Challenges in assessing outcomes for pediatric asthma

Abstract

Although progress has been made in understanding asthma, much remains unknown; as a result, diagnosis and treatment are not optimal. Information is still needed about the natural history of asthma to better understand which patients are at risk of inflammation and at what point during their disease that inflammation occurs. In the case of children, a greater understanding of the disease's natural history is needed to establish criteria for early diagnosis and to evaluate progressive aspects of the disease so that appropriate measures of progression can be defined. The safety of various medications used as long-term controllers must also be evaluated. The growing information about medication effects on asthma provides unique opportunities to design studies that will guide improvement in asthma care. A number of disease outcomes can be used as surrogate markers of clinically significant endpoints, and a number of adverse effects may serve as surrogate markers. The consequences of poor control of pediatric asthma can be observed in a variety of clinical markers, including clinical features of the disease that worsen as it progresses, pulmonary function deterioration, lung hyperexpansion, and inflammation increases. The Childhood Asthma Management Program is an ongoing clinical trial designed to improve the diagnosis and treatment of asthma in children. Its hypothesis is that anti-inflammatory treatment of childhood asthma will not only relieve morbidity but will also improve lung growth.