UP-TO-DATE MANAGEMENT OF LUNG DISEASE IN CHILDREN AND ADOLESCENTS WITH CYSTIC FIBROSIS

Abstract

Cystic fibrosis (CF) is a multi-organ disease,  affecting mostly lungs and gastrointestinal tract. Data from patient registries show that the survival of patients with CF has progressively improved over the past several decades, as a result of advances in antibiotic treatment, supplementation of pancreatic enzymes, better nutrition and a holistic approach to treatment in CF centres. The purpose of this review is to survey recent developments in the treatment of lung disease  in children and adolescents with CF. We describe newborn screening for CF. When chronic respiratory insufficiency occurs, lung transplantation becomes a very important issue. Lung disease is the most common cause of morbidity and mortality in CF patients. Emerging new therapies are targeted at all points in the pathogenesis of lung disease, from drugs that treat infection and inflammation in the airways to gene transfer studies  and to drugs that augment airway surface liquid height. A number of antibacterial agents formulated for inhalation are at various stages of study and there are several anti-inflammatory candidate drugs in  clinical trials.  The most important development  in the recent years is  modulation of the abnormal protein that causes CF, the cystic fibrosis transmembrane regulator (CFTR), where drugs are targeted at specific defects in the transcription, processing or functioning. When chronic respiratory insufficiency occurs, lung transplantation becomes a very important issue. The role of the CF nurse, who has responsibilities in educating and teaching clinical skills to patients and families, is described.