Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Achalasia
Treatment: Per Oral Endoscopic Myotomy (POEM)
The health plan should not cover the POEM.

The requested POEM is not likely to be more beneficial than any standard treatment(s) for the patient's condition or disease.

In a prospective case series, von Renteln and colleagues (2013) reported on outcomes of 70 patients who underwent POEM at five centers in Europe and North America. The mean followup period was ten months (range, three to twelve months). Follow-up evaluation at six months and one year showed sustained treatment success of 89% and 82%, respectively. The mean Eckhardt score pretreatment was 6.9 compared with 1.3 at six months and 1.7 at one year (p<0.001 for both comparisons). Multivariate analysis showed that neither age, previous treatment (Botox/dilatation), length of the myotomy, pre-procedure lower esophageal sphincter (LES) pressure, initial Eckardt score, sex, procedure duration, nor full-thickness dissection during POEM were significant predictors of treatment failure at one year. At three months after POEM, esophagitis was observed in 42% of cases. However, the severity of esophagitis was only minor (Grade A or B) and all patients could be managed adequately with proton pump inhibitor (PPI) therapy. At three months, 22% of patients required occasional and 12% required daily PPI therapy. The one-year follow-up evaluation showed overall rates of gastroesophageal reflux disease (GERD) of 37%, and PPI use of 29%. Other complication rates of POEM ranged from 1%-4%.

The study by Ren and colleagues (2012) highlights some of these POEM-specific complications. In their series of 119 cases, 23% of patients developed subcutaneous emphysema intraoperatively and an additional 56% postoperatively. Three of these patients required treatment with subcutaneous needle decompression. Additionally, three percent of their patients developed a pneumothorax intraoperatively and another 25% postoperatively. Postoperatively, the incidence of thoracic effusion was 49%, and of mild inflammation or segmental atelectasis of the lungs was 50%. All complications were resolved with conservative treatment.

At least two small case series have evaluated the efficacy and feasibility of POEM for patients with failed Heller myotomy/achalasia recurrence; success rates have been reported in over 90% of cases up to ten months after rescue POEM. Studies have also been undertaken comparing different POEM techniques; comparable outcomes have been reported between patients undergoing full-thickness versus circular myotomy. 

The current evidence for POEM in individuals with achalasia includes nonrandomized comparative studies and case series. Relevant outcomes are symptoms, functional outcomes, health status measures, resource utilization, and treatment-related morbidity. The comparative studies showed mostly similar outcomes with POEM versus Heller myotomy for the outcome of symptom relief as assessed by the Eckardt score. Some studies showed shorter length of stay and less postoperative pain with POEM. However, potential imbalance in patient characteristics in these nonrandomized studies may bias the comparisons between treatments. In the case series studies, treatment success at short follow-up periods was reported for a high proportion of patients treated with POEM. However, incidence of adverse events was relatively high, with POEM specific complications, including subcutaneous emphysema, pneumothorax, and thoracic effusion, reported across studies. Additionally, a substantial proportion of patients undergoing POEM developed esophagitis requiring treatment. The case series studies do not allow conclusions of the efficacy of POEM relative to established treatment. Long-term outcomes of the procedure are not well described in the literature. The evidence is insufficient to determine the effects of the technology on health outcomes at this time.