Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis:  Type III Achalasia
Treatment:  POEM
The health plan should cover the POEM.

The patient is a female with ineffective esophageal motility and type III achalasia.  The attending physician (AP) is requesting treatment with peroral endoscopic myotomy (POEM).
The requested health service or procedure is likely to be more beneficial than any standard treatment(s) for the patient's condition or disease.

POEM is approved for the treatment of achalasia.  POEM is highly efficacious in the short-term management of achalasia.  According to a 2014 summary of 14 studies of POEM, clinical success, defined as a post-treatment Eckardt score of less than three (< 3) and/or a greater than (>) 50 percent decrease in the lower esophageal sphincter (LES) pressure, was achieved in 82 to 100 percent of patients.  Other studies documented similar patient improvements after POEM using either timed barium esophagram or quality of life assessment. 

Three studies provided data on intermediate to long-term efficacies of POEM: 

In a German retrospective study of 80 patients who were followed for greater than two years after POEM, 77 patients (96 percent) achieved initial clinical response, of which 14 (18 percent) developed recurrent achalasia, making the overall success rate of POEM 78 percent at 24 to 49 months. 

In a Japanese retrospective study of 500 consecutive patients (of which 61 patients were followed for three years or longer), clinical success was observed in 91 percent at one to two years after POEM, which decreased to 88 percent at three years. 

In an American retrospective study of 115 patients who underwent POEM at a single high-volume center, the overall success rate was 92 percent at an average of 28 months (range 12 to 52 months).  The efficacy and safety of POEM is comparable with those of laparoscopic Heller myotomy. 

In a meta-analysis of 486 patients in seven nonrandomized comparative studies, there were no significant differences between patients who underwent POEM versus laparoscopic Heller myotomy in terms of reduction in Eckardt score, operative time, postoperative pain or analgesic requirements, and POEM had less complications. 

Per Inoue, H., et al. (2015), "Per-oral endoscopic myotomy was successfully completed in all cases, even when extended indications (extremes of age, previous interventions, or sigmoid esophagus) were used. Adverse events were rare (3.2%), and there were no mortalities.  Significant improvements in Eckardt scores and LES pressures were seen at 2 months, 1 year, and 3 years post-POEM.  Based on our large series, POEM is a safe and effective treatment for achalasia; there are relatively few contraindications, and the procedure may be used as either first- or second-line therapy." 

Patel, K., et al. (2016), "In conclusion, POEM is a safe and effective treatment for achalasia, showing significant improvements in objective metrics and achalasia-related symptoms.  Randomized comparative studies of LHM and POEM are required to determine the most effective treatment modality, with additional investigation of more novel metrics such as EndoFLIP and objective measures of reflux (e.g. impedance studies) pre- and post-treatment."

Lastly, per Zaninotto, G., et al. (2018), "Recommendations: we conditionally recommend POEM as an effective therapy (control of symptoms) for achalasia both in short- and medium-term follow-up with results comparable to Heller myotomy for symptom improvement." 

Therefore, the requested POEM is likely to be more beneficial than any standard treatment or treatments for the patient's condition or disease, and is a safe and effective treatment for achalasia, and should be covered by the health plan.