Efficacy of Vonoprazan 10 mg and 20 mg for Patients With Proton Pump Inhibitor-Refractory Functional Dyspepsia: A Double-Blinded, Randomized Study.

BACKGROUND

A proportion of patients with functional dyspepsia (FD) have inadequate symptom control with proton pump inhibitors (PPIs) treatment. Vonoprazan demonstrates higher efficacy in acid reduction than PPI; however, the existing efficacy data for vonoprazan in treating PPI-refractory FD is limited.

METHODS

This double-blinded, randomized controlled trial study was conducted at Rajavithi Hospital, Bangkok between December 2022 and 2023. Patients with FD who were unresponsive to the standard dose PPI were randomly assigned (1:1) to receive either 10 mg or 20 mg of vonoprazan for a 4-week duration, with a subsequent 4-week follow-up after treatment. The primary outcome was changes in the Global Overall Symptoms Scale (GOSS).

RESULTS

Sixty patients were randomized without significant differences in baseline characteristics between both groups. The mean GOSS between the 10-mg vonoprazan and the 20-mg vonoprazan arm were 25.73 and 26.17 at week 0, 14.33 and 15.50 at week 2, 9.37 and 10.04 at week 4, and 9.79 and 9.33 at week 8, respectively (all  < 0.001 vs. baseline and  > 0.05 between groups). The quality of life was improved, with the Nepean dyspepsia index changing -4.13 and -4.25 at week 4, respectively (all  < 0.001 vs. baseline;  = 0.853 between groups). Symptom response rates (> 50% improvement in GOSS) were 72.4% and 75.9% at week 8, respectively ( = 0.24 between groups). No serious adverse events were observed.

CONCLUSION

Vonoprazan demonstrated significant effects in the alleviation of symptoms in PPI-refractory FD patients. There was no statistically significant difference in symptom alleviation between the 10 mg and 20 mg doses of vonoprazan.