Despite the abundance of literature on management of acha- lasia, there appears to be a relative paucity of prospective, randomized controlled studies that directly compare different treatment modalities. Until recently, there have only been two really effective methods of treating patients with achalasia: forceful dilation and surgery. Data from uncontrolled retrospective trials have, in general, suggested that success rates were higher after surgery. In a prospective, randomized, controlled trial comparing PD (using a Mosher bag) with surgical anterior esophagomyotomy, Csendes et al demonstrated a five-year response rate of 65% versus 95% in clear favour of surgery.
The common limitation of PD and BTX injection is the lack of long term efficacy, with the need for repeat intervention. The literature has tried to compare the efficacy of one technique with the other. The definition of long term and short term efficacy appears arbitrary because it varies between authors. While some will define one-year follow-up data as short term, others will define a similar time span as long term. Annese et al were the first to publish a randomized, controlled, double-blind study comparing BTX with placebo injections and with PD of treatment failures. One or two BTX injections appeared as effective as PD in short term relief of dysphagia. However, 87% (seven of eight) of the patients required a second injection within one year of the first injection. Studies published in abstract form comparing BTX with PD in a prospective, randomized manner are summarized in Table 1. Preliminary data from Bansal et al suggested that the ‘short term efficacy’ (less than 12 months) of PD and BTX injections are equivalent, but initial treatment failure was significantly higher with BTX (six of 12 patients). On the contrary, Vaezi et al found a significantly higher “long term” efficacy (less than 12 months) with PD compared with BTX. Initial treatment failures, however, were similar for both treatment modalities in this study. Although reviews and meta-analyses clearly suggest a longer lasting effect from a single dilation than from a single BTX injection, a recent study by Prakash et al using survival analysis suggested that BTX injections given as needed have an efficacy similar to a single PD within the first two years of injection. In addition, retreatment with BTX injections may delay the need for subsequent injections.
A major problem in comparative studies is the relative rarity of the disease and the difficulty in maintaining long term follow-up in patients over a five- to 10-year period. The Csendes et al trial took about 15 years to complete and is unlikely to be reproduced. The debate should perhaps now be refocused, not only on efficacy of treatment regimens, but on issues of cost effectiveness and quality of life. As an example, it has been estimated that the long term (seven years) cumulative costs of surgery (approximately $20,000) are nearly two-and-a-half times more than those of PD, even taking into account the perforation rate and need for retreat- ment. A preliminary cost analysis of BTX injection, PD and laparoscopic Heller myotomy with fundoplication using decision analysis found PD to be the ‘least costly initial strategy’.
CONCLUSIONS AND RECOMMENDATIONS
It should be clear from this brief review that the final word on treatment of achalasia is far from being written. Despite some recent advances, therapy remains palliative and each method has a significant drawback. Nevertheless, most patients can expect to gain good to excellent symptomatic relief of their obstructive symptoms and improvement in their quality of life. With the variety of options available, it is now increasingly possible to tailor the treatment to the needs of the patient. Thus, most young patients, desirous of a single permanent treatment, may be offered a laparascopic myotomy with the caveat that they should be prepared for long term reflux monitoring and treatment. Others may opt for PD with good expectations from one or two dilations in the first five years. Patients fearful of perforation or those felt to be at high risk for more aggressive therapy may be given a choice of BTX injection, with the understanding that repeat injections will be required periodically.
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