Review of the Paediatric Pleuropulmonary Blastoma French Series (PPB)

Pleuropulmonary blastoma is a rare embryonic malignant tumour that remains the most frequent malignant tumour of the lung in childhood. The International Pleuro pulmonary Blastoma Registry (IPPB) found only 220 cases in 2009 and 350 in 2015. In France, 20 cases were identified in 2009.

Three histologies are described: type 1 purely cystic, type 2 combined and type 3 solid. Median age at diagnostic is 12 months, 35 months and 41 months respectively. Evolution is possible from type 1 to type 2 or 3 in 10% of the cases.

Since 2009, DICER 1 mutations research is proposed systematically to all families.

PPB symptoms are usually non-specific. Diagnostic is evoked when imaging work up shows bubbles or solid lesions, and confirmed by pathological analysis. However the diagnosis can be difficult because of the proximity with congenital cystic adenomatoid malformation.

The French society of paediatric oncology recommends surgery at first instance. PPB type 1 remains a problem because some are still misdiagnosed as CCAM, a benign lesion. Chemotherapy depends on the PPB type and the quality of the resection. There is a real interest to analyse the French series.

The prognosis of type 2 and 3 is low with a 5 years survival rate of 45-60%, whereas type 1 survival rate is 91%. The French experience reports a 100% survival rate in type 1 and 48% in type 2 and 3. Other prognostic factors are initial size of the tumour, extra pulmonary invasion and quality of surgery.

Early local relapses are possible and late ones concern more often type 2 and 3 with more cerebral metastasis.

In 2009, the french cases were collected, but no update has been performed since. The aim of this retrospective review of the cases since 2000, is to audit the care of PPB patients in France and update the French rare tumour database.

Evoking PPB diagnosis is difficult when imaging shows a neonatal cystic lesion. There are no radiologic criteria in the literature that differentiate congenital pulmonary cystic lesion and PPB type 1. Radiological presentation is however overlapping. Another aim of this study will be to look for a predictive sign of type 1 PPB.

Study Overview

• Status: Completed
• Conditions: Pleuropulmonary Blastoma
• Intervention / Treatment: Other: PPB

Detailed Description

Multicentre retrospective study

• Study Type: Observational
• Enrollment (Actual): 41

Contacts and Locations

Study Locations

• France
  • Angers, France, 49000
    • Angers University Hospital
  • Bordeaux, France, 33000
    • Bordeaux university hospital
  • Caen, France, 14000
    • CAEN university Hospital
  • Grenoble, France, 38000
    • Grenoble University Hospital
  • Le Kremlin-Bicêtre, France, 94000
    • Kremlin-Bicêtre Hospital
  • Lille, France, 59000
    • Lille University Hospital
  • Lyon, France, 69000
    • Lyon Universty Hospital
  • Marseille, France, 13000
    • Marseille University Hospital
  • Montpellier, France, 34000
    • Montpellier University Hospital
  • Nantes, France, 44000
    • Nantes university hospital
  • Nice, France, 06000
    • Nice University Hospital
  • Paris, France, 75000
    • Necher Hospital
  • Poitiers, France, 86000
    • Poitiers university hospital
  • Saint-Cloud, France, 92000
    • Curie Institute
  • Saint-Paul, France, 97000
    • Réunion University Hospital
  • Toulouse, France, 31000
    • Toulouse University Hospital
  • Tours, France, 37000
    • Tours university Hospital
  • Villejuif, France, 94000
    • Gustave Roussy Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients born between 01/01/2000 and 01/01/2016, followed up for PPB, treated in a French department of paediatric oncology or paediatric surgery.

Description

Inclusion Criteria:

• Patients born between 01/01/2000 and 01/01/2016 ;
• Followed up for PPB
• Treated in a French department of paediatric oncology or paediatric surgery
• Study agreement

Exclusion Criteria:

• Part of the care out of France
• Study disagreement

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pleuropulmonary Blastoma; Patients born between 01/01/2000 and 01/01/2016 ;; Followed up for PPB; Treated in a French department of paediatric oncology or paediatric surgery; Study agreement	Other: PPB; Global current care

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival		Within 5 years from the diagnosis
Progression free survival	Characterize the evolution and the global care of PPB in the French series	Within 5 years from the diagnosis

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
State of PPB surgical care		Within 5 years from the diagnosis
Radiology sign	Identification of a type 1 PPB predictive radiological sign	Within 5 years from the diagnosis

Collaborators and Investigators

• Sponsor: Rennes University Hospital
• Investigators: Principal Investigator: Alexis ARNAUD, MD, Rennes University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 9, 2017
• Primary Completion (Actual): February 22, 2018
• Study Completion (Actual): July 9, 2018

Study Registration Dates

• First Submitted: January 16, 2017
• First Submitted That Met QC Criteria: February 3, 2017
• First Posted (Estimate): February 7, 2017

Study Record Updates

• Last Update Posted (Actual): August 12, 2020
• Last Update Submitted That Met QC Criteria: August 11, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Respiratory Tract Neoplasms; Thoracic Neoplasms; Lung Neoplasms; Neoplasms, Complex and Mixed; Pulmonary Blastoma
• Other Study ID Numbers: 35RC15_3009_PPB; 15.18 (Other Identifier: Comité d'Ethique Régional de Rennes); 15.468 (Other Identifier: CCTIRS); 915465 (Other Identifier: CNIL)