Hermelijn Sergei, Kersten Casper, Mullassery Dhanya, Muthialu Nagarajan, Cobanoglu Nazan, Gartner Silvia, Bagolan Pietro, Mesas Burgos Carmen, Sgro Alberto, Heyman Stijn, Till Holger, Suominen Janne, Schurink Maarten, Desender Liesbeth, Losty Paul, Ertresvag Kjetil, Tiddens Harm A W M, Wijnen Rene M H, Schnater Marco
Pediatric Surgery, Erasmus MC Sophia Children's Hospital, Rotterdam, The Netherlands.
Pediatric Surgery, Great Ormond Street Hospital for Children, London, UK.
BMJ Open. 2021 Apr 12;11(4):e044544. doi: 10.1136/bmjopen-2020-044544.
A worldwide lack of consensus exists on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM) even though the incidence is increasing. Either a surgical resection is performed or a wait-and-see policy is employed, depending on the treating physician. Management is largely based on expert opinion and scientific evidence is scarce. Wide variations in outcome measures are seen between studies making comparison difficult thus highlighting the lack of universal consensus in outcome measures as well. We aim to define a core outcome set which will include the most important core outcome parameters for paediatric patients with an asymptomatic CPAM.
This study will include a critical appraisal of the current literature followed by a three-stage Delphi process with two stakeholder groups. One surgical group including paediatric as well as thoracic surgeons, and a non-surgeon group including paediatric pulmonologists, intensive care and neonatal specialists. All participants will score outcome parameters according to their level of importance and the most important parameters will be determined by consensus.
Electronic informed consent will be obtained from all participants. Ethical approval is not required. After the core outcome set has been defined, we intend to design an international randomised controlled trial: the COllaborative Neonatal NEtwork for the first CPAM Trial, which will be aimed at determining the optimal management of patients with asymptomatic CPAM.
尽管无症状先天性肺气道畸形（CPAM）的发病率在上升，但全球对于其最佳治疗方案仍未达成共识。治疗方案要么是进行手术切除，要么是采取观察等待策略，具体取决于主治医生。治疗很大程度上基于专家意见，科学证据匮乏。各研究之间的结局指标差异很大，难以进行比较，这也凸显了在结局指标方面缺乏普遍共识。我们旨在确定一个核心结局集，其中将包括无症状CPAM儿科患者最重要的核心结局参数。
本研究将首先对当前文献进行批判性评估，然后对两个利益相关者群体进行三阶段德尔菲法。一个手术组包括儿科和胸外科医生，一个非手术组包括儿科肺科医生、重症监护和新生儿专家。所有参与者将根据结局参数的重要程度进行评分，最重要的参数将通过共识确定。
将从所有参与者那里获得电子知情同意书。无需伦理批准。在确定核心结局集之后，我们打算设计一项国际随机对照试验：首个CPAM试验的协作新生儿网络试验，该试验旨在确定无症状CPAM患者的最佳治疗方案。
