Farmacotherapie bij adolescente en volwassen patienten met cystische fibrose

Abstract

Cystic fibrosis (CF) is the most common potentially fatal inherited disease in the white population. In patients with CF, abnormal chloride transport across the apical membrane of epithelial cells has been identified, causing inspissated secretions in the airways, pancreas and other organs. Clinical manifestations result from obstruction in ducts or organs followed by tissue destruction and inflammatory response due to viscid secretions. Most morbidity and mortality arises from pulmonary disease. Pulmonary infection usually occurs with Haemophilus influenzae, Staphylococcus aureus and Pseudomonas aeruginosa. Pharmacotherapeutic treatment modalities have been focused on substituting pancras enzymes and suppression of the chronic pulmonary infection. However, in case Burkholderia cepacia is isolated from the airways, this is associated with an increased deterioration of the pulmonary function and a high mortality. There is an urgent need for antibiotics with sufficient activity against B. cepacia. New therapeutic modalities are directed towards the normalization of the ion transport across the membranes and the suppression of the pulmonary inflammatory response. If the pulmonary ion transport could be normalized, it should be possible to treat the pulmonary manifestations of the disease