Cargando…

COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial

INTRODUCTION: Consensus is lacking on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM). For future studies, the CONNECT consortium (the COllaborative Neonatal Network for the first European CPAM Trial)—an international collaboration of specialised caregivers—has...

Descripción completa

Detalles Bibliográficos
Autores principales: Kersten, Casper M, Hermelijn, Sergei M, Dossche, Louis W J, Muthialu, Nagarajan, Losty, Paul D, Schurink, Maarten, Rietman, André B, Poley, Marten J, van Rosmalen, Joost, Zanen - van den Adel, Tabitha P L, Ciet, Pierluigi, von der Thüsen, Jan, Brosens, Erwin, Ijsselstijn, Hanneke, Tiddens, Harm A W M, Wijnen, Rene M H, Schnater, J Marco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10030930/
https://www.ncbi.nlm.nih.gov/pubmed/36931672
http://dx.doi.org/10.1136/bmjopen-2023-071989
_version_ 1784910485517238272
author Kersten, Casper M
Hermelijn, Sergei M
Dossche, Louis W J
Muthialu, Nagarajan
Losty, Paul D
Schurink, Maarten
Rietman, André B
Poley, Marten J
van Rosmalen, Joost
Zanen - van den Adel, Tabitha P L
Ciet, Pierluigi
von der Thüsen, Jan
Brosens, Erwin
Ijsselstijn, Hanneke
Tiddens, Harm A W M
Wijnen, Rene M H
Schnater, J Marco
author_facet Kersten, Casper M
Hermelijn, Sergei M
Dossche, Louis W J
Muthialu, Nagarajan
Losty, Paul D
Schurink, Maarten
Rietman, André B
Poley, Marten J
van Rosmalen, Joost
Zanen - van den Adel, Tabitha P L
Ciet, Pierluigi
von der Thüsen, Jan
Brosens, Erwin
Ijsselstijn, Hanneke
Tiddens, Harm A W M
Wijnen, Rene M H
Schnater, J Marco
author_sort Kersten, Casper M
collection PubMed
description INTRODUCTION: Consensus is lacking on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM). For future studies, the CONNECT consortium (the COllaborative Neonatal Network for the first European CPAM Trial)—an international collaboration of specialised caregivers—has established consensus on a core outcome set of outcome parameters concerning respiratory insufficiency, surgical complications, mass effect and multifocal disease. These outcome parameters have been incorporated in the CONNECT trial, a randomised controlled trial which, in order to develop evidence-based practice, aims to compare conservative and surgical management of patients with an asymptomatic CPAM. METHODS AND ANALYSIS: Children are eligible for inclusion after the CPAM diagnosis has been confirmed on postnatal chest CT scan and they remain asymptomatic. On inclusion, children are randomised to receive either conservative or surgical management. Subsequently, children in both groups are enrolled into a standardised, 5-year follow-up programme with three visits, including a repeat chest CT scan at 2.5 years and a standardised exercise tolerance test at 5 years. The primary outcome is exercise tolerance at age 5 years, measured according to the Bruce treadmill protocol. Secondary outcome measures are molecular genetic diagnostics, validated questionnaires—on parental anxiety, quality of life and healthcare consumption—, repeated imaging and pulmonary morbidity during follow-up, as well as surgical complications and histopathology. This trial aims to end the continuous debate surrounding the optimal management of asymptomatic CPAM. ETHICS AND DISSEMINATION: This study is being conducted in accordance with the Declaration of Helsinki. The Medical Ethics Review Board of Erasmus University Medical Centre Rotterdam, The Netherlands, has approved this protocol (MEC-2022–0441). Results will be disseminated through peer-reviewed scientific journals and conference presentations. TRIAL REGISTRATION NUMBER: NCT05701514.
format Online
Article
Text
id pubmed-10030930
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher BMJ Publishing Group
record_format MEDLINE/PubMed
spelling pubmed-100309302023-03-23 COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial Kersten, Casper M Hermelijn, Sergei M Dossche, Louis W J Muthialu, Nagarajan Losty, Paul D Schurink, Maarten Rietman, André B Poley, Marten J van Rosmalen, Joost Zanen - van den Adel, Tabitha P L Ciet, Pierluigi von der Thüsen, Jan Brosens, Erwin Ijsselstijn, Hanneke Tiddens, Harm A W M Wijnen, Rene M H Schnater, J Marco BMJ Open Paediatrics INTRODUCTION: Consensus is lacking on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM). For future studies, the CONNECT consortium (the COllaborative Neonatal Network for the first European CPAM Trial)—an international collaboration of specialised caregivers—has established consensus on a core outcome set of outcome parameters concerning respiratory insufficiency, surgical complications, mass effect and multifocal disease. These outcome parameters have been incorporated in the CONNECT trial, a randomised controlled trial which, in order to develop evidence-based practice, aims to compare conservative and surgical management of patients with an asymptomatic CPAM. METHODS AND ANALYSIS: Children are eligible for inclusion after the CPAM diagnosis has been confirmed on postnatal chest CT scan and they remain asymptomatic. On inclusion, children are randomised to receive either conservative or surgical management. Subsequently, children in both groups are enrolled into a standardised, 5-year follow-up programme with three visits, including a repeat chest CT scan at 2.5 years and a standardised exercise tolerance test at 5 years. The primary outcome is exercise tolerance at age 5 years, measured according to the Bruce treadmill protocol. Secondary outcome measures are molecular genetic diagnostics, validated questionnaires—on parental anxiety, quality of life and healthcare consumption—, repeated imaging and pulmonary morbidity during follow-up, as well as surgical complications and histopathology. This trial aims to end the continuous debate surrounding the optimal management of asymptomatic CPAM. ETHICS AND DISSEMINATION: This study is being conducted in accordance with the Declaration of Helsinki. The Medical Ethics Review Board of Erasmus University Medical Centre Rotterdam, The Netherlands, has approved this protocol (MEC-2022–0441). Results will be disseminated through peer-reviewed scientific journals and conference presentations. TRIAL REGISTRATION NUMBER: NCT05701514. BMJ Publishing Group 2023-03-17 /pmc/articles/PMC10030930/ /pubmed/36931672 http://dx.doi.org/10.1136/bmjopen-2023-071989 Text en © Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. https://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Paediatrics
Kersten, Casper M
Hermelijn, Sergei M
Dossche, Louis W J
Muthialu, Nagarajan
Losty, Paul D
Schurink, Maarten
Rietman, André B
Poley, Marten J
van Rosmalen, Joost
Zanen - van den Adel, Tabitha P L
Ciet, Pierluigi
von der Thüsen, Jan
Brosens, Erwin
Ijsselstijn, Hanneke
Tiddens, Harm A W M
Wijnen, Rene M H
Schnater, J Marco
COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial
title COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial
title_full COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial
title_fullStr COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial
title_full_unstemmed COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial
title_short COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial
title_sort collaborative neonatal network for the first european cpam trial (connect): a study protocol for a randomised controlled trial
topic Paediatrics
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10030930/
https://www.ncbi.nlm.nih.gov/pubmed/36931672
http://dx.doi.org/10.1136/bmjopen-2023-071989
work_keys_str_mv AT kerstencasperm collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT hermelijnsergeim collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT dosschelouiswj collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT muthialunagarajan collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT lostypauld collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT schurinkmaarten collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT rietmanandreb collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT poleymartenj collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT vanrosmalenjoost collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT zanenvandenadeltabithapl collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT cietpierluigi collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT vonderthusenjan collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT brosenserwin collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT ijsselstijnhanneke collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT tiddensharmawm collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT wijnenrenemh collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial
AT schnaterjmarco collaborativeneonatalnetworkforthefirsteuropeancpamtrialconnectastudyprotocolforarandomisedcontrolledtrial