Outcome measures for clinical trials in cystic fibrosis. Summary of a Cystic Fibrosis Foundation consensus conference.

The CFF Consensus Conference concluded with a summary of those outcome measures that would be most useful in studies of patients 6 years of age and older and those measures that would be most useful in studies of the younger population (< 6 years of age) (Table). These measures were further divided into biologic markers most appropriate for initial (phase I and phase II) clinical trials and those especially useful in large, multicenter (phase III) pivotal trials. There is an ongoing need to improve the accuracy and validity of currently available measures of biologic activity and clinical efficacy in CF, especially in the younger population. The conference participants recommended that the following eight issues be addressed as soon as possible: (1) definition of pulmonary exacerbation, (2) broadly applicable methods of testing pulmonary function in small children (ideally a single test for all ages), (3) a comprehensive severity-of-disease score for young children, (4) reliable methods of quantifying chest x-ray and CT scan changes in young patients, (5) simple, inexpensive measures of lung inflammation, (6) a centralized, uniform approach to the establishment of data monitoring committees, (7) a quality of well-being scale for small children, and (8) reliable, reproducible aerosol delivery systems with defined characteristics. In addition, participants recommended that better methods be developed for assessing patients' adherence to research protocols.