POEM Trial: Multi-center Study Comparing Endoscopic Pneumodilation and Per Oral Endoscopic Myotomy (POEM)

Study Overview
=================
Brief Summary
-----------------
The aim of the study is to compare the efficacy of per-oral endoscopic myotomy (POEM) to the efficacy of pneumodilation as the initial treatment of symptomatic idiopathic achalasia. It is hypothesized that POEM has a higher long-term efficacy than pneumodilation in treatment of therapy-naive patients with idiopathic achalasia.

Official Title
-----------------
A Prospective Randomized Multi-center Study Comparing Endoscopic Pneumodilation and Per Oral Endoscopic Myotomy (POEM) as Treatment of Idiopathic Achalasia

Conditions
-----------------
Achalasia

Intervention / Treatment
-----------------
* Procedure: PD
* Procedure: POEM


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Between 18 and 75 yr of age Manometric diagnosis of achalasia Eckardt score > 3 Informed consent Exclusion Criteria: Severe cardio-pulmonary disease or other serious disease leading to unacceptable surgical risk previous treatment, except treatment with nitroderivatives, Ca++ channel blockers or sildenafil, or dilation with Savary bougies or balloons of 2 cm diameter or smaller. Pseudo-achalasia Mega-esophagus (> 7 cm) and/or sigmoid-like esophagus Previous esophageal or gastric surgery (except for gastric perforation) Not capable to fill out questionnaires (f.e. due to language barrier) Not available for follow-up Esophageal diverticula in the distal esophagus Malignant or premalignant esophageal lesions Patients with liver cirrhosis and/or esophageal varices Pregnancy

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: PD<br>pneumodilation | Procedure: PD<br>* pneumodilation<br>|
| Active Comparator: POEM<br>peroral endoscopic myotomy | Procedure: POEM<br>* peroral endoscopic myotomy<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| therapeutic succes | The time till failure is the primary outcome parameter. The time is measured from date of pneumatic dilation (last session) or POEM to date of relapse of symptoms (date of establishment). Symptoms will be evaluated using the Eckardt scoring system (Table 1.). Therapeutic success is defined as a drop in Eckardt score to ≤ 3 after treatment and symptom control at yearly follow-up which is also defined as an Eckardt score of ≤ 3. If symptoms recur within patients randomized for pneumatic dilation, retreatment with pneumatic dilation is allowed according to the protocol. If retreatment does not result in reduction of the symptom score below 4, the patient will be considered as a treatment failure. | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| quality of life | The quality of life is determined using a general QoL questionnaire, namely the SF36 questionnaire. In addition, a more disease specific questionnaire, i.e. the EORTC QLQ-OES24 questionnaire, is filled out. These questionnaires have scored the QoL at fixed time points during follow-up (1 month, 3 months and yearly). | 2 years | 
| complication rate | Complications (perforation, bleeding, etc) occurring during or immediately following the procedure are recorded by the attending phycisian. In addition, gastroesophageal reflux is measured using 24h pHmetry after 3 months and 1 year. The number of patients with pathological acid exposure (>4.5% of time a pH<4) will be assessed. Endoscopy is performed after 1 year to detect esophagitis. Esophagitis rates and LA grades of esophagitis will be assessed. | 2 years | 
| need for retreetment | Retreatment is allowed in the pneumodilation group when symptoms recur, which is defined as an Eckardt score of > 3. Symptom scores are assessed after one month, three months and then yearly. | 2 years |