Skip to main content
Bulletin of the World Health Organization logoLink to Bulletin of the World Health Organization
. 1994;72(3):341–352.

Therapeutic approaches to cystic fibrosis: memorandum from a joint WHO/ICF(M)A meeting.

PMCID: PMC2486697  PMID: 7520369

Abstract

Cystic fibrosis is one of the commonest genetic diseases among Caucasians and represents an important cause of suffering and death among children and adults. In the past two decades marked prolongation of the life of patients with cystic fibrosis has been achieved as the result of improved case-finding and an extensive regimen of therapies. More recently, a variety of new approaches to therapy have been developed or proposed as the result of advances in cell physiology and molecular biology. This article summarizes the presentations and discussions made at a joint WHO/ICF(M)A (International Cystic Fibrosis (Mucoviscidosis) Association) meeting, held in Washington, DC, on 14 October 1992, and reviews the current status of possible therapies for cystic fibrosis and their implications for treatment in various countries of the world.

Full text

PDF
341

Selected References

These references are in PubMed. This may not be the complete list of references from this article.

  1. Anderson M. P., Berger H. A., Rich D. P., Gregory R. J., Smith A. E., Welsh M. J. Nucleoside triphosphates are required to open the CFTR chloride channel. Cell. 1991 Nov 15;67(4):775–784. doi: 10.1016/0092-8674(91)90072-7. [DOI] [PubMed] [Google Scholar]
  2. Bartholomew L. K., Parcel G. S., Seilheimer D. K., Czyzewski D., Spinelli S. H., Congdon B. Development of a health education program to promote the self-management of cystic fibrosis. Health Educ Q. 1991 Winter;18(4):429–443. doi: 10.1177/109019819101800403. [DOI] [PubMed] [Google Scholar]
  3. Cheng S. H., Gregory R. J., Marshall J., Paul S., Souza D. W., White G. A., O'Riordan C. R., Smith A. E. Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis. Cell. 1990 Nov 16;63(4):827–834. doi: 10.1016/0092-8674(90)90148-8. [DOI] [PubMed] [Google Scholar]
  4. Crystal R. G. Gene therapy strategies for pulmonary disease. Am J Med. 1992 Jun 22;92(6A):44S–52S. doi: 10.1016/0002-9343(92)90607-d. [DOI] [PubMed] [Google Scholar]
  5. Drumm M. L., Pope H. A., Cliff W. H., Rommens J. M., Marvin S. A., Tsui L. C., Collins F. S., Frizzell R. A., Wilson J. M. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell. 1990 Sep 21;62(6):1227–1233. doi: 10.1016/0092-8674(90)90398-x. [DOI] [PubMed] [Google Scholar]
  6. Hubbard R. C., McElvaney N. G., Birrer P., Shak S., Robinson W. W., Jolley C., Wu M., Chernick M. S., Crystal R. G. A preliminary study of aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis. N Engl J Med. 1992 Mar 19;326(12):812–815. doi: 10.1056/NEJM199203193261207. [DOI] [PubMed] [Google Scholar]
  7. Jensen T., Pedersen S. S., Høiby N., Koch C., Flensborg E. W. Use of antibiotics in cystic fibrosis. The Danish approach. Antibiot Chemother (1971) 1989;42:237–246. [PubMed] [Google Scholar]
  8. Knowles M. R., Church N. L., Waltner W. E., Yankaskas J. R., Gilligan P., King M., Edwards L. J., Helms R. W., Boucher R. C. A pilot study of aerosolized amiloride for the treatment of lung disease in cystic fibrosis. N Engl J Med. 1990 Apr 26;322(17):1189–1194. doi: 10.1056/NEJM199004263221704. [DOI] [PubMed] [Google Scholar]
  9. Knowles M. R., Clarke L. L., Boucher R. C. Activation by extracellular nucleotides of chloride secretion in the airway epithelia of patients with cystic fibrosis. N Engl J Med. 1991 Aug 22;325(8):533–538. doi: 10.1056/NEJM199108223250802. [DOI] [PubMed] [Google Scholar]
  10. Lannefors L., Wollmer P. Mucus clearance with three chest physiotherapy regimes in cystic fibrosis: a comparison between postural drainage, PEP and physical exercise. Eur Respir J. 1992 Jun;5(6):748–753. [PubMed] [Google Scholar]
  11. Madden B. P., Hodson M. E., Tsang V., Radley-Smith R., Khaghani A., Yacoub M. Y. Intermediate-term results of heart-lung transplantation for cystic fibrosis. Lancet. 1992 Jun 27;339(8809):1583–1587. doi: 10.1016/0140-6736(92)91842-v. [DOI] [PubMed] [Google Scholar]
  12. Mador J. A., Smith D. H. The psychosocial adaptation of adolescents with cystic fibrosis. A review of the literature. J Adolesc Health Care. 1989 Mar;10(2):136–142. doi: 10.1016/0197-0070(89)90104-6. [DOI] [PubMed] [Google Scholar]
  13. Marks M. I. Antibiotic therapy for bronchopulmonary infections in cystic fibrosis. The American approach. Antibiot Chemother (1971) 1989;42:229–236. doi: 10.1159/000417624. [DOI] [PubMed] [Google Scholar]
  14. Oberwaldner B., Evans J. C., Zach M. S. Forced expirations against a variable resistance: a new chest physiotherapy method in cystic fibrosis. Pediatr Pulmonol. 1986 Nov-Dec;2(6):358–367. doi: 10.1002/ppul.1950020608. [DOI] [PubMed] [Google Scholar]
  15. Pearson D. A., Pumariega A. J., Seilheimer D. K. The development of psychiatric symptomatology in patients with cystic fibrosis. J Am Acad Child Adolesc Psychiatry. 1991 Mar;30(2):290–297. doi: 10.1097/00004583-199103000-00019. [DOI] [PubMed] [Google Scholar]
  16. Quinton P. M., Reddy M. M. Control of CFTR chloride conductance by ATP levels through non-hydrolytic binding. Nature. 1992 Nov 5;360(6399):79–81. doi: 10.1038/360079a0. [DOI] [PubMed] [Google Scholar]
  17. Ramsey B. W., Farrell P. M., Pencharz P. Nutritional assessment and management in cystic fibrosis: a consensus report. The Consensus Committee. Am J Clin Nutr. 1992 Jan;55(1):108–116. doi: 10.1093/ajcn/55.1.108. [DOI] [PubMed] [Google Scholar]
  18. Riordan J. R., Rommens J. M., Kerem B., Alon N., Rozmahel R., Grzelczak Z., Zielenski J., Lok S., Plavsic N., Chou J. L. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989 Sep 8;245(4922):1066–1073. doi: 10.1126/science.2475911. [DOI] [PubMed] [Google Scholar]
  19. Shak S., Capon D. J., Hellmiss R., Marsters S. A., Baker C. L. Recombinant human DNase I reduces the viscosity of cystic fibrosis sputum. Proc Natl Acad Sci U S A. 1990 Dec;87(23):9188–9192. doi: 10.1073/pnas.87.23.9188. [DOI] [PMC free article] [PubMed] [Google Scholar]

Articles from Bulletin of the World Health Organization are provided here courtesy of World Health Organization

RESOURCES