Simen Vergote1, Felix De Bie1,2, Jan Bosteels1, Holly Hedrick2, James Duffy3,4, Beverley Power5, Alexandra Benachi6, Paolo De Coppi7,8, Caraciolo Fernandes9, Kevin Lally10, Irwin Reiss11, Jan Deprest12,13. 1. Department of Development and Regeneration cluster Woman and Child, Biomedical Sciences, KU Leuven, Herestraat 49 - Box 805, B-3000, Leuven, Belgium. 2. Department of Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA. 3. Institute of Women's Health, University College of London, London, UK. 4. Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, UK. 5. CDH UK, The Denes, Norfolk, UK. 6. Service de Gynécologie-Obstétrique, Hôpital Antoine Béclère, AP-HP, Clamart, France. 7. Department of Specialist Neonatal and Pediatric Surgery, Great Ormond Street Hospital for Children, Great Ormond Street, London, UK. 8. Stem Cells and Regenerative Medicine Section, Institute of Child Health, University College London, London, UK. 9. Texas Children's Fetal Center, Texas Children's Hospital and Department of Pediatrics - Newborn Section, Baylor College of Medicine, Houston, TX, USA. 10. Department of Pediatric Surgery, McGovern Medical School at UT Health and Children's Memorial Hermann Hospital, Houston, TX, USA. 11. Department of Pediatrics, Division of Neonatology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands. 12. Department of Development and Regeneration cluster Woman and Child, Biomedical Sciences, KU Leuven, Herestraat 49 - Box 805, B-3000, Leuven, Belgium. jan.deprest@uzleuven.be. 13. Institute of Women's Health, University College of London, London, UK. jan.deprest@uzleuven.be.
Abstract
BACKGROUND: Congenital diaphragmatic hernia (CDH) is, depending of the severity, a birth defect associated with significant mortality and morbidity. Prenatal screening by ultrasound may detect this condition and comprehensive assessment of severity is possible, allowing for in utero referral to an experienced centre for planned delivery. In an effort to improve outcomes, prenatal interventions to stimulate lung development were proposed. Along the same lines, new postnatal management strategies are being developed. In order to enable proper comparison of novel perinatal interventions as well as outcomes, a set of uniform and relevant outcome measures is required. Core outcome sets (COS) are agreed, clearly defined sets of outcomes to be measured in a standardised manner and reported consistently. Herein we aim to describe the methodology we will use to define a COS for perinatal and neonatal outcomes of foetuses and newborns with congenital diaphragmatic hernia and to draft a dissemination and implementation plan. METHODS: We will use the methodology described in the Core Outcome Measures in Effectiveness Trials (COMET) Initiative Handbook. An international steering group will be created to guide the development of the COS. We are systematically reviewing the literature to identify all potential relevant pre- and neonatal outcomes previously used in studies on perinatal interventions for CDH. We will build a consensus on these core outcomes in a stakeholder group using the Delphi method. After completion, a stakeholder meeting will decide on a final COS, using a modified Nominal Group Technique. Thereafter, we will review potential definitions and measurements of these outcomes, and again a consensus meeting will be organised, to finalise the COS before dissemination. DISCUSSION: We have started a procedure to develop a COS for studies on perinatal interventions for congenital diaphragmatic hernia, with the purpose of improving the quality of research, guide clinical practice and improve patient care and eventual use in future clinical trials, systematic reviews and clinical practice guidelines. TRIAL REGISTRATION: We prospectively registered this study in the International Prospective Register of Systematic Reviews (PROSPERO) (registration number: CRD42019124399 ) and The Core Outcome Measures in Effectiveness Trials (COMET) Initiative (registration number: 1296 ).
BACKGROUND:Congenital diaphragmatic hernia (CDH) is, depending of the severity, a birth defect associated with significant mortality and morbidity. Prenatal screening by ultrasound may detect this condition and comprehensive assessment of severity is possible, allowing for in utero referral to an experienced centre for planned delivery. In an effort to improve outcomes, prenatal interventions to stimulate lung development were proposed. Along the same lines, new postnatal management strategies are being developed. In order to enable proper comparison of novel perinatal interventions as well as outcomes, a set of uniform and relevant outcome measures is required. Core outcome sets (COS) are agreed, clearly defined sets of outcomes to be measured in a standardised manner and reported consistently. Herein we aim to describe the methodology we will use to define a COS for perinatal and neonatal outcomes of foetuses and newborns with congenital diaphragmatic hernia and to draft a dissemination and implementation plan. METHODS: We will use the methodology described in the Core Outcome Measures in Effectiveness Trials (COMET) Initiative Handbook. An international steering group will be created to guide the development of the COS. We are systematically reviewing the literature to identify all potential relevant pre- and neonatal outcomes previously used in studies on perinatal interventions for CDH. We will build a consensus on these core outcomes in a stakeholder group using the Delphi method. After completion, a stakeholder meeting will decide on a final COS, using a modified Nominal Group Technique. Thereafter, we will review potential definitions and measurements of these outcomes, and again a consensus meeting will be organised, to finalise the COS before dissemination. DISCUSSION: We have started a procedure to develop a COS for studies on perinatal interventions for congenital diaphragmatic hernia, with the purpose of improving the quality of research, guide clinical practice and improve patient care and eventual use in future clinical trials, systematic reviews and clinical practice guidelines. TRIAL REGISTRATION: We prospectively registered this study in the International Prospective Register of Systematic Reviews (PROSPERO) (registration number: CRD42019124399 ) and The Core Outcome Measures in Effectiveness Trials (COMET) Initiative (registration number: 1296 ).
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