A randomised controlled trial of surgery versus no treatment to RESTORE cardiopulmonary function in severe pectus excavatum – The RESTORE Trial.

Research question: Does corrective surgery for pectus excavatum (PE) improve functional cardiopulmonary outcomes and is it cost effective? Background: There is no high-quality evidence on the effectiveness of surgery in providing functional improvements in patients with severe PE and concomitant physiological impairment. Consequently, in 2019 the NHS England Clinical Panel decommissioned PE surgery in England, although it is still funded in the devolved nations. There is an urgent need for high quality evidence to inform guidelines, practice, and future commissioning decisions. Aims: to establish the impact of corrective surgery for PE on physical function, cardiopulmonary function and its cost effectiveness. Primary Objective: To determine whether corrective surgery for PE is superior to no surgery as measured by change in SF-36v2 physical function score at 1 year. Primary Economic Objective: To estimate the cost effectiveness of corrective for PE compared to no surgery over 1 year. Design: Prospective, Pragmatic, Superiority, Randomised Controlled Trial, and an observational cohort. RESTORE will recruit 200 participants with severe Pectus Excavatum to the randomised controlled trial from 12 centres over 24 months. An observational cohort will recruit up to 100 participants over the same period. Internal Pilot: to confirm recruitment rates and trial processes in the randomised trial. Methods: Patients aged 12 years or over who have severe PE defined as a Haller index more than 3.25, from 12 NHS cardiothoracic surgical centres who have already agreed to participate, will be eligible to join the study if they have any physical symptoms that might be attributable to their PE abnormality. Following consent, baseline tests will be performed and patients will be randomly allocated to immediate surgery (intervention), or delayed surgery which will occur at least one year after randomisation (control). The primary outcome measure will be the difference in the change from baseline of SF-36v2 physical function score 1 year after randomisation between the two groups. Secondary outcomes, including cardiopulmonary function measured by cardiopulmonary exercise testing, surgical complications including repeat surgery, surgical success, impact on symptoms, health related quality of life, and psychological outcomes, will be reported at 1 year after randomisation and 3 years after surgery. These same outcome measures will also be applied to a cohort group of very severe patients approved for surgery by the national MDT, and who are not willing to undergo randomisation. We will take consent from all patients to follow them beyond the time of the trial up to 5 years post-surgery. Timelines for delivery: The trial length will be 76 months. Primary Outcome Reporting: Early data lock will allow the primary analysis and reporting of the primary outcome in Month 51, to enable NHS commissioners in England to make an expedited decision. Anticipated Impact and Dissemination: This will be the first ever randomised controlled trial for PE internationally. As such, it will directly influence international guidelines and practice. If we demonstrate that PE surgery is clinically and cost effective, it will have immediate impact on NHS England commissioning, allowing patients with severe PE and symptoms, to gain routine access to a surgical treatment, addressing the disparity in access which currently exists across the devolved nations.