A trial of surgery versus no surgery in patients with severe pectus excavatum: looking at improvements in heart and lung function
| ISRCTN | ISRCTN11359779 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN11359779 |
| IRAS number | 331910 |
| Secondary identifying numbers | CPMS 57603, NIHR158749, IRAS 331910 |
- Submission date
- 26/04/2024
- Registration date
- 30/04/2024
- Last edited
- 04/06/2024
- Recruitment status
- Recruiting
- Overall study status
- Ongoing
- Condition category
- Surgery
Plain English Summary
Background and study aims
Pectus Excavatum (PE), also known as funnel chest, is a condition where the ribs and breastbone grow inwards forming a dent in the chest. It can be found in between 1 in 400 and 1 in 1000 people. People with severe PE, where the space between the spine and the chest is limited, can experience symptoms such as breathlessness, dizziness, fainting and pain with exercising. This can be very restrictive in daily life.
Treatment for PE includes surgery, which lifts the sternum up, relieving these symptoms. There are no new or “experimental” procedures being tested in this study: the two types of surgeries called the Nuss and Ravitch procedures are both well-established and regularly performed in patients across the world.
In 2019 the NHS England decommissioned pectus surgery, although it is still fully-funded in the devolved nations. This decision was based on the lack of high-quality comparative data showing an improvement in physical health or heart-lung function. More recently in 2023, NHS England have started funding surgery for only the most severe cases, as assessed by a national expert Multi-Disciplinary Team (MDT), which are then performed at two hospitals in England.
The purpose of this study is to see how surgery to treat PE affects a participant’s ability to be physically active. We will recruit 300 participants overall and compare the risks and benefits of surgery against no surgery as measured by a change in physical health after a year. We will also look at how much it costs the NHS overall.
Who can participate?
Patients with pectus excavatum aged 12 years or older.
What does the study involve?
Participants will have a series of data collected when they join the trial, most data should be available from their medical records, additionally, they will need to complete a number of questionnaires. If they have consented to the trial, then they will be allocated randomly (by a computer) to either the early or the delayed surgery arms. If they are part of the observational study, they will have the surgery as already planned.
Participants will then be followed initially for up to approximately 3 years post-surgery (at 6 months, 1 year and 3 years after surgery, and for the delayed-surgery group also at 6 months and 1 year after their allocation) and, if further funding is available, then for up to 5 years after surgery. At these follow-ups, participants will be asked about their current status, medications, any hospitalisations, adverse events and other procedures they might have undergone since their last contact point, additionally they will be asked to complete the same questionnaires as at baseline. Participants will also be asked to undergo exercise tests to measure their heart-lung function at 1 and 3 years after surgery, and for the delayed-surgery group also at 1 year after their allocation and before their surgery.
There are two types of surgery, one called Nuss and one called Ravitch. Both types are included in this study, and the surgeon will discuss them with the participant to help decide the best option. Both surgical procedures are well-established practices for the treatment of PE. As such, there are routine care practices to be followed.
What are the possible benefits and risks of participating?
The main benefit for participants in England in groups 1 and 2 is that being part of the study enables access to surgery that may not be available to them as part of NHS care; and it is expected that the surgery will improve their condition. However, there are risks associated with the surgery, including prolonged pain, infection, and complications of the procedure. In the routine surgical follow-ups, there is also the need for a CT scan, which involves a low dose of radiation; with any radiation exposure there is the risk that it may cause cancer many years later. Additional risks and burdens as research participants include the potential loss of confidentiality, as we will collect a lot of personal data, although we will recorded most of this against a code. Some people will also feel uncomfortable when answering questions about their health or healthcare.
Where is the study run from?
South Tees Hospitals NHS Foundation Trust (UK)
When is the study starting and how long is it expected to run for?
February 2024 to May 2030
Who is funding the study?
National Institute for Health and Care Research (NIHR) (UK).
Who is the main contact?
RESTORE trial team, Dr Lisa Chang
stees.pectusrestoretrial@nhs.net
Contact information
Public
South Tees NHS Foundation Trust
Cardiovascular Clinical Research Facility
James Cook University Hospital
Marton Road
Middlesbrough
TS4 3BW
United Kingdom
| Phone | +44 1642 850850 |
|---|---|
| stees.pectusrestoretrial@nhs.net |
Scientific, Principal Investigator
South Tees NHS Foundation Trust
Cardiovascular Clinical Research Facility
James Cook University Hospital
Marton Road
Middlesbrough
TS4 3BW
United Kingdom
 ORCID ID |
0000-0002-2429-3579 |
|---|---|
| Phone | +44 1642 850850 |
| stees.pectusrestoretrial@nhs.net |
Study information
| Study design | Interventional randomized controlled trial with observational cohort |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | To follow |
| Scientific title | A randomised trial of surgery versus no treatment to RESTORE cardiopulmonary function in severe pectus excavatum |
| Study acronym | RESTORE |
| Study hypothesis | Surgery improves physical functioning in patients with pectus excavatum, as measured at one-year. |
| Ethics approval(s) |
Approved 03/06/2024, East of Scotland Research Ethics Service (Tayside Medical Science Centre, George Pirie Way, Ninewells Hospital, Dundee, DD1 9SY, United Kingdom; +44 1382 383871; tay.eosres@nhs.scot), ref: 24/ES/0034 |
| Condition | Cardiothoracic surgery, pectus excavatum |
| Intervention | If a potential participant consents to the randomised trial, a series of screening and baseline assessments will be conducted and recorded. These include capturing data from routine care on medical history, physical exercise and lung function tests, existing scans and images that have been performed to diagnose their condition and the severity of it. In addition to capturing this routine data, participants will be asked to complete a series of questionnaires. Where the patients have had an exercise test some time ago (more than 12 months’ ago for people 16 years and over, and more than 6 months’ ago for people under 16 years) we will ask them to do a repeat test, as their condition might have changed. If meeting the eligibility criteria, participants will be allocated (randomised) into one of two groups: to have early surgery (within 3 months of randomisation), or to have surgery after a 12-month delay. For the late-surgery group, they will be contacted at 6 and 12 months after randomisation to ask about their health and complete the same questionnaires they had at baseline. At 12-months they will also be asked to repeat the exercise test. For all groups, after surgery, as part of their routine care, it is recommended in routine care that a low-dose CT scan is performed to measure the impact of the surgery, this takes place in a timeframe of up to 6 months after surgery and we will collect this data if it is available. At 6 and 12 months after surgery, all participants will be contacted to ask about their health and complete the same questionnaires as at baseline. At 12-months after surgery they will also be asked to repeat the exercise test. If patients undergo the Nuss surgical procedure (which involves the insertion of bars into their chest), then at any time from 2.5 - 3 years after surgery they will have the bars removed. These participants will be contacted approximately 6 months after the bars are removed to ask about their health and complete the same questionnaires. They will also be asked to repeat the exercise test. If patients undergo the Ravitch surgical procedure, they will be contacted 3 years after surgery to ask about their health, complete the same questionnaires and to repeat the exercise test. |
| Intervention type | Procedure/Surgery |
| Primary outcome measure | 1. SF-36v2 Physical Function score change between testing before randomisation and 1 year later (control group) or 1 year after surgery (experimental group). 2. Incremental cost per QALY at 1 year. QALY assessed through quality of life questionnaires (EQ-5D-5L, SF-36v2, HADS) completed at baseline and 1 year. |
| Secondary outcome measures | 1. Measures of cardiopulmonary function from cardiopulmonary exercise tests (CPET), at baseline, 1 year (post-surgery for all groups and also post-randomisation for the late surgery group), 3 years post-surgery. 2. Quality of life measures including those that of impact on mental well-being (EQ-5D-5L and SF36v2 mental component scores, and HADS), at baseline, 6 months, 1 year (post-surgery for all groups and also post-randomisation for the late surgery group), 3 years post-surgery. 3. Symptoms measured by Nuss and Phoenix Comprehensive Assessment of Pectus Excavatum Symptoms (PCAPES) Questionnaires, at baseline, 6 months, 1 year (post-surgery for all groups and also post-randomisation for the late surgery group), 3 years post-surgery. 4. Body image measured by the Body Image Disturbance Questionnaire (BIDQ), at baseline, 6 months, 1 year (post-surgery for all groups and also post-randomisation for the late surgery group), 3 years post-surgery. 5. Need for revision surgery (complications including the need for unplanned redo surgery and syncope), data collected across the duration of the patient’s participation in the study (up to 3 years). 6. Adverse events of special interest, data collected across the duration of the patient’s participation in the study (up to 3 years). 7. Major surgical complications, data collected across the duration of the patient’s participation in the study (up to 3 years). 8. Costs to the NHS and patients at 1 year and over the patients’ lifetime, measured by Health Resource Usage Questionnaire. 9. QALYs at 1 year and over the patients’ lifetime, measured by quality of life questionnaires (EQ-5D-5L, SF-36v2, HADS). 10. Incremental cost per QALY gained over the patients’ lifetime, measured by quality of life questionnaires and healthcare resource utilisation. 11. Patient preference measured by a discrete choice experiment, to be conducted at month 16 post-randomisation. 12. Net benefit of the intervention, measured by a combination of outcome measures listed above. |
| Overall study start date | 01/02/2024 |
| Overall study end date | 31/05/2030 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Mixed |
| Lower age limit | 12 Years |
| Sex | Both |
| Target number of participants | Planned Sample Size: 300; UK Sample Size: 300 |
| Participant inclusion criteria | For inclusion in the randomised trial: 1. ≥ 12 years old. 2. A PE deformity with a Haller Index of >3.25, as measured by the internal width of the chest measured at the widest point divided by the distance from the back of the sternum to the anterior vertebral body at its minimum point on CT scan. 3. The participant must satisfy at least one of the following criteria: 3.1. Significant level of shortness of breath or exercise ability perceived to be below that of their peers (e.g., limited by vigorous activities such as running or lifting heavy objects). 3.2. Presyncope or syncope on exercise. 3.3. Arrhythmias on ECG that may be due to the pectus abnormality. 3.4. Dysphagia or swallowing abnormalities in the absence of any other cause. 4. Provide informed consent/assent and agree to 5 years of follow up. 5. Fit to undergo surgery. For inclusion in the observational cohort: 1. >=12 years old. 2. Confirmed as eligible and fit for surgery via the national MDT surgical pathway. 3. Providing informed consent to take part in the embedded observational cohort and agreeing to 5 years follow up. |
| Participant exclusion criteria | For the randomised trial: 1. Patients not fulfilling the inclusion criteria. 2. Symptoms relating to causes other than PE. 3. Unwilling to have surgery for PE. For the observational cohort: 1. Patients not fulfilling the inclusion criteria. 2. Patients who are willing to join the full randomised trial group of the RESTORE Trial. |
| Recruitment start date | 01/08/2024 |
| Recruitment end date | 31/08/2026 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centres
Marton Road
Middlesbrough
TS4 3BW
United Kingdom
Eaton Road
West Derby
Liverpool
L12 2AP
United Kingdom
Blackshaw Road
Tooting
London
SW17 0QT
United Kingdom
80 Newark Street
London
E1 2ES
United Kingdom
Mindelsohn Way
Edgbaston
Birmingham
B15 2GW
United Kingdom
Beckett Street
Leeds
LS9 7TF
United Kingdom
Westminster Bridge Road
London
SE1 7EH
United Kingdom
Cambridge Biomedical Campus
Cambridge
CB2 0AY
United Kingdom
Infirmary Square
Leicester
LE1 5WW
United Kingdom
Headley Way
Headington
Oxford
OX3 9DU
United Kingdom
Queens Medical Centre
Derby Road
Nottingham
NG7 2UH
United Kingdom
Oxford Road
Manchester
M13 9WL
United Kingdom
Westcliff-on-sea
SS0 0RY
United Kingdom
Tremona Road
Southampton
SO16 6YD
United Kingdom
Sponsor information
Hospital/treatment centre
James Cook University Hospital, Marton Road
Middlesbrough
TS4 3BW
England
United Kingdom
| Phone | +44 7788 360 287 |
|---|---|
| david.rollins@nhs.net | |
| Website | http://southtees.nhs.uk/ |
"ROR" |
https://ror.org/02js17r36 |
Funders
Funder type
Government
No information available
Results and Publications
| Intention to publish date | 31/12/2028 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | The findings of the study will be disseminated to stakeholders for the RESTORE research, including patients, NHS England and the clinical community nationally and internationally. The Patient Advisory Group and lay co-applicants will work with the trial team to inform the dissemination plans to patients and the public. Findings will be reported to the funder, Society for Cardiothoracic Surgery (SCTS), the writing committee of the European Society of Thoracic Surgery (ESTS), National Institute for Health and Care Excellence (NICE) and presented at conferences including the European Society of Thoracic Surgeons and the Chest Wall International Group. The Chief Investigator and co-Chief Investigator will be responsible for ensuring the results of RESTORE are disseminated through peer reviewed journals once the analyses are complete. |
| IPD sharing plan | The datasets generated during and/or analysed during the current study are available from the corresponding author on reasonable request Academic Cardiovascular Unit, South Tees Hospitals NHS Foundation Trust stees.pectusrestoretrial@nhs.net Aggregated coded datasets will be available to be shared upon request, following publication of the main findings. The data will be available, as described in the participant information sheet, and according to the consent provided by the participant. Requesters must provide an outline of their analysis plans and agree to adhere to UK data privacy laws as a minimum, access will be granted after consideration of the plan by the study chief investigator, trials unit and sponsor. |
Editorial Notes
04/06/2024: Ethics approval details added.
26/04/2024: Trial's existence confirmed by the National Institute for Health and Care Research (NIHR) (UK).
