Bx Infection common At least one infection in 78% by 6 years Successful eradication of P. aeruginosa if treated early Early infection on BAL associated with Structural lung disease Abnormal lung function in infancy Low BMI NEUTROPHILIC INFLAMMATION Activated neutrophils secrete both pro- and antiinflammatory compounds H 2 O 2 Elastase Myeloperoxidase Calprotectin NETS NE NE Activation MPO H 2 O 2 6
7 H 2 O 2 MPO H 2 O 2 + Cl GSH MPO HOCl GSSG + GSA (40%) (32%) HOCl Protein cleavage 3Cl-Tyr NE Elastin destruction desmosines ROS DNA oxidation 8-oxodG Neutrophilic inflammation as an early disease driver 3 months (n=127) 12 months (n=109) 2 years (n=92) 3 years (n=81) BMI Z-score -1.34± ± ± ±0.96 NE positive 28 (23.3%) 19 (18.1%) 20 (21.7%) 19 (25.0%) Neutrophils x10 3 /ml 416± ± ± ±5618 Any infection 22.4% 21.1% 40.2% 46.9% S. aureus 6.4% 5.5% 10.9% 22.2% P. aeruginosa 5.6% 8.3% 5.4% 9.9% Bx point prevalence 29.3% 31.5% 44.0% 61.5% Bx ever (cumulative) 29.3% 46.6% 63.0% 83.7% GT point prevalence 68.0% 68.5% 71.6% 69.5% Sly et al NEJM
8 Longitudinal risk factors for Bx from 3m to 3y Multivariate analysis Odds Ratio (95% CI) GEE with binomial family, logit link and AR(1) correlation matrix Sly et al NEJM 2013 Those with NE in BAL develop Bx earlier Sly et al NEJM
9 Persistent Bx Persistent Negative Negative Resolved Persistent Acquired Acquired Resolved Initial scan Subsequent scan Bx label Detected Persistent Detected Not detected Resolved Not detected Detected Not detected Acquired Negative Sly et al NEJM 2013 Risk factors at 3m for persistent Bx: 12m Odds Ratio (95% CI) Logistic regression; Blue=univariate; Red=multivariate Sly et al NEJM
10 Risk factors at 3m for persistent Bx: 3y Odds Ratio (95% CI) Logistic regression; Blue=univariate; Red=multivariate Sly et al NEJM 2013 Neutrophil-induced oxidative stress as an driver of early lung disease Kettle ERJ
11 Oxidation of GSH is increased in CF Abnormal protein binding of GSH in CF Kettle ERJ 2014 Oxidation of calprotectin anti-bacterial function Magon 2015 submitted 11
12 Active calprotectin (MLTELEK) oxidized in CF More oxidation with pulmonary infection Magon 2015 submitted DRIVERS OF EARLY DISEASE IN CF Poor nutrition despite NBS [AREST CF] 42 infants studied from diagnosis (3m) to 3y 16 (38%) male, 36 NBS, 6 Mec Ileus, 39 PI 3 m (n=36) 1 y (n=42) 2y (n=42) 3y (n=42) Respiratory symptoms 38% 36% 31% 36% Anti-staph prophylaxis, n (%) 17 (42.5) 31 (73.8) 26 (61.9) 24 (57.1) S. Aureus, n (%) 9 (25.0) 3 (7.1) 6 (14.3) 7 (17.5) P. aeruginosa, n (%) 3 (8.3) 4 (9.5) 4 (9.5) 4 (9.5) Ranganathan, Thorax
13 Initial poor growth that normalizes by 1y (group data) Ranganathan, Thorax 2011 Free NE activity associated with worse growth over 3y Staph infection associated with worse growth over 3y Staph prophylaxis associated with better growth over 3y, p=0.013 Ranganathan, Thorax
14 MINIMIZING EARLY LUNG DISEASE Treatment options depend on disease mechanisms Inflammation: anti-inflammatories anti-neutrophil therapies: AZM? NE inhibitors? Infection: antibiotic prophylaxis Thicker mucus: mucolytics Dehydrated ELF: HS, enac blockers Impaired mucociliary clearance: HS, DNAse Impaired anti-oxidant defence: anti-oxidants; GSH Gene defect: gene therapy, CFTR correctors, potentiators Personal assessment may be more informative GOALS OF CF TREATMENT In the era of disease-altering therapy the goals of CF treatment should be: preserving lung function optimizing nutrition inimizing co-morbidities / complications until patient-specific disease-altering therapy is available. Personalized assessment of disease mechanisms and optimal therapeutic strategies likely to be required Point-of-care biomarkers of active disease in early life would be helpful 14
Enabling CF Therapeutic Development
Enabling CF Therapeutic Development PRESTON W. CAMPBELL, III, M.D. Executive Vice President for Medical Affairs No Disclosures Cystic Fibrosis In 1955 In 1955 most children with CF did not live long enough
More information"Management and Treatment of Patients with Cystic fibrosis (CF)
"Management and Treatment of Patients with Cystic fibrosis (CF) Dr. Malena Cohen-Cymberknoh Pediatric Pulmonology and CF Center Hadassah Hebrew-University Medical Center Jerusalem, Israel Afula, March
More informationRespiratory infection rates differ between geographically distant paediatric cystic fibrosis cohorts
ORIGINAL ARTICLE CYSTIC FIBROSIS Respiratory infection rates differ between geographically distant paediatric cystic fibrosis cohorts Kathryn A. Ramsey 1,2,7, Emily Hart 3,4,7, Lidija Turkovic 1, Marc
More informationA bs tr ac t. n engl j med 368;21 nejm.org may 23,
The new england journal of medicine established in 1812 may 23, 2013 vol. 368 no. 21 Risk Factors for Bronchiectasis in Children with Cystic Fibrosis Peter D. Sly, M.D., D.Sc., Catherine L. Gangell, Ph.D.,
More informationRespiratory Pharmacology: Treatment of Cystic Fibrosis
Respiratory Pharmacology: Treatment of Cystic Fibrosis Dr. Tillie-Louise Hackett Department of Anesthesiology, Pharmacology and Therapeutics University of British Columbia Associate Head, Centre of Heart
More informationCystic Fibrosis. Cystic Fibrosis. Cystic Fibrosis 5/01/2011 CYSTIC FIBROSIS OF THE PANCREAS AND ITS RELATION TO CELIAC DISEASE. D ANDERSEN.
1938 OF THE PANCREAS AND ITS RELATION TO CELIAC DISEASE. D ANDERSEN. American Journal Diseases Children. : The beginning May 1938: 49 cases 25 20 15 Nos of cases 10 5 0 0.5 1 1.5 2 2.5 3 3.5 4 4.5 5 Age
More informationTransformational Treatments. PRESTON W. CAMPBELL, III, M.D. Executive Vice President for Medical Affairs
Transformational Treatments PRESTON W. CAMPBELL, III, M.D. Executive Vice President for Medical Affairs Symptom-based CF Therapies 45 Median Predicted Survival Age of US Patients with Cystic Fibrosis 41
More informationCystic Fibrosis the future
Cystic Fibrosis the future Pathophysiologic cascade Abnormal Gene Abnormal CFTR Therapy Gene replacement Protein replacement Gene read through therapy Abnormal sodium chloride & water movement through
More informationWhat is Cystic Fibrosis? CYSTIC FIBROSIS. Genetics of CF
What is Cystic Fibrosis? CYSTIC FIBROSIS Lynne M. Quittell, M.D. Director, CF Center Columbia University Chronic, progressive and life limiting autosomal recessive genetic disease characterized by chronic
More informationCystic Fibrosis: Progress in Treatment Management. Patrick A. Flume, M.D. Medical University of South Carolina
Cystic Fibrosis: Progress in Treatment Management Patrick A. Flume, M.D. Medical University of South Carolina Disclosures Grant support Mpex Pharmaceuticals, Inc Gilead Sciences, Inc Bayer Healthcare AG
More informationKey Points: References: Canadian data from the Canadian Cystic Fibrosis Registry 2015 Annual Report normal
1 2 3 Cystic fibrosis is a rare life-long genetic disease that affects approximately 4,000 people in Canada and about 70,000 worldwide regardless of race or ethnicity but is more common in Caucasians 1,2
More informationImpact of lung disease on respiratory impedance in young children with cystic fibrosis
ERJ Express. Published on September 24, 2015 as doi: 10.1183/13993003.00156-2015 ORIGINAL ARTICLE IN PRESS CORRECTED PROOF Impact of lung disease on respiratory impedance in young children with cystic
More informationCystic Fibrosis as it relates to the neonate MARIANNE MUHLEBACH, MD PROFESSOR, DEPT. PEDIATRICS UNC CHAPEL HILL
Cystic Fibrosis as it relates to the neonate MARIANNE MUHLEBACH, MD PROFESSOR, DEPT. PEDIATRICS UNC CHAPEL HILL Objectives: At the end of the presentation the listeners will Be able to describe neonatal
More informationChanges in the management of children with Cystic Fibrosis. Caroline Murphy & Deirdre O Donovan CF Nurses
Changes in the management of children with Cystic Fibrosis Caroline Murphy & Deirdre O Donovan CF Nurses What Is Cystic Fibrosis? Cystic fibrosis (CF) is an inherited chronic disease that primarily affects
More information1. Telethon Kids Institute, University of Western Australia, Subiaco, Western Australia
Title: Early respiratory infection is associated with reduced spirometry in children with cystic fibrosis Authors: Kathryn A Ramsey 1, Sarath Ranganathan 2,3,4, Judy Park 1, Billy Skoric 2,3, Anne- Marie
More informationPediatrics Grand Rounds 18 Sept University of Texas Health Science Center. + Disclosure. + Learning Objectives.
Disclosure Dr Donna Willey Courand receives research support from Cystic Fibrosis Therapeutics The Cystic Fibrosis Foundation Children with Special Health Care Needs Cystic Fibrosis 05: Improving Survival
More informationCystic Fibrosis Foundation Patient Registry 2013
5/9/2015 Targeting CFTR to Treat Cystic Fibrosis: Small Molecule Therapy Mary Ellen Kleinhenz, MD Director, UCSF Adult Cystic Fibrosis Program Professor of Medicine UCSF Division of Pulmonary, Critical
More informationCystic Fibrosis. Presented by: Chris Belanger & Dylan Medd
Cystic Fibrosis Presented by: Chris Belanger & Dylan Medd Outline What is Cystic Fibrosis? Signs, Symptoms & Diagnosis Who does it effect? General effects on daily life Managing Cystic Fibrosis Exercise
More informationDisclosures. Advances in the Management of Cystic Fibrosis: A Closer Look at the Roles of CFTR Modulation Therapy 10/28/2016
Advances in the Management of Cystic Fibrosis: A Closer Look at the Roles of CFTR Modulation Therapy Susanna A McColley, MD Associate Chief Research Officer Stanley Manne Children s Research Institute
More informationProgressive ventilation inhomogeneity in infants with cystic fibrosis after pulmonary infection
ORIGINAL ARTICLE CYSTIC FIBROSIS Progressive ventilation inhomogeneity in infants with cystic fibrosis after pulmonary infection Shannon J. Simpson 1, Sarath Ranganathan 2,3,4, Judy Park 1, Lidija Turkovic
More information1. Introduction. Obstructive lung disease remains the leading cause of morbidity and mortality in cystic fibrosis
1. Introduction Obstructive lung disease remains the leading cause of morbidity and mortality in cystic fibrosis (CF) [1]. With time it has become increasingly clear that CF lung disease is present very
More informationCystic fibrosis: hitting the target
Cystic fibrosis: hitting the target Heartland Collaborative Annual Meeting Friday, October 5, 2012 Thomas Ferkol MD 1938 1953 Cystic fibrosis: a historical timeline Cystic fibrosis (CF) of the pancreas
More informationPharmacogenomics in Rare Diseases: Development Strategy for Ivacaftor as a Therapy for Cystic Fibrosis
Pharmacogenomics in Rare Diseases: Development Strategy for Ivacaftor as a Therapy for Cystic Fibrosis Federico Goodsaid Vice President Strategic Regulatory Intelligence Vertex Pharmaceuticals Is there
More informationCystic fibrosis: From childhood to adulthood. Eitan Kerem Department of Pediatrics and CF Center Hadassah University Hospital Jerusalem Israel
Cystic fibrosis: From childhood to adulthood Eitan Kerem Department of Pediatrics and CF Center Hadassah University Hospital Jerusalem Israel Vas deference H 2 O Cl - Na + H 2 O Na + Cl - Cl - Cl -
More informationPediatrics Grand Rounds 13 November University of Texas Health Science Center at San Antonio. Learning Objectives
Nationwide Newborn Screening for Cystic Fibrosis: Finally Creating an Opportunity for All Patients to Have Better Outcomes Philip M Farrell, MD, PhD* University of Wisconsin-Madison *No disclosures other
More informationNebulised hypertonic saline for cystic fibrosis.
Revista: Cochrane Database Syst Rev. 2003;(1):CD001506. Nebulised hypertonic saline for cystic fibrosis. Autor: Wark PA, McDonald V. Source: Department of Respiratory Medicine, John Hunter Hospital, Locked
More informationChapter 3 The Role of Nutrition in CF Care
Chapter 3 The Role of Nutrition in CF Care S. King, N. Saxby & N. Sander Cystic fibrosis is the most common lethal autosomal recessive genetic condition affecting Caucasians 186,187. Over 3500 Australians
More informationCystic Fibrosis. Jennifer McDaniel, BS, RRT-NPS
Cystic Fibrosis Jennifer McDaniel, BS, RRT-NPS Overview Cystic fibrosis is the most common fatal, inherited disease in the U. S. CF results from a defective autosomal recessive gene One copy of gene =
More informationOpening Doors to CF Clinical Research: Change is Coming
Opening Doors to CF Clinical Research: Change is Coming Disclosures Contracts with sponsors for clinical trials conducted at UNC-Chapel Hill: CFFT NIH/NHLBI AbbVie Inc. Aptalis Pharma US, Inc. Janssen
More informationThe Future of CF Therapy
The Future of CF Therapy Peter J. Mogayzel, Jr., M.D., Ph.D. Eudowood Division of Pediatric Respiratory Sciences The Johns Hopkins School of Medicine Overview The Future of CF Therapy Personalized therapy
More informationINHALED ANTIBIOTICS THERAPY IN NON-CF LUNG DISEASE
INHALED ANTIBIOTICS THERAPY IN NON-CF LUNG DISEASE Hannah Blau MBBS Pulmonary Institute and Graub CF Center SCHNEIDER CHILDREN S MEDICAL CENTER OF ISRAEL Chipap 18 th February 2015 Inhaled antibiotic therapy:
More informationA Genetic Approach to the Treatment of Cystic Fibrosis
A Genetic Approach to the Treatment of Cystic Fibrosis Peter Mueller, PhD Chief Scientific Officer and Executive Vice President Global Research and Development Vertex Pharmaceuticals, Incorporated March
More informationCYSTIC FIBROSIS OBJECTIVES NO CONFLICT OF INTEREST TO DISCLOSE
CYSTIC FIBROSIS Madhu Pendurthi MD MPH Staff Physician, Mercy Hospital Springfield, MO NO CONFLICT OF INTEREST TO DISCLOSE OBJECTIVES Epidemiology of Cystic Fibrosis (CF) Genetic basis and pathophysiology
More informationMedical Policy An independent licensee of the Blue Cross Blue Shield Association
Cystic Fibrosis Transmembrane Page 1 of 11 Medical Policy An independent licensee of the Blue Cross Blue Shield Association Title: Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Prime Therapeutics
More informationKeywords: multiple-breath washout; child; inflammation; infection; cystic fibrosis
Multiple-Breath Washout Outcomes Are Sensitive to Inflammation and Infection in Children with Cystic Fibrosis Kathryn A. Ramsey 1,2,3, Rachel E. Foong 1, Jasmine Grdosic 1, Alana Harper 1, Billy Skoric
More informationEvaluation of Patients with Diffuse Bronchiectasis
Evaluation of Patients with Diffuse Bronchiectasis Dr. Patricia Eshaghian, MD Assistant Clinical Professor of Medicine Director, UCLA Adult Cystic Fibrosis Affiliate Program UCLA Division of Pulmonary
More informationCYSTIC FIBROSIS Risk Factors Epidemiology Pathogenesis Defective protein synthesis (10%) Abnormal protein folding, processing & trafficking
CYSTIC FIBROSIS Risk Factors Caucasian Family history of CF Infection Exposure to allergens and tobacco Epidemiology Carrier frequency of 1 in 25 for Caucasians The most common lethal genetic disease affecting
More informationMedical Policy An independent licensee of the Blue Cross Blue Shield Association
Cystic Fibrosis Transmembrane Page 1 of 13 Medical Policy An independent licensee of the Blue Cross Blue Shield Association Title: Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Prime Therapeutics
More informationCost-effectiveness of Ivacaftor (Kalydeco ) for the treatment of cystic fibrosis in patients age 6 years and older who have the G551D mutation
Cost-effectiveness of Ivacaftor (Kalydeco ) for the treatment of cystic fibrosis in patients age 6 years and older who have the G551D mutation January 2013 1. A rapid review submission on the drug ivacaftor
More informationSupplementary appendix
Supplementary appendix This appendix formed part of the original submission and has been peer reviewed. We post it as supplied by the authors. Supplement to: Moss RB, Flume PA, Elborn JS, et al, on behalf
More informationIVACAFTOR THE ISRAELI EXPERIENCE ADI DAGAN MD THE ISRAELI CF CENTER SHEBA MEDICAL CENTER, TEL-HASHOMER
IVACAFTOR THE ISRAELI EXPERIENCE ADI DAGAN MD THE ISRAELI CF CENTER SHEBA MEDICAL CENTER, TEL-HASHOMER February 21, 2014 U.S. Food and Drug Administration Approves KALYDECO (ivacaftor) for Use in Eight
More informationCystic Fibrosis Diagnosis and Treatment
Cystic Fibrosis Diagnosis and Treatment Financial Disclosures Personal financial relationships with commercial interests relevant to medicine, within the past 3 years: NJH site PI for AstraZeneca. As faculty
More informationBriefing Document. FDA Pulmonary - Allergy Drugs Advisory Committee
FDA Advisory Committee Briefing Materials Page 1 of 157 Briefing Document FDA Pulmonary - Allergy Drugs Advisory Committee Ivacaftor for the Treatment of Cystic Fibrosis in Patients Age 6 Years and Older
More informationOxidation contributes to low glutathione in the airways of children with cystic fibrosis
ORIGINAL ARTICLE CYSTIC FIBROSIS Oxidation contributes to low glutathione in the airways of children with cystic fibrosis Anthony J. Kettle 1, Rufus Turner 1, Catherine L. Gangell 2, D. Timothy Harwood
More informationThe Bacteriology of Bronchiectasis in Australian Indigenous children
The Bacteriology of Bronchiectasis in Australian Indigenous children Kim Hare, Amanda Leach, Peter Morris, Heidi Smith-Vaughan, Anne Chang Presentation outline What is bronchiectasis? Our research at Menzies
More informationGoals Basic defect Pathophysiology Clinical i l signs and symptoms Therapy
CYSTIC FIBROSIS Lynne M. Quittell, M.D. Director, CF Center Columbia University Goals Basic defect Pathophysiology Clinical i l signs and symptoms Therapy What is Cystic Fibrosis? Chronic, progressive
More informationCase Study What is the Relationship Between the Cell Membrane and Cystic Fibrosis?
Names: Date: Case Study What is the Relationship Between the Cell Membrane and Cystic Fibrosis? Dr. Weyland examined a six month old infant that had been admitted to University Hospital earlier in the
More informationClinical Trials for Rare Diseases in CDER
Clinical Trials for Rare Diseases in CDER CDER Antibacterial Task Force/CTTI Think Tank October 11, 2012 Kathryn O Connell, MD PhD Medical Officer, Rare Diseases Program Office of New Drugs, CDER, FDA
More informationCOPD: From Phenotypes to Endotypes. MeiLan K Han, M.D., M.S. Associate Professor of Medicine University of Michigan, Ann Arbor, MI
COPD: From Phenotypes to Endotypes MeiLan K Han, M.D., M.S. Associate Professor of Medicine University of Michigan, Ann Arbor, MI Presenter Disclosures MeiLan K. Han Consulting Research support Novartis
More informationEndpoints for Clinical Trials in Young Children with Cystic Fibrosis
Endpoints for Clinical Trials in Young Children with Cystic Fibrosis Stephanie D. Davis 1, Alan S. Brody 2, Mary J. Emond 3, Lyndia C. Brumback 3, and Margaret Rosenfeld 4 1 Division of Pulmonology, Department
More informationNational Horizon Scanning Centre. Mannitol dry powder for inhalation (Bronchitol) for cystic fibrosis. April 2008
Mannitol dry powder for inhalation (Bronchitol) for cystic fibrosis April 2008 This technology summary is based on information available at the time of research and a limited literature search. It is not
More informationNON-CF BRONCHIECTASIS IN ADULTS
Séminaire de Pathologie Infectieuse Jeudi 25 juin 2008 Cliniques Universitaires UCL de Mont-Godinne, Yvoir NON-CF BRONCHIECTASIS IN ADULTS Dr Robert Wilson Royal Brompton Hospital, London, UK Aetiology
More informationMacrolide therapy in cystic fibrosis: new developments in clinical use
Macrolide therapy in cystic fibrosis: new developments in clinical use Clin. Invest. (2013) 3(12), 1179 1186 Macrolide therapy, in particular azithromycin, has been shown to improve aspects of lung health
More informationE-BOOK # PANCREATIC ENZYMES FOR CYSTIC FIBROSIS ARCHIVE
10 June, 2018 E-BOOK # PANCREATIC ENZYMES FOR CYSTIC FIBROSIS ARCHIVE Document Filetype: PDF 220.67 KB 0 E-BOOK # PANCREATIC ENZYMES FOR CYSTIC FIBROSIS ARCHIVE If you have a systemic illness like lupus
More informationA Case of Cystic Fibrosis
Name(s) Date A Case of Cystic Fibrosis Dr. Weyland examined a six month old infant that had been admitted to University Hospital earlier in the day. The baby's parents had brought young Zoey to the emergency
More informationHIT-CF New clinical trial design in Cystic Fibrosis
HIT-CF New clinical trial design in Cystic Fibrosis Peter van Mourik, MD, PhD-student Pediatric Pulmonology, University Medical Center Utrecht, the Netherlands Contents - Difficulties of clinical trials
More informationLung Disease in Pediatrics: is it all in the Genes?
Lung Disease in Pediatrics: is it all in the Genes? Jay K. Kolls, M.D. Chair, Department of Genetics LSU Health Sciences Center New Orleans, LA Children s s Hospital of Pittsburgh Severe combined immunodeficiency
More informationabstract SPECIAL ARTICLE
Clinical Practice Guidelines From the Cystic Fibrosis Foundation for Preschoolers With Cystic Fibrosis Thomas Lahiri, MD, a Sarah E. Hempstead, MS, b, c Cynthia Brady, DNP, d Carolyn L. Cannon, MD, PhD,
More informationImpact of bronchiectasis and trapped air on quality of life and exacerbations in cystic fibrosis
ORIGINAL ARTICLE CYSTIC FIBROSIS Impact of bronchiectasis and trapped air on quality of life and exacerbations in cystic fibrosis Leonie A. Tepper 1,2, Elisabeth M.W.J. Utens 3, Daan Caudri 1, Aukje C.
More informationSGRQ Questionnaire assessing respiratory disease-specific quality of life. Questionnaire assessing general quality of life
SUPPLEMENTARY MATERIAL e-table 1: Outcomes studied in present analysis. Outcome Abbreviation Definition Nature of data, direction indicating adverse effect (continuous only) Clinical outcomes- subjective
More informationTitle: Lung clearance index and structural lung disease on computed tomography in early
Title: Lung clearance index and structural lung disease on computed tomography in early cystic fibrosis Authors: Kathryn A Ramsey 1,2*, Tim Rosenow 1,3*, Lidija Turkovic 1, Billy Skoric 4,5, Georgia Banton
More informationScottish Paediatric Cystic Fibrosis MCN. Protocols / Guidelines. Ivacaftor: A guideline for use in paediatric CF patients in Scotland
Scottish Paediatric Cystic Fibrosis MCN Protocols / Guidelines Ivacaftor: A guideline for use in paediatric CF patients in Scotland Authors: Dr Carol Dryden Dr Jane Wilkinson Miss Julie Crocker, Registered
More informationTargeted therapies to improve CFTR function in cystic fibrosis
Brodlie et al. Genome Medicine (2015) 7:101 DOI 10.1186/s13073-015-0223-6 REVIEW Targeted therapies to improve CFTR function in cystic fibrosis Malcolm Brodlie 1*, Iram J. Haq 2, Katie Roberts 2 and J.
More information4.6 Small airways disease
4.6 Small airways disease Author: Jean-Marc Fellrath 1. INTRODUCTION Small airways are defined as any non alveolated and noncartilaginous airway that has an internal diameter of 2 mm. Several observations
More informationNorth American Cystic Fibrosis Conference 27 October Noreen R Henig, MD Chief Development Officer ProQR Therapeutics
Proof of Concept Study to Demonstrate the Effects of QR-010 on Nasal Potential Difference in Subjects With Cystic Fibrosis with the F508del CFTR Mutation Noreen R Henig, MD Chief Development Officer ProQR
More informationWe strive to develop innovative solutions that improve and extend the lives of people with cystic fibrosis
Nivalis Therapeutics, Inc., October 2015 Analyst Breakfast at NACFC We strive to develop innovative solutions that improve and extend the lives of people with cystic fibrosis Disclaimer Regarding Forward
More informationClinical Commissioning Policy: Ivacaftor for Cystic Fibrosis (named mutations)
Clinical Commissioning Policy: Ivacaftor for Cystic Fibrosis (named mutations) Reference: NHS England: A01/P/c NHS England INFORMATION READER BOX Directorate Medical Commissioning Operations Patients and
More informationA Cure for All: Leaving No One Behind. Assuring Effective Therapies for All Patients with Cystic Fibrosis
A Cure for All: Leaving No One Behind Assuring Effective Therapies for All Patients with Cystic Fibrosis Topics for Today s Presentation Demographics of the CF patient population in the modulator era
More informationThe ecology of disease interactions in CF
1/44 The ecology of disease in CF University of Utah Department of Mathematics and Department of Biology September 4, 2014 2/44 The big questions in ecology Why are there so many species? Charley Harper
More informationCYSTIC FIBROSIS FOUNDATION INFO-POD Information You Need to Make Benefits Decisions
CYSTIC FIBROSIS FOUNDATION INFO-POD Information You Need to Make Benefits Decisions Issue 1: Hypertonic Saline Summary: Preserving lung function is a crucial element in the care of the individual with
More informationAnti-inflammatory Therapy Targets Orphan Lung Disease-June 17
Anti-inflammatory Therapy Targets Orphan Lung Disease-June 17 Disease Modification in Cystic Fibrosis (CF) Targeted Approach in Pulmonary Hypertension (PH) Experienced Management Team Greg Duncan President/CEO
More informationCaregiver burden and quality of life of parents of young children with cystic fibrosis
Caregiver burden and quality of life of parents of young children with cystic fibrosis Professor Patricia Fitzpatrick 1 S George 1, R Somerville 1, B Linnane 2, C Fitzgerald 1 1 UCD School of Public Health,
More informationConference Bronchiectasis A Growing Problem
Conference 2015 Bronchiectasis A Growing Problem Bronchiectasis (in Children) What is it? Why such a concern in NZ? What to look out for? Management Positives? Just a note that the bar for diagnosis, referral
More informationOral Cystic Fibrosis Modulators
Oral Cystic Fibrosis Modulators Goals: To ensure appropriate drug use and limit to patient populations in which they have demonstrated to be effective and safe. To monitor for clinical response for appropriate
More informationTITLE: Hypertonic Saline Nebules for Patients with Cystic Fibrosis and Bronchioectasis: A Review of the Clinical and Cost-Effectiveness
TITLE: Hypertonic Saline Nebules for Patients with Cystic Fibrosis and Bronchioectasis: A Review of the Clinical and Cost-Effectiveness DATE: 16 March 2009 CONTEXT AND POLICY ISSUES: Hypertonic saline
More informationA review of Cystic Fibrosis
A review of Cystic Fibrosis Jennifer Landry md F.R.C.P.(C) Pulmonary & Critical Care Medicine McGill University Health Center Cystic Fibrosis One of the most common lethal inherited AR disorders in the
More informationStatins in lung disease
Statins in lung disease Associate Professor Robert Young BMedSc, MBChB, DPhil (Oxon), FRACP, FRCP University of Auckland, New Zealand Smoking and its complications Respiratory COPD Cardiovascular CAD Smoking
More informationT he cystic fibrosis transmembrane conductance regulator
971 RESPIRATORY PHYSIOLOGY Nasal airway ion transport is linked to the cystic fibrosis phenotype in adult patients I Fajac, D Hubert, D Guillemot, I Honoré, T Bienvenu, F Volter, J Dall Ava-Santucci, D
More informationThe role of Lipoxin A4 in Cystic Fibrosis Lung Disease
Volume No: 6, Issue: 7, March 2013, e201303018, http://dx.doi.org/10.5936/csbj.201303018 CSBJ The role of Lipoxin A4 in Cystic Fibrosis Lung Disease Valérie Urbach a,b,*, Gerard Higgins a, Paul Buchanan
More informationPULMONARY SURFACTANT, ALPHA 1 ANTITRYPSIN INHIBITOR DEFICIENCY, AND CYSTIC FIBROSIS DR. NABIL BASHIR BIOCHEMISTRY/RESPIRATORY SYSTEM
PULMONARY SURFACTANT, ALPHA 1 ANTITRYPSIN INHIBITOR DEFICIENCY, AND CYSTIC FIBROSIS DR. NABIL BASHIR BIOCHEMISTRY/RESPIRATORY SYSTEM Pulmonary surfactant Pulmonary surfactant is (phospholipoprotein) complex
More informationCystic fibrosis: From the gene to the disease
Cystic fibrosis: From the gene to the disease Christiane Knoop, MD, PhD Institut de Mucoviscidose de l ULB Hôpital Erasme christiane.knoop@erasme.ulb.ac.be Cystic fibrosis «The infant that tastes of salt
More informationEfficacy of NaCl nebulized hypertonic solutions in cystic fibrosis
Acta Biomed 2014; Vol. 85, Supplement 4: 10-18 Mattioli 1885 Original article Efficacy of NaCl nebulized hypertonic solutions in cystic fibrosis Azienda Ospedaliero Universitaria Policlinico, DAI Scienze
More informationDisclosures. Learning Objectives. What is Cystic Fibrosis? Background
39 th National Conference on Pediatric Health Care March 19-22, 2018 CHICAGO Disclosures The Vision and the Journey of Cystic Fibrosis: Newborn Screening to Breakthrough Therapy March 20, 2018 Cynthia
More informationCyanide in bronchoalveolar lavage is not diagnostic for Pseudomonas aeruginosa in children with cystic fibrosis
Eur Respir J 2011; 37: 553 558 DOI: 10.1183/09031936.00024210 CopyrightßERS 2011 Cyanide in bronchoalveolar lavage is not diagnostic for Pseudomonas aeruginosa in children with cystic fibrosis M.D. Stutz*,#,
More informationAUSTRALIAN CYSTIC FIBROSIS DATA REGISTRY ANNUAL REPORT This publication was produced with the support of Cystic Fibrosis Australia.
AUSTRALIAN CYSTIC FIBROSIS DATA REGISTRY ANNUAL REPORT 2017 This publication was produced with the support of Cystic Fibrosis Australia. CONTENTS FOREWORD 1 INTRODUCTION 2 DATA PERIOD 2 ABBREVIATIONS 2
More informationPA Update: Oral Cystic Fibrosis Modulators
Copyright 2012 Oregon State University. All Rights Reserved Drug Use Research & Management Program Oregon State University, 500 Summer Street NE, E35 Salem, Oregon 97301-1079 Phone 503-947-5220 Fax 503-947-1119
More informationHow to Form a Research Question. Insight CF Registry Research Project
How to Form a Research Question Insight CF Registry Research Project Insight CF Registry Research Project Thank you for your interest in the Insight CF Registry Research Project. The goal of this training
More informationCystic fibrosis and survival to 40 years: a study of cystic fibrosis transmembrane conductance regulator function
Eur Respir J 2011; 37: 1076 1082 DOI: 10.1183/09031936.00079010 CopyrightßERS 2011 Cystic fibrosis and survival to 40 years: a study of cystic fibrosis transmembrane conductance regulator function N.J.
More informationDr Conroy Wong. Professor Richard Beasley. Dr Sarah Mooney. Professor Innes Asher
Professor Richard Beasley University of Otago Director Medical Research Institute of New Zealand Wellington Dr Sarah Mooney Physiotherapy Advanced Clinician Counties Manukau Health NZ Respiratory and Sleep
More informationBronchiectasis. What is bronchiectasis? What causes bronchiectasis?
This factsheet explains what bronchiectasis is, what causes it, and how it is diagnosed and managed. More detailed information is available on the Bronchiectasis Patient Priorities website: www.europeanlunginfo.org/bronchiectasis
More informationA GUIDE TO: CYSTIC FIBROSIS FOR HEALTH PROFESSIONALS
A GUIDE TO: CYSTIC FIBROSIS FOR HEALTH PROFESSIONALS CYSTIC FIBROSIS FOR HEALTH PROFESSIONALS Contents 1. INTRODUCTION 03 2. THE CF GENE 04 3. CF DIAGNOSIS AND SCREENING 05 4. CLINICAL MANIFESTATIONS 06
More informationThe role of serum Pseudomonas aeruginosa antibodies in the diagnosis and follow-up of cystic fibrosis
The Turkish Journal of Pediatrics 2013; 55: 50-57 Original The role of serum Pseudomonas aeruginosa antibodies in the diagnosis and follow-up of cystic fibrosis Deniz Doğru 1, Sevgi Pekcan 1, Ebru Yalçın
More informationA GUIDE TO: CYSTIC FIBROSIS FOR HEALTH PROFESSIONALS
A GUIDE TO: CYSTIC FIBROSIS FOR HEALTH PROFESSIONALS Contents 1. Introduction 1. INTRODUCTION 03 2. THE CF GENE 04 8. PREVENTION OF 13 CROSS INFECTION 9. LUNG TRANSPLANTATION 13 Cystic Fibrosis (CF) is
More informationCystic Fibrosis. Advances and Asian Perspective Dr AS Paul 13 October 06
Cystic Fibrosis Advances and Asian Perspective Dr AS Paul 13 October 06 Cystic Fibrosis An Overview Asian Perspective Advances Cystic Fibrosis An Overview Epidemiology and Pathogenesis Most common severe
More informationDiseases of the gastrointestinal system. H Awad Lecture 2: small intestine/ part 2 and appendix
Diseases of the gastrointestinal system H Awad Lecture 2: small intestine/ part 2 and appendix Malabsorption most important causes of malabsorption: Celiac disease tropical sprue Lactase deficiency Whipple
More informationGlossary. Acronyms used in cystic fibrosis peer review reports
Glossary Acronyms used in cystic fibrosis peer review reports Acronym Definition Notes AC(T) Airway clearance (techniques) A term used in physiotherapy ACB Association of Clinical Biochemists Professional
More informationLong term azithromycin therapy in patients with cystic fibrosis
The Turkish Journal of Pediatrics 2016; 58: 34-40 Original Long term azithromycin therapy in patients with cystic fibrosis Nagehan Emiralioğlu 1, Zeynelabidin Öztürk 2, Ebru Yalçın 1, Deniz Doğru 1, Uğur
More informationSupplementary appendix
Supplementary appendix This appendix formed part of the original submission and has been peer reviewed. We post it as supplied by the authors. Supplement to: Wells JM, Farris RF, Gosdin TA, et al. Pulmonary
More informationClinical Commissioning Policy: Dornase alfa inhaled therapy for primary ciliary dyskinesia (all ages)
Clinical Commissioning Policy: Dornase alfa inhaled therapy for primary ciliary dyskinesia (all ages) Reference: NHS England: 16029/P NHS England INFORMATION READER BOX Directorate Medical Operations and
More information