Cystic fibrosis

References

Key articles

Farrell PM, White TB, Ren CL, et al. Diagnosis of cystic fibrosis: consensus guidelines from the Cystic Fibrosis Foundation. J Pediatr. 2017 Feb;181S:S4-15.e1.Full text  Abstract

Mogayzel PJ Jr, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013 Apr 1;187(7):680-9.Full text  Abstract

Flume PA, Robinson KA, O'Sullivan BP, et al; Clinical Practice Guidelines for Pulmonary Therapies Committee. Cystic fibrosis pulmonary guidelines: airway clearance therapies. Respir Care. 2009 Apr;54(4):522-37.Full text  Abstract

Ramos KJ, Smith PJ, McKone EF, et al. Lung transplant referral for individuals with cystic fibrosis: Cystic Fibrosis Foundation consensus guidelines. J Cyst Fibros. 2019 May;18(3):321-33.Full text  Abstract

Kapnadak SG, Dimango E, Hadjiliadis D, et al. Cystic Fibrosis Foundation consensus guidelines for the care of individuals with advanced cystic fibrosis lung disease. J Cyst Fibros. 2020 May;19(3):344-54.Full text  Abstract

Reference articles

1. Heneghan M, Southern KW, Murphy J, et al. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966.Full text  Abstract

2. Orenstein DM, Rosenstein BJ, Stern RC. Cystic fibrosis: medical care. Philadelphia, PA: Lippincott Williams & Wilkins; 2000:365.

3. Cystic Fibrosis Foundation. 2021 patient registry annual data report. Bethesda, MD: Cystic Fibrosis Foundation; 2022.Full text

4. Scotet V, Gutierrez H, Farrell PM. Newborn screening for CF across the globe - where is it worthwhile? Int J Neonatal Screen. 2020 Mar;6(1):18.Full text  Abstract

5. Southern KW, Munck A, Pollitt R, et al. A survey of newborn screening for cystic fibrosis in Europe. J Cyst Fibros. 2007 Jan;6(1):57-65.Full text  Abstract

6. Hale JE, Parad RB, Comeau AM. Newborn screening showing decreasing incidence of cystic fibrosis. N Engl J Med. 2008 Feb 28;358(9):973-4. Abstract

7. Scotet V, L'Hostis C, Férec C. The changing epidemiology of cystic fibrosis: incidence, survival and impact of the CFTR gene discovery. Genes (Basel). 2020 May 26;11(6):589.Full text  Abstract

8. Castellani C, Picci L, Tamanini A, et al. Association between carrier screening and incidence of cystic fibrosis. JAMA. 2009 Dec 16;302(23):2573-9.Full text  Abstract

9. Castellani C, Massie J. Newborn screening and carrier screening for cystic fibrosis: alternative or complementary? Eur Respir J. 2014 Jan;43(1):20-3.Full text  Abstract

10. Cystic Fibrosis Foundation. 2022 Cystic Fibrosis Foundation patient registry highlights. Bethesda, MD: Cystic Fibrosis Foundation; 2023.Full text

11. Valapour M, Lehr CJ, Schladt DP, et al. OPTN/SRTR 2021 annual data report: lung. Am J Transplant. 2023 Feb;23(2 suppl 1):S379-S442.Full text  Abstract

12. Salsgiver EL, Fink AK, Knapp EA, et al. Changing epidemiology of the respiratory bacteriology of patients with cystic fibrosis. Chest. 2016 Feb;149(2):390-400. Abstract

13. Hatziagorou E, Orenti A, Drevinek P, et al. Changing epidemiology of the respiratory bacteriology of patients with cystic fibrosis-data from the European cystic fibrosis society patient registry. J Cyst Fibros. 2020 May;19(3):376-83.Full text  Abstract

14. Reynolds D, Kollef M. The epidemiology and pathogenesis and treatment of Pseudomonas aeruginosa infections: an update. Drugs. 2021 Dec;81(18):2117-31.Full text  Abstract

15. Langton Hewer SC, Smith S, Rowbotham NJ, et al. Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis. Cochrane Database Syst Rev. 2023 Jun 2;6(6):CD004197.Full text  Abstract

16. Tarran R, Button B, Picher M, et al. Normal and cystic fibrosis airway surface liquid homeostasis. The effects of phasic shear stress and viral infections. J Biol Chem. 2005 Oct 21;280(42):35751-9.Full text  Abstract

17. Moskwa P, Lorentzen D, Excoffon KJ, et al. A novel host defense system of airways is defective in cystic fibrosis. Am J Respir Crit Care Med. 2007 Jan 15;175(2):174-83.Full text  Abstract

18. Matsui H, Grubb BR, Tarran R, et al. Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airways disease. Cell. 1998 Dec 23;95(7):1005-15. Abstract

19. Knowles MR, Stutts MJ, Spock A, et al. Abnormal ion permeation through cystic fibrosis respiratory epithelium. Science. 1983 Sep 9;221(4615):1067-70. Abstract

20. Knowles M, Gatzy J, Boucher R. Increased bioelectric potential difference across respiratory epithelia in cystic fibrosis. N Engl J Med. 1981 Dec 17;305(25):1489-95. Abstract

21. Joo NS, Irokawa T, Robbins RC, et al. Hyposecretion, not hyperabsorption, is the basic defect of cystic fibrosis airway glands. J Biol Chem. 2006 Mar 17;281(11):7392-8.Full text  Abstract

22. Conner GE, Wijkstrom-Frei C, Randell SH, et al. The lactoperoxidase system links anion transport to host defense in cystic fibrosis. FEBS Lett. 2007 Jan 23;581(2):271-8.Full text  Abstract

23. Boucher RC. New concepts of the pathogenesis of cystic fibrosis lung disease. Eur Respir J. 2004 Jan;23(1):146-58.Full text  Abstract

24. Bals R, Weiner DJ, Meegalla RL, et al. Salt-independent abnormality of antimicrobial activity in cystic fibrosis airway surface fluid. Am J Respir Cell Mol Biol. 2001 Jul;25(1):21-5.Full text  Abstract

25. Ballard ST, Trout L, Mehta A, et al. Liquid secretion inhibitors reduce mucociliary transport in glandular airways. Am J Physiol Lung Cell Mol Physiol. 2002 Aug;283(2):L329-35.Full text  Abstract

26. Picci L, Cameran M, Marangon O, et al. A 10-year large-scale cystic fibrosis carrier screening in the Italian population. J Cyst Fibros. 2010 Jan;9(1):29-35.Full text  Abstract

27. Hamosh A, FitzSimmons SC, Macek M Jr, et al. Comparison of the clinical manifestations of cystic fibrosis in black and white patients. J Pediatr. 1998 Feb;132(2):255-9. Abstract

28. Farrell PM, White TB, Ren CL, et al. Diagnosis of cystic fibrosis: consensus guidelines from the Cystic Fibrosis Foundation. J Pediatr. 2017 Feb;181S:S4-15.e1.Full text  Abstract

29. Whitcomb DC, Buchner AM, Forsmark CE. AGA clinical practice update on the epidemiology, evaluation, and management of exocrine pancreatic insufficiency: expert review. Gastroenterology. 2023 Nov;165(5):1292-301.Full text  Abstract

30. Berke G, Gede N, Szadai L, et al. Bicarbonate defective CFTR variants increase risk for chronic pancreatitis: a meta-analysis. PLoS One. 2022;17(10):e0276397.Full text  Abstract

31. Eigen H, Rosenstein BJ, FitzSimmons S, et al. A multicenter study of alternate-day prednisone therapy in patients with cystic fibrosis. Cystic Fibrosis Foundation Prednisone Trial Group. J Pediatr. 1995 Apr;126(4):515-23. Abstract

32. Comeau AM, Accurso FJ, White TB, et al. Guidelines for implementation of cystic fibrosis newborn screening programs: Cystic Fibrosis Foundation workshop report. Pediatrics. 2007 Feb;119(2):e495-518. Abstract

33. American College of Obstetricians and Gynecologists. Committee opinion no. 691: carrier screening for genetic conditions. Mar 2017 [internet publication].Full text  Abstract

34. Seidler D, Griffin M, Nymon A, et al. Throat swabs and sputum culture as predictors of P. aeruginosa or S. aureus lung colonization in adult cystic fibrosis patients. PLoS One. 2016;11(10):e0164232.Full text  Abstract

35. Watson MS, Cutting GR, Desnick RJ, et al. Cystic fibrosis population carrier screening: 2004 revision of American College of Medical Genetics mutation panel. Genet Med. 2004 Sep-Oct;6(5):387-91.Full text  Abstract

36. Hopkins MK, Dugoff L, Kuller JA. Guidelines for cystic fibrosis carrier screening in the prenatal/preconception period. Obstet Gynecol Surv. 2022 Oct;77(10):606-10. Abstract

37. American College of Medical Genetics. Standards and guidelines for clinical genetics laboratories, 2008 edition. March 2011 [internet publication].Full text

38. American College of Obstetricians and Gynecologists. Committee opinion no. 690: carrier screening in the age of genomic medicine. Mar 2017 [internet publication].Full text  Abstract

39. Castellani C, Southern KW, Brownlee K, et al. European best practice guidelines for cystic fibrosis neonatal screening. J Cyst Fibros. 2009 May;8(3):153-73. Abstract

40. Ren CL, Borowitz DS, Gonska T, et al. Cystic fibrosis transmembrane conductance regulator-related metabolic syndrome and cystic fibrosis screen positive, inconclusive diagnosis. J Pediatr. 2017 Feb;181S:S45-S51.e1.Full text  Abstract

41. Barben J, Castellani C, Munck A, et al. Updated guidance on the management of children with cystic fibrosis transmembrane conductance regulator-related metabolic syndrome/cystic fibrosis screen positive, inconclusive diagnosis (CRMS/CFSPID). J Cyst Fibros. 2021 Sep;20(5):810-9. Abstract

42. Castellani C, Duff AJA, Bell SC, et al. ECFS best practice guidelines: the 2018 revision. J Cyst Fibros. 2018 Mar;17(2):153-78.Full text  Abstract

43. National Institute for Health and Care Excellence. Cystic fibrosis: diagnosis and management. Oct 2017 [internet publication].Full text

44. Mogayzel PJ Jr, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013 Apr 1;187(7):680-9.Full text  Abstract

45. Wilson LM, Morrison L, Robinson KA. Airway clearance techniques for cystic fibrosis: an overview of Cochrane systematic reviews. Cochrane Database Syst Rev. 2019 Jan 24;(1):CD011231.Full text  Abstract

46. Main E, Rand S. Conventional chest physiotherapy compared to other airway clearance techniques for cystic fibrosis. Cochrane Database Syst Rev. 2023 May 5;5(5):CD002011.Full text  Abstract

47. Warnock L, Gates A. Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis. Cochrane Database Syst Rev. 2023 Apr 12;4(4):CD001401.Full text  Abstract

48. McIlwaine M, Button B, Nevitt SJ. Positive expiratory pressure physiotherapy for airway clearance in people with cystic fibrosis. Cochrane Database Syst Rev. 2019 Nov 27;(11):CD003147.Full text  Abstract

49. Hill AT, Barker AF, Bolser DC, et al. Treating cough due to non-CF and CF bronchiectasis with nonpharmacological airway clearance: CHEST Expert Panel report. Chest. 2018 Apr;153(4):986-93.Full text  Abstract

50. Wilson LM, Saldanha IJ, Robinson KA. Active cycle of breathing technique for cystic fibrosis. Cochrane Database Syst Rev. 2023 Feb 2;2(2):CD007862.Full text  Abstract

51. Burnham P, Stanford G, Stewart R. Autogenic drainage for airway clearance in cystic fibrosis. Cochrane Database Syst Rev. 2021 Dec 15;12(12):CD009595.Full text  Abstract

52. Flume PA, Robinson KA, O'Sullivan BP, et al; Clinical Practice Guidelines for Pulmonary Therapies Committee. Cystic fibrosis pulmonary guidelines: airway clearance therapies. Respir Care. 2009 Apr;54(4):522-37.Full text  Abstract

53. Morrison L, Milroy S. Oscillating devices for airway clearance in people with cystic fibrosis. Cochrane Database Syst Rev. 2020 Apr 30;(4):CD006842.Full text  Abstract

54. Cystic Fibrosis Trust (UK). Standards of care and good clinical practice for the physiotherapy management of cystic fibrosis. Fourth ed. Nov 2020 [internet publication].Full text

55. Zisi D, Chryssanthopoulos C, Nanas S, et al. The effectiveness of the active cycle of breathing technique in patients with chronic respiratory diseases: a systematic review. Heart Lung. 2022 May-Jun;53:89-98.Full text  Abstract

56. Cystic Fibrosis Trust. ​A statement on the physiotherapy Vest by the Association of Chartered Physiotherapists in Cystic Fibrosis (ACPCF) [internet pubication].Full text

57. Stanford G, Morrison L, Brown C. Nebuliser systems for drug delivery in cystic fibrosis. Cochrane Database Syst Rev. 2023 Nov 9;11(11):CD007639.Full text  Abstract

58. Moran F, Bradley JM, Piper AJ. Non-invasive ventilation for cystic fibrosis. Cochrane Database Syst Rev. 2017 Feb 20;(2):CD002769.Full text  Abstract

59. Smith S, Rowbotham NJ, Edwards CT. Short-acting inhaled bronchodilators for cystic fibrosis. Cochrane Database Syst Rev. 2022 Jun 24;6(6):CD013666.Full text  Abstract

60. Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;(3):CD001127.Full text  Abstract

61. Wark P, McDonald VM, Smith S. Nebulised hypertonic saline for cystic fibrosis. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD001506.Full text  Abstract

62. Tiddens HAWM, Chen Y, Andrinopoulou ER, et al. The effect of inhaled hypertonic saline on lung structure in children aged 3-6 years with cystic fibrosis (SHIP-CT): a multicentre, randomised, double-blind, controlled trial. Lancet Respir Med. 2022 Jul;10(7):669-78. Abstract

63. Mogayzel PJ Jr, Naureckas ET, Robinson KA, et al; Cystic Fibrosis Foundation Pulmonary Clinical Practice Guidelines Committee. Cystic Fibrosis Foundation pulmonary guideline. Pharmacologic approaches to prevention and eradication of initial Pseudomonas aeruginosa infection. Ann Am Thorac Soc. 2014 Dec;11(10):1640-50.Full text  Abstract

64. Langton Hewer SC, Smyth AR, Brown M, et al. Intravenous or oral antibiotic treatment in adults and children with cystic fibrosis and Pseudomonas aeruginosa infection: the TORPEDO-CF RCT. Health Technol Assess. 2021 Nov;25(65):1-128.Full text  Abstract

65. Hewer SCL, Smyth AR, Brown M, et al. Intravenous versus oral antibiotics for eradication of Pseudomonas aeruginosa in cystic fibrosis (TORPEDO-CF): a randomised controlled trial. Lancet Respir Med. 2020 Oct;8(10):975-86.Full text  Abstract

66. Smith S, Rowbotham NJ. Inhaled anti-pseudomonal antibiotics for long-term therapy in cystic fibrosis. Cochrane Database Syst Rev. 2022 Nov 14;11(11):CD001021.Full text  Abstract

67. Langton Hewer SC, Smyth AR. Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis. Cochrane Database Syst Rev. 2017 Apr 25;(4):CD004197.Full text  Abstract

68. Palser S, Smith S, Nash EF, et al. Treatments for preventing recurrence of infection with Pseudomonas aeruginosa in people with cystic fibrosis. Cochrane Database Syst Rev. 2019 Dec 17;(12):CD012300.Full text  Abstract

69. Rosenfeld M, Rayner O, Smyth AR. Prophylactic anti-staphylococcal antibiotics for cystic fibrosis. Cochrane Database Syst Rev. 2020 Sep 30;(9):CD001912.Full text  Abstract

70. Lo DK, Muhlebach MS, Smyth AR. Interventions for the eradication of meticillin-resistant Staphylococcus aureus (MRSA) in people with cystic fibrosis. Cochrane Database Syst Rev. 2022 Dec 13;12(12):CD009650.Full text  Abstract

71. Bhatt J, Jahnke N, Smyth AR. Once-daily versus multiple-daily dosing with intravenous aminoglycosides for cystic fibrosis. Cochrane Database Syst Rev. 2019 Sep 4;(9):CD002009.Full text  Abstract

72. Kowalik A, de Monestrol I, Sorjonen K, et al. Antibiotic hypersensitivity in cystic fibrosis - low frequency of anaphylaxis over 16 000 courses. Br J Clin Pharmacol. 2022 Nov;88(11):4845-53.Full text  Abstract

73. Shao H, Shi D, Dai Y. Linezolid and the risk of QT interval prolongation: a pharmacovigilance study of the Food and Drug Administration Adverse Event Reporting System. Br J Clin Pharmacol. 2023 Apr;89(4):1386-92. Abstract

74. Dovey M, Aitken ML, Emerson J, et al. Oral corticosteroid therapy in cystic fibrosis patients hospitalized for pulmonary exacerbation: a pilot study. Chest. 2007 Oct;132(4):1212-8. Abstract

75. Auerbach HS, Williams M, Kirkpatrick JA, et al. Alternate-day prednisone reduces morbidity and improves pulmonary function in cystic fibrosis. Lancet. 1985 Sep 28;2(8457):686-8. Abstract

76. Saiman L, Marshall BC, Mayer-Hamblett N, et al; Macrolide Study Group. Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA. 2003 Oct 1;290(13):1749-56.Full text  Abstract

77. Stick SM, Foti A, Ware RS, et al. The effect of azithromycin on structural lung disease in infants with cystic fibrosis (COMBAT CF): a phase 3, randomised, double-blind, placebo-controlled clinical trial. Lancet Respir Med. 2022 Aug;10(8):776-84. Abstract

78. Fennell PB, Quante J, Wilson K, et al. Use of high-dose ibuprofen in a pediatric cystic fibrosis center. J Cyst Fibros. 2007 Apr;6(2):153-8. Abstract

79. Lands LC, Stanojevic S. Oral non-steroidal anti-inflammatory drug therapy for lung disease in cystic fibrosis. Cochrane Database Syst Rev. 2019 Sep 9;(9):CD001505.Full text  Abstract

80. Balfour-Lynn IM, Welch K, Smith S. Inhaled corticosteroids for cystic fibrosis. Cochrane Database Syst Rev. 2019 Jul 4;(7):CD001915.Full text  Abstract

81. Ren CL, Morgan RL, Oermann C, et al. Cystic Fibrosis Foundation pulmonary guidelines. Use of cystic fibrosis transmembrane conductance regulator modulator therapy in patients with cystic fibrosis. Ann Am Thorac Soc. 2018 Mar;15(3):271-80.Full text  Abstract

82. Rosenfeld M, Wainwright CE, Higgins M, et al; ARRIVAL study group. 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 Jul;6(7):545-53.Full text  Abstract

83. Davies J, Wang LT, Panorchan P, et al. WS06-4 ivacaftor (IVA) treatment in patients 6 to <12 months old with cystic fibrosis with a CFTR gating mutation: results of a 2-part, single-arm, phase 3 study. Paper presented at: 42nd European Cystic Fibrosis Conference. 5-8 Jun 2019. Liverpool, UK. J Cyst Fibros. 2019 Jun 1;18(suppl 1):S11.Full text

84. Davies JC, Wainwright CE, Sawicki GS, et al. Ivacaftor in infants aged 4 to &lt;12 months with cystic fibrosis and a gating mutation. Results of a two-part phase 3 clinical trial. Am J Respir Crit Care Med. 2021 Mar 1;203(5):585-93.Full text  Abstract

85. Skilton M, Krishan A, Patel S, et al. Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2019 Jan 7;(1):CD009841.Full text  Abstract

86. Mitchell RM, Jones AM, Stocking K, et al. Longitudinal effects of ivacaftor and medicine possession ratio in people with the Gly551Asp mutation: a 5-year study. Thorax. 2021 Sep;76(9):874-9. Abstract

87. Bailey J, Rozga M, McDonald CM, et al. Effect of CFTR modulators on anthropometric parameters in individuals with cystic fibrosis: an Evidence Analysis Center systematic review. J Acad Nutr Diet. 2021 Jul;121(7):1364-78.e2. Abstract

88. Chilvers MA, Davies JC, Milla C, et al. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-32. Abstract

89. Walker S, Flume P, McNamara J, et al. A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis. J Cyst Fibros. 2019 Sep;18(5):708-13. Abstract

90. Sawicki GS, Chilvers M, McNamara J, et al. A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant. J Cyst Fibros. 2022 Jul;21(4):675-83.Full text  Abstract

91. Flume PA, Biner RF, Downey DG, et al; VX14-661-110 study group. Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study. Lancet Respir Med. 2021 Jul;9(7):733-46. Abstract

92. Kapouni N, Moustaki M, Douros K, et al. Efficacy and safety of elexacaftor-tezacaftor-ivacaftor in the treatment of cystic fibrosis: a systematic review. Children (Basel). 2023 Mar 15;10(3):554.Full text  Abstract

93. Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single phe508del allele. N Engl J Med. 2019 Nov 7;381(19):1809-19.Full text  Abstract

94. Goralski JL, Hoppe JE, Mall MA, et al. Phase 3 open-label clinical trial of elexacaftor/tezacaftor/ivacaftor in children aged 2-5 years with cystic fibrosis and at least one F508del allele. Am J Respir Crit Care Med. 2023 Jul 1;208(1):59-67.Full text  Abstract

95. Zemanick ET, Taylor-Cousar JL, Davies J, et al. A phase 3 open-label study of elexacaftor/tezacaftor/ivacaftor in children 6 through 11 years of age with cystic fibrosis and at least one F508del allele. Am J Respir Crit Care Med. 2021 Jun 15;203(12):1522-32.Full text  Abstract

96. Heijerman HGM, McKone EF, Downey DG, et al. 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. 2019 Nov 23;394(10212):1940-8. Abstract

97. Barry PJ, Mall MA, Álvarez A, et al. Triple therapy for cystic fibrosis Phe508del-gating and -residual function genotypes. N Engl J Med. 2021 Aug 26;385(9):815-25.Full text  Abstract

98. Sutharsan S, McKone EF, Downey DG, et al. Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial. Lancet Respir Med. 2022 Mar;10(3):267-77. Abstract

99. Mayer-Hamblett N, Ratjen F, Russell R, et al. Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials. Lancet Respir Med. 2023 Apr;11(4):329-40. Abstract

100. Okroglic L, Sohier P, Martin C, et al. Acneiform eruption following elexacaftor-tezacaftor-ivacaftor treatment in patients with cystic fibrosis. JAMA Dermatol. 2023 Jan 1;159(1):68-72. Abstract

101. Baroud E, Chaudhary N, Georgiopoulos AM. Management of neuropsychiatric symptoms in adults treated with elexacaftor/tezacaftor/ivacaftor. Pediatr Pulmonol. 2023 Jul;58(7):1920-30. Abstract

102. Bilton D, Canny G, Conway S, et al. Pulmonary exacerbation: towards a definition for use in clinical trials. Report from the EuroCareCF Working Group on outcome parameters in clinical trials. J Cyst Fibros. 2011 Jun;10 Suppl 2:S79-81.Full text  Abstract

103. Stanford GE, Dave K, Simmonds NJ. Pulmonary exacerbations in adults with cystic fibrosis: a grown-up issue in a changing cystic fibrosis landscape. Chest. 2021 Jan;159(1):93-102.Full text  Abstract

104. Abbott L, Plummer A, Hoo ZH, et al. Duration of intravenous antibiotic therapy in people with cystic fibrosis. Cochrane Database Syst Rev. 2019 Sep 5;(9):CD006682.Full text  Abstract

105. Aldossary S, Shah A. Healthcare Utilization and impact of antifungal stewardships within respiratory care settings: a systematic literature review. Mycopathologia. 2021 Oct;186(5):673-84.Full text  Abstract

106. Saadh MJ, Lohrasbi A, Ghasemian E, et al. The status of carbapenem resistance in cystic fibrosis: a systematic review and meta-analysis. Yale J Biol Med. 2022 Dec;95(4):495-506. Abstract

107. Bonyadi P, Saleh NT, Dehghani M, et al. Prevalence of antibiotic resistance of Pseudomonas aeruginosa in cystic fibrosis infection: A systematic review and meta-analysis. Microb Pathog. 2022 Apr;165:105461.Full text  Abstract

108. Smith S, Rowbotham NJ, Charbek E. Inhaled antibiotics for pulmonary exacerbations in cystic fibrosis. Cochrane Database Syst Rev. 2022 Aug 1;8(8):CD008319.Full text  Abstract

109. Somayaji R, Parkins MD, Shah A, et al; Antimicrobial Resistance in Cystic Fibrosis International Working Group. Antimicrobial susceptibility testing (AST) and associated clinical outcomes in individuals with cystic fibrosis: a systematic review. J Cyst Fibros. 2019 Mar;18(2):236-43.Full text  Abstract

110. Frost F, Shaw M, Nazareth D. Antibiotic therapy for chronic infection with Burkholderia cepacia complex in people with cystic fibrosis. Cochrane Database Syst Rev. 2019 Jun 13;6(6):CD013079.Full text  Abstract

111. Aaron SD, Vandemheen KL, Ferris W, et al. Combination antibiotic susceptibility testing to treat exacerbations of cystic fibrosis associated with multiresistant bacteria: a randomised, double-blind, controlled clinical trial. Lancet. 2005 Aug 6-12;366(9484):463-71. Abstract

112. Nicholson TT, Smith A, McKone EF, et al. Duration of intravenous antibiotic treatment for acute exacerbations of cystic fibrosis: a systematic review. J Cyst Fibros. 2022 Jul;21(4):562-73. Abstract

113. Ramos KJ, Smith PJ, McKone EF, et al. Lung transplant referral for individuals with cystic fibrosis: Cystic Fibrosis Foundation consensus guidelines. J Cyst Fibros. 2019 May;18(3):321-33.Full text  Abstract

114. Shah P, Lowery E, Chaparro C, et al. Cystic fibrosis foundation consensus statements for the care of cystic fibrosis lung transplant recipients. J Heart Lung Transplant. 2021 Jul;40(7):539-56.Full text  Abstract

115. Koutsokera A, Varughese RA, Sykes J, et al. Pre-transplant factors associated with mortality after lung transplantation in cystic fibrosis: a systematic review and meta-analysis. J Cyst Fibros. 2019 May;18(3):407-15.Full text  Abstract

116. Holmes M, Murphy V, Taylor M, et al. Intussusception in cystic fibrosis. Arch Dis Child. 1991 Jun;66(6):726-7.Full text  Abstract

117. Ng C, Major G, Smyth AR. Timing of pancreatic enzyme replacement therapy (PERT) in cystic fibrosis. Cochrane Database Syst Rev. 2021 Aug 2;8(8):CD013488.Full text  Abstract

118. Gilchrist FJ, Green J, Carroll W. Interventions for treating distal intestinal obstruction syndrome (DIOS) in cystic fibrosis. Cochrane Database Syst Rev. 2021 Dec 22;12(12):CD012798.Full text  Abstract

119. Shidrawi RG, Murugan N, Westaby D, et al. Emergency colonoscopy for distal intestinal obstruction syndrome in cystic fibrosis patients. Gut. 2002 Aug;51(2):285-6.Full text  Abstract

120. El Boghdady M, Ewalds-Kvist BM. Laparoscopic surgery in patients with cystic fibrosis: a systematic review. Asian J Endosc Surg. 2021 Jul;14(3):327-34. Abstract

121. Ng J, Friedmacher F, Pao C, et al. Gastroesophageal reflux disease and need for antireflux surgery in children with cystic fibrosis: a systematic review on incidence, surgical complications, and postoperative outcomes. Eur J Pediatr Surg. 2021 Feb;31(1):106-14.Full text  Abstract

122. Dana J, Debray D, Beaufrère A, et al. Cystic fibrosis-related liver disease: clinical presentations, diagnostic and monitoring approaches in the era of CFTR modulator therapies. J Hepatol. 2022 Feb;76(2):420-34.Full text  Abstract

123. Sasame A, Stokes D, Bourke B, et al. The impact of liver disease on mortality in cystic fibrosis - a systematic review. J Cyst Fibros. 2022 Mar;21(2):202-11.Full text  Abstract

124. Kapnadak SG, Dimango E, Hadjiliadis D, et al. Cystic Fibrosis Foundation consensus guidelines for the care of individuals with advanced cystic fibrosis lung disease. J Cyst Fibros. 2020 May;19(3):344-54.Full text  Abstract

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