Review article: endpoints used in functional dyspepsia drug therapy trials.

INDIGESTION treatment; GASTROINTESTINAL disease treatment; METABOLIC disorders; HEALTH outcome assessment; CLINICAL trials

Background The measurement of patient-reported outcomes (PRO) in treatment trials for functional gastrointestinal disorders is a matter of controversy. Aim To focus on instruments and endpoints that have been used to evaluate the efficacy of therapeutic agents in functional dyspepsia (FD) trials, also considering the newly defined Rome III FD criteria. Methods A Medline search was conducted to identify relevant studies pertaining to FD treatment, with particular emphasis on the studies to date which have used validated outcome measures. Results Currently available outcome measures are heterogeneous across studies. They include global binary endpoints, analogue or categorical scoring scales, uni- or multi-dimensional disease specific questionnaires, global outcome evaluations and quality of life questionnaires. Across the available outcome measures, substantial heterogeneity is found, not only in the type of endpoint measure, but also in the number and types of symptoms that are considered to be part of the FD symptom complex. Especially based on content validity, none of the existing questionnaires or endpoints can be considered sufficiently validated to be recommended unequivocally as the primary outcome measure for FD trials according to the Rome III criteria. On the other hand, existing well-validated multi-dimensional questionnaires that include many non-FD symptoms can be narrowed down to evaluate only the cardinal symptoms according to Rome III. Conclusions There is an urgent need to develop Rome III-based patient-reported outcomes for functional dyspepsia. Well-validated multi-dimensional questionnaires may serve as a guidance for this purpose, and could also be considered for use in ongoing clinical trials. [ABSTRACT FROM AUTHOR]