Review
Copyright ©The Author(s) 2015. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastrointest Pharmacol Ther. Nov 6, 2015; 6(4): 145-155
Published online Nov 6, 2015. doi: 10.4292/wjgpt.v6.i4.145
Pharmacotherapy for the management of achalasia: Current status, challenges and future directions
Ammar Nassri, Zeeshan Ramzan
Ammar Nassri, Department of Internal Medicine, University of Texas at Austin Dell Medical School, Austin, TX 78701, United States
Zeeshan Ramzan, Gastrointestinal Section, Department of Internal Medicine, VA North Texas Healthcare System, Dallas, TX 75216, United States
Zeeshan Ramzan, Department of Medicine, Division of Gastroenterology and Hepatology, University of Texas Southwestern Medical Center at Dallas, Dallas, TX 75390, United States
Author contributions: Nassri A and Ramzan Z contributed equally to this work.
Conflict-of-interest statement: The authors have no conflicts of interest.
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: Zeeshan Ramzan, MD, Assistant Professor, Gastrointestinal Section, Department of Internal Medicine, VA North Texas Healthcare System, 4500 S. Lancaster Road, Dallas, TX 75216, United States. zeeshanramzan@hotmail.com
Telephone: +1-214-8571591
Received: June 24, 2015
Peer-review started: June 26, 2015
First decision: August 26, 2015
Revised: September 6, 2015
Accepted: October 16, 2015
Article in press: October 19, 2015
Published online: November 6, 2015
Processing time: 141 Days and 11.6 Hours
Abstract

This article reviews currently available pharmacological options available for the treatment of achalasia, with a special focus on the role of botulinum toxin (BT) injection due to its superior therapeutic effect and side effect profile. The discussion on BT includes the role of different BT serotypes, better pharmacological formulations, improved BT injection techniques, the use of sprouting inhibitors, designer recombinant BT formulations and alternative substances used in endoscopic injections. The large body of ongoing research into achalasia and BT may provide a stronger role for BT injection as a form of minimally invasive, cost effective and efficacious form of therapy for patients with achalasia. The article also explores current issues and future research avenues that may prove beneficial in improving the efficacy of pharmacological treatment approaches in patients with achalasia.

Keywords: Botulinum toxin; Pharmacotherapy; Botox; Achalasia; Sprouting inhibitors

Core tip: Botulinum toxin (BT) injection is the most common and effective pharmacological therapy used in the treatment of achalasia, and is commonly used in the elderly, those with multiple comorbidities, patients at high risk for surgery and as a salvage therapy. This article discusses new advances related to the pharmacological management of achalasia that may help to optimize minimally invasive treatment approaches in patients with achalasia, and discusses improvements in endoscopic injection techniques, the use of sclerosants, new BT formulations, alternate serotypes, sprouting inhibitors and designer recombinant BT formulations.