Clinical trial NCT03044834: Review of the Paediatric Pleuropulmonary Blastoma French Series
Official title: Review of the Paediatric Pleuropulmonary Blastoma French Series
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pleuropulmonary Blastoma
Lead sponsor: Rennes University Hospital
Sponsor class: OTHER
First posted: 2017-02-07
Enrollment: 41
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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.

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.

Detailed description: Multicentre retrospective study

Eligibility criteria: 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

Primary outcomes:
measure: Overall survival
timeFrame: Within 5 years from the diagnosis
measure: Progression free survival
timeFrame: Within 5 years from the diagnosis
Secondary outcomes:
measure: State of PPB surgical care
timeFrame: Within 5 years from the diagnosis
measure: Radiology sign
timeFrame: Within 5 years from the diagnosis
Other outcomes:
Locations:
city: Angers
country: France
facility: Angers University Hospital
city: Bordeaux
country: France
facility: Bordeaux University Hospital
city: Caen
country: France
facility: Caen University Hospital
city: Grenoble
country: France
facility: Grenoble University Hospital
city: Le Kremlin-Bicêtre
country: France
facility: Kremlin-Bicêtre Hospital
city: Lille
country: France
facility: Lille University Hospital
city: Lyon
country: France
facility: Lyon Universty Hospital
city: Marseille
country: France
facility: Marseille University Hospital
city: Montpellier
country: France
facility: Montpellier University Hospital
city: Nantes
country: France
facility: Nantes University Hospital
city: Nice
country: France
facility: Nice University Hospital
city: Paris
country: France
facility: Necher Hospital
city: Poitiers
country: France
facility: Poitiers University Hospital
city: Saint-Cloud
country: France
facility: Curie Institute
city: Saint-Paul
country: France
facility: Réunion University Hospital
city: Toulouse
country: France
facility: Toulouse University Hospital
city: Tours
country: France
facility: Tours University Hospital
city: Villejuif
country: France
facility: Gustave Roussy Institute
Study officials:
affiliation: Rennes University Hospital
name: Alexis ARNAUD, MD
role: PRINCIPAL_INVESTIGATOR
References: