Evidence-based medicines for children: Important implications for new therapies at all ages. Society for Clinical Respiratory Physiology

摘要

It has long been known that 50-90% of medications used in children are "off-label" or unlicensed, and this is associated with an increased risk of adverse events [1-14]. This means that some of the most vulnerable people in the European Union (EU), sick children, are being treated in a non-evidence-based, unregulated fashion. Even commonly prescribed respiratory medicines, such as short-acting p2-agonists and antipyretics, have not been adequately studied in young children [15]. The risks of the present situation include: death or serious adverse events from overdosing; nonavailability to children of important medications; under-dosing leading to loss of efficacy; and the use of poor-quality formulae. Side-effects that may not affect adults might be very important in children; the effects of inappropriately prescribed inhaled steroids on growth is well known [16, 17], but could the use of parenteral steroids affect alveolar development in the first 2 years of life [18, 19]? Furthermore, there may be fundamental differences between adult and paediatric diseases; several studies have established that adult asthma is a chronic inflammatory disease, but many wheezing phenotypes in early childhood are not inflammatory and are thus poorly responsive to corticosteroids [20-29]. Some welcome steps have been taken to address this, with the publication of the evidence-based Medicines for Children by the Royal College of Paediatrics and Child Health [30], and the first edition of a Paediatric British National Formulary [31] replacing the generic one. However, evidence-based therapeutics requires evidence and this has been lacking.