Kersten Casper M, Hermelijn Sergei M, Mullassery Dhanya, Muthialu Nagarajan, Cobanoglu Nazan, Gartner Silvia, Bagolan Pietro, Mesas Burgos Carmen, Sgrò Alberto, Heyman Stijn, Till Holger, Suominen Janne, Schurink Maarten, Desender Liesbeth, Losty Paul, Steyaert Henri, Terheggen-Lagro Suzanne, Metzelder Martin, Bonnard Arnaud, Sfeir Rony, Singh Michael, Yardley Iain, Rikkers-Mutsaerts Noor R V M, van der Ent Cornelis K, Qvist Niels, Cox Des W, Peters Robert, Bannier Michiel A G E, Wessel Lucas, Proesmans Marijke, Stanton Michael, Hannon Edward, Zampoli Marco, Morini Francesco, Tiddens Harm A W M, Wijnen René M H, Schnater Johannes M
Department of Pediatric Surgery, Erasmus MC Sophia Children's Hospital, 3015 GD Rotterdam, The Netherlands.
Department of Pediatric Surgery, Great Ormond Street Hospital, London WC1N 3JH, UK.
Children (Basel). 2022 Jul 30;9(8):1153. doi: 10.3390/children9081153.
Consensus on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM) is lacking, and comparison between studies remains difficult due to a large variety in outcome measures. We aimed to define a core outcome set (COS) for pediatric patients with an asymptomatic CPAM. An online, three-round Delphi survey was conducted in two stakeholder groups of specialized caregivers (surgeons and non-surgeons) in various European centers. Proposed outcome parameters were scored according to level of importance, and the final COS was established through consensus. A total of 55 participants (33 surgeons, 22 non-surgeons) from 28 centers in 13 European countries completed the three rounds and rated 43 outcome parameters. The final COS comprises seven outcome parameters: respiratory insufficiency, surgical complications, mass effect/mediastinal shift (at three time-points) and multifocal disease (at two time-points) The seven outcome parameters included in the final COS reflect the diversity in priorities among this large group of European participants. However, we recommend the incorporation of these outcome parameters in the design of future studies, as they describe measurable and validated outcomes as well as the accepted age at measurement.
目前对于无症状先天性肺气道畸形（CPAM）的最佳管理尚无共识，且由于结局指标差异很大，不同研究之间的比较仍然困难。我们旨在为无症状CPAM的儿科患者定义一个核心结局集（COS）。在欧洲多个中心的两个利益相关者群体（外科医生和非外科医生）中进行了三轮在线德尔菲调查。根据重要性水平对提出的结局参数进行评分，并通过共识确定最终的COS。来自欧洲13个国家28个中心的55名参与者（33名外科医生，22名非外科医生）完成了三轮调查，并对43个结局参数进行了评分。最终的COS包括七个结局参数：呼吸功能不全、手术并发症、肿块效应/纵隔移位（在三个时间点）和多灶性疾病（在两个时间点）。最终COS中包含的七个结局参数反映了这一大批欧洲参与者之间优先事项的多样性。然而，我们建议在未来研究的设计中纳入这些结局参数，因为它们描述了可测量和经过验证的结局以及公认的测量年龄。
