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Ivacaftor

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https://www.readbyqxmd.com/read/28325531/real-life-initiation-of-lumacaftor-ivacaftor-combination-in-adults-with-cystic-fibrosis-homozygous-for-the-phe508del-cftr-mutation-and-severe-lung-disease
#1
Dominique Hubert, Raphaël Chiron, Boubou Camara, Dominique Grenet, Anne Prévotat, Laurence Bassinet, Stéphane Dominique, Gilles Rault, Julie Macey, Isabelle Honoré, Reem Kanaan, Sylvie Leroy, Nadine Desmazes Dufeu, Pierre-Régis Burgel
OBJECTIVE: To investigate the short-term adverse events and effectiveness of lumacaftor/ivacaftor combination treatment in adults with cystic fibrosis (CF) and severe lung disease in a real life setting. METHODS: A multicentre observational study investigated adverse events, treatment discontinuation, FEV1 and body mass index (BMI) one month and three months after lumacaftor/ivacaftor initiation in adults with CF and FEV1 below 40% predicted. RESULTS: Respiratory adverse events (AEs) were reported by 27 of 53 subjects (51%) and 16 (30%) discontinued treatment...
March 15, 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/28314539/immediate-effects-of-lumacaftor-ivacaftor-administration-on-lung-function-in-patients-with-severe-cystic-fibrosis-lung-disease
#2
Natalia Popowicz, Jamie Wood, Anna Tai, Sue Morey, Siobhain Mulrennan
Safety-data for lumacaftor/ivacaftor (LUM/IVA) combination therapy in patients with severe lung disease (percent predicted forced expiratory volume in 1s [ppFEV1] <40) remain limited. We report immediate post-dose respiratory-related adverse events in 12 patients with severe cystic fibrosis (CF) lung disease (median [IQR] ppFEV1: 34 [31-36]) prescribed LUM/IVA. All patients experienced a decline in ppFEV1 from baseline at 2-hours (median [IQR] relative change: -19 [-21 to -11]%, p<0.001) that persisted at 24-hours but recovered in most patients at 1-month...
March 14, 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/28300821/impact-of-cftr-modulation-on-intestinal-ph-motility-and-clinical-outcomes-in-patients-with-cystic-fibrosis-and-the-g551d-mutation
#3
Daniel Gelfond, Sonya Heltshe, Changxing Ma, Steven M Rowe, Carla Frederick, Ahmet Uluer, Leonard Sicilian, Michael Konstan, Elizabeth Tullis, R N Christine Roach, Katherine Griffin, Elizabeth Joseloff, Drucy Borowitz
OBJECTIVES: A defect in bicarbonate secretion contributes to the pathophysiology of gastrointestinal complications in patients with cystic fibrosis (CF). We measured gastrointestinal pH, clinical outcomes, and intestinal transit profiles in patients with the G551D mutation before and after treatment with ivacaftor, a CF transmembrane regulator channel (CFTR) potentiator. METHODS: Observational studies of ivacaftor effectiveness were conducted in the United States and Canada...
March 16, 2017: Clinical and Translational Gastroenterology
https://www.readbyqxmd.com/read/28222269/restoring-cftr-function-reduces-airway-bacteria-and-inflammation-in-people-with-cystic-fibrosis-and-chronic-lung-infections
#4
Katherine B Hisert, Sonya L Heltshe, Christopher Pope, Peter Jorth, Xia Wu, Rachael M Edwards, Matthew Radey, Frank J Accurso, Daniel J Wolter, Gordon Cooke, Ryan J Adam, Suzanne Carter, Brenda Grogan, Jan L Launspach, Seamas C Donnelly, Charles Gallagher, James E Bruce, David Stoltz, Michael J Welsh, Lucas R Hoffman, Edward F McKone, Pradeep K Singh
RATIONALE: Previous work indicates that ivacaftor improves CFTR activity and lung function in people with cystic fibrosis (CF) and G551D-CFTR mutations, but does not reduce density of bacteria or markers of inflammation in the airway. These findings raise the possibility that infection and inflammation may progress independently of CFTR activity once CF lung disease is established. OBJECTIVES: To better understand the relationship between CFTR activity, airway microbiology and inflammation, and lung function in subjects with CF and chronic airway infections...
February 21, 2017: American Journal of Respiratory and Critical Care Medicine
https://www.readbyqxmd.com/read/28190780/pregnancy-among-cystic-fibrosis-women-in-the-era-of-cftr-modulators
#5
Sonya L Heltshe, Emily M Godfrey, Tatiana Josephy, Moira L Aitken, Jennifer L Taylor-Cousar
BACKGROUND: Little is known about how new therapies that partially correct the basic cystic fibrosis (CF) defect (ivacaftor and lumacaftor) might alter hormonal contraceptive effectiveness, impact pregnancy outcomes, or affect pregnancy timing. Examination of pregnancy rates among CF women during periods of CFTR modulator therapy initiation will provide foundation for further research in this area. METHODS: The Cystic Fibrosis Foundation Patient Registry was used to examine pregnancy rates and outcomes by genotype class before, during, and after the introduction of CFTR modulator therapies between 2005 and 2014...
February 9, 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/28143919/growth-in-prepubertal-children-with-cystic-fibrosis-treated-with-ivacaftor
#6
Michael S Stalvey, Jesse Pace, Minoo Niknian, Mark N Higgins, Valerie Tarn, Joy Davis, Sonya L Heltshe, Steven M Rowe
BACKGROUND AND OBJECTIVES: Cystic fibrosis (CF) is known for its impact on the lung and pancreas of individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may affect growth outcomes. METHODS: In this post hoc analysis, we assessed linear growth and weight in 83 children (aged 6-11 years) enrolled in 2 clinical trials, the longitudinal-observation GOAL study and the placebo-controlled ENVISION study, to evaluate the effects of ivacaftor, a CFTR potentiator...
February 2017: Pediatrics
https://www.readbyqxmd.com/read/28132845/use-of-hyperpolarized-helium-3-mri-to-assess-response-to-ivacaftor-treatment-in-patients-with-cystic-fibrosis
#7
Talissa A Altes, Mac Johnson, Meredith Fidler, Martyn Botfield, Nicholas J Tustison, Carlos Leiva-Salinas, Eduard E de Lange, Deborah Froh, John P Mugler
BACKGROUND: This pilot study evaluated the effect of short- and long-term ivacaftor treatment on hyperpolarized (3)He-magnetic resonance imaging (MRI)-defined ventilation defects in patients with cystic fibrosis aged ≥12years with a G551D-CFTR mutation. METHODS: Part A (single-blind) comprised 4weeks of ivacaftor treatment; Part B (open-label) comprised 48weeks of treatment. The primary outcome was change from baseline in total ventilation defect (TVD; total defect volume:total lung volume ratio)...
March 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/28107795/adherence-to-therapies-in-cystic-fibrosis-a-targeted-literature-review
#8
Siva Narayanan, Jochen G Mainz, Smeet Gala, Harold Tabori, Daniel Grossoehme
Cystic fibrosis (CF) is a life-shortening condition with no cure. Available therapies relieving the symptoms of CF are complex and time-consuming. A comprehensive review assessing adherence to different CF therapies, association of adherence with outcomes, and factors influencing adherence could inform optimal patient management strategies. Areas covered: A targeted literature review of studies published from 2010-2016 assessed adherence to CF therapies. Nineteen studies qualified for inclusion. Adherence to CF therapies was sub-optimal, and varied by treatment, mode of treatment administration, age, season, time and method of adherence measurement...
January 20, 2017: Expert Review of Respiratory Medicine
https://www.readbyqxmd.com/read/28087700/two-small-molecules-restore-stability-to-a-sub-population-of-the-cystic-fibrosis-transmembrane-conductance-regulator-with-the-predominant-disease-causing-mutation
#9
Xin Meng, Yiting Wang, Xiaomeng Wang, Joe A Wrennall, Tracy L Rimington, Hongyu Li, Zhiwei Cai, Robert C Ford, David N Sheppard
Cystic fibrosis (CF) is caused by mutations that disrupt the plasma membrane expression, stability, and function of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel. Two small molecules, the CFTR corrector lumacaftor and the potentiator ivacaftor, are now used clinically to treat CF, although some studies suggest that they have counteracting effects on CFTR stability. Here, we investigated the impact of these compounds on the instability of F508del-CFTR, the most common CF mutation...
January 13, 2017: Journal of Biological Chemistry
https://www.readbyqxmd.com/read/28068001/in-vivo-and-in-vitro-ivacaftor-response-in-cystic-fibrosis-patients-with-residual-cftr-function-n-of-1-studies
#10
Meghan E McGarry, Beate Illek, Ngoc P Ly, Lorna Zlock, Sabrina Olshansky, Courtney Moreno, Walter E Finkbeiner, Dennis W Nielson
RATIONALE: Ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, decreases sweat chloride concentration, and improves pulmonary function in 6% of cystic fibrosis (CF) patients with specific CFTR mutations. Ivacaftor increases chloride transport in many other CFTR mutations in non-human cells, if CFTR is in the epithelium. Some CF patients have CFTR in the epithelium with residual CFTR function. The effect of ivacaftor in these patients is unknown. METHODS: This was a series of randomized, crossover N-of-1 trials of ivacaftor and placebo in CF patients ≥8 years old with potential residual CFTR function (intermediate sweat chloride concentration, pancreatic sufficient, or mild bronchiectasis on chest CT)...
January 9, 2017: Pediatric Pulmonology
https://www.readbyqxmd.com/read/28042521/cftr-modulator-therapies-in-pediatric-cystic-fibrosis-focus-on-ivacaftor
#11
Elizabeth L Kramer, John P Clancy
INTRODUCTION: Mutations in the cystic fibrosis transmembrane conductance regulator protein (CFTR) cause cystic fibrosis (CF), a disease with life threatening pulmonary and gastrointestinal manifestations. Recent breakthrough therapies restore function to select disease-causing CFTR mutations. Ivacaftor is a small molecule that increases the open channel probability of certain CFTR mutations, producing clear evidence of bioactivity and efficacy in pediatric CF patients. CFTR modulators represent a significant advancement in CF treatment...
October 2016: Expert Opinion on Orphan Drugs
https://www.readbyqxmd.com/read/28012258/functional-defect-of-variants-in-the-adenosine-triphosphate-binding-sites-of-abcb4-and-their-rescue-by-the-cystic-fibrosis-transmembrane-conductance-regulator-potentiator-ivacaftor-vx-770
#12
Jean-Louis Delaunay, Alix Bruneau, Brice Hoffmann, Anne-Marie Durand-Schneider, Véronique Barbu, Emmanuel Jacquemin, Michèle Maurice, Chantal Housset, Isabelle Callebaut, Tounsia Aït-Slimane
ABCB4 (MDR3) is an adenosine triphosphate (ATP)-binding cassette (ABC) transporter expressed at the canalicular membrane of hepatocytes, where it mediates phosphatidylcholine (PC) secretion. Variations in the ABCB4 gene are responsible for several biliary diseases, including progressive familial intrahepatic cholestasis type 3 (PFIC3), a rare disease that can be lethal in the absence of liver transplantation. In this study, we investigated the effect and potential rescue of ABCB4 missense variations that reside in the highly conserved motifs of ABC transporters, involved in ATP binding...
February 2017: Hepatology: Official Journal of the American Association for the Study of Liver Diseases
https://www.readbyqxmd.com/read/28011037/assessment-of-safety-and-efficacy-of-long-term-treatment-with-combination-lumacaftor-and-ivacaftor-therapy-in-patients-with-cystic-fibrosis-homozygous-for-the-f508del-cftr-mutation-progress-a-phase-3-extension-study
#13
Michael W Konstan, Edward F McKone, Richard B Moss, Gautham Marigowda, Simon Tian, David Waltz, Xiaohong Huang, Barry Lubarsky, Jaime Rubin, Stefanie J Millar, David J Pasta, Nicole Mayer-Hamblett, Christopher H Goss, Wayne Morgan, Gregory S Sawicki
BACKGROUND: The 24-week safety and efficacy of lumacaftor/ivacaftor combination therapy was shown in two randomised controlled trials (RCTs)-TRAFFIC and TRANSPORT-in patients with cystic fibrosis who were aged 12 years or older and homozygous for the F508del-CFTR mutation. We aimed to assess the long-term safety and efficacy of extended lumacaftor/ivacaftor therapy in this group of patients in PROGRESS, the long-term extension of TRAFFIC and TRANSPORT. METHODS: PROGRESS was a phase 3, parallel-group, multicentre, 96-week study of patients who completed TRAFFIC or TRANSPORT in 191 sites in 15 countries...
February 2017: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/27990075/pharmaceutical-approval-update
#14
Michele B Kaufman
Lisinopril oral solution (Qbrelis) for the treatment of hypertension, heart failure, and acute myocardial infarction; etanercept-szzs (Erelzi) for multiple autoimmune disorders; and lumacaftor/ivacaftor (Orkambi) for cystic fibrosis.
December 2016: P & T: a Peer-reviewed Journal for Formulary Management
https://www.readbyqxmd.com/read/27976892/fatty-acid-cysteamine-conjugates-as-novel-and-potent-autophagy-activators-that-enhance-the-correction-of-misfolded-f508del-cystic-fibrosis-transmembrane-conductance-regulator-cftr
#15
Chi B Vu, Robert J Bridges, Cecilia Pena-Rasgado, Antonio E Lacerda, Curtis Bordwell, Abby Sewell, Andrew J Nichols, Sachin Chandran, Pallavi Lonkar, Dominic Picarella, Amal Ting, Allison Wensley, Maisy Yeager, Feng Liu
A depressed autophagy has previously been reported in cystic fibrosis patients with the common F508del-CFTR mutation. This report describes the synthesis and preliminary biological characterization of a novel series of autophagy activators involving fatty acid cysteamine conjugates. These molecular entities were synthesized by first covalently linking cysteamine to docosahexaenoic acid. The resulting conjugate 1 synergistically activated autophagy in primary homozygous F508del-CFTR human bronchial epithelial (hBE) cells at submicromolar concentrations...
January 12, 2017: Journal of Medicinal Chemistry
https://www.readbyqxmd.com/read/27970879/cost-effectiveness-of-ivacaftor-and-lumacaftor-combination-for-the-treatment-of-patients-with-cystic-fibrosis-in-the-united-states
#16
D Sharma, S Xing, Y Hung, R N Caskey, M L Dowell, D R Touchette
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27898234/lumacaftor-ivacaftor-treatment-of-patients-with-cystic-fibrosis-heterozygous-for-f508del-cftr
#17
Steven M Rowe, Susanna A McColley, Ernst Rietschel, Xiaolei Li, Scott C Bell, Michael W Konstan, Gautham Marigowda, David Waltz, Michael P Boyle
RATIONALE: In a prior study, lumacaftor/ivacaftor treatment (≤28 d) in patients with cystic fibrosis (CF) heterozygous for F508del-CFTR did not improve lung function. OBJECTIVES: To evaluate an optimized lumacaftor/ivacaftor dosing regimen with a longer duration in a cohort of patients heterozygous for F508del-CFTR. METHODS: Patients aged 18 years or older with a confirmed CF diagnosis and percent predicted FEV1 (ppFEV1) of 40 to 90 were randomized to lumacaftor/ivacaftor (400 mg/250 mg every 12 h) or placebo daily for 56 days...
February 2017: Annals of the American Thoracic Society
https://www.readbyqxmd.com/read/27894875/effect-of-bronchodilators-in-healthy-individuals-receiving-lumacaftor-ivacaftor-combination-therapy
#18
Gautham Marigowda, Fang Liu, David Waltz
In an open-label, single-center phase 1 pharmacokinetic study in healthy subjects who received lumacaftor (LUM) in combination with ivacaftor (IVA), review of spirometry data showed a transient decline in percent predicted forced expiratory volume in 1s (ppFEV1) within 4h of drug administration. An additional cohort of healthy subjects with normal baseline ppFEV1 values was studied to evaluate the ppFEV1 response to LUM/IVA administration and assess the effect of long-acting bronchodilators (LABDs) and short-acting bronchodilators (SABDs) on ppFEV1 response...
March 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/27875677/a-case-report-of-pregnancy-during-use%C3%A2-of-targeted-therapeutics-for-cystic%C3%A2-fibrosis
#19
Sigrid Ladores, Traci M Kazmerski, Steven M Rowe
New therapeutics, such as ivacaftor, and the combination drug lumacaftor/ivacaftor that target the underlying genetic cause of cystic fibrosis are being hailed as game-changers in this era of personalized medicine. Although these drugs improve lung function, their effects on female fertility have not been studied. In this case report we describe one woman's experience with ivacaftor and her unanticipated pregnancy. Implications related to comprehensive sexual and reproductive health care for women with cystic fibrosis are presented...
January 2017: Journal of Obstetric, Gynecologic, and Neonatal Nursing: JOGNN
https://www.readbyqxmd.com/read/27812499/the-use-of-ivacaftor-in-cftr-mutations-resulting-in-residual-functioning-protein
#20
S Guigui, J Wang, R I Cohen
INTRODUCTION: Ivacaftor, a cystic fibrosis transmembrane regulator (CFTR) potentiator is currently approved for use in individuals with class III gating mutations and the R117H mutation, a non-gating mutation with residual functioning CFTR. Nevertheless, ivacaftor may also be effective in individuals who have CF mutations giving rise to a residual functioning protein. However, aside from case reports involving a single patient, little data exist on the use of ivacaftor in such individuals...
2016: Respiratory Medicine Case Reports
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