The Management of Asymptomatic Congenital Pulmonary Airway Malformation: Results of a European Delphi Survey

Casper M Kersten, Sergei M Hermelijn, Dhanya Mullassery, Nagarajan Muthialu, Nazan Cobanoglu, Silvia Gartner, Pietro Bagolan, Carmen Mesas Burgos, Alberto Sgrò, Stijn Heyman, Holger Till, Janne Suominen, Maarten Schurink, Liesbeth Desender, Paul Losty, Henri Steyaert, Suzanne Terheggen-Lagro, Martin Metzelder, Arnaud Bonnard, Rony Sfeir, Michael Singh, Iain Yardley, Noor R V M Rikkers-Mutsaerts, Cornelis K van der Ent, Niels Qvist, Des W Cox, Robert Peters, Michiel A G E Bannier, Lucas Wessel, Marijke Proesmans, Michael Stanton, Edward Hannon, Marco Zampoli, Francesco Morini, Harm A W M Tiddens, René M H Wijnen, Johannes M Schnater, Casper M Kersten, Sergei M Hermelijn, Dhanya Mullassery, Nagarajan Muthialu, Nazan Cobanoglu, Silvia Gartner, Pietro Bagolan, Carmen Mesas Burgos, Alberto Sgrò, Stijn Heyman, Holger Till, Janne Suominen, Maarten Schurink, Liesbeth Desender, Paul Losty, Henri Steyaert, Suzanne Terheggen-Lagro, Martin Metzelder, Arnaud Bonnard, Rony Sfeir, Michael Singh, Iain Yardley, Noor R V M Rikkers-Mutsaerts, Cornelis K van der Ent, Niels Qvist, Des W Cox, Robert Peters, Michiel A G E Bannier, Lucas Wessel, Marijke Proesmans, Michael Stanton, Edward Hannon, Marco Zampoli, Francesco Morini, Harm A W M Tiddens, René M H Wijnen, Johannes M Schnater

Abstract

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.

Keywords: congenital lung abnormalities; congenital pulmonary airway malformation; consensus; core outcome set; outcome parameters.

Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Flowchart of the 3-round Delphi Survey.

References

    1. Morini F., Zani A., Conforti A., van Heurn E., Eaton S., Puri P., Rintala R., Lukac M., Kuebler J.F., Friedmacher F., et al. Current Management of Congenital Pulmonary Airway Malformations: A “European Pediatric Surgeons’ Association” Survey. Eur. J. Pediatr. Surg. 2018;28:1–5. doi: 10.1055/s-0037-1604020.
    1. Stanton M. The argument for a non-operative approach to asymptomatic lung lesions. Semin. Pediatr. Surg. 2015;24:183–186. doi: 10.1053/j.sempedsurg.2015.01.014.
    1. Singh R., Davenport M. The argument for operative approach to asymptomatic lung lesions. Semin. Pediatr. Surg. 2015;24:187–195. doi: 10.1053/j.sempedsurg.2015.02.003.
    1. Hermelijn S.M., Elders B.B.L.J., Ciet P., Wijnen R.M.H., Tiddens H., Schnater J.M. A clinical guideline for structured assessment of CT-imaging in congenital lung abnormalities. Paediatr. Respir. Rev. 2021;37:80–88. doi: 10.1016/j.prrv.2019.12.004.
    1. Stanton M., Njere I., Ade-Ajayi N., Patel S., Davenport M. Systematic review and meta-analysis of the postnatal management of congenital cystic lung lesions. J. Pediatr. Surg. 2009;44:1027–1033. doi: 10.1016/j.jpedsurg.2008.10.118.
    1. MacSweeney F., Papagiannopoulos K., Goldstraw P., Sheppard M.N., Corrin B., Nicholson A.G. An assessment of the expanded classification of congenital cystic adenomatoid malformations and their relationship to malignant transformation. Am. J. Surg. Pathol. 2003;27:1139–1146. doi: 10.1097/00000478-200308000-00012.
    1. Baird R., Puligandla P.S., Laberge J.M. Congenital lung malformations: Informing best practice. Semin. Pediatr. Surg. 2014;23:270–277. doi: 10.1053/j.sempedsurg.2014.09.007.
    1. Kunisaki S.M., Lal D.R., Saito J.M., Fallat M.E., St Peter S.D., Fox Z.D., Heider A., Chan S.S., Boyd K.P., Burns R.C., et al. Pleuropulmonary Blastoma in Pediatric Lung Lesions. Pediatrics. 2021;147:e2020028357. doi: 10.1542/peds.2020-028357.
    1. Hsieh C.C., Chao A.S., Chang Y.L., Kuo D.M., Hsieh T.T., Hung H.T. Outcome of congenital cystic adenomatoid malformation of the lung after antenatal diagnosis. Int. J. Gynecol. Obstet. 2005;89:99–102. doi: 10.1016/j.ijgo.2004.11.031.
    1. Cook J., Chitty L.S., De Coppi P., Ashworth M., Wallis C. The natural history of prenatally diagnosed congenital cystic lung lesions: Long-term follow-up of 119 cases. Arch. Dis Child. 2017;102:798–803. doi: 10.1136/archdischild-2016-311233.
    1. Benjamin D.R., Cahill J.L. Bronchioloalveolar carcinoma of the lung and congenital cystic adenomatoid malformation. Am. J. Clin. Pathol. 1991;95:889–892. doi: 10.1093/ajcp/95.6.889.
    1. Papagiannopoulos K.A., Sheppard M., Bush A.P., Goldstraw P. Pleuropulmonary blastoma: Is prophylactic resection of congenital lung cysts effective? Ann. Thorac. Surg. 2001;72:604–605. doi: 10.1016/S0003-4975(00)02539-X.
    1. Peters R.T., Burge D.M., Marven S.S. Congenital lung malformations: An ongoing controversy. Ann. R. Coll. Surg. Engl. 2013;95:144–147. doi: 10.1308/003588412X13373405387735.
    1. Eber E. Antenatal diagnosis of congenital thoracic malformations: Early surgery, late surgery, or no surgery? Semin. Respir. Crit. Care Med. 2007;28:355–366. doi: 10.1055/s-2007-981656.
    1. David M., Lamas-Pinheiro R., Henriques-Coelho T. Prenatal and Postnatal Management of Congenital Pulmonary Airway Malformation. Neonatology. 2016;110:101–115. doi: 10.1159/000440894.
    1. Kantor N., Wayne C., Nasr A. Symptom development in originally asymptomatic CPAM diagnosed prenatally: A systematic review. Pediatr. Surg. Int. 2018;34:613–620. doi: 10.1007/s00383-018-4264-y.
    1. Criss C.N., Musili N., Matusko N., Baker S., Geiger J.D., Kunisaki S.M. Asymptomatic congenital lung malformations: Is nonoperative management a viable alternative? J. Pediatr. Surg. 2018;53:1092–1097. doi: 10.1016/j.jpedsurg.2018.02.065.
    1. Kapralik J., Wayne C., Chan E., Nasr A. Surgical versus conservative management of congenital pulmonary airway malformation in children: A systematic review and meta-analysis. J. Pediatr. Surg. 2016;51:508–512. doi: 10.1016/j.jpedsurg.2015.11.022.
    1. Tocchioni F., Lombardi E., Ghionzoli M., Ciardini E., Noccioli B., Messineo A. Long-term lung function in children following lobectomy for congenital lung malformation. J. Pediatr. Surg. 2017;52:1891–1897. doi: 10.1016/j.jpedsurg.2017.08.059.
    1. Spoel M., van de Ven K.P., Tiddens H.A.W.M., Hop W.C.J., Wijnen R.M.H., Tibboel D., Ijsselstijn H. Lung function of infants with congenital lung lesions in the first year of life. Neonatology. 2013;103:60–66. doi: 10.1159/000342224.
    1. Hijkoop A., van Schoonhoven M.M., van Rosmalen J., Tibboel D., van der Cammen-van Zijp M.H.M., Pijnenburg M.W., Cohen-Overbeek T.E., Schnater J.M., IJsselstijn H. Lung function, exercise tolerance, and physical growth of children with congenital lung malformations at 8 years of age. Pediatr. Pulmonol. 2019;54:1326–1334. doi: 10.1002/ppul.24345.
    1. Naito Y., Beres A., Lapidus-Krol E., Ratjen F., Langer J.C. Does earlier lobectomy result in better long-term pulmonary function in children with congenital lung anomalies? A prospective study. J. Pediatr. Surg. 2012;47:852–856. doi: 10.1016/j.jpedsurg.2012.01.037.
    1. Dincel A., Yilmaz Yegit C., Ergenekon A.P., Erdem Eralp E., Gokdemir Y., Kiyan G., Karadag B. Long-term respiratory outcomes of post-op congenital lung malformations. Pediatr. Int. 2020;63:704–709. doi: 10.1111/ped.14488.
    1. Calzolari F., Braguglia A., Valfrè L., Dotta A., Bagolan P., Morini F. Outcome of infants operated on for congenital pulmonary malformations. Pediatr. Pulmonol. 2016;51:1367–1372. doi: 10.1002/ppul.23472.
    1. Pinter A., Kalman A., Karsza L., Verebely T., Szemledy F. Long-term outcome of congenital cystic adenomatoid malformation. Pediatr. Surg. Int. 1999;15:332–335. doi: 10.1007/s003830050593.
    1. Williamson P.R., Altman D.G., Bagley H., Barnes K.L., Blazeby J.M., Brookes S.T., Clarke M., Gargon E., Gorst S., Harman N., et al. The COMET Handbook: Version 1.0. Trials. 2017;18:280. doi: 10.1186/s13063-017-1978-4.
    1. Knaapen M., Hall N.J., van der Lee J.H., Butcher N.J., Offringa M., Van Heurn E.W.E., Bakx R., Gorter R.R. Establishing a core outcome set for treatment of uncomplicated appendicitis in children: Study protocol for an international Delphi survey. BMJ Open. 2019;9:e028861. doi: 10.1136/bmjopen-2018-028861.
    1. Sherratt F.C., Eaton S., Walker E., Beasant L., Blazeby J.M., Young B., Crawley E., Wood W.W., Hall N.J. Development of a core outcome set to determine the overall treatment success of acute uncomplicated appendicitis in children: A study protocol. BMJ Paediatr Open. 2017;1:e000151. doi: 10.1136/bmjpo-2017-000151.
    1. Blackwood B., Ringrow S., Clarke M., Marshall J.C., Connolly B., Rose L., McAuley D.F. A Core Outcome Set for Critical Care Ventilation Trials. Crit. Care Med. 2019;47:1324–1331. doi: 10.1097/CCM.0000000000003904.
    1. Hermelijn S., Kersten C., Mullassery D., Muthialu N., Cobanoglu N., Gartner S., Bagolan P., Mesas Burgos C., Sgro A., Heyman S., et al. Development of a core outcome set for congenital pulmonary airway malformations: Study protocol of an international Delphi survey. BMJ Open. 2021;11:e044544. doi: 10.1136/bmjopen-2020-044544.
    1. Eyes D. Welphi-Home. [(accessed on 16 February 2021)]. Available online: .
    1. de Magalhães S.T. The European Union’s General Data Protection Regulation (GDPR) [(accessed on 16 February 2021)]. Available online: .
    1. Sinha I.P., Smyth R.L., Williamson P.R. Using the Delphi technique to determine which outcomes to measure in clinical trials: Recommendations for the future based on a systematic review of existing studies. PLoS Med. 2011;8:e1000393. doi: 10.1371/journal.pmed.1000393.
    1. Sauvat F., Michel J.L., Benachi A., Emond S., Revillon Y. Management of asymptomatic neonatal cystic adenomatoid malformations. J. Pediatr. Surg. 2003;38:548–552. doi: 10.1053/jpsu.2003.50119.
    1. Davenport M., Warne S.A., Cacciaguerra S., Patel S., Greenough A., Nicolaides K. Current outcome of antenally diagnosed cystic lung disease. J. Pediatr. Surg. 2004;39:549–556. doi: 10.1016/j.jpedsurg.2003.12.021.
    1. Miller J.A., Corteville J.E., Langer J.C. Congenital cystic adenomatoid malformation in the fetus: Natural history and predictors of outcome. J. Pediatr. Surg. 1996;31:805–808. doi: 10.1016/S0022-3468(96)90138-4.
    1. Bunduki V., Ruano R., da Silva M.M., Miguelez J., Miyadahira S., Maksoud J.G., Zugaib M. Prognostic factors associated with congenital cystic adenomatoid malformation of the lung. Prenat. Diagn. 2000;20:459–464. doi: 10.1002/1097-0223(200006)20:6<459::AID-PD851>;2-F.
    1. Mallmann M.R., Graham V., Rösing B., Gottschalk I., Müller A., Gembruch U., Geipel A., Berg C. Thoracoamniotic Shunting for Fetal Hydrothorax: Predictors of Intrauterine Course and Postnatal Outcome. Fetal Diagn. Ther. 2017;41:58–65. doi: 10.1159/000446110.
    1. Shulman R., Sparks T.N., Gosnell K., Blat C., Norton M.E., Lee H., Gonzalez-Velez J., Goldstein R.B. Fetal Congenital Pulmonary Airway Malformation: The Role of an Objective Measurement of Cardiomediastinal Shift. Am. J. Perinatol. 2019;36:225–232.
    1. Aziz D., Langer J.C., Tuuha S.E., Ryan G., Ein S.H., Kim P.C. Perinatally diagnosed asymptomatic congenital cystic adenomatoid malformation: To resect or not? J. Pediatr. Surg. 2004;39:329–334. doi: 10.1016/j.jpedsurg.2003.11.021.
    1. Hermelijn S.M., Dragt O.V., Bosch J.J., Hijkoop A., Riera L., Ciet P., Wijnen R.M.H., Schnater J.M., Tiddens H. Congenital lung abnormality quantification by computed tomography: The CLAQ method. Pediatr. Pulmonol. 2020;55:3152–3161. doi: 10.1002/ppul.25032.
    1. Ryu J.H., Tian X., Baqir M., Xu K. Diffuse cystic lung diseases. Front. Med. 2013;7:316–327. doi: 10.1007/s11684-013-0269-z.
    1. Priest J.R., Williams G.M., Hill D.A., Dehner L.P., Jaffé A. Pulmonary cysts in early childhood and the risk of malignancy. Pediatr. Pulmonol. 2009;44:14–30. doi: 10.1002/ppul.20917.
    1. Nasr A., Himidan S., Pastor A.C., Taylor G., Kim P.C. Is congenital cystic adenomatoid malformation a premalignant lesion for pleuropulmonary blastoma? J. Pediatr. Surg. 2010;45:1086–1089. doi: 10.1016/j.jpedsurg.2010.02.067.
    1. Oliveira C., Himidan S., Pastor A.C., Nasr A., Manson D., Taylor G., Yanchar N.L., Brisseau G., Kim P.C. Discriminating preoperative features of pleuropulmonary blastomas (PPB) from congenital cystic adenomatoid malformations (CCAM): A retrospective, age-matched study. Eur. J. Pediatr. Surg. 2011;21:2–7. doi: 10.1055/s-0030-1267923.
    1. Leblanc C., Baron M., Desselas E., Phan M.H., Rybak A., Thouvenin G., Lauby C., Irtan S. Congenital pulmonary airway malformations: State-of-the-art review for pediatrician′s use. Eur. J. Pediatr. 2017;176:1559–1571. doi: 10.1007/s00431-017-3032-7.
    1. Lee A., Davies A., Young A.E. Systematic review of international Delphi surveys for core outcome set development: Representation of international patients. BMJ Open. 2020;10:e040223. doi: 10.1136/bmjopen-2020-040223.

Source: PubMed

Sponsors et collaborateurs

Les conditions médicales

Interventions en matière de drogue

3
S'abonner