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Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol

AIM: Necrotizing enterocolitis (NEC) is the most lethal disease of the gastrointestinal tract of preterm infants. New and existing management strategies need clinical evaluation. Large heterogeneity exists in the selection, measurement, and reporting of outcome measures in NEC intervention studies....

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Autores principales: Klerk, Daphne H., van Varsseveld, Otis C., Offringa, Martin, Modi, Neena, Lacher, Martin, Zani, Augusto, Pakarinen, Mikko P., Koivusalo, Antti, Jester, Ingo, Spruce, Marie, Derikx, Joep P. M., Bakx, Roel, Ksia, Amine, Vermeulen, Marijn J., Kooi, Elisabeth M. W., Hulscher, Jan B. F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10230797/
https://www.ncbi.nlm.nih.gov/pubmed/37259112
http://dx.doi.org/10.1186/s13063-023-07413-x
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author Klerk, Daphne H.
van Varsseveld, Otis C.
Offringa, Martin
Modi, Neena
Lacher, Martin
Zani, Augusto
Pakarinen, Mikko P.
Koivusalo, Antti
Jester, Ingo
Spruce, Marie
Derikx, Joep P. M.
Bakx, Roel
Ksia, Amine
Vermeulen, Marijn J.
Kooi, Elisabeth M. W.
Hulscher, Jan B. F.
author_facet Klerk, Daphne H.
van Varsseveld, Otis C.
Offringa, Martin
Modi, Neena
Lacher, Martin
Zani, Augusto
Pakarinen, Mikko P.
Koivusalo, Antti
Jester, Ingo
Spruce, Marie
Derikx, Joep P. M.
Bakx, Roel
Ksia, Amine
Vermeulen, Marijn J.
Kooi, Elisabeth M. W.
Hulscher, Jan B. F.
author_sort Klerk, Daphne H.
collection PubMed
description AIM: Necrotizing enterocolitis (NEC) is the most lethal disease of the gastrointestinal tract of preterm infants. New and existing management strategies need clinical evaluation. Large heterogeneity exists in the selection, measurement, and reporting of outcome measures in NEC intervention studies. This hampers meta-analyses and the development of evidence-based management guidelines. We aim to develop a Core Outcome Set (COS) for NEC that includes the most relevant outcomes for patients and physicians, from moment of diagnosis into adulthood. This COS is designed for use in NEC treatment trials, in infants with confirmed NEC. METHODS: This study is designed according to COS-STAD (Core Outcome Set-STAndards for Development) recommendations and the COMET (Core Outcome Measures in Effectiveness Trials) Initiative Handbook. We obtained a waiver from the Ethics Review Board and prospectively registered this study with COMET (Study 1920). We will approach 125 clinicians and/or researchers from low-middle and high-income countries based on their scientific output (using SCIVAL, a bibliometric tool). Patients and parents will be approached through local patient organisations. Participants will be separated into three panels, to assess differences in priorities between former patients and parents (1. lay panel), clinicians and researchers involved in the neonatal period (2. neonatal panel) and after the neonatal period (3. post-neonatal panel). They will be presented with outcomes currently used in NEC research, identified through a systematic review, in a Delphi process. Eligible outcome domains are also identified from the patients and parents’ perspectives. Using a consensus process, including three online Delphi rounds and a final face-to-face consensus meeting, the COS will be finalised and include outcomes deemed essential to all stakeholders: health care professionals, parents and patients’ representatives. The final COS will be reported in accordance with the COS-Standards for reporting (COS-STAR) statement. CONCLUSIONS: Development of an international COS will help to improve homogeneity of outcome measure reporting in NEC, will enable adequate and efficient comparison of treatment strategies, and will help the interpretation and implementation of clinical trial results. This will contribute to high-quality evidence regarding the best treatment strategy for NEC in preterm infants. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13063-023-07413-x.
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spelling pubmed-102307972023-06-01 Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol Klerk, Daphne H. van Varsseveld, Otis C. Offringa, Martin Modi, Neena Lacher, Martin Zani, Augusto Pakarinen, Mikko P. Koivusalo, Antti Jester, Ingo Spruce, Marie Derikx, Joep P. M. Bakx, Roel Ksia, Amine Vermeulen, Marijn J. Kooi, Elisabeth M. W. Hulscher, Jan B. F. Trials Study Protocol AIM: Necrotizing enterocolitis (NEC) is the most lethal disease of the gastrointestinal tract of preterm infants. New and existing management strategies need clinical evaluation. Large heterogeneity exists in the selection, measurement, and reporting of outcome measures in NEC intervention studies. This hampers meta-analyses and the development of evidence-based management guidelines. We aim to develop a Core Outcome Set (COS) for NEC that includes the most relevant outcomes for patients and physicians, from moment of diagnosis into adulthood. This COS is designed for use in NEC treatment trials, in infants with confirmed NEC. METHODS: This study is designed according to COS-STAD (Core Outcome Set-STAndards for Development) recommendations and the COMET (Core Outcome Measures in Effectiveness Trials) Initiative Handbook. We obtained a waiver from the Ethics Review Board and prospectively registered this study with COMET (Study 1920). We will approach 125 clinicians and/or researchers from low-middle and high-income countries based on their scientific output (using SCIVAL, a bibliometric tool). Patients and parents will be approached through local patient organisations. Participants will be separated into three panels, to assess differences in priorities between former patients and parents (1. lay panel), clinicians and researchers involved in the neonatal period (2. neonatal panel) and after the neonatal period (3. post-neonatal panel). They will be presented with outcomes currently used in NEC research, identified through a systematic review, in a Delphi process. Eligible outcome domains are also identified from the patients and parents’ perspectives. Using a consensus process, including three online Delphi rounds and a final face-to-face consensus meeting, the COS will be finalised and include outcomes deemed essential to all stakeholders: health care professionals, parents and patients’ representatives. The final COS will be reported in accordance with the COS-Standards for reporting (COS-STAR) statement. CONCLUSIONS: Development of an international COS will help to improve homogeneity of outcome measure reporting in NEC, will enable adequate and efficient comparison of treatment strategies, and will help the interpretation and implementation of clinical trial results. This will contribute to high-quality evidence regarding the best treatment strategy for NEC in preterm infants. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13063-023-07413-x. BioMed Central 2023-05-31 /pmc/articles/PMC10230797/ /pubmed/37259112 http://dx.doi.org/10.1186/s13063-023-07413-x Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Study Protocol
Klerk, Daphne H.
van Varsseveld, Otis C.
Offringa, Martin
Modi, Neena
Lacher, Martin
Zani, Augusto
Pakarinen, Mikko P.
Koivusalo, Antti
Jester, Ingo
Spruce, Marie
Derikx, Joep P. M.
Bakx, Roel
Ksia, Amine
Vermeulen, Marijn J.
Kooi, Elisabeth M. W.
Hulscher, Jan B. F.
Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol
title Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol
title_full Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol
title_fullStr Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol
title_full_unstemmed Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol
title_short Development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol
title_sort development of an international core outcome set for treatment trials in necrotizing enterocolitis—a study protocol
topic Study Protocol
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10230797/
https://www.ncbi.nlm.nih.gov/pubmed/37259112
http://dx.doi.org/10.1186/s13063-023-07413-x
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