dysfagie idiopatic achalasie sphincter into the esophagus
Conditions
Interventions
POEM and PD
Sponsors
University Hospital Leuven
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: - Between 18 and 75 year of age - Manometric diagnosis of achalasia - Eckardt score >3 - signed Informed consent
Exclusion criteria
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-esophageal (> 7 cm) and/or sigmoid-like esophagus -Previous esophageal or gastric surgery (except for gastric perforation) - Not capable to fill out questionnaires (e.g. 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Therapeutic success This study is designed to evaluate which of the two treatment modalities results in the highest success rate after 2 years. Thereafter, follow-up will continue and a similar analysis will be performed 5 years after treatment (and every 5 years thereafter) to determine the long-term outcome of both treatment arms. As described in more detail later, symptoms will be evaluated using the Eckardt scoring system. Therapeutic success is defined as a reduction to an Eckardt score of 3 or less. This implies that only patients with an Eckardt score > 3 will be included in order to be able to detect a relevant drop in symptom score. Two years after treatment, the number of patients with a successful symptom control will be determined for both groups of treatment. If symptoms recur within the first year of follow-up in patients randomized to pneumatic dilation, retreatment with pneumatic dilation is allowed. If retreatment however does not result in reduction of the symptom score below 4, the patient will be considered as a failure. In case of a failure, patients will be offered the choice to undergo an alternative treatment (surgery, botox), and will be excluded from the study. Data collection will however be continued. When patients successfully treated with pneumatic dilation develop recurrent symptoms during follow-up, retreatment is allowed. Again, treatment is started with a 35 mm balloon, and symptoms are evaluated 4 weeks later. In case the Eckardt score is still above 3, redilation with a 40 mm balloon will be performed. After this second series of pneumodilation, symptoms should be controlled for at least 2 year before a third and last session is allowed. If symptoms occur before this 2 year interval, the patient is considered as a failure. In total, only three dilation sessions will be allowed. If symptoms recur after the third series of dilations, this patient is considered as a failure. If symptoms recur after POEM, | — |
Secondary
| Measure | Time frame |
|---|---|
| Quality of life The quality of life will be determined using a general QoL questionnaire, namely the SF36 questionnaire. In addition, a more disease specific questionnaire, i.e. the EORTC QLQ-OES24, the Reflux Disease Questionnair RDQ and the Achalasia DSQL questionnaire, will be filled out. These questionnaires will score the QoL at fixed time points during follow-up (3 months, yearly) and if possible before withdrawing from the study for patients who wish to stop their participation. Complication rate (related to the procedure, GERD) As discussed in detail in section 3.1. and 3.2, complications (perforation, bleeding, aspiration, infection, sepsis, *) occurring during or immediately following the procedure will be recorded. In addition, reflux symptoms will be scored using a disease-specific questionnaire (HRQL) and gastroesophageal reflux will be measured using 24h pHmetry / endoscopy (see table) Need for retreatment As described above, retreatment is allowed to some degree. The number of retreatment sessions after the initial therapy (pneumodilation) will be compared between the two groups. Retreatment is accepted in PD under restricted conditions (see above) whereas the need for retreatment is considered a failure in case of POEM. Costs Costs will be calculated for each procedure in each center separately. Functional parameters (Lower esophageal sphincter pressure (LESP), esophageal emptying, distensibility) To evaluate the impact on LES function, LES function will be assessed via manometry (LESP), timed barium esophagogram (emptying). Depending on the expertise, patients will undergo an endoFLIP (distensibility) study to assess distensibility. These data are of crucial importance to determine predictors of clinical outcome. | — |
Countries
Netherlands
Outcome results
None listed