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References
- Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.N Engl J Med. 2010; 363: 1991-2003
- Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.Lancet, London, England)2019
- Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.N Engl J Med. 2019; 381: 1809-1819
- A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.N Engl J Med. 2011; 365: 1663-1672
- Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis.N Engl J Med. 2017; 377: 2024-2035
- Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.N Engl J Med. 2015; 373: 220-231
- Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype.Pediatr Pulmonol. 2021; 56: 1496-1503
- Health Disparities among adults cared for at an urban cystic fibrosis program.Orphanet J rare Dis. 2021; 16: 332
- Impact of socioeconomic status, race, and ethnicity on quality of life in patients with cystic fibrosis in the United States.Chest. 2010; 137: 642-650
- Insights into patient variability during ivacaftor-lumacaftor therapy in cystic fibrosis.Front Pharmacol. 2021; 12: 577263
- CFTR modulators: does one dose fit all?.J Personalized Med. 2021; 11
- Variable cellular ivacaftor concentrations in people with cystic fibrosis on modulator therapy.J Cystic Fibrosis. 2020; 19: 742-745
- Expression of cystic fibrosis transmembrane conductance regulator in a model epithelium.Am J Physiol. 1994; 266: L405-L413
- A functional CFTR assay using primary cystic fibrosis intestinal organoids.Nat Med. 2013; 19: 939-945
- Correction of CFTR function in intestinal organoids to guide treatment of Cystic Fibrosis.Eur Respir J. 2020; 57: 1902426
- CRISPR-based adenine editors correct nonsense mutations in a cystic fibrosis organoid biobank.Cell stem cell. 2020; 26: 503-510
- Stratifying infants with cystic fibrosis for disease severity using intestinal organoid swelling as a biomarker of CFTR function.Eur Respir J. 2018; 52
- Intestinal organoids and personalized medicine in cystic fibrosis: a successful patient-oriented research collaboration.Curr Opin Pulm Med. 2016; 22: 610-616
- Individualized medicine using intestinal responses to CFTR potentiators and correctors.Pediatr Pulmonol. 2016; 51: S23-S34
- A bioassay using intestinal organoids to measure CFTR modulators in human plasma.J Cystic Fibrosis. 2015; 14: 178-181
- Functional restoration of CFTR nonsense mutations in intestinal organoids.J Cyst Fibro. 2021; 21: 246-253
- CFTR rescue in intestinal organoids with GLPG/ABBV-2737, ABBV/GLPG-2222 and ABBV/GLPG-2451 triple therapy.Front Mol Biosci. 2021; 8: 698358
- Comparison of organoid swelling and in vivo biomarkers of CFTR function to determine effects of lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for the F508del mutation.Am J Respir Crit Care Med. 2020; 202: 1589-1592
- Use of primary cultures of human bronchial epithelial cells isolated from cystic fibrosis patients for the pre-clinical testing of CFTR modulators.Methods Mol Biol (Clifton, NJ). 2011; 741: 39-54
- Brushed nasal epithelial cells are a surrogate for bronchial epithelial CFTR studies.JCI Insight. 2018; 3
- Comparison of three different brushing techniques to isolate and culture primary nasal epithelial cells from human subjects.Exp Lung Res. 2014; 40: 327-332
- Culturing of human nasal epithelial cells at the air liquid interface.J Vis Exp. 2013; 80
- Development of primary human nasal epithelial cell cultures for the study of cystic fibrosis pathophysiology.Am J Physiol Cell Physiol. 2012; 303: C1173-C1179
- Feasibility of nasal epithelial brushing for the study of airway epithelial functions in CF infants.J Cystic Fibros. 2008; 7: 44-53
- Radiation induces diffusible feeder cell factor(s) that cooperate with ROCK inhibitor to conditionally reprogram and immortalize epithelial cells.Am J Pathol. 2013; 183: 1862-1870
- ROCK inhibitor and feeder cells induce the conditional reprogramming of epithelial cells.Am J Pathol. 2012; 180: 599-607
- Detection of CFTR function and modulation in primary human nasal cell spheroids.J Cyst Fibros. 2017; 17: 26-33
- CFTR function and clinical response to modulators parallel nasal epithelial organoid swelling.Am J Physiol Lung Cell Mol Physiol. 2021; 321: L119-L129
- Human nasal epithelial organoids for therapeutic development in cystic fibrosis.Genes (Basel). 2020; 11: 603
- Nasospheroids permit measurements of CFTR-dependent fluid transport.JCI insight. 2017; 2: e95734
- In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N-of-1 studies.Pediatr Pulmonol. 2017; 52: 472-479
- Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia.NPJ Genomic Med. 2017; 2: 12
- Might brushed nasal cells Be a surrogate for CFTR modulator clinical response?.Am J Respir Crit Care Med. 2019; 199: 123-126
- Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators.Scientific Rep. 2017; 7: 7375
- Personalised CFTR pharmacotherapeutic response testing and therapy of cystic fibrosis.Eur Respir J. 2018; 51: 1702457
- Therapeutic challenges posed by critical drug-drug interactions in cystic fibrosis.Pediatr Pulmonol. 2016; 51: S61-S70
- Pharmacokinetic variability of clarithromycin and differences in CYP3A4 activity in patients with cystic fibrosis.J Cystic Fibros. 2014; 13: 179-185
- Individual pharmacokinetic variation leads to underdosing of ciprofloxacin in some cystic fibrosis patients.Pediatr Pulmonol. 2017; 52: 319-323
- Drug disposition in cystic fibrosis.Clin Pharmacokinet. 1998; 35: 313-329
- Pharmacokinetics and pharmacodynamics of antibiotics in cystic fibrosis: a narrative review.Int J Antimicrob Agents. 2021; 58: 106381
- Clinical use and bioavailability of tacrolimus in heart-lung and double lung transplant recipients with cystic fibrosis.Transplant Proc. 1998; 30: 1519-1520
- Therapeutic drug monitoring of the beta-lactam antibiotics: what is the evidence and which patients should we be using it for?.J Antimicrob Chemother. 2015; 70: 3178-3183
- Bayesian forecasting for intravenous tobramycin dosing in adults with cystic fibrosis using one versus two serum concentrations in a dosing interval.Ther Drug Monit. 2021; 43: 505-511
- Evaluation of vancomycin dosing in pediatric cystic fibrosis patients.J Pediatr Pharmacol Ther. 2016; 21: 155-161
- Pharmacokinetic and pharmacodynamic optimisation of intravenous tobramycin dosing among children with cystic fibrosis.J Pharmacokinet Pharmacodyn. 2014; 41: 71-79
- A retrospective 'real-world' cohort study of azole therapeutic drug monitoring and evolution of antifungal resistance in cystic fibrosis.JAC Antimicrob Resist. 2021; 3: dlab026
- Treating allergic bronchopulmonary aspergillosis with short-term prednisone and itraconazole in cystic fibrosis.J Allergy Clin Immunol Pract. 2020; 8: 2608-2614 e2603
- Clinical pharmacokinetics and dose recommendations for posaconazole gastroresistant tablets in children with cystic fibrosis.J Antimicrob Chemother. 2021; 76: 3247-3254
- Pharmacokinetics of oral antimycobacterials and dosing guidance for Mycobacterium avium complex treatment in cystic fibrosis.J Cyst Fibros. 2021; 20: 772-778
- Administration and monitoring of clofazimine for NTM infections in children with and without cystic fibrosis.J Cyst Fibros. 2021; 21: 348-352
- Sensitivity of ivacaftor to drug-drug interactions with rifampin, a cytochrome P450 3A4 inducer.Pediatr Pulmonol. 2018; 53: E6-E8
- The cystic fibrosis foundation patient registry. design and methods of a national observational disease registry.Ann Am Thorac Soc. 2016; 13: 1173-1179
- Association of high-dose ibuprofen use, lung function decline, and long-term survival in children with cystic fibrosis.Ann Am Thorac Soc. 2018; 15: 485-493
- Oral non-steroidal anti-inflammatory drug therapy for lung disease in cystic fibrosis.Cochrane database Syst Rev. 2019; 9: CD001505
- Impact of a cystic fibrosis transmembrane conductance regulator (CFTR) modulator on high-dose ibuprofen therapy in pediatric cystic fibrosis patients.Pediatr Pulmonol. 2018; 53: 1035-1039
- Isotope dilution LC-MS/MS quantification of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor, lumacaftor, tezacaftor, elexacaftor, and their major metabolites in human serum.Clin Chem Lab Med. 2021; 60: 82-91
- Development of HPLC and LC-MS/MS methods for the analysis of ivacaftor, its major metabolites and lumacaftor in plasma and sputum of cystic fibrosis patients treated with ORKAMBI or KALYDECO.J Chromatogr B Analyt Technol Biomed Life Sci. 2016; 1038: 57-62
- Multiple reaction monitoring mass spectrometry for the drug monitoring of ivacaftor, tezacaftor, and elexacaftor treatment response in cystic fibrosis: a high-throughput method.ACS Pharmacol Transl Sci. 2020; 3: 987-996
- Drug approval package: trikafta.(Available at:) (Accessed July 30, 2020)
- Drug approval package: kalydeco (ivacaftor).(Available at:) (Accessed February 12, 2019)
- Accumulation and persistence of ivacaftor in airway epithelia with prolonged treatment.J Cyst Fibros. 2020; 19: 746-751
- Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770.Proc Natl Acad Sci U S A. 2009; 106: 18825-18830
- Potentiator ivacaftor abrogates pharmacological correction of DeltaF508 CFTR in cystic fibrosis.Sci translational Med. 2014; 6 (246ra296)
- Mutation-specific downregulation of CFTR2 variants by gating potentiators.Hum Mol Genet. 2017; 26: 4873-4885
- Breast development in a 7 year old girl with CF treated with ivacaftor: an indication for personalized dosing?.J Cyst Fibros. 2021; 20: e63-e66
- A case of self-limited drug induced liver injury under treatment with elexacaftor/tezacaftor/ivacaftor: when it is worth taking the risk.J Cyst Fibros. 2021; 20: 712-714
- Drug-induced liver injury from elexacaftor/ivacaftor/tezacaftor.J Cyst Fibros. 2021; 21: e99-e101
- Treatment complexity in cystic fibrosis (CF): an increasing multifaceted challenge.Pediatr Pulmonol. 2018; 53: 1174-1176
- Ivacaftor-elexacaftor-tezacaftor and tacrolimus combination in cystic fibrosis.J Cyst Fibros. 2021; 21: e8-e10
- Use of elexacaftor/tezacaftor/ivacaftor in liver transplant patients with cystic fibrosis.J Cyst Fibros. 2021; 21: 227-229
- Lumacaftor/ivacaftor initiation in two liver transplantation patients under tacrolimus and antifungal azoles.Clin Case Rep. 2019; 7: 616-618
- Entry of cystic fibrosis transmembrane conductance potentiator ivacaftor into the developing brain and lung.J Cyst Fibros. 2021; 20: 857-864
- Maternal and fetal outcomes following elexacaftor-tezacaftor-ivacaftor use during pregnancy and lactation.J Cyst Fibros. 2021; 20: 402-406
- Applying whole-genome sequencing in relation to phenotype and outcomes in siblings with cystic fibrosis.Cold Spring Harb Mol Case Stud. 2020; 6
- Pharmacogenetics of cystic fibrosis treatment.Pharmacogenomics. 2016; 17: 1453-1463
- The clinical pharmacogenetics implementation consortium: 10 Years later.Clin Pharmacol Ther. 2020; 107: 171-175
- Pharmacogenetic considerations for optimizing tacrolimus dosing in liver and kidney transplant patients.World J Gastroenterol. 2013; 19: 9156-9173
- A randomized controlled trial comparing the efficacy of cyp3a5 genotype-based with body-weight-based tacrolimus dosing after living donor kidney transplantation.Am J Transplant. 2016; 16: 2085-2096
- Long-term clinical impact of adaptation of initial tacrolimus dosing to CYP3A5 genotype.Am J Transplant. 2016; 16: 2670-2675
- Influence of combined CYP3A4 and CYP3A5 single-nucleotide polymorphisms on tacrolimus exposure in kidney transplant recipients: a study according to the post-transplant phase.Pharmacogenomics. 2015; 16: 2045-2054
- A new functional CYP3A4 intron 6 polymorphism significantly affects tacrolimus pharmacokinetics in kidney transplant recipients.Clin Chem. 2011; 57: 1574-1583
- The CYP3A4∗22 allele affects the predictive value of a pharmacogenetic algorithm predicting tacrolimus predose concentrations.Br J Clin Pharmacol. 2013; 75: 1545-1547
- Effect of a new functional CYP3A4 polymorphism on calcineurin inhibitors' dose requirements and trough blood levels in stable renal transplant patients.Pharmacogenomics. 2011; 12: 1383-1396
- 2020 Annual data report. Cystic Fibrosis Foundation, Bethesda, Maryland2020
- Risk factors for lung function decline in a large cohort of young cystic fibrosis patients.Pediatr Pulmonol. 2015; 50: 763-770
- A systematic review of studies examining the rate of lung function decline in patients with cystic fibrosis.Paediatr Respir Rev. 2016; 20: 55-66
- Association between Staphylococcus aureus alone or combined with Pseudomonas aeruginosa and the clinical condition of patients with cystic fibrosis.J Cyst Fibros. 2013; 12: 497-503
- Factors associated with FEV1 decline in cystic fibrosis: analysis of the ECFS patient registry.Eur Respir J. 2014; 43: 125-133
- Clinical outcomes after initial pseudomonas acquisition in cystic fibrosis.Pediatr Pulmonol. 2015; 50: 42-48
- Clinical effectiveness of elexacaftor/tezacftor/ivacaftor in people with cystic fibrosis.Am J Respir Crit Care Med. 2021; 205: 529-539
- Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis.Am J Respir Crit Care Med. 2014; 190: 175-184
- Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation.Am J Respir Crit Care Med. 2013; 187: 1219-1225
- Lung clearance index is a sensitive, repeatable and practical measure of airways disease in adults with cystic fibrosis.Thorax. 2008; 63: 135-140
- Multiple-breath washout as a marker of lung disease in preschool children with cystic fibrosis.Am J Respir Crit Care Med. 2005; 171: 249-256
- Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function.Thorax. 2010; 65: 379-383
- Hyperpolarized (129)Xe for investigation of mild cystic fibrosis lung disease in pediatric patients.J Cyst Fibros. 2017; 16: 275-282
- Hyperpolarised (129)Xe magnetic resonance imaging to monitor treatment response in children with cystic fibrosis.Eur Respir J. 2019; 53: 1802188
- Dynamic perfluorinated gas MRI reveals abnormal ventilation despite normal FEV1 in cystic fibrosis.JCI insight. 2020; 5
- Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: results from the PROSPECT MCC sub-study.J Cyst Fibros. 2021; 21: 143-145
- Intranasal micro-optical coherence tomography imaging for cystic fibrosis studies.Sci translational Med. 2019; 11: eaav3505
- Functional anatomic imaging of the airway surface.Ann Am Thorac Soc. 2018; 15: S177-S183
- Effect of ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis patients with G551D-CFTR.JCI Insight. 2018; 3
- Correlation of sweat chloride and percent predicted FEV(1) in cystic fibrosis patients treated with ivacaftor.J Cyst Fibros. 2017; 16: 41-44
- Measuring the impact of CFTR modulation on sweat chloride in cystic fibrosis: rationale and design of the CHEC-SC study.J Cyst Fibros. 2021; 20: 965-971
- BAL inflammatory markers can predict pulmonary exacerbations in children with cystic fibrosis.Chest. 2020; 158: 2314-2322
- Induced sputum matrix metalloproteinase-9 correlates with lung function and airway inflammation in children with cystic fibrosis.Pediatr Pulmonol. 2005; 39: 224-232
- Airway profile of bioactive lipids predicts early progression of lung disease in cystic fibrosis.J Cyst Fibros. 2020; 19: 902-909
- SPLUNC1: a novel marker of cystic fibrosis exacerbations.Eur Respir J. 2021; 58
- Cystic fibrosis point of personalized detection (CFPOPD): an interactive web application.JMIR Med Inform. 2020; 8: e23530
- Association between number of intravenous antipseudomonal antibiotics and clinical outcomes of pediatric cystic fibrosis pulmonary exacerbations.Clin Infect Dis. 2021; 73: 1589-1596
- Outcomes of adults with cystic fibrosis infected with antibiotic-resistant Pseudomonas aeruginosa.Respiration. 2006; 73: 27-33
- Targeted exhaled breath analysis for detection of Pseudomonas aeruginosa in cystic fibrosis patients.J Cyst Fibros. 2021; 21: e28-e34
- Exhaled breath hydrogen cyanide as a marker of early Pseudomonas aeruginosa infection in children with cystic fibrosis.ERJ Open Res. 2015; 1: 00044-02015
- Cystic Fibrosis Foundation consensus guidelines for the care of individuals with advanced cystic fibrosis lung disease.J Cyst Fibros. 2020; 19: 344-354
- Lung transplant referral for individuals with cystic fibrosis: cystic Fibrosis Foundation consensus guidelines.J Cyst Fibros. 2019; 18: 321-333
- Cystic fibrosis foundation pulmonary guidelines. use of cystic fibrosis transmembrane conductance regulator modulator therapy in patients with cystic fibrosis.Ann Am Thorac Soc. 2018; 15: 271-280
- The future of cystic fibrosis care: a global perspective.Lancet Respir Med. 2020; 8: 65-124
- Testing the effects of combining azithromycin with inhaled tobramycin for P. aeruginosa in cystic fibrosis: a randomised, controlled clinical trial.Thorax, 2021
- A randomized trial of antimicrobial duration for cystic fibrosis pulmonary exacerbation treatment.Am J Respir Crit Care Med. 2021; 204: 1295-1305
- Effect of high-dose ibuprofen in patients with cystic fibrosis.N Engl J Med. 1995; 332: 848-854
- A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis.N Engl J Med. 2006; 354: 229-240
- Evaluating the impact of stopping chronic therapies after modulator drug therapy in cystic fibrosis: the simplify clinical trial study design.Ann Am Thorac Soc. 2021; 18: 1397-1405
- Inhaled hypertonic saline in preschool children with cystic fibrosis (SHIP): a multicentre, randomised, double-blind, placebo-controlled trial.Lancet Respir Med. 2019; 7: 802-809
- Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial.JAMA. 2003; 290: 1749-1756
- Disease progression in patients with cystic fibrosis treated with ivacaftor: data from national US and UK registries.J Cyst Fibros. 2020; 19: 68-79
- Reduced mortality in cystic fibrosis patients treated with tobramycin inhalation solution.Pediatr Pulmonol. 2012; 47: 44-52
- Quantifying long-term changes in lung function and exacerbations after initiation of azithromycin in cystic fibrosis.Ann Am Thorac Soc. 2020; 17: 195-201
- Pulmonary outcomes associated with long-term azithromycin therapy in cystic fibrosis.Am J Respir Crit Care Med. 2020; 201: 430-437
- Inhaled tobramycin effectively reduces FEV1 decline in cystic fibrosis. An instrumental variables analysis.Ann Am Thorac Soc. 2013; 10: 205-212
- Relationship between high-dose ibuprofen use and rate of decline in FEV1 among young patients with mild lung disease in the CFF Registry.Pediatr Pulmonol. 2004; 27 (322): A385
- Rapid improvement after starting elexacaftor-tezacaftor-ivacaftor in patients with cystic fibrosis and advanced pulmonary disease.Am J Respir Crit Care Med. 2021; 204: 64-73
- Efficacy of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease.Eur Respir J. 2021; 57
- Mucus clearance and lung function in cystic fibrosis with hypertonic saline.N Engl J Med. 2006; 354: 241-250
- Restoring cystic fibrosis transmembrane conductance regulator function reduces airway bacteria and inflammation in people with cystic fibrosis and chronic lung infections.Am J Respir Crit Care Med. 2017; 195: 1617-1628
- Pseudomonas aeruginosa in cystic fibrosis patients with G551D-CFTR treated with ivacaftor.Clin Infect Dis : official Publ Infect Dis Soc America. 2015; 60: 703-712
- Azithromycin for early pseudomonas infection in cystic fibrosis. The OPTIMIZE Randomized Trial.Am J Respir Crit Care Med. 2018; 198: 1177-1187
- Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled trial.JAMA. 2010; 303: 1707-1715
- An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5years (KLIMB).J Cyst Fibros. 2019; 18: 838-843
- Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study.Lancet Respir Med. 2018; 6: 545-553
- Ethical dilemma: elexacaftor-tezacaftor-ivacaftor or lung transplantation in cystic fibrosis and end-stage lung disease?.Chest. 2021; 161: 773-780
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