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Inequalities in diagnosis and registration of pediatric very rare tumors: a European study on pleuropulmonary blastoma

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Abstract

Very rare tumors (VRTs) account for up to 11% of childhood cancers. Dedicated national groups and registries only exist in some European countries. Pleuropulmonary blastoma (PPB) is a very rare intrathoracic pediatric tumor with a potentially severe prognosis. Due to its rarity, it sometimes goes unrecognized. We investigated PPB diagnostic capability and possible correlations between diagnostic performance and VRT-dedicated activities. The number of cases of PPB registered between 2000 and 2014 at pediatric oncology centers in Europe was compared with the number of expected cases. Data sources included VRT registries, population-based cancer registries, and hospital registries. Data were obtained for 25 countries, grouped into 4 geographical regions. The expected cases were 111, and the observed cases were 129. The observed-to-expected ratio was 1.86 for Northern Europe, 1.33 for Southern Europe, 1.22 for Central Europe, and 0.65 for Eastern Europe. More cases than expected were registered in all countries with an official VRT registry.

Conclusion: The number of cases observed is consistent with expectations, but disparities exist across Europe. Difficulties in diagnosing PPB emerged in most Eastern countries. The incidence rate of PPB may be underestimated. The creation of VRT-dedicated groups and a European Registry for VRTs could help to reduce inequalities.

What is Known:

• Very rare pediatric tumors are often not recognized, despite representing almost 11% of childhood cancers .

• Pleuropulmonary blastoma is a rare pediatric tumor with a poor prognosis.

What is New:

• The ability to diagnose and register pleuropulmonary blastoma varies in Europe.Registries dedicated to very rare pediatric tumors improve the diagnostic rates.

• The incidence rate of pleuropulmonary blastoma may currently be underestimated

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Abbreviations

PPB:

Pleuropulmonary blastoma

VRT:

Very rare tumor

CPAM:

Congenital pulmonary airway malformation

PARTNER:

Pediatric Rare Tumors Network–European Registry

References

  1. Gatta G, van der Zwan JM, Casali PG, Siesling S, Dei Tos AP, Kunkler I, Otter R, Licitra L, Mallone S, Tavilla A, Trama A, Capocaccia R, RARECARE working group (2011) Rare cancers are not so rare: the rare cancer burden in Europe. Eur J Cancer 47(17):2493–2511. https://doi.org/10.1016/j.ejca.2011.08.008

    Article  PubMed  Google Scholar 

  2. Steliarova-Foucher E, Colombet M, Ries LAG, Moreno F, Dolya A, Bray F, Hesseling P, Shin HY, Stiller CA (2017) International incidence of childhood cancer, 2011-10: a population-based registry study. Lancet Oncol 18(6):719–731. https://doi.org/10.1016/S1470-2045(17)30186-9

    Article  PubMed  PubMed Central  Google Scholar 

  3. Ferrari A, Bisogno G, De Salvo GL, Indolfi P, Perilongo G, Cecchetto G, Italian Study on Rare Tumours in Paediatric Age (TREP), Associazione Italiana Ematologia Oncologia Pediatrica (AIEOP) (2007) The challenge of very rare tumors in childhood: the Italian TREP project. Eur J Cancer 43:654–659. https://doi.org/10.1016/j.ejca.2006.08.028

    Article  PubMed  Google Scholar 

  4. Ferrari A, Brecht IB, Gatta G, Schneider DT, Orbach D, Cecchetto G et al (2019) Defining and listing very rare cancers of paediatric age: consensus of the joint action on rare cancers in cooperation with the European cooperative study group for pediatric rare tumors. Eur J Cancer 110:120–126. https://doi.org/10.1016/j.ejca.2018.12.031

    Article  PubMed  Google Scholar 

  5. Brecht IB, Bremensdorfer C, Schneider DT, Frühwald MC, Offenmüller S, Mertens R, Vorwerk P, Koscielniak E, Bielack SS, Benesch M, Hero B, Graf N, von Schweinitz D, Kaatsch P (2014) Rare malignant pediatric tumors registered in the German Childhood Cancer Registry 2001-2010. Pediatr Blood Cancer 61:1202–1209. https://doi.org/10.1002/pbc.2499

    Article  PubMed  Google Scholar 

  6. Balcerska A, Godziński J, Bień E, Stachowicz-Stencel T, Sulka W, Rapała M et al (2004) Rare tumors - are they really rare in the Polish children population? Przegl Lek 61(Suppl 2):57–61

    PubMed  Google Scholar 

  7. Réguerre Y, Lacour B, André N, Claude L, Hameury F, Lavrand F, Kalfa N, Peuchmaur M, Orbach D (2010) Epidemiology and management of rare pediatric tumors within the framework of the French Society for Children Cancer. Bull Cancer 97(9):1041–1045. https://doi.org/10.1684/bdc.2010.1169

    Article  PubMed  Google Scholar 

  8. Bisogno G, Ferrari A, Bien E, Brecht IB, Brennan B, Cecchetto G, Godzinski J, Orbach D, Reguerre Y, Stachowicz-Stencel T, Schneider DT (2012) Rare cancers in children - the EXPeRT initiative: a report from the European cooperative study group on pediatric rare tumors. Klin Padiatr 224:416–420

    Article  CAS  PubMed  Google Scholar 

  9. ExPO-r-Net (2008) ExPO-r-Net - European Expert Paediatric Oncology Reference Network for Diagnostics and Treatment. [online] Available at: https://www.expornet.eu/ [Accessed 4 Dec. 2018]

  10. Manivel JC, Priest JR, Watterson J, Steiner M, Woods WG, Wick MR, Dehner LP (1988) Pleuropulmonary blastoma the so-called pulmonary blastoma of childhood. Cancer 62:1516–1526. https://doi.org/10.1002/1097-0142(19881015)62:8<1516::aid-cncr2820620812>3.0.co;2-3

    Article  CAS  PubMed  Google Scholar 

  11. Messinger YH, Stewart DR, Priest JR, Williams GM, Harris AK, Schultz KA, Yang J, Doros L, Rosenberg PS, Hill DA, Dehner LP (2015) Pleuropulmonary blastoma: a report on 350 central pathology-confirmed pleuropulmonary blastoma cases by the international pleuropulmonary blastoma registry. Cancer 121(2):276–285. https://doi.org/10.1002/cncr.29032

    Article  PubMed  Google Scholar 

  12. Bisogno G, Brennan B, Orbach D, Stachowicz-Stencel T, Cecchetto G, Indolfi P, Bien E, Ferrari A, Dommange-Romero F (2014) Treatment and prognostic factors in pleuropulmonary blastoma: an EXPeRT report. Eur J Cancer 50:178–184. https://doi.org/10.1016/j.ejca.2013.08.015

    Article  PubMed  Google Scholar 

  13. Schultz KAP, Williams GM, Kamihara J, Stewart DR, Harris AK, Bauer AJ, Turner J, Shah R, Schneider K, Schneider KW, Carr AG, Harney LA, Baldinger S, Frazier AL, Orbach D, Schneider DT, Malkin D, Dehner LP, Messinger YH, Hill DA (2018) DICER1 and associated conditions: identification of at-risk individuals and recommended surveillance strategies. Clin Cancer Res 24(10):2251–2261. https://doi.org/10.1158/1078-0432.CCR-17-3089

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  14. Feinberg A, Hall NJ, Williams GM, , Schultz KA, Miniati D, Hill DA, Dehner LP, Messinger YH, Langer JC (2016) Can congenital pulmonary airway malformation be distinguished from type I pleuropulmonary blastoma based on clinical and radiological features? J Pediatr Surg 51(1):33–37. https://doi.org/10.1016/j.jpedsurg.2015.10.019

    Article  PubMed  Google Scholar 

  15. Pai S, Eng HL, Lee SY, Hsaio CC, Huang WT, Huang SC, Hill DA, Dehner LP, Priest JR (2007) Correction: pleuropulmonary blastoma, not rhabdomyosarcoma in a congenital lung cyst. Pediatr Blood Cancer 48:370–371. https://doi.org/10.1002/pbc.20965

    Article  PubMed  Google Scholar 

  16. Surveillance, Epidemiology and End Results (SEER) Program Populations (1992-2007) (www.seer.cancer.gov/popdata). National Cancer Institute, DCCPS, Surveillance Research Program, Cancer Statistics Branch, released 2010

  17. Pastore G, De Salvo GL, Bisogno G, Dama E, Inserra A, Cecchetto G, Ferrari A, TREP Group, CSD of Epidemiology Biostatistics, AIEOP (2009) Evaluating access to pediatric cancer care centers of children and adolescents with rare tumors in Italy: the TREP project. Pediatr Blood Cancer 53(2):152–155. https://doi.org/10.1002/pbc.22049

    Article  PubMed  Google Scholar 

  18. Sparber-Sauer M, Seitz G, Kirsch S, Vokuhl C, Leuschner I, Dantonello TM, Scheer M, von Kalle T, Ljungman G, Bielack SS, Klingebiel T, Fuchs J, Koscielniak E, CWS Study Group (2017) The impact of local control in the treatment of type II/III pleuropulmonary blastoma. Experience of the Cooperative Weichteilsarkom Studiengruppe (CWS). J Surg Oncol 115(2):164–172. https://doi.org/10.1002/jso.24416

    Article  PubMed  Google Scholar 

  19. Schneider D, Brecht I, Ferrari A (2012) Rare tumors in children and adolescents. Springer, New York, pp 95–108

    Book  Google Scholar 

  20. nopho.org [homepage on the Internet] Childhood cancer in the Nordic countries. Report on epidemiologic and therapeutic results from registries and working groups. NOPHO Annual Report 2016. Available from: www.nopho.org/annualreport [Accessed 01.05.2019]

  21. Steliarova-Foucher E, Colombet M, Ries LAG, Moreno F, Dolya A, Bray F, Hesseling P, Shin HY, Stiller CA, IICC-3 contributors (2017) International incidence of childhood cancer, 2001-10: a population-based registry study. Lancet Oncol 18(6):719–731. https://doi.org/10.1016/S1470-2045(17)30186-9

    Article  PubMed  PubMed Central  Google Scholar 

  22. Gatta G, Botta L, Rossi S, Aareleid T, Bielska-Lasota M, Clavel J, Dimitrova N, Jakab Z, Kaatsch P, Lacour B, Mallone S, Marcos-Gragera R, Minicozzi P, Sánchez-Pérez MJ, Sant M, Santaquilani M, Stiller C, Tavilla A, Trama A, Visser O, Peris-Bonet R, EUROCARE Working Group (2014) Childhood cancer survival in Europe 1999–2007: results of EUROCARE-5—a population-based study. Lancet Oncol 15:35–47. https://doi.org/10.1016/S1470-2045(13)70548-5

    Article  PubMed  Google Scholar 

  23. Panagopoulou P, Georgakis MK, Baka M, Moschovi M, Papadakis V, Polychronopoulou S, Kourti M, Hatzipantelis E, Stiakaki E, Dana H, Tragiannidis A, Bouka E, Antunes L, Bastos J, Coza D, Demetriou A, Agius D, Eser S, Gheorghiu R, Šekerija M, Trojanowski M, Žagar T, Zborovskaya A, Ryzhov A, Dessypris N, Morgenstern D, Petridou ET (2018) Persisting inequalitis in survival patterns of childhood neuroblastoma in Southern and Eastern Europe and the effect of socio-economic development compared with those of the US. Eur J Cancer 96:44–53. https://doi.org/10.1016/j.ejca.2018.03.003

    Article  PubMed  Google Scholar 

  24. Gatta G, Peris-Bonet R, Visser O, Stiller C, Marcos-Gragera R, Sánchez MJ, Lacour B, Kaatsch P, Berrino F, Rutkowski S, Botta L, EUROCARE-5 Working Group (2017) Geographical variability in survival of European children with central nervous system tumors. Eur J Cancer 82:137–148. https://doi.org/10.1016/j.ejca.2017.05.028

    Article  CAS  PubMed  Google Scholar 

  25. European Commission, Chafea Health Programmes Database; ERN-Paedcan Partner: Pediatric Rare tumors Network – European Registry [PARTNER]; project description available at https://webgate.ec.europa.eu/chafea_pdb/health/projects/777336/summary [accessed 01.05.2019]

Download references

Acknowledgments

The authors wish to thank Brigitte Lacour, Alexandra Kolenova, Maja Cesen Mazic, Christine Devalck, Catherine Rechnitzer, Heidi Glosli, Dobrin Konstantinov, Jelena Rascon, Jelena Roganovic, and Zsuzsanna Jakab for their contribution to data collection in their respective countries. We also extend our sincere thanks to Beatrice Coppadoro and Ilaria Zanetti for their help with data analysis, Serena Mancini for managing the PARTNER project, and Silvia Sorbara for managing the ExPO-r-Net project.

Funding

This work stems from the activities of the ExPO-r-Net project (Project 2013 12 07-CUP I94G 13,000,010,002) and was undertaken as part of the “777,336/PARTNER” project, which received funding from the European Union’s Health Programme (2014–2020). The content of this paper represents the views of the author only and is his/her sole responsibility; it can not be considered to reflect the views of the European Commission and/or the Consumers, Health, Agriculture and Food Executive Agency or any other body of the European Union. The European Commission and the Agency do not accept any responsibility for use that may be made of the information it contains. The Italian TREP Registry is supported by the Fondazione Celeghin. The French Fracture database is supported by the “Enfant, cancers et santé” association. The German (SoTiSaR), registry was supported by the Deutsche Kinderkrebsstiftung, Bonn, Germany, grant no. A2007/13DKS2009.08, and by the Foerderkreis Krebskranke Kinder Stuttgart, Germany.

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Authors and Affiliations

  1. Pediatric Hematology and Oncology Division, Department of Women’s and Children’s Health, Padua University Hospital, Padua, Italy

    V. Grigoletto, A. Tagarelli & G. Bisogno

  2. Klinikum Stuttgart-Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pediatrics 5 (Oncology, Hematology, Immunology), Stuttgart, Germany

    M. Sparber-Sauer & E. Koscielniak

  3. Department of Pediatric Hematology and Oncology, Children’s Hospital, Tuebingen, Germany

    E. Koscielniak

  4. SIREDO Oncology Center (Care, Innovation and Research for Children, Adolescents and Young Adults with Cancer), Institut Curie, Université PSL, Paris, France

    D. Orbach

  5. Pediatric Hematology-Oncology-Immunology Unit, Angers University Hospital, Angers, France

    M. Duplan

  6. Pediatric Hematology and Oncology Unit, Department of Pediatrics, Medical University of Gdansk, Gdańsk, Poland

    T. Stachowicz-Stencel & E. Bien

  7. Pediatric Oncology and Hematology Unit, Cruces University Hospital, Barakaldo, Spain

    R. López-Almaraz

  8. Pediatric Hematology-Oncology Unit, Kaplan Medical Center, Rehovot, Israel

    T. Ben-Ami

  9. Pediatric Oncology Unit, Aglaia Kyriakou Children’s Hospital, Athens, Greece

    A. Pourtsidis

  10. Pediatric Hematology and Oncology Unit, The Queen Silvia Children’s Hospital, Sahlgrenska University Hospital, Goteborg, Sweden

    G. Österlundh

  11. Department of Pediatric Oncology, Faculty of Medicine, Masaryk University, Brno, Czech Republic

    P. Mazanek

  12. Pediatric Oncology Unit, Princess Maxima Center for Pediatric Oncology, Utrechtx, Netherlands

    J. Zsiros

  13. Pediatric Hematology and Oncology Unit, Centro Hospitalar de Sao Joao, Porto, Portugal

    N. Farinha

  14. Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy

    A. Ferrari

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  1. V. Grigoletto
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  2. A. Tagarelli
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  3. M. Sparber-Sauer
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  4. E. Koscielniak
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  16. A. Ferrari
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Contributions

Bisogno: Supervision, study design, method, investigation, data curation and analysis, and manuscript revision

Grigoletto and Tagarelli: Study design, investigation, data curation and analysis, manuscript drafting and revision

Sparber-Sauer, Koscielniak, Orbach, Duplan, Stachowicz-Stencel, Bien, Lopez, Ben-Ami, Pourtsidis, Österlundh, Mazanek, Zsiros, Farinha, Ferrari: Study design, investigation, data analysis, and manuscript review

Corresponding author

Correspondence to V. Grigoletto.

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This article contains no studies on human participants or animals performed by any of the authors.

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Informed consent was obtained from all individual participants in the study and their parents.

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Communicated by Peter de Winter

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Grigoletto, V., Tagarelli, A., Sparber-Sauer, M. et al. Inequalities in diagnosis and registration of pediatric very rare tumors: a European study on pleuropulmonary blastoma. Eur J Pediatr 179, 749–756 (2020). https://doi.org/10.1007/s00431-019-03566-7

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