This study (International – Patient reported outcome of Achalasia Symptom Score) is being run by Professor Giovanni Zaninotto from Imperial College and Assistant Professor Sheraz Markar. It aims to clarify treatments and the patient’s outcome to try and standardise the best possible treatment.
The contact point is Sara Jamel S.Jamel@imperial.ac.uk
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.
Presentation on I-PASS from Prof Giovanni Zaninotto
Professor Giovanni Zaninotto gave an introduction to a new project, I-PASS, which stands for International – Patient reported outcome of Achalasia Symptom Score.
The presentation is available as a pdf download here.
The following are some general notes and a slide by slide description from the presentation.
Achalasia is
- Hard to diagnose
- There is no standardised treatment
- No cure, treatment is palliative and aims to improve symptoms
Prof Zaninotto is a specialist in oesophageal disease with an interest in Achalasia. Two years ago started developing guidelines for Achalasia treatment, there is currently no standardised way to establish if treatment brings improvement and how to measure the symptomatic improvement
The I-PASS project is creating a questionnaire that will enable patients to report the outcomes of treatment. It will be used by patients at before their treatment and again after treatment. The data will be used to understand, and drive improvements in, the efficacy of treatments for Achalasia. Having a standardised, internationally available Patient Reported Outcome (PRO) is important to enable future projects to develop treatments and improve care for people with Achalasia (for example, major funding organisations only support projects where there is a PRO). The PRO will enable randomised control trials of future treatments to have objective measurement of the outcome.
Stages of the I-PASS project
- 15 Achalasia experts were asked to develop a set of questions that would help demonstrate the efficacy of any treatment. This used the Delphi process (multiple rounds of questions circulated and amended based on feedback). This stage has been completed.
- A group of 15 people, from different countries, with Achalasia will read the questions and be interviewed by a psychologist to ensure that the questions are fit for purpose and to improve the questionnaire.
- Pilot study. 50 people who have not had treatment will be asked to complete the questionnaire, and then repeat it after treatment. Then the questionnaire will be refined. The project is currently funded to this stage.
- Validation study. 750 people who have not had treatment will be asked to complete the questionnaire, and then repeat it after treatment.
I-PASS would like the support of the Achalasia group in the following ways.
- 5 volunteers for cognitive interview (people in the meeting gave their details, the study will interview a cross section. A further call out will follow if people of a different demographic (eg older or younger, male or female are required)
- support for the pilot study (recommend patients to take part)
- help in asking for grants for larger study by endorsing the work (a previous similar study cost around £25k)
- help recruiting treated patients for the validation study
- There will be more information from the study after a meeting with one of the projects funders (ISDE) in November.
The meeting unanimously voted to support the project with a show of hands.
Other interesting points raised during the presentation & questions and answer session.
- Many patients have multiple treatments for Achalasia
- The first record of dilatation, most likely for achalasia was in 1650, using a whalebone with a sponge on it’s tip, the first Heller’s Myotomy was in 1913
- There is no difference in occurrence of Achalasia in adults globally (that we currently know of) based on race, age, sex. There is a lower incidence of achalasia in children than adults.
- There is currently an increase in cases in South America because of Chagas disease
- There are 6 trials comparing surgery and dilation that Mr Hashemi is aware of – none of them are of the best standard or comparing like with like.
- The Eckhardt score was never validated for outcomes of treatment – only to assess the initial diagnosis and severity of Achalasia. The test scores the severity of symptoms on the following factors
- Dysphagia (difficulty swallowing)
- Frequency of regurgitation
- Chest pain
- Weight loss
- There is the potential to create a European network , Achalasia is recognised by the EU as a rare disease, some special funds being available for research on rare diseases.
- The chronic nature of condition was raised, it changes throughout life. The new PRO will just be a snapshot. There was discussion about a longitudinal study being ideal. The PRO is a good starting point for this.
- There was a point raised about people who have not had intervention but have improved their condition over time, receiving good non-surgical advice. The questionnaire will probably include space for people to share their experiences. In the same way tips and experiences are shared at the meetings this study could share that. The international nature of this study may enable sharing of useful, different approaches from other countries.
- Manometry and barium swallow aren’t pleasant tests and you don’t want to repeat them without good cause. The PRO could help to create a threshold for further investigation / treatment.
- On the question of spasms, an important issue for those with achalasia, Prof Zaninotto said that chest pain was included in the study.