Review of the Paediatric Pleuropulmonary Blastoma French Series

Pleuropulmonary Blastoma Clinical Trial

— PPB  

Official title:

Review of the Paediatric Pleuropulmonary Blastoma French Series

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03044834
• Other study ID #: 35RC15_3009_PPB
• Secondary ID: 15.1815.46891546
• Status: Completed
• Start date: January 9, 2017
• Est. completion date: July 9, 2018

Study information

• Verified date: August 2020
• Source: Rennes University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

Multicentre retrospective study

Recruitment information / eligibility

• Status: Completed
• Enrollment: 41
• Est. completion date: July 9, 2018
• Est. primary completion date: February 22, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Pleuropulmonary Blastoma

Intervention

Other:
• PPB
Global current care

Locations

Country	Name	City	State
France	Angers University Hospital	Angers
France	Bordeaux University Hospital	Bordeaux
France	Caen University Hospital	Caen
France	Grenoble University Hospital	Grenoble
France	Kremlin-Bicêtre Hospital	Le Kremlin-Bicêtre
France	Lille University Hospital	Lille
France	Lyon Universty Hospital	Lyon
France	Marseille University Hospital	Marseille
France	Montpellier University Hospital	Montpellier
France	Nantes University Hospital	Nantes
France	Nice University Hospital	Nice
France	Necher Hospital	Paris
France	Poitiers University Hospital	Poitiers
France	Curie Institute	Saint-Cloud
France	Réunion University Hospital	Saint-Paul
France	Toulouse University Hospital	Toulouse
France	Tours University Hospital	Tours
France	Gustave Roussy Institute	Villejuif

Sponsors (1)

Lead Sponsor	Collaborator
Rennes University Hospital

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall survival		Within 5 years from the diagnosis
Primary	Progression free survival	Characterize the evolution and the global care of PPB in the French series	Within 5 years from the diagnosis
Secondary	State of PPB surgical care		Within 5 years from the diagnosis
Secondary	Radiology sign	Identification of a type 1 PPB predictive radiological sign	Within 5 years from the diagnosis