Challenges in the development of new therapies for bronchiectasis

Expert Opinion on Pharmacotherapy

Volumn 16, Issue 6, 2015, Pages 833-850

  Chalmers, James D ^a   Loebinger, Michael ^b,c   Aliberti, Stefano ^d  

^a UNIVERSITY OF DUNDEE   (United Kingdom)

^b ROYAL BROMPTON AND HAREFIELD NHS FOUNDATION TRUST   (United Kingdom)

^c NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

^d UNIVERSITY OF MILANO BICOCCA   (Italy)

Author keywords

Antibiotics; Bronchiectasis; Clinical trials; Pseudomonas

Indexed keywords

ANTIBIOTIC AGENT; ANTIINFLAMMATORY AGENT; AZTREONAM; BETA LACTAM ANTIBIOTIC; CIPROFLOXACIN; COLISTIN; GENTAMICIN; MACROLIDE; MUCOLYTIC AGENT; TOBRAMYCIN; ANTIINFECTIVE AGENT;

BRONCHIECTASIS; COMPUTER ASSISTED TOMOGRAPHY; DISEASE EXACERBATION; FORCED EXPIRATORY VOLUME; HUMAN; IONIZING RADIATION; LUNG CLEARANCE INDEX; LUNG FUNCTION; MICROBIOME; MULTICENTER STUDY (TOPIC); NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); RANDOMIZED CONTROLLED TRIAL (TOPIC); RESPIRATORY TRACT PARAMETERS; REVIEW; SPIROMETRY; SPUTUM; BIOASSAY; MUCOCILIARY CLEARANCE; PATHOPHYSIOLOGY;

ANTI-BACTERIAL AGENTS; ANTI-INFLAMMATORY AGENTS; BRONCHIECTASIS; CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; ENDPOINT DETERMINATION; HUMANS; MUCOCILIARY CLEARANCE; RANDOMIZED CONTROLLED TRIALS AS TOPIC;

EID: 84925876929     PISSN: 14656566     EISSN: 17447666     Source Type: Journal    
DOI: 10.1517/14656566.2015.1019863     Document Type: Review

References (100)

1. Bilton D, Loebinger MR, Wilson R.Non-cystic fibrosis bronchiectasis: an evidence-based for new therapies.Lancet Respir Med 2014; 2(12): 958-60
2. Seitz AE, Olivier KN, Adjemian J, et al.Trends in bronchiectasis among medicare beneficiaries in the United States, 2000 to 2007.Chest 2012; 142: 432-9
3. Ringshausen FC, de Roux A, Pletz MW, et al.Bronchiectasis-Associated hospitalizations in Germany, 2005-2011: a population-based study of disease burden and trends.PLoS One 2013; 8(8): e71109
4. Martinez-Garcia MA, Soler-Cataluna JJ, Donat Sanz Y, et al.Factors associated with bronchiectasis in patients with COPD.Chest 2011; 140(5): 1130-7
5. Joish VN, Spilsbury-Cantalupo M, Operschall E, et al.Economic burden of non-cystic fibrosis bronchiectasis in the first year after diagnosis from a US health plan perspective.Appl Health Econ Health Policy 2013; 11(3): 299-304
6. Goeminne PC, Nawrot TS, Ruttens D, et al.Mortality in non-cystic fibrosis bronchiectasis: a prospective cohort analysis.Respir Med 2014; 108(2): 287-96
7. Kelly MG, Murphy S, Elborn JS.Bronchiectasis in secondary care: a comprehensive profile of a neglected disease.Eur J Intern Med 2003; 14(8): 488-92
8. Chalmers JD, Goemmine PC, Aliberti S, et al.The Bronchiectasis Severity Index: an international derivation and validation study.Am J Respir Crit Care Med 2014; 189(5): 576-85
9. Gao Y, Guan W, Xu G, et al.Sleep disturbances and health-related quality of life in adults with steady-state bronchiectasis.PLoS One 2014; 9(7): e102970
10. Chalmers JD, McHugh BJ, Doherty CJ, et al.Mannose binding lectin deficiency and disease severity in non-CF bronchiectasis: a prospective study.Lancet Respir Med 2013; 1(3): 175-274
11. Chan SL, Chan-Yeung MM, Ooi GC, et al.Validation of the Hong Kong Chinese version of the St.George Respiratory Questionnaire in patients with bronchiectasis.Chest 2002; 122(6): 2030-7
12. Stockley R, De Soyza A, Gunawardena K, et al.Phase II study of a neutrophil elastase inhibitor (AZD9668) in patients with bronchiectasis.Respir Med 2013; 107(4): 524-33
13. Kupczyk M, ten Brinke A, Sterk PJ, et al.Frequent exacerbators - A distinct phenotype of severe asthma.Clin Exp Allergy 2014; 44(2): 212-21
14. Wedzicha JA, Singh D, Vestbo J, et al.Extrafine beclomethasone/formoterol in severe COPD patients with history of exacerbations.Respir Med 2014; 108(8): 1153-62
15. Agusti A, de Teresa L, De Backer W, et al.A comparison of the efficacy and safety of once-daily fluticasone furoate/.vilaterol with twice-daily fluticasone propionate/salmeterol in moderate to very severe COPD.Eur Respir J 2014; 43(3): 763-72
16. Han MK, Swigris J, Liu L, et al.Gender influences health-related quality of life in IPF.Respir Med 2010; 104(5): 724-30
17. Loebinger MR, Wells AU, Hansell DM, et al.Mortality in bronchiectasis: a longterm study assessing the factors influencing survival.Eur Respir J 2009; 34(4): 843-9
18. Roberts HJ, Hubbard R.Trends in bronchiectasis mortality in England and Wales.Respir Med 2010; 104(7): 981-5
19. Cole PJ.Inflammation: a two-edged sword-The model of bronchiectasis.Eur J Respir Dis Suppl 1986; 147: 6-15
20. Chalmers JD.Bronchiectasis trials: losing the battle but winning the war?.Lancet Respir Med 2014; 2(9): 679-81
21. Doring G, Flume P, Heijerman H, Elborn JS.Treatment of lung infection in patients with cystic fibrosis: current and future strategies.J Cyst Fibros 2012; 11: 461-79
22. Stockley RA, Hill SL, Burnett D.Nebulized amoxicillin in chronic purulent bronchiectasis.Clin Ther 1985; 7: 593-9
23. Barker AF, Couch L, Fiel SB, et al.Tobramycin solution for inhalation reduces sputum Pseudomonas aeruginosa density in bronchiectasis.Am J Respir Crit Care Med 2000; 162: 481-5
24. Drobnic ME, Sune P, Montoro JB, et al.Inhaled tobramycin in non-cystic fibrosis with chronic and chronic bronchial infection with Pseudomonas aeruginosa.Ann Pharmacother 2005; 39(1): 39-44
25. Scheinberg P, Shore E.A pilot study of the safety and efficacy of tobramycin solution for inhalation in patients with severe bronchiectasis.Chest 2005; 127(4): 1420-6
26. Haworth CS, Foweraker JE, Wilkinson P, et al.Inhaled colistin in patients with bronchiectasis and chronic pseudomonas aeruginosa infection.Am J Respir Crit Care Med 2014; 189(8): 975-82
27. Available from: http://www.isrctn.com/.ISRCTN49790596 [Accessed 20 December 2014
28. McCoy KS, Quittner AL, Oermann CM, et al.Inhaled aztreonam lysine for chronic airway Pseudomonas aeruginosa in cystic fibrosis.Am J Respir Crit Care Med 2008; 178: 921-8
29. Barker AF, O'Donnell AE, Flume P, et al.Aztreonam for inhalation solution in patients with non-cystic fibrosis bronchiectasis (AIR-BX1 and AIR-BX2): two randomised double-blind, placebocontrolled phase 3 trials.Lancet Respir Med 2014; 2(9): 738-49
30. Murray MP, Govan JRW, Docherty CJ, et al.A randomised controlled trial of nebulised gentamicin in non-cystic fibrosis bronchiectasis.Am J Respir Crit Care Med 2011; 183(4): 491-9
31. Wilson R, Welte T, Polverino E, et al.Ciprofloxacin dry powder for inhalation in non-cystic fibrosis bronchiectasis: a phase II randomised study.Eur Respir J 2013; 41(5): 1108-15
32. Serisier DJ, Bilton D, De Soyza A, et al.Inhaled, dual release liposomal ciprofloxacin in non-cystic fibrosis bronchiectasis (ORBIT-2): a randomised, double-blind, placebo-controlled trial.Thorax 2013; 68(9): 812-17
33. Bilton D, Henig N, Morrissey B, Gottfried M.Addition of inhaled tobramycin to ciprofloxacin for acute exacerbations of Pseudomonas aeruginosa in adult bronchiectasis.Chest 2006; 130(5): 1503-10
34. Wong C, Jayaram L, Karalus N, et al.Azithromycin for prevention of exacerbations in non-cystic fibrosis bronchiectasis (EMBRACE): a randomised, double-blind, placebocontrolled trial.Lancet 2012; 380(9842): 660-7
35. Altenburg J, de Graaff CS, Stienstra Y, et al.Effect of azithromycin maintenance treatment on infectious exacerbations among patients with non-cystic fibrosis bronchiectasis: the BAT randomized controlled trial.JAMA 2013; 309: 1251-9
36. Serisier DJ, Martin ML, McGuckin MA, et al.Effect of long-Term, low-dose erythromycin on pulmonary exacerbations among patients with noncystic fibrosis bronchiectasis: the BLESS randomized controlled trial.JAMA 2013; 309: 1260-7
37. Wu Q, Shen W, Cheng H, Zhou X.Long-Term macrolides for non-cystic fibrosis bronchiectasis: a systematic review and meta-Analysis.Respirology 2014; 19(3): 321-9
38. McCullough AR, Tunney MM, Elborn JS, et al.Treatment adherence and health outcomes in patients with bronchiectasis.BMC Pulm Med 2014; 14: 107
39. Haworth CS, Bilton D, Elborn JS.Long-Term macrolide maintenance therapy in non-CF bronchiectasis: evidence and questions.Respir Med 2014; 108(10): 1397-408
40. Ray WA, Murray KT, Hall K, et al.Azithromycin and the risk of cardiovascular death.N Engl J Med 2012; 366(20): 1881-90
41. Martinez-Garcia MA, Soler-Cataluna JJ, Catalan-Serra P, et al.Clinical efficacy and safety of budesonide-formoterol in non-cystic fibrosis bronchiectasis.Chest 2012; 141(2): 461-8
42. Kapur N, Bell S, Kolbe J, Chang AB.Inhaled steroids for bronchiectasis.Cochrane Database Syst Rev 2009(1): CD000996
43. Singanayagam A, Chalmers JD, Akram AR, Hill AT.Impact of inhaled corticosteroid use on outcome in COPD patients admitted with pneumonia.Eur Respir J 2011; 38(1): 36-41
44. Andrejak C, Nielson R, Thomson VO, et al.Chronic respiratory disease, inhaled corticosteroids and risk of nontuberculous mycobacteriosis.Thorax 2013; 68(3): 256-62
45. Pasteur MC, Bilton D, Hill AT.British Thoracic Society guideline for non-CF bronchiectasis.Thorax 2010; 65(Suppl 1): i1-58
46. Chalmers JD, Hill AT.Mechanisms of immune dysfunction and bacterial persistence in non-cystic fibrosis bronchiectasis.Mol Immunol 2013; 55: 27-34
47. Pavord I, De Soyza A, Elborn JS, et al.Efficacy and safety of AZD5069, a CXCR2 antagonist in adult bronchiectasis.Eur Respir J 2013; 42(Suppl 57): P1593
48. Konstan MW, Doring G, Heltsche SL, et al.A randomized double blind, placebo controlled phase 2 trial of BIIL284BS (an LTB4 receptor antagonist) for the treatment of lung disease in children and adults with cystic fibrosis.J Cyst Fibros 2014; 13(2): 148-55
49. Doring G, Bragonzi A, Paroni M, et al.BIL 284 reduces neutrophil numbers but increases P.aeruginosa bacteremia and inflammation in mouse lungs.J Cyst Fibros 2014; 13(2): 156-63
50. Rennard SI, Fogarty C, Kelsen S, et al.The safety and efficacy of infliximab in moderate to severe chronic obstructive pulmonary disease.Am J Respir Crit Care Med 2007; 175(9): 926-34
51. Aitkey ML, Bellon G, De Boeck K, et al.Long-Term inhaled dry powder mannitol in cystic fibrosis: an international randomized study.Am J Respir Crit Care Med 2012; 185(6): 645-52
52. Bilton D, Bellon G, Charlton B, et al.Pooled analysis of two large randomised phase III inhaled mannitol studies in cystic fibrosis.J Cyst Fibros 2013; 12(4): 367-76
53. Bilton D, Daviskas E, Anderson SD, et al.Phase 3 randomized study of the efficacy and safety of inhaled dry powder mannitol for the symptomatic treatment of non-cystic fibrosis bronchiectasis.Chest 2013; 144(1): 215-25
54. Bilton D, Tino G, Barker AF, et al.Inhaled mannitol for non-cystic fibrosis bronchiectasis: a randomised, controlled trial.Thorax 2014; 69(12): 1073-9
55. Nicolson CH, Stirling RG, Borg BM, et al.The long term effect of inhaled hypertonic saline 6% in non-cystic fibrosis bronchiectasis.Respir Med 2012; 106(5): 661-7
56. O'Donnell AE, Barker AF, Ilowite JS, Fick RB.Treatment of idiopathic bronchiectasis with aerosolized recombinant human DNase I.rhDNase Study Group.Chest 1998; 113: 1329-34
57. Young RL, Malcolm KC, Kret JE, et al.Neutrophil extracellular trap (NET)-mediated killing of Pseudomonas aeruginosa: evidence of acquired resistance within the CF airway, independent of CFTR.PLoS One 2011; 6(9): e23637
58. Dubois AV, Gauthier A, Brea D, et al.Influence of DNA on the activities and inhibition of neutrophil serine proteases in cystic fibrosis sputum.Am J Respir Cell Mol Biol 2012; 47(1): 80-6
59. Hill AT, Welham S, Reid K, Bucknall CE.British Thoracic Society national bronchiectasis audit 2010 and 2011.Thorax 2012; 67(10): 928-30
60. Paganin F, Seneterre E, Chanez P, et al.Computed tomography of the lungs in asthma: influence of disease severity and etiology.Am J Respir Crit Care Med 1996; 153: 110-14
61. Blasi F, Chalmers JD, Aliberti S.COPD and Bronchiectasis: phenotype, endotype or co-morbidity?.COPD 2014; 11(6): 603-4
62. Swinson DR, Symmons D, Suresh U, et al.Decreased survival in patients with co-existent rheumatoid arthritis and bronchiectasis.Br J Rheumatol 1997; 36(6): 689-91
63. Griffin DE.Nontuberculous mycobacterial lung disease.Curr Opin Infect Dis 2010; 23(2): 185-90
64. McShane PJ, Naureckas ET, Strek ME.Bronchiectasis in a diverse US population: effects of ethnicity on etiology and sputum culture.Chest 2012; 142(1): 159-67
65. Redding GJ, Singleton RJ, Valery PC, et al.Respiratory exacerbations in indigenous children from two countries with non-cystic fibrosis chronic suppurative lung disease/bronchiectasis.Chest 2014; 146(3): 762-74
66. Martinez Garcia MA, de Gracia J, Vendrell Relat M, et al.Multidimensional approach to non-cystic fibrosis bronchiectasis; the FACED score.Eur Respir J 2014; 43(5): 1357-67
67. Nick JA, Moskowitz SM, Chmiel JF, et al.Azithromycin may antagonize inhaled tobramycin when targeting Pseudomonas aeruginosa in cystic fibrosis.Ann Am Thorac Soc 2014; 11(3): 342-50
68. Chang AB, Bell SC, Torzillo PJ, et al.Chronic suppurative lung disease and bronchiectasis in children and adults in Australia and New Zealand Thoracic Society of Australia and New Zealand guidelines.Med J Aust 2015; 202(1): 21-3
69. Murray MP, Turnbull K, Macquarrie S, Hill AT.Assessing response to treatment of exacerbations of bronchiectasis in adults.Eur Respir J 2009; 33(2): 312-18
70. Langsetmo L, Platt RW, Ernst P, Bourbeau J.Underreporting exacerbation of chronic obstructive pulmonary disease in a longitudinal cohort.Am J Respir Crit Care Med 2008; 177(4): 396-401
71. Wilson CB, Jones PW, O'Leary CJ, et al.Validation of the St.Georges Respiratory Questionnaire in bronchiectasis.Am J Respir Crit Care Med 1997; 156(2 Pt 1): 536-41
72. Quittner AL, O'Donnell AE, Salathe MA, et al.Quality of life questionnaire-Bronchiectasis: final psychometric analyses and determination of minimal important difference scores.Thorax 2015; 70(1): 12-20
73. Mandal P, Chalmers JD, Graham C, et al.Atorvastatin as a stable treatment in bronchiectasis: a randomised controlled trial.Lancet Respir Med 2014; 2(6): 455-63
74. Chalmers JD, Smith MP, McHugh B, et al.Short and long term antibiotic therapy reduces airway and systemic inflammation in non-CF bronchiectasis.Am J Respir Crit Care Med 2012; 186(7): 657-65
75. Horsley AR, Gustafsson PM, Macleod KA, et al.Lung clearance index is a sensitive, repeatable and practical measure of airways disease in adults with cystic fibrosis.Thorax 2008; 63(2): 135-40
76. Rowan SA, Bradley JM, Bradbury I, et al.Lung clearance index is a repeatable and sensitive indicator of radiological changes in bronchiectasis.Am J Respir Crit Care Med 2014; 189(5): 586-92
77. Gonem S, Scadding A, Soares M, et al.Lung clearance index in adults with noncystic fibrosis bronchiectasis.Respir Res 2014; 15: 59
78. Tsang KW, Chan K, Ho P, et al.Sputum elastase in steady-state bronchiectasis.Chest 2000; 117(2): 420-6
79. Chalmers JD, McHugh BJ, Doherty CJ, et al.Vitamin-D deficiency is associated with chronic bacterial colonisation and disease severity in non-CF bronchiectasis.Thorax 2012; 68(1): 39-47
80. Shoemark A, Devaraj A, Meister M, et al.Elevated peripheral airway nitric oxide in bronchiectasis reflects disease severity.Respir Med 2011; 105(6): 885-91
81. Loeve M, Krestin GP, Rosenfeld M, et al.Chest computed tomography: a validated surrogate endpoint of cystic fibrosis lung disease?.Eur Respir J 2013; 42(3): 844-57
82. Kuo W, Clet P, Tiddens HA, et al.Monitoring cystic fibrosis lung disease by computed tomography: radiation risk in perspective.Am J Respir Crit Care Med 2014; 189(11): 1328-36
83. Wielputz MO, Puderbach M, Kopp-Schneider A, et al.Magnetic resonance imaging detects changes in structure and perfusion and response to therapy in early cystic fibrosis lung disease.Am J Respir Crit Care Med 2014; 189(8): 956-65
84. Tunney MM, Einarsson GG, Wei L, et al.Lung microbiota and bacterial abundance in patients with bronchiectasis when clinically stable and during exacerbation.Am J Respir Crit Care Med 2013; 187(10): 1118-26
85. Rogers GB, van der Gast CJ, Cuthbertson L, et al.Clinical measures of disease in adult non-CF bronchiectasis correlate with airway microbiota composition.Thorax 2013; 68(8): 731-7
86. Rogers GB, Bruce KD, Martin ML, et al.The effect of long-Term macrolide treatment on respiratory microbiota composition in non-cystic fibrosis bronchiectasis: an analysis from the randomized, double-blind placebocontrolled BLESS trial.Lancet Respir Med 2014; 2(12): 988-96
87. Available from: www.bronchiectasis.eu [Accessed 20 December 2014
88. Aksamit TR, Carretta E, Daley CL, et al.The Bronchiectasis Research Registry: a collaborative research cohort for noncystic fibrosis bronchiectasis.Am J Respir Crit Care Med 2012; A3654
89. Ramsey BW, Pepe MS, Quan JM, et al.Intermittent administration of inhaled tobramycin in patients with cystic fibrosis.Cystic Fibrosis Inhaled Tobramycin Study Group.N Engl J Med 1999; 340(1): 23-30
90. Ramsey BW, Dorkin HC, Eisenberg JD, et al.Efficacy of aerolised tobramycin in patients with cystic fibrosis.N Engl J Med 1993; 328: 1740-6
91. Clancy JP, Dupont L, Konstan MW, et al.Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection.Thorax 2013; 68(9): 818-25
92. Available from: https://clinicaltrials.gov/.ct2/show/NCT00775138 [Accessed 20 December 2014
93. Alothman GA, Ho B, Alsaadi MM, et al.Bronchial constriction and inhaled colistin in cystic fibrosis.Chest 2005; 127: 522-9
94. Jensen T, Pedersen SS, Garne S, et al.Colistin inhalation therapy in cystic fibrosis patients with chronic Pseudomonas aeruginosa lung infection.J Antimicrob Chemother 1987; 19: 831-8
95. Saiman L, Marshall BC, Mayer-Hamblett N, et al.Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial.JAMA 2003; 290(13): 1749-56
96. Saiman L, Anstead M, Mayer-Hamblett N, et al.Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled trial.JAMA 2010; 303(17): 1707-15
97. Fuchs HJ, Borowitz DS, Christiansen DH, et al.Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis.N Engl J Med 1994; 331: 637-42
98. Quan JM, Tiddens HA, Sy JP, et al.A two-year randomized, placebocontrolled trial of dornase alfa in young patients with cystic fibrosis with mild lung function abnormalities.J Pediatr 2001; 139: 813-20
99. Elkins MR, Robinson M, Rose BR, et al.A controlled trial of long term inhaled hypertonic saline in patients with cystic fibrosis.N Engl J Med 2006; 354: 229-40
100. Donaldson SH, Bennett WD, Zeman KL, et al.Mucus clearance and lung function in cystic fibrosis with hypertonic saline.N Engl J Med 2006; 354(3): 241-50