CURRENT APPROACHES TO THE THERAPY OF CYSTIC FIBROSIS


Cite item

Full Text

Abstract

The article presents the main trends in the treatment of cystic fibrosis: traditional methods with well-proven efficacy for survival and preservation of pulmonary function, as well as innovative technology for optimization of antibacterial and anti-inflammatory therapy, pharmacological modeling of ion transport, functional recovery of protein - cystic fibrosis transmembrane regulator.

References

  1. Капранов Н.И., Каширская Н.Ю., Шерман В.Д. и др. Муковисцидоз. Современные достижения и актуальные проблемы. Методические рекомендации. М., 2011. С. 64-5.
  2. CF Foundation Patient Registry 2007 (ECFS Patient Registry Report 2007 data).
  3. Dodge JA, Lewis PA, Stanton M, Wilsher J. Cystic fibrosis mortality and survival in the UK: 1947-2003. Eur Respir J 2007;29:522-26.
  4. Doring G, Hoiby N, for the Consensus Study Group. Early intervention and prevention of lung disease in cystic fibrosis: a European consensus. J Cyst Fibros 2004;3:67-91.
  5. Красовский C.А., Черняк А.В., Амелина Е.Л. и др. Survival analysis of cystic fibrosis patient in Moscow region of Russia in 2000-2010. Abstr. 372; J Cyst. Fibr 2011;10(1):95.
  6. Sinaasappel M, Stern M, Littlewood J, et al. Nutrition in patient with cystic fibrosis: a European Consensus. J Cyst Fibros 2002;1:64.
  7. Hodson M, Duncan G, Bush A. Cystic fibrosis. Third ed. by London: Edward Arnold (Publishers) Ltd, 2007:477.
  8. Gibson RL, Emerson J, Mayer-Hamblett N, et al. Duration of treatment effect after tobramycin solution for inhalation in young children with cystic fibrosis. Pediatr Pulmonol 2007;42:610-23.
  9. Капранов Н.И., Каширская Н.Ю., Радионович А.М., Амелина Е.Л., Чучалин А.Г. и др. Клиническое значение специальной аэрозольной формы тобрамицина в лечении хронического бронхолегочного процесса у больных муковисцидозом // Пульмонология 2008 № 3. С. 20-6.
  10. Retsch-Bogart GZ, Quittner A, Montgomery AB, et al. Phase 3 trial (AIR-CF1) measuring improvement in respiratory symptoms in patients with cystic fibrosis (CF) following treatment with aztreonam lysine for inhalation (AZLI). Pediatr Pulmonol 2007;42(30):310.
  11. FDA Grants Orphan Drug Status to Ciprofloxacin Dry Powder Inhaler for the Treatment of Cystic Fibrosis: http://insciences.org/article.
  12. Meers P, Neville M, Malinin V, et al. Biofilm penetration, triggered release and in vivo activity of inhaled liposomal amikacin in chronic Pseudomonas aeruginosa lung infections. J Antimicrob Chemother 2008;61(4):859-68.
  13. Geller DE, Flume PA, Staab D, et al. Levofloxacin Inhalation Solution (MP-376) in Patients with Cystic Fibrosis with Pseudomonas aeruginosa. Am J Respir Crit Care Med 2011;183(11):1510-16.
  14. Cystic Fibrosis in children and adults. The Leeds Method of Management. November 7, 2008, St James's University Hospital, UK.
  15. Капустина Т.Ю. Изменения печени и их коррекция при муковисцидозе у детей: Дисс. канд. мед. наук. М., 2001.
  16. Каширская Н.Ю. Состояние желудочно-кишечного тракта, поджелудочной железы и гепатобилиарной системы у больных муковисцидозом. Дисс. докт. мед. наук. М., 2001.
  17. Dondos V, Westaby D. Liver, biliary and pancreatic disease. Cystic Fibrosis, Third edition, Edited by Hodson M, Geddes D, Bush A. London, 2007:225-35.
  18. Konstan MW, Berger M. Infection and inflammation of the lung in cystic fibrosis. In: Davis PB, editor. Cystic fibrosis. New York: Marcel Dekker Inc, 1993.
  19. Капранов Н.И., Передерко Л.В., Каширская Н.Ю., Пухальский А.Л., Толстова В.Д., Шмарина Г.В. Системные и ингаляционные кортикостероиды в комплексном лечении бронхолегочных поражений у детей с муковисцидозом // Педиатрия им. Г.Н. Сперанского 2009. № 1. С. 60-8.
  20. van Haren EHJ, Lammers JWJ, Festen J, et al. The effects of the inhaled corticosteroid budesonide on lung function and bronchial hyperresponsiveness in adult patients with cystic fibrosis. Resp Med 1995;89:209-14.
  21. Balfour-Lynn IM. Multicenter randomised controlled trial of withdrawal of inhaled corticosteroids in cystic fibrosis. Am J Respir Crit Care Med 2006;173:1356-62.
  22. Patrick A. Flume, Brian P, O'Sullivan, Karen A, Robinson, et al. Cystic Fibrosis Pulmonary Guidelines. Am J Respir Crit Care Med 2007;176:957-69.
  23. Bush A. Rubin BK. Macrolides as biologic response modifiers in cystic fibrosis and bronchiectasis. Semin Resp Crit Care Med 2003;24:737-47.
  24. Equi A. Balfour, Lynn I, Bush A. Rosenthal M. Long term azithromycin in children with cystic fibrosis: a randomized, placebo-controlled crossover trial. Lancet 2002;360:978-84.
  25. Saiman S, Marshall BC, Meyer-Hamblett N, et al. Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA 2003;290:1749-56.
  26. Радионович А.М., Каширская Н.Ю., Капранов Н.И. Длительное применение макролидов при хронической синегнойной инфекции у больных муковисцидозом // Consilium medicum. Частные вопросы педиатрии 2006. Т. 8. № 1. С. 68-72.
  27. McElvaney NG, Hubbard RC, Birrer P, et al. Aerosol ?1-antitrypsin treatment for cystic fibrosis. Lancet 1991;4:337-92.
  28. McElvaney NG, Doujaiji B, Moan MJ, et al. Pharmacokinetics of recombinant secretory leukoprotease inhibitor aerosolised to normals and individuals with cystic fibrosis. Am Rev Respir Dis 1993;148:1056-60.
  29. Vogelmeier C, Gillissen A, Buhl R. Use of secretory leukoprotease inhibitor to augment lung antineutrophil elastase activity. Chest 1996;110:261-66.
  30. Martin SL, Downey D, Bilton D, et al. Safety and efficacy of recombinant alpha1-antitrypsin therapy in cystic fibrosis. Pediatr Pulmonol 2006;41:177-83.
  31. Griese M, Latzin P, Kappler M, et al. ?1-antitrypsin inhalation reduces airway inflammation in cystic fibrosis patients. Eur Respir J 2007;29:240-50.
  32. Brennan S. Revisiting ?1-antitrypsin therapy in cystic fibrosis: can it still offer promise? Eur Respir J 2007;29:229-30.
  33. Гинтер Е.К., Капранов Н.И., Н.В.Петрова и др. Муковисцидоз / В кн. Генетика бронхолегочных заболеваний. М., 2010. C. 51-78.
  34. Frederiksen B, Pressler T, Hansen A, Koch C. Effect of aerosolized rhDNase (Pulmozyme) on pulmonary colonisation in patients with cystic fibrosis. Acta Paediatr 2006;95:11.
  35. Paul K, Rietschel E, Ballman M, et al. Effect of treatment with dornase alpha on airway inflammation in patients with cystic fibrosis. Bronchoalveolar lavage for the Evaluation of Anti-inflammatory Treatment Study Group. Am J Respir Crit Care Med 2004;169:719-25.
  36. Богданова Т.А., Каширская Н.Ю., Толстова В.Д., Капранов Н.И. Клиническая эффективность и безопасность пульмозима (дорназа альфа) у детей раннего возраста, больных муковисцидозом // Российский вестник перинатологии и педиатрии 2008. Т. 53. № 4. С. 35-42.
  37. Donaldson SH. Bennett WD, Zeman KL, et al. Boucher RC. Mucus clearanceand lung function in cystic fibrosis with hypertonic saline. N Engl J Med 2006;354:241-50.
  38. Hughes T, Clifton IJ, Peckham DG, et al. A retrospective study to assess the response to nebulised salbutamol during a period of stability in adult patients with cystic fibrosis. J Cyst Fibros 2006;5(1):44.
  39. Proesmans M, Vermeulen F, De Boeck K. What's new in cystic fibrosis? From treating symptoms to correction of the basic defect. Eur J Pediatr 2008;167(8):839-49.
  40. Kerem E. Mutation specific therapy in CF. Paediatr Respir Rev 2006;7:(1):166-69.
  41. Wilschanski M, Yahav Y, Yaakov Y, Blau H et al. Gentamicin-induced correction of of CFTR function in patients with cystic fibrosis and CFTR stop mutations. N Engl J Med 2003;349:1433-41.
  42. Sermet-Gaudilus I, Renouil M, Fajac A, et al In vitro prediction of stop codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study. BMC Med 2007;5:5.
  43. Linde L, Boelz S, Nissim-Rafinia M, et al. Ninsense-mediated mRNA decay affects nonsense transcript levels and governs response of cystic fibrosis patients to gentamicin. J Clin Invest 2007;117:1-9.
  44. Kerem E, Hirawat S, Armoni S, et al Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. Lancet 2008;372:719-27.
  45. Wilschanski M, Miller LL, Shoseyov D, et al. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur Respir J 2011;38(1):59-69.
  46. Accurso F. The Tiger-1 clinical trial on denufosol in cystic fibrosis. Ped Pumonol 2008;31:191.
  47. Grasemann H, Stehling F, Brunar H, et al Inhalation of moli1901 in patients with cystic fibrosis. Chest 2007;131:1461-66.

Supplementary files

Supplementary Files
Action
1. JATS XML

This website uses cookies

You consent to our cookies if you continue to use our website.

About Cookies