Clinical Question: Is itopride (not available in the United States, U.S. Food and Drug Administration approval pending) effective for the treatment of functional (nonulcer) dyspepsia?

Setting: Outpatient (any)

Study Design: Randomized controlled trial (double-blinded)

Allocation: Concealed

Synopsis: Like its cousin cisapride (Propulsid, removed from the market because of cardiac side effects), itopride is a prokinetic agent used in Japan to treat functional dyspepsia. In this study, the first well-designed clinical trial of itopride for that indication, the authors randomized (allocation concealed) 554 patients with functional dyspepsia based on the Rome II criteria to receive itopride 50 mg three times daily, 100 mg three times daily, or 200 mg three times daily, or placebo. All underwent ultrasonography and upper endoscopy to exclude ulcer, malignancy, or other structural causes for their symptoms.

The groups were balanced at the start of the study; the mean age of participants was 47 years, with a range of 18 to 94 years. Approximately two thirds of participants were women. Outcomes were assessed blindly, and analysis was by intention to treat.

The primary outcome was the score on the validated Leeds Dyspepsia Questionnaire (LDQ), which rates eight symptoms from 0 (not present) to 5 (very severe). There were no significant differences between the three dosages of itopride, so the results reported for all three doses were pooled. This is reasonable given the statistical homogeneity of those findings.

After eight weeks, the average patient’s LDQ score improved 4.5 points in the placebo group and 6.2 points in the itopride group, a change that is of borderline clinical significance. Patients also assessed the global degree of improvement (marked or complete) and were more likely to respond to itopride than to placebo (59.9 versus 41.2 percent; P < .001; number needed to treat = 6). There was no difference among groups regarding minor or serious adverse events during the trial.

Bottom Line: Itopride was somewhat effective for functional dyspepsia. The drug appears to be safe on the basis of this small, short study. (Level of Evidence: 1b)