Efficacy and safety of azithromycin maintenance therapy in primary ciliary dyskinesia (BESTCILIA): a multicentre, double-blind, randomised, placebo-controlled phase 3 trial
Tài liệu tham khảo
Knowles, 2013, Primary ciliary dyskinesia. Recent advances in diagnostics, genetics, and characterization of clinical disease, Am J Respir Crit Care Med, 188, 913, 10.1164/rccm.201301-0059CI
Lucas, 2014, Diagnosis and management of primary ciliary dyskinesia, Arch Dis Child, 99, 850, 10.1136/archdischild-2013-304831
Barbato, 2009, Primary ciliary dyskinesia: a consensus statement on diagnostic and treatment approaches in children, Eur Respir J, 34, 1264, 10.1183/09031936.00176608
Goutaki, 2016, Clinical manifestations in primary ciliary dyskinesia: systematic review and meta-analysis, Eur Respir J, 48, 1081, 10.1183/13993003.00736-2016
Marthin, 2010, Lung function in patients with primary ciliary dyskinesia: a cross-sectional and 3-decade longitudinal study, Am J Respir Crit Care Med, 181, 1262, 10.1164/rccm.200811-1731OC
Strippoli, 2012, Management of primary ciliary dyskinesia in European children: recommendations and clinical practice, Eur Respir J, 39, 1482, 10.1183/09031936.00073911
Shapiro, 2016, Diagnosis, monitoring, and treatment of primary ciliary dyskinesia: PCD foundation consensus recommendations based on state of the art review, Pediatr Pulmonol, 51, 115, 10.1002/ppul.23304
Werner, 2015, Diagnosis and management of primary ciliary dyskinesia, Cilia, 4, 2, 10.1186/s13630-014-0011-8
Paff, 2017, A randomised controlled trial on the effect of inhaled hypertonic saline on quality of life in primary ciliary dyskinesia, Eur Respir J, 49, 10.1183/13993003.01770-2016
Koh, 2000, The effect of regular salbutamol on lung function and bronchial responsiveness in patients with primary ciliary dyskinesia, Chest, 117, 427, 10.1378/chest.117.2.427
Kudoh, 1998, Improvement of survival in patients with diffuse panbronchiolitis treated with low-dose erythromycin, Am J Respir Crit Care Med, 157, 1829, 10.1164/ajrccm.157.6.9710075
Spagnolo, 2013, Long-term macrolide treatment for chronic respiratory disease, Eur Respir J, 42, 239, 10.1183/09031936.00136712
Wilms, 2012, Azithromycin maintenance therapy in patients with cystic fibrosis: a dose advice based on a review of pharmacokinetics, efficacy, and side effects, Pediatr Pulmonol, 47, 658, 10.1002/ppul.21620
Southern, 2012, Macrolide antibiotics for cystic fibrosis, Cochrane Database Syst Rev, 11
Wong, 2012, Azithromycin for prevention of exacerbations in non-cystic fibrosis bronchiectasis (EMBRACE): a randomised, double-blind, placebo-controlled trial, Lancet, 380, 660, 10.1016/S0140-6736(12)60953-2
Altenburg, 2013, Effect of azithromycin maintenance treatment on infectious exacerbations among patients with non-cystic fibrosis bronchiectasis: the BAT randomized controlled trial, JAMA, 309, 1251, 10.1001/jama.2013.1937
Valery, 2013, Long-term azithromycin for Indigenous children with non-cystic-fibrosis bronchiectasis or chronic suppurative lung disease (Bronchiectasis Intervention Study): a multicentre, double-blind, randomised controlled trial, Lancet Respir Med, 1, 610, 10.1016/S2213-2600(13)70185-1
Kobbernagel, 2016, Study protocol, rationale and recruitment in a European multi-centre randomized controlled trial to determine the efficacy and safety of azithromycin maintenance therapy for 6 months in primary ciliary dyskinesia, BMC Pulm Med, 16, 104, 10.1186/s12890-016-0261-x
Lucas, 2015, A quality-of-life measure for adults with primary ciliary dyskinesia: QOL-PCD, Eur Respir J, 46, 375, 10.1183/09031936.00216214
Dell, 2016, Primary ciliary dyskinesia: first health-related quality-of-life measures for pediatric patients, Ann Am Thorac Soc, 13, 1726
Behan, 2017, Validation of a health-related quality of life instrument for primary ciliary dyskinesia (QOL-PCD), Thorax, 72, 832, 10.1136/thoraxjnl-2016-209356
Behan, 2019, Validation of pediatric health-related quality of life instruments for primary ciliary dyskinesia (QOL-PCD), Pediatr Pulmonol, 54, 2011, 10.1002/ppul.24507
Quanjer, 2012, Multi-ethnic reference values for spirometry for the 3–95-yr age range: the global lung function 2012 equations, Eur Respir J, 40, 1324, 10.1183/09031936.00080312
Koopman, 2011, Reference values for paediatric pulmonary function testing: the Utrecht dataset, Respir Med, 105, 15, 10.1016/j.rmed.2010.07.020
Verbanck, 2016, Transfer factor, lung volumes, resistance and ventilation distribution in healthy adults, Eur Respir J, 47, 166, 10.1183/13993003.00695-2015
Robinson, 2012, Clarithromycin therapy for patients with cystic fibrosis: a randomized controlled trial, Pediatr Pulmonol, 47, 551, 10.1002/ppul.21613
Clement, 2006, Long term effects of azithromycin in patients with cystic fibrosis: a double blind, placebo controlled trial, Thorax, 61, 895, 10.1136/thx.2005.057950
Saiman, 2010, Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled trial, JAMA, 303, 1707, 10.1001/jama.2010.563
Sunther, 2016, Recovery of baseline lung function after pulmonary exacerbation in children with primary ciliary dyskinesia, Pediatr Pulmonol, 51, 1362, 10.1002/ppul.23479
Saiman, 2003, Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial, JAMA, 290, 1749, 10.1001/jama.290.13.1749
Albert, 2011, Azithromycin for prevention of exacerbations of COPD, N Engl J Med, 365, 689, 10.1056/NEJMoa1104623
Shah, 2016, A longitudinal study characterising a large adult primary ciliary dyskinesia population, Eur Respir J, 48, 441, 10.1183/13993003.00209-2016
Green, 2012, Ventilation inhomogeneity in children with primary ciliary dyskinesia, Thorax, 67, 49, 10.1136/thoraxjnl-2011-200726
Kobbernagel, 2019, One-year evolution and variability in multiple-breath washout indices in children and young adults with primary ciliary dyskinesia, Eur Clin Respir J, 6, 10.1080/20018525.2019.1591841
Kent, 2014, Lung clearance index: evidence for use in clinical trials in cystic fibrosis, J Cyst Fibros, 13, 123, 10.1016/j.jcf.2013.09.005
Renna, 2011, Azithromycin blocks autophagy and may predispose cystic fibrosis patients to mycobacterial infection, J Clin Invest, 121, 3554, 10.1172/JCI46095
Lucas, 2019, Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials, ERJ Open Res, 5, 00147, 10.1183/23120541.00147-2018