Review of the Paediatric Pleuropulmonary Blastoma French Series

NCT03044834 | Completed

• 4 other identifiers: 35RC15_3009_PPB15.1815.468915465
• Study Type: observational
• Target: 41
• Locations: 1 country
• Sites: 18

Brief Summary

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.

Conditions

Pleuropulmonary Blastoma

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• State of PPB surgical care

  Within 5 years from the diagnosis

• Radiology sign

  Within 5 years from the diagnosis

Study Arms (1)

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

Interventions

PPB OTHER

Global current care

Pleuropulmonary Blastoma

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• 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

You may not qualify if:

• Part of the care out of France
• Study disagreement

Sponsors & Collaborators

• Rennes University Hospital: lead

Study Sites (18)

Angers University Hospital

Angers, 49000, France

Location

Bordeaux University Hospital

Bordeaux, 33000, France

Location

Caen University Hospital

Caen, 14000, France

Location

Grenoble University Hospital

Grenoble, 38000, France

Location

Kremlin-Bicêtre Hospital

Le Kremlin-Bicêtre, 94000, France

Location

Lille University Hospital

Lille, 59000, France

Location

Lyon Universty Hospital

Lyon, 69000, France

Location

Marseille University Hospital

Marseille, 13000, France

Location

Montpellier University Hospital

Montpellier, 34000, France

Location

Nantes University Hospital

Nantes, 44000, France

Location

Nice University Hospital

Nice, 06000, France

Location

Necher Hospital

Paris, 75000, France

Location

Poitiers University Hospital

Poitiers, 86000, France

Location

Curie Institute

Saint-Cloud, 92000, France

Location

Réunion University Hospital

Saint-Paul, 97000, France

Location

Toulouse University Hospital

Toulouse, 31000, France

Location

Tours University Hospital

Tours, 37000, France

Location

Gustave Roussy Institute

Villejuif, 94000, France

Location

MeSH Terms

Conditions

Pleuropulmonary blastoma

Study Officials

• Alexis ARNAUD, MD

  Rennes University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 16, 2017
• First Posted: February 7, 2017
• Study Start: January 9, 2017
• Primary Completion: February 22, 2018
• Study Completion: July 9, 2018
• Last Updated: August 12, 2020

Record last verified: 2020-08

Locations

FR (18)