Randomized Controlled Trial
Copyright ©The Author(s) 2018. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Apr 14, 2018; 24(14): 1550-1561
Published online Apr 14, 2018. doi: 10.3748/wjg.v24.i14.1550
Maintenance for healed erosive esophagitis: Phase III comparison of vonoprazan with lansoprazole
Kiyoshi Ashida, Katsuhiko Iwakiri, Naoki Hiramatsu, Yuuichi Sakurai, Tetsuharu Hori, Kentarou Kudou, Akira Nishimura, Eiji Umegaki
Kiyoshi Ashida, Department of Gastroenterology, Rakuwakai Otowa Hospital, Kyoto 607-8062, Japan
Katsuhiko Iwakiri, Department of Gastroenterology, Nippon Medical School Graduate School of Medicine, Tokyo 113-8603, Japan
Naoki Hiramatsu, Department of Gastroenterology and Hepatology, Osaka Rosai Hospital, Sakai, Osaka 591-8025, Japan
Yuuichi Sakurai, Tetsuharu Hori, Kentarou Kudou, Akira Nishimura, Takeda Pharmaceutical Company Limited, Osaka 540-8645, Japan
Eiji Umegaki, Department of Gastroenterology, Kobe University Graduate School of Medicine, Kobe, Hyogo 650-0017, Japan
Author contributions: Ashida K, Sakurai Y, Hori T and Nishimura A were involved in study conception and design; Hiramatsu N served as Medical Expert; Umegaki E, Iwakiri K and Ashida K served as the Central Adjudication Committee; Kudou K conducted statistical analyses; all authors were involved in the drafting and critical revision of the manuscript, and approved the final version, including the authorship list.
Institutional review board statement: The study was reviewed and approved by the institutional review board of each participating site.
Clinical trial registration statement: This study is registered at ClinicalTrials.gov. The registration identification number is NCT01459367.
Informed consent statement: All study participants provided written informed consent prior to study enrollment.
Conflict-of-interest statement: Kiyoshi Ashida has received fees and honoraria from Takeda Pharmaceutical Company Limited and Otsuka Pharmaceutical Company Limited; Katsuhiko Iwakiri has received grants, fees, and honoraria from Takeda Pharmaceutical Company Limited, and fees from Otsuka Pharmaceutical Company Limited; Yuuichi Sakurai, Tetsuharu Hori, Kentarou Kudou, and Akira Nishimura are full-time employees of Takeda Pharmaceutical Company Limited; Naoki Hiramatsu and Eiji Umegaki have no conflicts of interest to declare.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Kiyoshi Ashida, MD, PhD, Department of Gastroenterology, Rakuwakai Otowa Hospital, 2 Otowachinji-cho, Yamashina-ku, Kyoto 607-8062, Japan. rakuwadr1185@rakuwadr.com
Telephone: +81-75-5934111 Fax: +81-75-5934160
Received: November 30, 2017
Peer-review started: December 1, 2017
First decision: December 13, 2017
Revised: February 6, 2018
Accepted: March 7, 2018
Article in press: March 6, 2018
Published online: April 14, 2018
Processing time: 131 Days and 12.8 Hours
ARTICLE HIGHLIGHTS
Research background

Proton-pump inhibitors (PPIs) such as lansoprazole are widely accepted as the treatment of choice for acid-related disorders, including erosive esophagitis (EE). Nevertheless, agents of this class are associated with notable shortcomings, which include: significant inter-individual variability in the time to onset of action; reduced night-time efficacy in preventing acid regurgitation, leading to nocturnal acid breakthrough; and differences in plasma concentrations and acid-inhibitory effects in extensive versus poor CYP2C19 metabolizers.

Vonoprazan fumarate (TAK-438) belongs to a relatively new class of acid suppressants known as potassium-competitive acid blockers (P-CABs), which, by virtue of their novel mechanism of action, offer a number of potential advantages over PPIs in the treatment of acid-related disorders. In animal studies, vonoprazan provided more potent and sustained suppression of gastric acid secretion than lansoprazole, while studies in healthy human volunteers demonstrated rapid, sustained, and dose-related suppression of 24-h gastric acid secretion. The present study adds to these earlier findings by confirming that vonoprazan is non-inferior to lansoprazole in preventing EE recurrence in Japanese patients with healed EE.

Research motivation

As a result of the increasingly widespread adoption of a westernized lifestyle and the general aging of the population, EE is now the most common acid-related disorder in Japan. Typical symptoms of EE include heartburn, acid reflux, difficulty swallowing, and sore throat, which can negatively impact patients’ quality of life. In Japan, as elsewhere, PPIs remain the mainstay of treatment for EE and other acid-related disorders; however, in view of the limitations of PPIs mentioned above, there is a need for new treatment modalities that offer greater efficacy and more consistent outcomes. Any treatments that improve outcomes in EE may also be beneficial in gastroesophageal reflux disease, duodenal ulcer, and other acid-related disorders, and could become the focus of a new area of research.

Research objectives

The main objective of the research described in this paper was to demonstrate that the efficacy of vonoprazan in preventing EE recurrence is comparable to that of lansoprazole at its established maintenance dose. This objective was realized, with the results obtained confirming that vonoprazan, at doses of 10 and 20 mg once daily, is non-inferior to lansoprazole 15 mg once daily as maintenance therapy for healed EE. In addition, the safety profile of vonoprazan was shown to be similar to that of lansoprazole at the doses investigated. These findings suggest that vonoprazan may be a viable alternative to PPIs in the maintenance of EE healing, and provide a basis for future clinical trials to establish the optimal positioning of this new agent in the treatment of acid-related disorders.

Research methods

To establish the non-inferiority of vonoprazan 10 and 20 mg to lansoprazole 15 mg as maintenance therapy in Japanese patients with endoscopically-confirmed healed EE, we designed and conducted a multicenter, double-blind, randomized, phase III clinical trial. Eligible patients received vonoprazan 10 or 20 mg, or lansoprazole 15 mg, once daily for 24 wk. The primary and secondary endpoints were the rate of EE recurrence at Weeks 24 and 12, respectively; safety outcomes were also evaluated. Based on EE recurrence rates in previous studies, it was calculated that 174 patients per treatment group would be required to provide > 90% power to confirm the non-inferiority of vonoprazan 10 and 20 mg to lansoprazole 15 mg.

Research results

We found that vonoprazan, administered at a dose of 10 or 20 mg once daily, is non-inferior to lansoprazole 15 mg once daily in maintaining EE healing in Japanese patients over a period of 24 wk, and demonstrates a comparable safety profile. Post-hoc analyses also confirmed that both doses of vonoprazan investigated provide more consistent EE healing than lansoprazole, even in patients who are CYP2C19 extensive metabolizers and those with severe (Los Angeles grade C/D) EE. These results add to our previous findings that vonoprazan 5-40 mg once daily is non-inferior to lansoprazole 30 mg once daily in terms of EE healing rates over a 4-wk period, and that vonoprazan 20 mg once daily is non-inferior to lansoprazole 30 mg once daily in terms of 8-wk EE healing rates. As the maintenance period in this study was relatively short, further studies are needed to establish the long-term efficacy and safety characteristics of vonoprazan in the maintenance of EE healing.

Research conclusions

To our knowledge, this study is the first to confirm that vonoprazan is non-inferior to lansoprazole once daily in maintaining EE healing in Japanese patients. Importantly, it is also the first to show that vonoprazan is more consistent in maintaining EE healing, even in extensive CYP2C19 metabolizers and patients with more severe disease. These findings appear to confirm that the novel mechanism of action of vonoprazan is associated with advantages versus PPIs in the treatment of acid-related disorders, and suggest that vonoprazan could be an important new addition to the range of treatment options available to clinicians.

Research perspectives

This study confirms that vonoprazan demonstrates efficacy comparable with that of lansoprazole not only in healing EE, but also in maintaining the healing of EE over 24 wk. Future research should focus on evaluating the longer-term efficacy and safety of vonoprazan in this indication. In addition to randomized controlled trials, observational studies should be undertaken to gather valuable real-life data and inform decisions regarding the optimal positioning of vonoprazan in the management of EE.