Medical Device Daily National Editor
The patient has severe heartburn after eating or late at night – sometimes with symptoms even suggesting heart attack – goes to the doctor and is told he or she has reflux, or, more technically, gastroesophageal reflux disease (GERD), one of the most common gastrointestinal conditions seen in primary care and GI clinics.
The patient is usually offered a group of standard things to do as a way of dealing with these painful episodes: lose weight (if overweight), stop smoking, alter the diet (away from spicy and fatty foods and others considered as digestive culprits); raise the head of the bed a few inches (to use gravity to retard the reflux); use and stick to a prescribed regimen of drugs known as proton pump inhibitors (PPIs).
And maybe the doctor suggests periodic endoscopic looks at the esophagus as further management and to check if the problem worsens.
Trouble is, except for the use of drugs to deal with simple heartburn or esophagitis – defined as inflammation of the esophagus, an "erosive" category – none of these common recommendations are based on evidence of effectiveness from randomized clinical trials (RCTs) or other rigorous validating methods. And even defining the line between simple heartburn and GERD, between GERD and more serious results of the disease, is hard to pin down.
These are the conclusions of a new report and set of guidelines from the American Gastroenterological Association (AGA; Bethesda, Maryland), which acknowledges and emphasizes the general lack of rigorous evidence to support the management of GERD, while attempting to offer some direction for better management based on what evidence is available, even if not RCT-based.
GERD is considered the failure of the sphincter at the bottom of the esophagus (lower esophageal sphincter, or LES) to work properly, allowing stomach acids to return (reflux) back up, causing pain and sometimes damaging the lining of the esophagus.
While the guidelines specify no particular controlled studies for the value of PPI use in GERD, Michael Vaezi, MD, a gastroenterologist at Vanderbilt University Medical Center (Nashville, Tennessee), and a member of the guidelines writing group, told Medical Device Daily that the main emphasis of the guidelines is to provide direction for managing those patients that do not respond to drug therapies, such as the use of PPIs.
"Where people do not improve," he said, "that's where the evidence is lacking."
While he said there is "strong evidence" for PPI therapy, the guidelines make no reference to RCTs proving this, and the document states: "Initiating empirical treatment with a PPI amounts to a pragmatic therapeutic trial; if a patient reports symptoms consistent with GERD and response to therapy for GERD, then he or she must have GERD" – but that this is not a clinical test.
The guidelines use this same circularity of approach in depending on the patient's report of symptoms to define GERD, rather than specific imaging or laboratory definitions.
The guidelines employ what is called "the Montreal definition" of GERD, which says the disease is best seen when it becomes "troublesome" to the patient and significantly interferes with daily activities. And it acknowledges as well that determination of the more serious stages of the disease must be considered "arbitrary."
In a press statement introducing the guidelines, the AGA says that while physicians "primarily rely on empirical trials of medications," they are only using "their own observations and experience" in doing this, because "high-quality clinical trials for GERD management strategies do not exist."
"There are many methods physicians are using to treat their patients with GERD, without knowing which is the best one," said John Allen, MD, chair of the AGA Institute's Clinical Practice and Quality Management Committee. Thus, the guidelines are an attempt "to encapsulate the major management issues leading" to consultation and treatment.
While the guidelines acknowledge that certain interventions appear to work with some patients, or sometimes many, this is only the result of "pragmatic" knowledge on the part of physicians.
Thus, while weight loss, smoking cessation, raising the bed and other "behavioral modifications" may at times show some effectiveness and do no harm, they are all labeled in the guidelines as "dubious," in terms of proven validity.
The use of PPIs is given the highest ("A") recommendation by the guideline writers, but they also note that PPI ineffectiveness fails to demonstrate either the presence or absence of GERD.
Even the diagnostic approach of endoscopy is put in an extremely marginal, if not entirely useless, category.
The guidelines say that "usually" PPIs are prescribed "empirically" before testing, and so "the sensitivity of endoscopy as a diagnostic test for GERD is poor but may be useful in management."
Acknowledged is the availability of endoscopic surveillance to screen for Barrett's esophagus – abnormal cell development in the esophagus considered as a precursor to cancer – in the GERD population. But such recommendations, according to the document, are based "solely on expert opinion," not on substantiation by "direct data."
Overall, the guidelines find no benefit for either primary testing or screening for Barrett's esophagus, "despite the ubiquity" of their use.
And the document is directly critical of diagnostic attempts to link GERD with "extrasophageal syndromes" (such as asthma, chronic cough and laryngitis), increasingly considered as symptoms GERD. It says the attempts at pinning down this association "have proven elusive, and the premature adoption of flawed diagnostic criteria has likely resulted in the over-diagnosis of extrasophagelal GERD syndromes."
The result, it adds, have been "multiple [often repeated] diagnostic tests and expensive unsuccessful therapies."
In its conclusion section, the guidelines provide rankings from Grade A ("strongly recommended," based on evidence) to Grade D ("Recommend against, fair evidence that it is ineffective or harms outweigh benefits.")
It provides only four conclusions in the "A" category, generally recommending the use of PPIs and only choosing anti-reflux surgery as an alternative to be "recommended" when drug therapy doesn't work.
But the body of the guidelines indicates anti-reflux surgery as only a last resort, given the possibility of troublesome after-effects. And as a Grade C conclusion ("Balance of benefits and harms is too close to justify a general recommendation"), the document says that the "deleterious effect" of anti-reflux surgery may include "sysphagia, flatulence, an inability to belch, and postsurgery bowel symptoms."
And it points to data indicating the general decline in surgical procedures for GERD, primarily known as laparoscopic Nissen fundoplication.
It says that "enthusiastic endorsement" of this procedure drove the number of these surgeries from 11,000 a year in 1985 to 31,695 in 1999, but then fell 30% to just under 24,000 procedures in 2003.
Vaezi told MDD he did not have an explanation for this but guessed that greater use by over-the-counter medications by patients had reduced the amount of esophagitis, thus reducing the need for anti-reflux surgeries.