Paediatric Surgery, Erasmus MC Sophia Children's Hospital, Rotterdam, Zuid-Holland, Netherlands.
Tracheal Team, Department of Cardiothoracic Surgery, Great Ormond Street Hospital for Children, London, UK.
BMJ Open. 2023 Mar 17;13(3):e071989. doi: 10.1136/bmjopen-2023-071989.
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.
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.
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.
NCT05701514.
对于无症状先天性肺气道畸形（CPAM）的最佳治疗方法，目前尚未达成共识。为了开展未来的研究，由专门医护人员组成的国际合作组织 CONNECT 联盟（即首个欧洲 CPAM 试验的协作性新生儿网络）就呼吸功能不全、手术并发症、肿块效应和多灶性疾病等与结果相关的参数达成了核心结局集的共识。这些结局参数已经纳入 CONNECT 试验，这是一项随机对照试验，旨在比较无症状 CPAM 患者的保守治疗和手术治疗，以便为制定循证实践提供依据。
在儿童的 CPAM 经产后胸部 CT 扫描确诊且无症状后，即可入组。入组时，儿童将被随机分配接受保守或手术治疗。随后，两组中的儿童都将纳入一个标准化的 5 年随访计划，该计划包括 3 次就诊，包括 2.5 岁时进行重复胸部 CT 扫描和 5 岁时进行标准的运动耐量测试。主要结局是 5 岁时的运动耐量，根据布鲁斯跑步机方案进行测量。次要结局指标包括分子遗传学诊断、经过验证的父母焦虑、生活质量和医疗保健消费问卷、随访期间的重复影像学检查和肺部发病率，以及手术并发症和组织病理学。本试验旨在结束围绕无症状 CPAM 最佳治疗方法的持续争论。
本研究符合《赫尔辛基宣言》的规定。荷兰鹿特丹伊拉斯谟大学医学中心的医学伦理审查委员会已经批准了该方案（MEC-2022-0441）。研究结果将通过同行评议的科学期刊和会议报告进行传播。
NCT05701514。
