Patients-reported-outcome for oesophageal achalasia symptom score
| ISRCTN | ISRCTN79962781 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN79962781 |
| IRAS number | 267141 |
| Secondary identifying numbers | IRAS 267141 |
- Submission date
- 01/09/2020
- Registration date
- 14/09/2020
- Last edited
- 24/08/2023
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Digestive System
Plain English summary of protocol
Background and study aims
Oesophageal Achalasia (OA) means that a person has difficulty swallowing food or liquids due to the muscles in their food-pipe not working properly. Patients may have chest pain, trouble swallowing food or liquids, weight loss, and may complain about food coming back into their mouth after eating. The incidence of OA is relatively low (1-2.5 new cases per 100.000 people per year), but because it is a chronic disease, it is estimated that total cases are around 10 patients per 100.000 people. OA is a chronic disease and currently, all treatment options are for the control of symptoms only and patients may require multiple interventions during their lifetime. There are currently three treatment options for OA: surgical or endoscopic division of the muscle between the oesophagus and the stomach, forceful pneumatic dilatation, and paralysis of the muscle by injecting botulinum toxin. The outcomes of these treatments are measured by symptom improvement; however, currently, there are no validated patient-reported outcome tools to measure achalasia symptoms.
With the help of seventeen international experts and cognitive interviews with patients, a new Patient Reported Outcome Tool has been created to quantify the symptoms in achalasia patients and to assess the efficacy of achalasia treatments. This is necessary for a period when different treatment options need to be compared in randomized controlled trials
Before embarking in a large study, a pilot study has been planned with the main aim of establishing the willingness of patients to fill in questionnaires related to their symptoms. This will allow the design of a larger study to test the capacity of the tool for detecting the success or failure of treatments. It is expected that the acceptance rate in the pilot study will be over 80%. If this target is achieved, a larger study to validate the I-PASS questionnaire will be created.
Who can participate?
Adult patients after their diagnosis has been confirmed by the usual tests for achalasia (High-resolution manometry, barium swallow, and UGI endoscopy). Patients younger than 18 years, not fluent in English, or with psychiatric or neurological disorders will be excluded. This study has been discussed and approved by the Association of Achalasia Patients in the UK.
What does the study involve?
Participants will be invited via an invitation letter, the study information leaflet, and consent form being posted to them. They will have a pre-paid envelope to return the documents. They will be provided with a contact number of the research team if they have any queries regarding the study. Participants will be given at least 1 month to decide whether on not they would like to participate in the study.
The patient will receive the I-PASS questionnaire and the short form 36 of the quality of life questionnaire once informed consent has been taken. The questionnaires will be posted to the participant with a prepaid envelope. Patients will fill in the two questionnaires individually (without any involvement of the nurse) however, the research team contact details will be available if the patient requires any further explanations. On completion of the questionnaires, the time needed to fill in the forms will be recorded by the participant. It is expected that the time needed to complete the questionnaires will be around 10 to 20 minutes.
The last questionnaire will then be filled to establish if the patient was satisfied with the questionnaires, if they understood all the questions, and whether they would be available to review the questionnaires again, after treatment.
What are the possible benefits and risks of participating?
There will be no direct or indirect benefit for the patients from their participation in the study, and there are not risks foreseen as a result of participation.
Where is the study run from?
Three NHS centers in London, Imperial College, University College, and Barts Health (UK)
When is the study starting and how long is it expected to run for?
From November 2019 to December 2022
Who is funding the study?
Fondazione Morgagni ONLUS (Italy)
Who is the main contact?
Dr. Sheraz Markar
s.markar@imperial.ac.uk
Contact information
Scientific
Imperial College London
10th floor QEQM Building
St Mary’s Hospital
South Warf Road
London
W2 1NY
United Kingdom
 ORCID ID |
0000-0001-8650-2017 |
|---|---|
| Phone | +44 (0)20 3312 2125 |
| s.markar@imperial.ac.uk |
Study information
| Study design | Observational pilot study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cross sectional study |
| Study setting(s) | Hospital |
| Study type | Diagnostic |
| Scientific title | International patients-reported-outcome for Achalasia Symptom Score |
| Study acronym | I-PASS |
| Study objectives | Oesophageal achalasia is a relatively rare disease characterized by the absence of opening of the cardia and the absence of peristaltic contractions along the oesophageal body. Consequently, when an achalasia patient swallows solid food or liquid, the bolus cannot pass into the stomach and he/she experiences dysphagia, regurgitation of undigested food into the mouth, or chest pain. It has been estimated that the incidence of OA is between 1 to 2.5 per 100.000 habitants per year, but the prevalence of the disease is ten times higher (10/100.000). Most patients will require more than one treatment during their lifetime. The medical community treats the symptoms caused by OA (no etiological therapy exists) by lowering the resting pressure of the cardia. This is achieved by cutting the muscle, either during surgery or endoscopy or by forceful stretching of the muscle or by “poisoning” the muscle by injecting botulinum toxin into it. The efficacy of any of these treatment options is measured by symptom improvement, however, there are no validated patient-reported outcome questionnaires for achalasia symptoms. At present, the so-called “Eckardt score” is used, but this tool is not based on patient self-reporting, and therefore may be influenced by the interviewer and more importantly, it has never been properly validated. When we developed the International Guidelines for Oesophageal Achalasia, the lack of a validated tool that helps to define the success/failure of treatment was highlighted. A validated patient report outcome tool is also essential to compare the different treatment methods in randomized clinical trials and may facilitate the medical community accurately selecting the most appropriate therapy option. We have created an international group of “achalasia experts” comprising of seventeen gastroenterologists and surgeons from the UK, USA, Canada, Australia, Sweden, Norway, the Netherlands, Belgium, Germany, and Italy with the aim of creating this new tool (International Patient-reported outcome for Achalasia Symptom Scoring – I-PASS). Through a 3-round Delphi process, we have prepared a questionnaire, translated it into lay words, and refined it with cognitive interviews with 10 achalasia patients. Our hypothesis is that this new tool will allow to objectively measure the outcome of the different type of treatments of achalasia. |
| Ethics approval(s) | Approved 21/07/2020, East Midlands - Nottingham 1 Research Ethics Committee (no address: +44 (0)207 104 8115; nottingham1.rec@hra.nhs.uk), ref: 20/EM/0167 |
| Health condition(s) or problem(s) studied | Oesophageal achalasia |
| Intervention | Achalasia patients will be recruited in high-volume Upper Gastrointestinal (UGI) surgical and gastroenterological units. The recruitment of the patients will occur once the patients have completed their investigations (high-resolution manometry testing and barium swallow) and have a confirmed diagnosis of achalasia. UGI endoscopy will exclude any malignancies in the esophagus and the stomach. Participants will be asked if they would like to participate in this research study via an invitation letter, the study information leaflet, and consent form being posted to them. They will have a pre-paid envelope to return the documents. They will be provided with a contact number of the research team if they have any queries regarding the study. Participants will be given at least 1 month to decide whether on not they would like to participate in the study. Once informed consent has been taken, questionnaires will be posted to the participant with a prepaid envelope. The patient will then receive the I-PASS questionnaire and the short form 36 of the quality of life questionnaire. The I-PASS questionnaire has been developed through a 3-round Delphi process and then translated into lay words and refined through cognitive interviews with 10 achalasia patients. Patients will fill in the two questionnaires individually (without any involvement of the nurse), however, the research team contact details will be available if the patient requires any further explanations. On completion of the questionnaires, the time needed to fill in the forms will be recorded by the participant. The last questionnaire will then be filled to establish if the patient was satisfied with the questionnaires, if they understood all the questions, and whether they would be available to review the questionnaires again, after treatment. It is expected that the time needed to complete the questionnaires will be around 10 to 20 mins. |
| Intervention type | Other |
| Primary outcome measure | The willingness of naïve achalasia patients to fill in questionnaires related to their symptoms measured by the proportion of the number of eligible patients invited to participate who complete the questionnaires |
| Secondary outcome measures | 1. The time required for the patient to fill in the questionnaires recorded by patients on completion of the questionnaires 2. Patient satisfaction with the questionnaires measured using a follow-up questionnaire |
| Overall study start date | 01/11/2019 |
| Completion date | 01/12/2022 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 50 |
| Key inclusion criteria | 1. Aged ≥18 years 2. Primary achalasia confirmed by High-Resolution manometry, Barium swallow and endoscopy 3. Not previously treated for achalasia |
| Key exclusion criteria | 1. Not fluent in English 2. Diagnosis of neurological or cognitive impairment, and/or severe psychiatric disorder |
| Date of first enrolment | 15/11/2020 |
| Date of final enrolment | 15/11/2022 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centres
South Warf Road
London
W2 1BL
United Kingdom
London
NW1 2PG
United Kingdom
Whitechapel
London
E1 1BB
United Kingdom
Sponsor information
Hospital/treatment centre
Medical School Building
Norfolk Place
London
W1 PG
England
United Kingdom
| Phone | +44 (0)2075941862 |
|---|---|
| r.nicholson@imperial.ac.uk | |
| Website | http://www.imperial.nhs.uk/ |
"ROR" |
https://ror.org/056ffv270 |
Funders
Funder type
Charity
No information available
Results and Publications
| Intention to publish date | 01/06/2023 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Other |
| Publication and dissemination plan | Planned publication of results in a high impact-factor journal |
| IPD sharing plan | The datasets generated and/or analysed during the current study will be included in the subsequent results publication. Data collected will be anonymized and stored in password-protected Imperial College PC. |
Editorial Notes
24/08/2023: The following changes have been made:
1. The IRAS number has been corrected.
2. The ethics approval has been added.
17/09/2021: Internal review.
04/09/2020: Trial’s existence confirmed by the Morgagni Foundation.
