Achalasia
Conditions
Keywords
Achalasia, Heller myotomy, Dysphagia, Peroral Endoscopic Myotomy
Brief summary
This study intends to investigate the feasibility, safety and efficacy of peroral endoscopic myotomy for the treatment of achalasia in a single center setting in Europe.
Detailed description
This pilot study intends to investigate the feasibility, safety and efficacy of peroral endoscopic myotomy for the treatment of achalasia in a single center setting in Europe. 16 patients are enrolled to evaluate feasibility, safety and efficacy of peroral endoscopic myotomy. Main outcome measurement is the eckhardt symptom score 3 month after therapy. Primary outcome: -eckhardt symptom score 3 month after therapy. Secondary outcomes: * Lower esophageal sphincter pressure at 3 month after therapy. * Reflux symptoms at 3 month after therapy. For this prospective study, inclusion criteria are achalasia, as diagnosed by established methods (contrast fluoroscopy, manometry, esophagal-gastro-duodenoscopy) and age greater than 18 years. Previous therapy, such as esophageal surgery or previous myotomy are exclusion criterion. A forward-viewing upper endoscope is used with a transparent distal cap attachment. Carbon dioxide gas is necessary for insufflation during the procedures. An endoscopic knife is used to access the submucosa, dissect the submucosal tunnel and also to divide circular muscle bundles over a length of approximately 10cm, extending 2-3cm onto the cardia. An electric generator is used with spray coagulation mode. A coagulating forceps is used for hemostasis as needed. Closure of the mucosal entry site is performed using standard endoscopic clips.
Interventions
A forward-viewing upper endoscope is used with a transparent distal cap attachment. Carbon dioxide gas is necessary for insufflation during the procedures. An endoscopic knife is used to access the submucosa, dissect the submucosal tunnel and also to divide circular muscle bundles over a length of approximately 10cm, extending 2-3cm onto the cardia. An electric generator is used with spray coagulation mode. A coagulating forceps is used for hemostasis as needed. Closure of the mucosal entry site is performed using standard endoscopic clips
Sponsors
Universitätsklinikum Hamburg-Eppendorf
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient with symptomatic achalasia and pre-op barium swallow, manometry and esophagal-gastro-duodenoscopy which are consistent with the diagnosis * persons of age \> 18 years with medical indication for surgical myotomy or Endoscopic balloon dilatation * Signed written informed consent.
Exclusion criteria
* Patients with previous surgery of the stomach or esophagus * Patients with known coagulopathy * Previous achalasia-treatment with surgery * Patients with liver cirrhosis and/or esophageal varices * Active esophagitis * Eosinophilic esophagitis * Barrett's esophagus * Pregnancy * Stricture of the esophagus * Malignant or premalignant esophageal lesion * Candida esophagitis * Hiatal hernia \> 2cm
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3 | 3 months after treatment | eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. with every meal giving 3 points, daily (2 points), sometimes (1 Point) or no (0 Points), as well as weight loss,(\>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Procedure-related Adverse Events | procedure to 3 months post procedure | procedure-related adverse events per protocol |
| mmHg of the Lower Esophageal Sphincter 3 Months After POEM Procedure | manometry at 3 month after therapy | esophageal manometry is done 3 months after POEM procedure to evaluate resting lower esophageal sphincter pressure |
| Medication 3 Months After POEM | 3 months | proton pump inhibitor (PPI) use at 3 months after POEM procedure |
| Number of Participants With Reflux Symptoms | during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment | Number of Participants with Reflux Symptoms during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment |
| Days Duration Hospitalization | days of hospitalization for POEM procedure, an average of 4 days | participants were followed for the duration of hospital stay, an average of 4 days |
| cm Myotomy Length | POEM procedure | myotomy length in cm |
| Percentage of Participants Who Achieved Treatment Success 3 Months After Treatment | 3 months after treatment | eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. with every meal giving 3 points, daily (2 points), sometimes (1 Point) or no (0 Points), as well as weight loss,(\>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms. Post-myotomy eckhardt score ≤ 3 n individuals has been reached in 15 individuals. |
| Duration Time Procedure | procedure | duration time of POEM procedures in minutes |
Countries
Germany
Participant flow
Recruitment details
Patients with symptomatic achalasia
Pre-assignment details
16 patients were included as per protocol, no patient was lost during follow-up over a 2 year period
Participants by arm
| Arm | Count |
|---|---|
| POEM Patients pilot study, first 16 patients who received POEM for treatment of achalasia | 16 |
| Total | 16 |
Baseline characteristics
| Characteristic | POEM Patients |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 3 Participants |
| Age, Categorical Between 18 and 65 years | 13 Participants |
| Age, Continuous | 45 years STANDARD_DEVIATION 16.22 |
| Region of Enrollment Germany | 16 participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 1 / 16 |
| serious Total, serious adverse events | 2 / 16 |
Outcome results
Primary
Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3
eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. with every meal giving 3 points, daily (2 points), sometimes (1 Point) or no (0 Points), as well as weight loss,(\>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms.
Time frame: 3 months after treatment
Population: Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| POEM Patients | Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3 | symptom relief 3 months after treatment | 1.4 Eckardt Score | Standard Deviation 2.217355783 |
| POEM Patients | Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3 | eckhardt score baseline | 8.8 Eckardt Score | Standard Deviation 1.28 |
Comparison: this pilot study was created ro evaluate sample size for the following multicenter study, based on manometric outcomes. Mean values between baseline and follow-up were compared using Student ' s t -test for paired samples. P values less then 0.05, two-sided, were considered significant.R 2.13.1(R Development Core Team (2011). Subgroups (partial vs. complete myotomy) were compared using an analysis of variance test adjusted for initial values.p-value: <0.001t-test, 2 sided
Secondary
cm Myotomy Length
myotomy length in cm
Time frame: POEM procedure
Population: Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| POEM Patients | cm Myotomy Length | 12 cm | Standard Deviation 2.704933764 |
Secondary
Days Duration Hospitalization
participants were followed for the duration of hospital stay, an average of 4 days
Time frame: days of hospitalization for POEM procedure, an average of 4 days
Population: Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| POEM Patients | Days Duration Hospitalization | 4.5625 days | Standard Deviation 0.892094913 |
Secondary
Duration Time Procedure
duration time of POEM procedures in minutes
Time frame: procedure
Population: Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| POEM Patients | Duration Time Procedure | 114 minutes | Standard Deviation 37.48238475 |
Secondary
Medication 3 Months After POEM
proton pump inhibitor (PPI) use at 3 months after POEM procedure
Time frame: 3 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| POEM Patients | Medication 3 Months After POEM | 1 participants |
Secondary
mmHg of the Lower Esophageal Sphincter 3 Months After POEM Procedure
esophageal manometry is done 3 months after POEM procedure to evaluate resting lower esophageal sphincter pressure
Time frame: manometry at 3 month after therapy
Population: Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| POEM Patients | mmHg of the Lower Esophageal Sphincter 3 Months After POEM Procedure | 11.8 mmHg | Standard Deviation 9.016200316 |
Secondary
Number of Participants With Procedure-related Adverse Events
procedure-related adverse events per protocol
Time frame: procedure to 3 months post procedure
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| POEM Patients | Number of Participants With Procedure-related Adverse Events | 2 Participants |
Secondary
Number of Participants With Reflux Symptoms
Number of Participants with Reflux Symptoms during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment
Time frame: during procedure, and 3 and 6 months, and 1, 2 and 5 years after treatment
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| POEM Patients | Number of Participants With Reflux Symptoms | 1 participants |
Secondary
Percentage of Participants Who Achieved Treatment Success 3 Months After Treatment
eckhardt score is a score to evaluate achalasia discomfort in patients. Patients are being interrogated for dysphagia, regurgitation, and retrosternal pain , correlated with the time frame of occurrence. with every meal giving 3 points, daily (2 points), sometimes (1 Point) or no (0 Points), as well as weight loss,(\>10 kg= 3 points, 5-10 kg=2 points, 0-5 kg=1 point, None=0 points. Scale range is from 0 Points (no achalasia) up to 12 points for the worst achalasia symptoms. Post-myotomy eckhardt score ≤ 3 n individuals has been reached in 15 individuals.
Time frame: 3 months after treatment
Population: Pilot study group to evaluate feasibility and safety of POEM procedure before initiating studies evaluating long-term efficacy.Patients with primary achalasia, diagnosed by established methods(contrast fluoroscopy, manometry, esophagogastroduodenoscopy(EGD)) and age greater than 18 years were included.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| POEM Patients | Percentage of Participants Who Achieved Treatment Success 3 Months After Treatment | 94 percentage of treated patients |