COMMENTARY

Achalasia Treatment: Latest Updates

David A. Johnson, MD

Disclosures

January 24, 2020

This transcript has been edited for clarity.

Hello. I'm Dr David Johnson, professor of medicine and chief of gastroenterology at Eastern Virginia Medical School in Norfolk, Virginia.

Achalasia is a diagnosis we all learned about during our medical training, and which we deal with not infrequently in gastroenterology focused on the esophagus. In the past week alone, I've had two cases of achalasia referred to me. One is a patient who had a diagnosis of food impaction, whose physician thought it was probably achalasia. They scheduled a manometry and administered Botox, followed by a referral to a general surgeon for evaluation for surgical myotomy. The second is a physician who had a diagnosis of achalasia suspected at the time of endoscopy and confirmed by impedance manometry, and wanted to know what the best treatment options are.

It just so happens that the treatment paradigm for achalasia is changing dramatically. This makes for a good opportunity to review the latest when it comes to treating this condition.

Weighing the Achalasia Treatment Options

A welcome clarification of treatment options was recently provided in the form of evidence-based practice guidelines from the American Society for Gastrointestinal Endoscopy (ASGE), as well as in a randomized controlled trial just published in The New England Journal of Medicine comparing a new option with an old option.

Before we discuss these, let's review the current treatment options for achalasia.

Botox is an option that can be administered via a standard endoscopy. The intramuscular injection of Botox inhibits the release of acetylcholine. The ASGE guidelines suggest that this has an efficacy in the range of 75%. The problem with this treatment is that it's not durable. It's relatively quick and simple to administer, but it just doesn't last.

The second option is pneumatic dilation, which has changed dramatically in recent years. Pneumatic dilation went from the old Rider-Moeller and Brown-McHardy dilators to the very graduated balloon dilation that is now in use. With this current technique, we start with a 30-mm balloon, then go to a 35-mm balloon, etc. (I personally never do a 40-mm balloon.) The safety profile has improved substantially with these graded dilations. Whereas the perforation risk with older techniques was around 3%, it's dramatically lower with graded balloon dilation; in my personal experience, perforation has never occurred. Treating this condition over time using a graded dilation seems to be remarkably safe.

The third option is the standard Heller myotomy generally followed by a fundoplication to prevent reflux. This procedure is associated with an efficacy in the range of 87%, so it's not perfect. The complications that I've personally seen with this procedure are the ones you'd expect when working around the esophagogastric junction, such as vagal nerve injury and gastroparesis. Therefore, this is not a procedure that should be done casually. It's critical that you seek an expert surgeon who has done this with regularity, not just occasionally.

The fourth option is peroral endoscopic myotomy (POEM). Professor Inoue from Tokyo, Japan, preformed the first case of POEM in 2008 with a natural orifice type of approach. In 2010 in the journal Endoscopy, Professor Inoue published results with the first 17 patients treated with POEM and showed a remarkable improvement following myotomy performed endoscopically. In doing so, he basically proved that POEM could be done. A recent endoscopic analysis combined with a meta-analysis looked at nearly 2400 patients, a number that was possible given how incredibly well recognized and popular this procedure has become. It showed that there was a remarkable efficacy achieved with POEM in types I through III achalasia. Importantly, POEM was recommended in a clinical practice update from the American Gastroenterological Association (AGA), particularly for spastic (type III) achalasia, as it was by an international consensus statement, both of which were published in 2017. So it's clear that POEM has become remarkably safe and effective, particularly in type III achalasia.

Direct Comparisons of Achalasia Treatments

The ASGE guideline went through available data regarding the treatment of achalasia.

When it comes to Botox versus pneumatic dilation, the latter is favored by a 30% difference in clinical success. Botox just doesn't work as well. Pneumatic dilation, in the hands of an expert, simply works better. Botox should be reserved for very fragile patients. You also have to weigh the risk for tachyphylaxis and the fact that patients can develop antibodies that lead to them not responding as well to Botox treatment. Therefore, Botox really should not be endorsed as a primary treatment over a longer period of time.

Secondly, pneumatic dilation has shown itself to be comparable to Heller myotomy with fundoplication. Randomized controlled trials have shown the value to be roughly on par for these two approaches.

But where does POEM fit in this arena of treatment options?

POEM has not really been systematically evaluated against pneumatic dilation. The three studies [two retrospective studies and one randomized clinical trial] included in the ASGE guidelines suggest that POEM has a numerical advantage, but the guideline committee decided that they did not meet the standards of a meta-analysis. Again, we should remember that pneumatic dilation should only be offered by experts, and this is even more true of POEM.

Then there's the question of how POEM compares with Heller myotomy coupled with an antireflux procedure. There was no randomized controlled trial on this subject when the ASGE guidelines were being put together, but there is one now published in The New England Journal of Medicine. This is a study of 221 patients prospectively randomized and not subject to the type of achalasia. No patients who received POEM were subject to conversion to Heller myotomy, and there were no major adverse events that required surgery as it relates to POEM. So I think this comparison shows that POEM is certainly noninferior to Heller myotomy.

For type III achalasia, we just don't do as well with anything that is not POEM. The reason is that POEM creates a longer myotomy. Also, regardless of whether we use pneumatic dilation, Botox, or Heller myotomy, we don't get the effectiveness for chest pain like we do with POEM. Chest pain is the primary posttreatment complaint of patients with type III achalasia, with the exception of those undergoing POEM, which is really now the treatment of choice in this indication.

Help Guide Your Patients to the Right Treatment

The ASGE guideline gives us a lot of good advice.

First of all, you need to discuss all three definitive options—pneumatic dilation, Heller myotomy, and now POEM—for all types of achalasia. For type III achalasia, POEM is absolutely the preferred strategy. I think there's a good consensus on this based on the AGA guideline, the expert recommendation, and the international consensus statement.

We need to put POEM on the map. This is a revolutionary new approach to the treatment of achalasia that appears remarkably safe. I think the surgical approach to achalasia is waning and the endoscopic approach is emerging.

But you also need to recognize that these procedures are extremely technical and high-risk. They require an expert, not only in endoscopy but also in anatomy. The POEM safety data have been remarkable, but they come from procedures performed by experts. There is a learning curve associated with POEM, with a minimum number of 20-25 procedures, with more recent evidence suggesting that this begins plateauing around nearly 75-100 procedures.

You need to choose your POEM expert well. Pick somebody whom you know and trust, but also look at experience, seeking those who perform these procedures in high-volume centers of expertise. Patients need to receive this treatment elsewhere if it cannot be performed to current best-practice recommendations. In my mind, it is not acceptable treatment for this disease to default to convenience rather than expertise, as this has important implications for long-term treatment success.

We need to discuss all of the treatment options with our patients, and even if patients do not avail themselves of this option [POEM], they at least need to have it discussed with them.

The treatment of achalasia has changed considerably over the past 40 years that I have been in the field. We now have a great guidance document from the ASGE, and a great randomized controlled trial showing that POEM is not inferior to Heller myotomy. These data are welcome and show that patients are subject to improved status and qualified benefit. Hopefully these data improve your discussions with patients with achalasia.

I look forward to talking with you again soon. Thanks for listening.

David A. Johnson, MD, a regular contributor to Medscape, is professor of medicine and chief of gastroenterology at Eastern Virginia Medical School in Norfolk, Virginia, and a past president of the American College of Gastroenterology. His primary focus is the clinical practice of gastroenterology. He has published extensively in the internal medicine/gastroenterology literature, with principal research interests in esophageal and colon disease, and more recently in sleep and microbiome effects on gastrointestinal health and disease.

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