Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Achalasia.
Treatment: Ambulatory surgery [Peroral Endoscopic Myotomy].
The insurer denied Ambulatory surgery [Peroral Endoscopic Myotomy].
The determination is overturned.

The patient has dysphagia, regurgitation, chest pain, and a weight loss (10 pounds [lbs]). She has had an esophagogastroduodenoscopy (EGD), and esophageal manometry revealing a basal lower esophageal sphincter (LES) pressure of 82.4 millimeters of mercury (mmHg). She has been diagnosed with Achalasia. Her Eckard Score is 9. She has a medical history significant for a cardiomyopathy, and hypertension. Her body mass index (BMI) is 41.6. She has been advised to have a POEM [per oral endoscopic myotomy] procedure for achalasia. At issue is whether the proposed health service or treatment ambulatory surgery [Peroral Endoscopic Myotomy] is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.
It is likely to be more beneficial than any standard treatment procedures, such as pneumatic dilatation, or Heller Myotomy (HM).
This patient has had dysphagia, regurgitation, chest pain, and a weight loss of 10 lbs. She had an EGD, and esophageal manometry revealing a basal LES pressure of 82.4 mmHg. She has been diagnosed with Achalasia. Her Eckardt score is 9. The POEM procedure [per oral endoscopic myotomy] for the treatment of her achalasia is considered a medical standard of care treatment at this time. It has been proven to be safe, and efficacious within evidence-based, peer-reviewed medical literature.
The Clinical Practice Updates Committee of the American Gastroenterological Association proposes the following recommendations: 1) in determining the need for achalasia therapy, patient-specific parameters (Chicago Classification subtype, comorbidities, early versus (vs) late disease, primary or secondary causes) should be considered along with published efficacy data; 2) given the complexity of this procedure, POEM should be performed by experienced physicians in high-volume centers because an estimated 20-40 procedures are needed to achieve competence; 3) if the expertise is available, POEM should be considered as primary therapy for type III achalasia; 4) if the expertise is available, POEM should be considered as treatment option comparable with laparoscopic Heller myotomy for any of the achalasia syndromes; and 5) post-POEM patients should be considered high risk to develop reflux esophagitis and advised of the management considerations (potential indefinite proton pump inhibitor therapy and/or surveillance endoscopy) of this before undergoing the procedure." [Kahrilas, Peter J., David Katzka, and Joel E. Richter. "Clinical practice update: the use of per-oral endoscopic myotomy in achalasia: expert review and best practice advice from the AGA Institute." Gastroenterology 153.5 (2017): 1205-1211].
"POEM is safe and effective for patients with achalasia who were not treated successfully by prior HM. Although the rate of clinical success in patients with prior HM is lower than in those without prior HM, the safety profile of POEM is comparable between groups." [Ngamruengphong, Saowanee, et al. "Efficacy and safety of peroral endoscopic myotomy for treatment of achalasia after failed Heller myotomy." Clinical Gastroenterology and Hepatology 15.10 (2017): 1531-1537].
"In this randomized clinical trial that compared POEM with pneumatic dilation as the initial treatment for treatment-naive patients with achalasia, POEM resulted in a significantly higher treatment success rate at 2 years. However, development of reflux esophagitis was more frequent after POEM than after pneumatic dilation, and POEM was associated with increased proton pump inhibitor (PPI) use. To our knowledge, this is the first randomized clinical trial that evaluated the use of POEM as an initial treatment for achalasia. The efficacy of POEM in this study was similar to the results reported in uncontrolled prospective and retrospective studies, which showed therapeutic success rates of 80% to 97% after 12 months or more. The definition of success in these studies was an Eckardt score less than or equal to 3, the need for re-treatment, or both. Some studies have suggested that the recurrence rate after POEM could further increase with time.9,28 However, most of the prospective studies were not restricted to treatment-naive patients with achalasia, which makes direct comparison difficult. In previous studies involving laparoscopic Heller myotomy, efficacy at 5 years decreased to 80% to 85%. Outcome data for POEM with such a long follow-up is not available, but it can be anticipated that POEM most likely will perform similarly to laparoscopic Heller myotomy because 1-year and 2-year follow-up data reveal similar success rates. Randomized clinical trials comparing POEM to laparoscopic Heller myotomy are necessary to answer that question. The observed success rate of 92% at 2 years in this trial should be considered as a medium-term outcome and follow-up data at 5 years will help to provide information about the duration of the treatment effect. The data confirmed that POEM was a technique with a low risk of major complications because SAEs were not observed in the POEM group. For pneumatic dilation, the rate of perforations was 1.5% despite the use of the smallest (30-millimeter (mm)) balloon for the initial pneumatic dilation. This finding was within the reported range of complication rates of previous studies. Although POEM is more invasive and requires more technical endoscopic skills, the risk of severe complications was not higher than with pneumatic dilation, especially when performed by experienced endoscopists. Treatment success of pneumatic dilation ranged from 54% to 80% during the study, which is on the lower end compared with other studies in which success ranged from 50% to 85%. One reason for this discrepancy could be the pneumatic dilation protocol that was followed in the current study. Patients were considered to have unsuccessful treatment after 1 or 2 pneumatic dilation procedures with a 35-mm or smaller balloon. Other studies included an additional pneumatic dilation with a 40-mm balloon in cases of clinical recurrence or extra dilation series with 2 or 3 pneumatic dilations. Some evidence suggests that repeated dilation is accepted and reflects daily clinical practice. However, patients will experience persistent or recurrent symptoms after previous pneumatic dilations as failed treatment. Pursuing another series of pneumatic dilations would be a second treatment. Furthermore, each time a pneumatic dilation is performed there is a perforation risk and multiple pneumatic dilation sessions form a potential bias in the comparison to 1 treatment intervention. Therefore, the effect of just 1 series of pneumatic dilations was compared with the effect of POEM. However, 23 of the 29 patients in the pneumatic dilation group whose treatment was unsuccessful in this study subsequently underwent pneumatic dilation with a 35-mm balloon, a 40-mm balloon, or both. Of these 23 patients, 9 (39%) still had persistent symptoms and underwent POEM. The additional pneumatic dilation increased the treatment success rate of pneumatic dilation to 76%, but this was still lower than the 92% success rate of POEM. Follow-up after re-treatment was less than 6 to 12 months in most patients, which cannot imply successful treatment. Previous data suggest that if symptoms do not improve after a pneumatic dilation series with a 30-mm and/or 35-mm balloon, it is unlikely that symptoms will improve after pneumatic dilation with a 40-mm balloon. The minimal expected effect observed after additional pneumatic dilation with a 40-mm balloon was another reason not to include subsequent dilation sessions in the protocol. The 2011 trial by Boeckxstaens et al differed from the current trial in that the former had a more aggressive dilation protocol and excluded patents with serious dilation complications after pneumatic dilation from further analysis. If that trial used the same definition of treatment success as the current study, the success rate of pneumatic dilation would be lower and comparable to the success rate of this study." [Ponds, Fraukje A., et al. "Effect of peroral endoscopic myotomy vs pneumatic dilation on symptom severity and treatment outcomes among treatment-naive patients with achalasia: a randomized clinical trial." JAMA 322.2 (2019): 134-144]
"POEM is safe and effective for challenging achalasia patients. SMF classification grade 2/3 was shown to be an independent risk factor for incomplete tunneling, adverse events, and procedure time greater than or equal to (>=)90 minutes. For these patients, POEM should be performed by experienced endoscopists, some cases are better served with traditional surgery, such as minimally invasive Heller with Dor fundoplication." [Xu, Shanshan, et al. "Outcomes of peroral endoscopic myotomy in challenging achalasia patients: a long-term follow-up study." Surgical Endoscopy (2020): 1-12]