Short- and long-term efficacy. Childhood asthma.

In clinical studies of asthma, great attention should be focused on the choice and validation of outcome measures. The outcome parameters to be used when evaluating efficacy of early intervention in childhood asthma treatment must be closely linked to the aims of the intervention and considered in the design of the study. In an interventional study, possible adverse consequences should also be considered as outcome measures when designing the study. The appropriate time span for assessing different types of interventions may vary from days to several decades, depending on the character of the intervention. Not only outcome parameters, such as improvement in symptom score and improvement of lung function, but also those that measure reduction in frequency and severity of acute exacerbations, reduction in morbidity and improvement in quality of life should be used. For some purposes, a more detailed approach to symptom severity is needed, such as separation of acute from chronic symptoms. Other outcome measures that need to be considered are: cost-effectiveness, normalization of inflammatory changes in the airways, control of airway hyperresponsiveness, airway growth and prevention of airway remodelling and importantly, whether an intervention against asthma can alter the natural course, or cure, the disease. Interventions should be evaluated to see to what extent such aims have been met.