{
  "abstract": "Introduction Severe pectus excavatum (PE) may impair cardiopulmonary and physical function. The effectiveness of surgical treatment to correct PE and restore physical function is widely debated due to a lack of high-quality comparative evidence. The RESTORE trial aims to determine the clinical and cost-effectiveness of corrective surgery for severe PE compared with conservative management for the first time in a randomised controlled trial (RCT).Methods and analysis RESTORE is a pragmatic, multicentre, RCT with an embedded observational cohort. 200 participants aged ≥12 years with severe PE will be recruited at around 12 National Health Service cardiothoracic surgical centres in England. Participants will be randomised 1:1 to receive either surgery within 3 months of randomisation (intervention arm) or no surgery until after the primary outcome measurement at 1 year (comparator arm). The primary outcome is change in physical functioning from baseline to 1 year as measured by the Short Form Health Survey (SF-36v2) physical function score. The primary economic outcome is cost-effectiveness. The key secondary outcome is change in % predicted VO 2peak at 1 year measured by cardiopulmonary exercise test (CPET). Outcomes will be assessed at 1 year post-randomisation in the comparator arm and 1 year post-surgery in the intervention arm. The primary analyses will be undertaken on an intention-to-treat population using a linear mixed-effects model, adjusted for stratification variables via a binary covariate. Other secondary outcomes will include change from baseline of cardiopulmonary function (CPET and spirometry), health-related quality of life using the EuroQol 5 Dimension 5 Level (EQ-5D-5L) and SF-36v2 questionnaires, Hospital Anxiety and Depression Scale and disease specific symptoms (Phoenix Comprehensive Assessment for Pectus Excavatum Symptoms and Pectus Excavatum Evaluation Questionnaire). Adverse events, complications from surgery and operative technical success (Haller and Compression Indices from preoperative and postoperative CT scans) will also be assessed. Health economic analysis will estimate the incremental cost per quality adjusted life year at 1 year.Ethics and dissemination The trial was approved by East of Scotland Research and Ethics Service (24/ES/0034). Participants who are ≥16 years of age will be required to provide written informed consent. For participants <16 years of age who are not judged to be Gillick competent, written assent and written informed consent from a parent/guardian will be required. Results will be submitted for publication in peer-reviewed journals and shared with participants, clinicians and commissioners.Trial registration number ISRCTN11359779.",
  "authors": [
    {
      "affiliations": [
        "Academic Cardiovascular Unit, South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK",
        "Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK"
      ],
      "name": "Rebecca Maier"
    },
    {
      "affiliations": [
        "South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK"
      ],
      "name": "Joel Dunning"
    },
    {
      "affiliations": [
        "Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK"
      ],
      "name": "James Wason"
    },
    {
      "affiliations": [
        "Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK"
      ],
      "name": "Thomas Chadwick"
    },
    {
      "affiliations": [
        "Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK"
      ],
      "name": "Andrew Bryant"
    },
    {
      "affiliations": [
        "Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK"
      ],
      "name": "Cristina Fernandez-Garcia"
    },
    {
      "affiliations": [
        "Global Health Economics Centre, London School of Hygiene & Tropical Medicine, London, UK"
      ],
      "name": "Luke Vale"
    },
    {
      "affiliations": [
        "South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK",
        "North Yorkshire Academic Alliance of Perioperative Medicine, York, UK"
      ],
      "name": "Gerard R Danjoux"
    },
    {
      "affiliations": [
        "North Tees and Hartlepool NHS Foundation Trust, Hartlepool, UK"
      ],
      "name": "Gillian Wallace"
    },
    {
      "affiliations": [
        "South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK"
      ],
      "name": "Alistair Levett-Renton"
    },
    {
      "affiliations": [
        "University of Birmingham, Birmingham, UK"
      ],
      "name": "Babu Naidu"
    },
    {
      "affiliations": [
        "PPI Representative, Salford, UK"
      ],
      "name": "Claire Pryor"
    },
    {
      "affiliations": [
        "University of Oxford, Oxford, UK"
      ],
      "name": "Peter McCulloch"
    },
    {
      "affiliations": [
        "Alder Hey Children’s NHS Foundation Trust, Liverpool, UK"
      ],
      "name": "Rebecca Thursfield"
    },
    {
      "affiliations": [
        "South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK"
      ],
      "name": "Jonathan Wyllie"
    },
    {
      "affiliations": [
        "Academic Cardiovascular Unit, South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK"
      ],
      "name": "Lisa Chang"
    },
    {
      "affiliations": [
        "Academic Cardiovascular Unit, South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK"
      ],
      "name": "Leanne Marsay"
    },
    {
      "affiliations": [
        "Academic Cardiovascular Unit, South Tees Hospitals NHS Foundation Trust, Middlesbrough, UK",
        "Newcastle University Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK"
      ],
      "name": "Enoch Akowuah"
    }
  ],
  "title": "Surgery versus conservative management for severe pectus excavatum (RESTORE): protocol for a multicentre, randomised, controlled superiority trial",
  "uid": "2624818f-b0d1-5d4c-92a1-ce7c586dd4da"
}