Review of the Paediatric Pleuropulmonary Blastoma French Series

NCT ID: NCT03044834

Last Updated: 2020-08-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

41 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-01-09

Study Completion Date

2018-07-09

Brief Summary

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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

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Multicentre retrospective study

Conditions

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Pleuropulmonary Blastoma

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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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

PPB

Intervention Type OTHER

Global current care

Interventions

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PPB

Global current care

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

1 Year

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Rennes University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Alexis ARNAUD, MD

Role: PRINCIPAL_INVESTIGATOR

Rennes University Hospital

Locations

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Angers University Hospital

Angers, , France

Site Status

Bordeaux University Hospital

Bordeaux, , France

Site Status

Caen University Hospital

Caen, , France

Site Status

Grenoble University Hospital

Grenoble, , France

Site Status

Kremlin-Bicêtre Hospital

Le Kremlin-Bicêtre, , France

Site Status

Lille University Hospital

Lille, , France

Site Status

Lyon Universty Hospital

Lyon, , France

Site Status

Marseille University Hospital

Marseille, , France

Site Status

Montpellier University Hospital

Montpellier, , France

Site Status

Nantes University Hospital

Nantes, , France

Site Status

Nice University Hospital

Nice, , France

Site Status

Necher Hospital

Paris, , France

Site Status

Poitiers University Hospital

Poitiers, , France

Site Status

Curie Institute

Saint-Cloud, , France

Site Status

Réunion University Hospital

Saint-Paul, , France

Site Status

Toulouse University Hospital

Toulouse, , France

Site Status

Tours University Hospital

Tours, , France

Site Status

Gustave Roussy Institute

Villejuif, , France

Site Status

Countries

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France

Other Identifiers

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15.18

Identifier Type: OTHER

Identifier Source: secondary_id

15.468

Identifier Type: OTHER

Identifier Source: secondary_id

915465

Identifier Type: OTHER

Identifier Source: secondary_id

35RC15_3009_PPB

Identifier Type: -

Identifier Source: org_study_id

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