Esophageal Dilation, Refractory Benign Esophageal Stricture
Conditions
Brief summary
This study is being done to see which treatment is more effective in improving the difficulty of swallowing. Researchers are comparing self-dilation to endoscopic dilation.
Detailed description
Benign esophageal strictures can be challenging condition to treat. The mainstay of treatment is endoscopic dilations. However, 30 to 40% of these strictures recur despite rigorous dilations. Although a consensus definition does not exist, a stricture is typically termed as a refractory benign esophageal stricture (RBES), when there is a failure to maintain luminal patency after at least 5 endoscopic dilations. Patients with RBES are extremely difficult to manage and the current armamentarium includes repeated endoscopic dilations, corticosteroid or mitomycin C injections, incisional therapy, and/ or temporary stent placement. These procedures are costly, their efficacy can be short-lived, and are associated with great burden both for the patient and clinician. Esophageal self -dilation therapy (ESDT) is where the patient learns to pass a polyvinyl dilator orally on a routine basis. In past, smaller studies, ESDT appears to be effective for RBES, reducing the number of endoscopic dilations from an average of 21.7 to an average of 1.
Interventions
DEVICEEsophageal self-dilation therapy
Patient learns to pass a polyvinyl dilator orally on a routine basis. Teaching will take over 1-3 training sessions by one of two esophageal physicians and a nurse. Patients will be instructed to start Esophageal self dilation therapy twice a day for at least one week.
PROCEDUREEndoscopic therapy with esophageal dilation
A long, flexible tube is inserted down the throat into the esophagus and the narrow area of the esophagus is dilated, or stretched.
Sponsors
Mayo Clinic
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Subjects who agreed to participate in the randomized trial were randomized to standard clinical care consisting of endoscopic dilation as needed versus esophageal self-dilation therapy in a 1:1 ratio using a block randomization approach. Subjects randomized to the standard clinical care group were offered cross-over to the esophageal self-dilation therapy if they failed stand of care. Subjects who agreed to take part in in the observational prospective study either commenced ESDT or continued standard clinical care based on shared decision making with their esophageal provider.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Refractory benign esophageal stricture defined as an esophageal stricture with persistent dysphagia despite undergoing 5 endoscopic dilations within a 1 year period. Persistent dysphagia will be considered if patients has solid food dysphagia at least once a week
Exclusion criteria
* Patient with malignant esophageal stricture * Angulated stricture which prevents safe passage of Maloney dilator in office setting * In ability to achieve an esophageal diameter of 10 mm with endoscopic dilation * Known significant esophageal motor disorder (i.e. achalasia, aperistalsis, functional obstruction, jackhammer, distal esophageal spasm)\* * The presence of esophageal stent * Inability to learn self-dilation secondary to blindness or cognitive dysfunction * Use of chronic anticoagulants
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Endoscopic Interventions | 6 months following serial dilation | Number of endoscopies required in a 6 month interval in subjects who achieved at least a 10-12 mm esophageal diameter during serial dilation for refractory benign esophageal stricture (RBES) and were subsequently treated with ESDT versus standard clinical care |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Length of Days Intervention Free | 6 months | Number of days from start of study participation to 1st endoscopic intervention for recurrent dysphagia |
| Mayo Dysphagia Questionnaire (MDQ-30) at Baseline | Baseline | The MDQ-30 provides a series of questions for patients regarding their swallowing difficulties over the past 30 days. Participants were asked to rate the severity of their troubles swallowing on a scale of 0 to 10; 0 being Not at all severe and 10 being Very severe |
| Mayo Dysphagia Questionnaire (MDQ-30) at 12 Months | 12 months | The MDQ-30 provides a series of questions for patients regarding their swallowing difficulties over the past 30 days. Participants were asked to rate the severity of their troubles swallowing on a scale of 0 to 10; 0 being Not at all severe and 10 being Very severe |
| Clinically Significant Adverse Events | 12 months | Number of clinically significant adverse events including perforation, bleeding and pain |
Countries
United States
Participant flow
Recruitment details
6 subjects randomized to self-dilation group and 6 subjects randomized to standard of care with repeat endoscopy and dilations as needed at symptoms recurrence. 5 subjects from the standard of care group crossed over to receive self-dilation because they were considered treatment failures due to requiring 2 endoscopic dilation after 3 months of study participation. 14 subjects choose not to participate in the randomized trial, but did agree to be followed in a prospective observational group.
Participants by arm
| Arm | Count |
|---|---|
| Standard Clinical Care Endoscopic Dilation Group Following standard clinical care consisting of serial endoscopic dilation to achieve an esophageal diameter of at least 10 mm, subjects will undergo repeat upper endoscopy with dilation as needed if their dysphagia relapses which is the current standard of care. A relapse will be considered if a patient developed solid food dysphagia at least once a week.
Endoscopic therapy with esophageal dilation: A long, flexible tube is inserted down the throat into the esophagus and the narrow area of the esophagus is dilated, or stretched. | 6 |
| Esophageal Self-Dilation Therapy (ESDT) Group Following standard clinical care consisting of serial endoscopic dilation to achieve an esophageal diameter of at least 10 mm, subjects are instructed to perform esophageal self-dilation twice a day. If dysphagia is adequately controlled, and there was no resistance with passing the dilator, patients will be asked to decrease the frequency of ESDT to daily, weekly, and monthly over an average period of 6 months.
Esophageal self-dilation therapy: Patient learns to pass a polyvinyl dilator orally on a routine basis. Teaching will take over 1-3 training sessions by one of two esophageal physicians and a nurse. Patients will be instructed to start Esophageal self dilation therapy twice a day for at least one week. | 6 |
| Observational Study Group Subjects undergo either esophageal self-dilation therapy or continued standard of clinical care base on shared decision making with their esophageal provider.
Esophageal self-dilation therapy: Patient learns to pass a polyvinyl dilator orally on a routine basis. Teaching will take over 1-3 training sessions by one of two esophageal physicians and a nurse. Patients will be instructed to start Esophageal self dilation therapy twice a day for at least one week.
Endoscopic therapy with esophageal dilation: A long, flexible tube is inserted down the throat into the esophagus and the narrow area of the esophagus is dilated, or stretched. | 14 |
| Standard Clinical Care Endoscopic Dilation, Then ESDT Subjects that received standard of clinical care endoscopic dilation who required two endoscopic dilations within 3 months of randomization were considered to have failed standard care and offered cross-over to the self-dilation therapy.
Esophageal self-dilation therapy: Patient learns to pass a polyvinyl dilator orally on a routine basis. Teaching will take over 1-3 training sessions by one of two esophageal physicians and a nurse. Patients will be instructed to start Esophageal self dilation therapy twice a day for at least one week.
Endoscopic therapy with esophageal dilation: A long, flexible tube is inserted down the throat into the esophagus and the narrow area of the esophagus is dilated, or stretched. | 5 |
| Total | 31 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Cross-over for Treatment Failure | Withdrawal by Subject | 0 | 0 | 0 | 1 |
| Initial Study Phase | Death | 0 | 1 | 0 | 0 |
| Initial Study Phase | Treatment failure | 5 | 0 | 0 | 0 |
| Initial Study Phase | Withdrawal by Subject | 1 | 1 | 2 | 0 |
Baseline characteristics
| Characteristic | Total | Standard Clinical Care Endoscopic Dilation, Then ESDT | Observational Study Group | Esophageal Self-Dilation Therapy (ESDT) Group | Standard Clinical Care Endoscopic Dilation Group |
|---|---|---|---|---|---|
| Age, Continuous Cross-over for Treatment Failure | 65 years STANDARD_DEVIATION 7 | 65 years STANDARD_DEVIATION 7 | — | — | — |
| Age, Continuous Initial Study Phase | 62.9 years STANDARD_DEVIATION 12.3 | — | 62 years STANDARD_DEVIATION 15 | 62.3 years STANDARD_DEVIATION 9.6 | 64.0 years STANDARD_DEVIATION 11 |
| Race and Ethnicity Not Collected | 0 Participants | — | — | — | — |
| Region of Enrollment United States | 26 participants | 5 participants | 14 participants | 6 participants | 6 participants |
| Sex: Female, Male Cross-over for Treatment Failure Female | 3 Participants | 3 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Cross-over for Treatment Failure Male | 2 Participants | 2 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Initial Study Phase Female | 14 Participants | 0 Participants | 7 Participants | 4 Participants | 3 Participants |
| Sex: Female, Male Initial Study Phase Male | 12 Participants | 0 Participants | 7 Participants | 2 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 1 / 6 | 0 / 14 | 0 / 5 |
| other Total, other adverse events | 0 / 6 | 0 / 6 | 0 / 14 | 0 / 5 |
| serious Total, serious adverse events | 0 / 6 | 0 / 6 | 0 / 14 | 1 / 5 |
Outcome results
Primary
Number of Endoscopic Interventions
Number of endoscopies required in a 6 month interval in subjects who achieved at least a 10-12 mm esophageal diameter during serial dilation for refractory benign esophageal stricture (RBES) and were subsequently treated with ESDT versus standard clinical care
Time frame: 6 months following serial dilation
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Standard Clinical Care Endoscopic Dilation Group | Number of Endoscopic Interventions | 12 endoscopies |
| Esophageal Self-Dilation Therapy (ESDT) Group | Number of Endoscopic Interventions | 11 endoscopies |
| Observational Study Group | Number of Endoscopic Interventions | 12 endoscopies |
| Standard Clinical Care Endoscopic Dilation, Then ESDT | Number of Endoscopic Interventions | 7 endoscopies |
Secondary
Clinically Significant Adverse Events
Number of clinically significant adverse events including perforation, bleeding and pain
Time frame: 12 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Standard Clinical Care Endoscopic Dilation Group | Clinically Significant Adverse Events | 0 events |
| Esophageal Self-Dilation Therapy (ESDT) Group | Clinically Significant Adverse Events | 0 events |
| Observational Study Group | Clinically Significant Adverse Events | 0 events |
| Standard Clinical Care Endoscopic Dilation, Then ESDT | Clinically Significant Adverse Events | 1 events |
Secondary
Length of Days Intervention Free
Number of days from start of study participation to 1st endoscopic intervention for recurrent dysphagia
Time frame: 6 months
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard Clinical Care Endoscopic Dilation Group | Length of Days Intervention Free | 7 days |
| Esophageal Self-Dilation Therapy (ESDT) Group | Length of Days Intervention Free | 82 days |
| Observational Study Group | Length of Days Intervention Free | 147 days |
Secondary
Mayo Dysphagia Questionnaire (MDQ-30) at 12 Months
The MDQ-30 provides a series of questions for patients regarding their swallowing difficulties over the past 30 days. Participants were asked to rate the severity of their troubles swallowing on a scale of 0 to 10; 0 being Not at all severe and 10 being Very severe
Time frame: 12 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Standard Clinical Care Endoscopic Dilation Group | Mayo Dysphagia Questionnaire (MDQ-30) at 12 Months | 5.55 score on a scale | Standard Deviation 2.07 |
| Esophageal Self-Dilation Therapy (ESDT) Group | Mayo Dysphagia Questionnaire (MDQ-30) at 12 Months | 5.83 score on a scale | Standard Deviation 3.6 |
| Observational Study Group | Mayo Dysphagia Questionnaire (MDQ-30) at 12 Months | 5.17 score on a scale | Standard Deviation 2.04 |
Secondary
Mayo Dysphagia Questionnaire (MDQ-30) at Baseline
The MDQ-30 provides a series of questions for patients regarding their swallowing difficulties over the past 30 days. Participants were asked to rate the severity of their troubles swallowing on a scale of 0 to 10; 0 being Not at all severe and 10 being Very severe
Time frame: Baseline
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Standard Clinical Care Endoscopic Dilation Group | Mayo Dysphagia Questionnaire (MDQ-30) at Baseline | 7.16 score on a scale | Standard Deviation 1.16 |
| Esophageal Self-Dilation Therapy (ESDT) Group | Mayo Dysphagia Questionnaire (MDQ-30) at Baseline | 7.56 score on a scale | Standard Deviation 1.44 |
| Observational Study Group | Mayo Dysphagia Questionnaire (MDQ-30) at Baseline | 5.36 score on a scale | Standard Deviation 2.17 |