NCT03044834: PPB

Review of the Paediatric Pleuropulmonary Blastoma French Series

Completed Last updated 12 August 2020

What this trial tests

trial testing PPB in Pleuropulmonary Blastoma in 41 participants. Completed in 9 July 2018.

Timeline

9 January 2017

Primary endpoint
22 February 2018

9 July 2018

Quick facts

Lead sponsor	Rennes University Hospital
Status	Completed
Study type	OBSERVATIONAL
Enrollment	41
Start date	9 January 2017
Primary completion	22 February 2018
Estimated completion	9 July 2018
Sites	18 locations across France

Drugs / interventions tested

• PPB

Conditions studied

• Pleuropulmonary Blastoma — all drugs for Pleuropulmonary Blastoma →

Sponsor

Rennes University Hospital

Who can join

Adults 1 to 17, any sex, with Pleuropulmonary Blastoma. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Type II pleuropulmonary blastoma in a fetus: case report of a rare mesenchymal mediastinal tumor and literature review.
   Moldovan VT, Sajin M, Habago SD, Ali L. · · 2024 · PMID 38919847 · DOI 10.22551/2024.43.1102.10286