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La fibrosis quística (FQ) era considerada, hasta hace pocos años, la enfermedad genética más frecuente en la infancia. Sin embargo, en la actualidad, su supervivencia ha aumentado de forma considerable, debido a la mejoría en las nuevas terapias. Entre éstas se hace mención a aquellas que ya están en vigencia, así como a otras que están en vías de investigación. En este artículo intentamos acercarnos más al conocimiento de esta enfermedad y a los tratamientos que han conseguido incrementar la calidad de vida de estos pacientes.
Palabras clave:
Fibrosis quística
Trasplante de pulmón
ADNrh recombinante
Terapia génica
Only a few years ago, cystic fibrosis (CF) was considered the most frequent genetic disease in childhood, although survival has increased considerably in recent years owing to improved treatment. We discuss treatments that are still relevant as well as others that are under investigation now, aiming for better understanding of the disease and the therapies that have improved quality of life for CF patients.
Key words:
Cystic fibrosis
Lung transplant
Recombinant DNArh
Gene therapy
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Bibliografía
[1.]
P.A. Lewis.
The epidemiology of cystic fibrosis.
Cystic fibrosis, pp. 1-14
[2.]
T.M. Egan, F.C. Detterbeck, M.R. Mill, L.J. Paradowski, R.P. Lackner, W.D. Ogden, et al.
Improved results of lung transplantation for patients with cystic fibrosis.
J Thorac Cardiovasc Surg, 109 (1995), pp. 224-235
[3.]
P.B. Davis.
Fibrosis quística: nuevas ideas, nuevas estrategias.
Hosp Prac, 8 (1993), pp. 21-35
[4.]
A. Harris.
Cystic fibrosis.
Q J Med, 88 (1995), pp. 229-232
[5.]
P.L. Shah, S.F. Scott, D.M. Geddes, M.E. Hodson.
Two years experience with recombinant human DNasa I in the treatment of pulmonary disease in cystic fibrosis.
Respir Med, 89 (1995), pp. 499-502
[6.]
R.P. Tomkiewicz, E.M. App, J.G. Zayas, O. Ramírez, N. Church, R.C. Boucher, et al.
Amiloride inhalation therapy in cystic fibrosis. Influence on ion content, hydration, and rheology of sputum.
Am Rev Respir Dis, 148 (1993), pp. 1.002-1.007
[7.]
P.G. Middleton, D.M. Geddes, E.W.F.W. Alton.
Effect of amiloride and saline on nasal mucociliary clearance and potential difference in cystic fibrosis and normal subjects.
Thorax, 48 (1993), pp. 812-816
[8.]
E.M. App, M. King, R. Helfesrieder, D. Kohler, H. Matthys.
Acute and long-term amiloride inhalation in cystic fibrosis lung disease.
Am Rev Respir Dis, 141 (1990), pp. 605-612
[9.]
A. Graham, A. Hasani, E.W.F.W. Alton, G.P. Martin, C. Marriott, M.E. Hodson, et al.
No added benefit from nebulized amiloride in patients with cystic fibrosis.
Eur Respir J, 6 (1993), pp. 1.243-1.248
[10.]
M.R. Knowles, N.L. Church, W.E. Waltner, J.R. Yankaskas, P. Gilligan, M. King, et al.
A pilot study of aerolized amiloride for the treatment of lung disease in cystic fibrosis.
N Engl J Med, 322 (1995), pp. 1.189-1.194
[11.]
M.R. Knowles, L.L. Clarke, R.C. Boucher.
Activation by extracellular nucleotides of chloride secretion in the airway epithelia of patients with cystic fibrosis.
N Engl J Med, 325 (1991), pp. 533-538
[12.]
I.M. Balfour-Lyn, R. Dinwiddie.
Role of corticoids in cystic fibrosis lung diseases.
J R Soc Med, 89 (1996), pp. 8-13
[13.]
N.J. Lewinston, R.B. Moss.
Circulating immune complexes decrease during corticosteroids therapy in cystic fibrosis.
Pediatr Res, 49 (1982), pp. 207-212
[14.]
P. Greally, M.J. Hussain, D. Vergani, J.F. Price.
Interleukin-1, soluble interleukin-2 receptor, and IgG concentrations in cystic fibrosis treated with prednisolone.
Arch Dis Child, 71 (1994), pp. 35-39
[15.]
H.S. Auerbach, M. Williams, J.A. Kirkpatrick, H.R. Cohen.
Alternate day prednisolone reduces the morbidity and improves pulmonary function in cystic fibrosis.
Lancet, 2 (1985), pp. 686-688
[16.]
B.J. Rosenstein, H. Eigen.
Risks of alternate-day prednisone in patients with cystic fibrosis.
Pediatrics, 87 (1991), pp. 245-246
[17.]
E.H.J. Van Haren, J.W.J. Lammers, J. Festen, H.G.M. Heijerman, C.A.R. Groot, C.L.A. Van Herwaarden.
The effects of the inhaled corticosteroid budesonide on lung function and bronchial hyperresponsiveness in adult patients with cystic fibrosis.
Respir Med, 89 (1995), pp. 209-214
[18.]
W.J. Nikoaizik, M.H. Schôlni.
Effects of inhaled corticosteroids on lung function-a prospective study.
Eur Respir J, 7 (1994), pp. 430
[19.]
W.H. Nikoaizik, M.H. Schölni.
Pilot study to asses the efect of inhaled corticosteroids on lung function in patients with cystic fibrosis.
J Pediatr, 128 (1996), pp. 271-274
[20.]
H.S. Auerbach, M. Williams, J.A. Kirkpatrick, H.R. Colten.
Alternateday prednisone reduces morbidity and improves pulmonary function in cystic fibrosis.
Lancet, 2 (1985), pp. 686-688
[21.]
B.J. Rosenstein, H. Eigen.
Risks of alternate-day prednosone in patients with cystic fibrosis.
Pediatrics, 87 (1991), pp. 245-246
[22.]
M.W. Konstan, P.J. Byard, C.L. Hoppel, P.B. Davis.
Effect of highdose ibuprofen in patients with cystic fibrosis.
N Engl J Med, 332 (1995), pp. 848-854
[23.]
M.W. Konstan, C.L. Hoppel, B.L. Chai, P.B. Davis.
Ibuprofen in children with cystic fibrosis: pharmacokinetics and adverse effects.
J Pediatr, 118 (1991), pp. 956-964
[24.]
H.R. Colten.
Airway inflammation in cystic fibrosis.
N Engl J Med, 332 (1995), pp. 886-887
[25.]
N.G. McElvaney, R.C. Hubbard, P. Birrer, M.S. Chemiack, D.B. Caplan, M.M. Frank, et al.
Aerosol alfa 1-antitrypsin treatment for cystic fibrosis.
Lancet, 337 (1991), pp. 392-394
[26.]
M. Robinson, J.A. Regnis, D.L. Bailey, M. King, G.J. Bautovich, P.T.P. Bye.
Effect of hypertonic saline, amiloride, and cough on mucociliary clearance in patients with cystic fibrosis.
Am J Respir Crit Care Med, 153 (1996), pp. 1.503-1.509
[27.]
M.E. Hodson, P.L. Shah.
DNasa trials in cystic fibrosis.
Eur Respir J, 8 (1995), pp. 1.786-1.791
[28.]
S.P. Range, A.J. Knox.
rhDNasa in cystic fibrosis.
Thorax, 59 (1995), pp. 321-322
[29.]
P.L. Shah, S.F. Scott, H.J. Fuchs, D.M. Geddes, M.E. Hodson.
Medium term treatment of stable stage cystic fibrosis with recombinant human DNasa I.
Thorax, 30 (1995), pp. 333-338
[30.]
H. Shennib.
Heart-lung and bilateral lung transplantation in children with cystic fibrosis.
Pediatr Pulmonol, 11 (1995), pp. 76-78
[31.]
B.P. Madden.
Lung transplantation.
Cystic fibrosis, pp. 329-346
[32.]
P. Helms.
Commentary to heart-lung transplantation: all the facts.
Arch Dis Child, 66 (1991), pp. 1.016-1.017
[33.]
J.M. Wilson.
Adenoviruses as gene-delivery vehicles.
N Engl J Med, 334 (1996), pp. 1.185-1.186
[34.]
Y. Yang, G. Trinchieri, J.M. Wilson.
Recombinant IL-I2 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repetead gene therapy to mouse lung.
Nat Med, 1 (1995), pp. 890-893
[35.]
J.M. Wilson.
Gene therapy for cystic fibrosis: challenges and future directions.
J Clin Invest, 96 (1995), pp. 2.547-2.554
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