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Ivacaftor

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https://www.readbyqxmd.com/read/29773589/ivacaftor-for-cystic-fibrosis
#1
Peter J Barry, Anna L Donaldson, Andrew M Jones
No abstract text is available yet for this article.
May 17, 2018: BMJ: British Medical Journal
https://www.readbyqxmd.com/read/29750923/ion-channel-modulators-in-cystic-fibrosis
#2
REVIEW
Martina Gentzsch, Marcus A Mall
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and remains one of the most common life-shortening genetic diseases affecting the lung and other organs. CFTR functions as a cAMP-dependent anion channel that transports chloride and bicarbonate across epithelial surfaces and disruption of these ion transport processes plays a central role in the pathogenesis of CF. These findings provided the rationale for pharmacological modulation of ion transport, either by targeting mutant CFTR or alternative ion channels that can compensate for CFTR dysfunction, as a promising therapeutic approach...
May 8, 2018: Chest
https://www.readbyqxmd.com/read/29748252/data-from-the-us-and-uk-cystic-fibrosis-registries-support-disease-modification-by-cftr-modulation-with-ivacaftor
#3
Leona Bessonova, Nataliya Volkova, Mark Higgins, Leif Bengtsson, Simon Tian, Christopher Simard, Michael W Konstan, Gregory S Sawicki, Ase Sewall, Stephen Nyangoma, Alexander Elbert, Bruce C Marshall, Diana Bilton
BACKGROUND: Ivacaftor is the first cystic fibrosis transmembrane conductance regulator (CFTR) modulator demonstrating clinical benefit in patients with cystic fibrosis (CF). As ivacaftor is intended for chronic, lifelong use, understanding long-term effects is important for patients and healthcare providers. OBJECTIVE: This ongoing, observational, postapproval safety study evaluates clinical outcomes and disease progression in ivacaftor-treated patients using data from the US and the UK CF registries following commercial availability...
May 10, 2018: Thorax
https://www.readbyqxmd.com/read/29733727/precision-medicine-in-action-the-impact-of-ivacaftor-on-cystic-fibrosis-related-hospitalizations
#4
Lisa B Feng, Scott D Grosse, Ridgely Fisk Green, Aliza K Fink, Gregory S Sawicki
Cystic fibrosis is a life-threatening genetic disease that causes severe damage to the lungs. Ivacaftor, the first drug that targeted the underlying defect of the disease caused by specific mutations, is a sterling example of the potential of precision medicine. Clinical trial and registry studies showed that ivacaftor improved outcomes and reduced hospitalizations. Our study used US administrative claims data to assess the real-world effectiveness of ivacaftor. Comparing twelve-month rates before and after starting the use of ivacaftor among people who initiated therapy during 2012-2015, we found that overall and cystic fibrosis-related inpatient admissions fell by 55 percent and 81 percent, respectively...
May 2018: Health Affairs
https://www.readbyqxmd.com/read/29717552/impact-of-a-cystic-fibrosis-transmembrane-conductance-regulator-cftr-modulator-on-high-dose-ibuprofen-therapy-in-pediatric-cystic-fibrosis-patients
#5
Brittany A Bruch, Sachinkumar B Singh, Laura J Ramsey, Timothy D Starner
BACKGROUND: This study was undertaken to determine if a clinically relevant drug-drug interaction occurred between ibuprofen and lumacaftor/ivacaftor. METHODS: Peak ibuprofen plasma concentrations were measured prior to and after lumacaftor/ivacaftor initiation. A Wilcoxon signed rank sum test was used to compare the values. RESULTS: Nine patients were included in the final analysis. Peak ibuprofen plasma concentrations decreased an average of 36...
May 1, 2018: Pediatric Pulmonology
https://www.readbyqxmd.com/read/29685811/effectiveness-of-ivacaftor-in-cystic-fibrosis-patients-with-non-g551d-gating-mutations
#6
Jennifer Guimbellot, George M Solomon, Arthur Baines, Sonya L Heltshe, Jill VanDalfsen, Elizabeth Joseloff, Scott D Sagel, Steven M Rowe
BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator ivacaftor is approved for patients with CF with gating and residual function CFTR mutations. We report the results of an observational study investigating its effects in CF patients with non-G551D gating mutations. METHODS: Patients with non-G551D gating mutations were recruited to an open-label study evaluating ivacaftor. Primary outcomes included: lung function, sweat chloride, weight gain, and quality of life scores...
April 20, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29627168/cystic-fibrosis-papers-of-the-year-2017
#7
REVIEW
Iolo Doull
The number of published articles on Cystic Fibrosis (CF) continues to increase year on year. The evidence base for small molecule therapies in CF has continued to expand, with evidence for lumacaftor/ivacaftor in younger patients and longer-term evidence in adults, and pivotal studies on tezacaftor/ivacaftor. There were reports on emerging CFTR mutation agnostic therapies, and new evidence for long standing therapies.
March 15, 2018: Paediatric Respiratory Reviews
https://www.readbyqxmd.com/read/29614238/changes-in-lung-clearance-index-in-preschool-cystic-fibrosis-patients-treated-with-ivacaftor
#8
Felix Ratjen, Michelle Klingel, Philip Black, Michael R Powers, Hartmut Grasemann, Melinda Solomon, Scott D Sagel, Scott H Donaldson, Steven M Rowe, Margaret Rosenfeld
No abstract text is available yet for this article.
April 3, 2018: American Journal of Respiratory and Critical Care Medicine
https://www.readbyqxmd.com/read/29595119/cytochrome-p450-3a4-induction-lumacaftor-versus-ivacaftor-potentially-resulting-in-significantly-reduced-plasma-concentration-of-ivacaftor
#9
Elena K Schneider
Since release of ivacaftor-lumacaftor several red-flags have been raised that highlight the clinical efficacy of this combination strategy may be be limited due to antagonistic drug-drug interactions. </p><p> Method: The effect of ivacaftor, its major metabolites M1 and M6, lumacaftor and the novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator tezacaftor at 10 µg/mL on the enzymatic activity of the major xenobiotic metabolizing enzymes CYP1A2 and CYP3A4 as well as the minor enzymes CYP2B6 and CYP2C9 was assayed...
March 27, 2018: Drug Metabolism Letters
https://www.readbyqxmd.com/read/29560360/the-potentially-beneficial-central-nervous-system-activity-profile-of-ivacaftor-and-its-metabolites
#10
Elena K Schneider, Rachel M McQuade, Vincenzo C Carbone, Felisa Reyes-Ortega, John W Wilson, Brenda Button, Ayame Saito, Daniel P Poole, Daniel Hoyer, Jian Li, Tony Velkov
Ivacaftor-lumacaftor and ivacaftor are two new breakthrough cystic fibrosis transmembrane conductance modulators. The interactions of ivacaftor and its two metabolites hydroxymethylivacaftor (iva-M1) and ivacaftorcarboxylate (iva-M6) with neurotransmitter receptors were investigated in radioligand binding assays. Ivacaftor displayed significant affinity to the 5-hydroxytryptamine (5-HT; serotonin) 5-HT2C receptor (p K i =6.06±0.03), β3 -adrenergic receptor (p K i =5.71±0.07), δ-opioid receptor (p K i =5...
January 2018: ERJ Open Research
https://www.readbyqxmd.com/read/29549268/sites-associated-with-kalydeco-binding-on-human-cystic-fibrosis-transmembrane-conductance-regulator-revealed-by-hydrogen-deuterium-exchange
#11
Laura J Byrnes, Yingrong Xu, Xiayang Qiu, Justin D Hall, Graham M West
Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). Mutations associated with CF cause loss-of-function in CFTR leading to salt imbalance in epithelial tissues. Kalydeco (also called VX-770 or ivacaftor) was approved for CF treatment in 2012 but little is known regarding the compound's interactions with CFTR including the site of binding or mechanisms of action. In this study we use hydrogen/deuterium exchange (HDX) coupled with mass spectrometry to assess the conformational dynamics of a thermostabilized form of CFTR in apo and ligand-bound states...
March 16, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29538046/cystic-fibrosis-transmembrane-conductance-regulator-modulators-precision-medicine-in-cystic-fibrosis
#12
Elizabeth B Burgener, Richard B Moss
PURPOSE OF REVIEW: The aim of this study was to describe the newest development in cystic fibrosis (CF) care, CF transmembrane conductance regulator (CFTR) modulator therapies. RECENT FINDINGS: Phase II results showing CFTR modulator triple therapies are more effective than current CFTR modulators. SUMMARY: CFTR modulator therapy targets the protein defective in CF and boosts its function, but the drug must match mutation pathobiology. Ivacaftor, a CFTR potentiator, was the first modulator approved in 2012, with impressive improvement in lung function and other measures of disease in patients with gating and other residual function mutations (∼10% of CF patients)...
June 2018: Current Opinion in Pediatrics
https://www.readbyqxmd.com/read/29512893/outcome-evaluation-of-a-pharmacy-based-therapy-management-program-for-patients-with-cystic-fibrosis
#13
Heather S Kirkham, Francis Staskon, Nishita Hira, Darren McLane, Karl M Kilgore, Alexis Parente, Seung Kim, Gregory S Sawicki
OBJECTIVE: To compare medication adherence, pulmonary exacerbations, healthcare utilization, and costs for patients with cystic fibrosis (CF) who utilized a pharmacy-based therapy management program to a matched control group. We hypothesized that patient management services would be associated with better medication adherence, and thus require fewer visits to the emergency room or hospitalizations. METHODS: This retrospective, observational cohort study used claims data from the MORE2 claims Registry®...
March 7, 2018: Pediatric Pulmonology
https://www.readbyqxmd.com/read/29497617/factors-influencing-readthrough-therapy-for-frequent-cystic-fibrosis-premature-termination-codons
#14
Iwona Pranke, Laure Bidou, Natacha Martin, Sandra Blanchet, Aurélie Hatton, Sabrina Karri, David Cornu, Bruno Costes, Benoit Chevalier, Danielle Tondelier, Emmanuelle Girodon, Matthieu Coupet, Aleksander Edelman, Pascale Fanen, Olivier Namy, Isabelle Sermet-Gaudelus, Alexandre Hinzpeter
Premature termination codons (PTCs) are generally associated with severe forms of genetic diseases. Readthrough of in-frame PTCs using small molecules is a promising therapeutic approach. Nonetheless, the outcome of preclinical studies has been low and variable. Treatment efficacy depends on: 1) the level of drug-induced readthrough, 2) the amount of target transcripts, and 3) the activity of the recoded protein. The aim of the present study was to identify, in the cystic fibrosis transmembrane conductance regulator (CFTR) model, recoded channels from readthrough therapy that may be enhanced using CFTR modulators...
January 2018: ERJ Open Research
https://www.readbyqxmd.com/read/29488691/sensitivity-of-ivacaftor-to-drug-drug-interactions-with-rifampin-a-cytochrome-p450-3a4-inducer
#15
Jennifer S Guimbellot, Edward P Acosta, Steven M Rowe
The CFTR potentiator ivacaftor is responsible for significant clinical improvements among a subset of patients with cystic fibrosis. Because it is a substrate of the cytochrome P450 system, specifically CYP3A4/5, ivacaftor is subject to significant drug-drug interactions, including due to commonly used antimicrobials such as rifampin. While the interaction of rifampin and ivacaftor has been examined in vitro, severe adverse events resulting from this interaction have not been reported in the literature. In this report, we describe the termination of steady, long-term improvement in a patient taking ivacaftor, resulting from the use of rifampin and precipitating a significant pulmonary exacerbation...
May 2018: Pediatric Pulmonology
https://www.readbyqxmd.com/read/29461009/metabolomic-responses-to-lumacaftor-ivacaftor-in-cystic-fibrosis
#16
Benjamin T Kopp, Scott McCulloch, Chandra L Shrestha, Shuzhong Zhang, Lisa Sarzynski, Frederick W Woodley, Don Hayes
BACKGROUND: Cystic fibrosis (CF) is a life-limiting disease caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Lumacaftor/Ivacaftor is a novel CFTR modulator approved for patients that are homozygous for Phe508del CFTR, but its clinical effectiveness varies amongst patients, making it difficult to determine clinical responders. Therefore, identifying biochemical biomarkers associated with drug response are clinically important for follow-up studies...
May 2018: Pediatric Pulmonology
https://www.readbyqxmd.com/read/29451946/first-experience-in-switzerland-in-phe508del-homozygous-cystic-fibrosis-patients-with-end-stage-pulmonary-disease-enrolled-in-a-lumacaftor-ivacaftor-therapy-trial-preliminary-results
#17
Christian Murer, Lars Christian Huber, Thomas Kurowski, Astrid Hirt, Cécile A Robinson, Urs Bürgi, Christian Benden
AIMS OF THE STUDY: Cystic fibrosis is the most common genetic disorder in Caucasians. The combination of the cystic fibrosis transmembrane conductance regulator (CFTR) corrector lumacaftor / potentiator ivacaftor (LUM/IVA) has been shown to increase forced expiratory volume in 1 second (FEV1) moderately, but predominantly reduce acute exacerbation rate (AER) in Phe508del homozygous cystic fibrosis patients; however, patients with FEV1 <40% predicted were excluded from studies. We used LUM/IVA on a "compassionate use" basis in cystic fibrosis patients with end-stage pulmonary disease...
February 16, 2018: Swiss Medical Weekly
https://www.readbyqxmd.com/read/29412515/l-methionine-anti-biofilm-activity-against-pseudomonas-aeruginosa-is-enhanced-by-the-cystic-fibrosis-transmembrane-conductance-regulator-potentiator-ivacaftor
#18
Do-Yeon Cho, Dong-Jin Lim, Calvin Mackey, Christopher G Weeks, Jaime A Peña Garcia, Daniel Skinner, Jessica W Grayson, Harrison S Hill, David K Alexander, Shaoyan Zhang, Bradford A Woodworth
BACKGROUND: Biofilms may contribute to refractory chronic rhinosinusitis (CRS), as they lead to antibiotic resistance and failure of effective clinical treatment. l-Methionine is an amino acid with reported biofilm-inhibiting properties. Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator with mild antimicrobial activity via inhibition of bacterial DNA gyrase and topoisomerase IV. The objective of this study was to evaluate whether co-treatment with ivacaftor and l-methionine can reduce the formation of Pseudomonas aeruginosa biofilms...
May 2018: International Forum of Allergy & Rhinology
https://www.readbyqxmd.com/read/29351449/personalized-medicine-in-cf-from-modulator-development-to-therapy-for-cystic-fibrosis-patients-with-rare-cftr-mutations
#19
Misak Harutyunyan, Yunjie Huang, Kyu-Shik Mun, Fanmuyi Yang, Kavisha Arora, Anjaparavanda P Naren
Cystic fibrosis (CF) is the most common life-shortening genetic disease affecting ~1 in 3,500 of the Caucasian population. CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. To date, more than 2,000 CFTR mutations have been identified, which produce a wide range of phenotypes. The CFTR protein, a chloride channel, is normally expressed on epithelial cells lining the lung, gut, and exocrine glands. Mutations in CFTR have led to pleiotropic effects in CF patients and have resulted in early morbidity and mortality...
April 1, 2018: American Journal of Physiology. Lung Cellular and Molecular Physiology
https://www.readbyqxmd.com/read/29342367/cystic-fibrosis-foundation-pulmonary-guidelines-use-of-cystic-fibrosis-transmembrane-conductance-regulator-modulator-therapy-in-patients-with-cystic-fibrosis
#20
Clement L Ren, Rebecca L Morgan, Christopher Oermann, Helaine E Resnick, Cynthia Brady, Annette Campbell, Richard DeNagel, Margaret Guill, Jeffrey Hoag, Andrew Lipton, Thomas Newton, Stacy Peters, Donna Beth Willey-Courand, Edward T Naureckas
RATIONALE: Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are a new class of medications targeting the underlying defect in CF. Ivacaftor (IVA) and IVA combined with lumacaftor (LUM; IVA/LUM) have been approved by the U.S. Food and Drug Administration (FDA) for use in patients with CF. However, the FDA label for these medications encompasses patient groups that were not studied as part of the drug approval process. CF clinicians, patients, and their families have recognized a need for recommendations to guide the use of these medications...
March 2018: Annals of the American Thoracic Society
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