REVIEW ARTICLE: ENDPOINTS USED IN FUNCTIONAL DYSPEPSIA DRUG THERAPY TRIALS

Daphne Ang (1) , Nicholas J Talley (2) , Magnus Simrén (3) , Pieter Janssen (4) , Guy E Boeckxstaens (5) , Jan Tack (6)

1 Gastroenterology
2 Faculty of Health
3 Dept of Internal Medicine
4 KULeuven
5 Pathophysiology
6 Center for Gastroenterological Research

Background: How to measure patient reported outcomes (PRO) in treatment trials for functional gastrointestinal disorders is a matter of controversy. This review focuses 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, analog 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. In conclusion, there is an urgent need to develop Rome III-based PROs 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.

Mots clés: Medicine

Daphne Ang, Nicholas J Talley, Magnus Simrén, Pieter Janssen, Guy E Boeckxstaens, et al.. REVIEW ARTICLE: ENDPOINTS USED IN FUNCTIONAL DYSPEPSIA DRUG THERAPY TRIALS. Alimentary Pharmacology and Therapeutics, 2011, 33 (6), pp.634. ⟨10.1111/j.1365-2036.2010.04566.x⟩. ⟨hal-00608029⟩