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Eur J Cancer. 2016 Jun;60:69-82. doi: 10.1016/j.ejca.2016.02.027. Epub 2016 Apr 13.

Outcome of extracranial malignant rhabdoid tumours in children registered in the European Paediatric Soft Tissue Sarcoma Study Group Non-Rhabdomyosarcoma Soft Tissue Sarcoma 2005 Study-EpSSG NRSTS 2005.

Author information

1
Paediatric Oncology, Royal Manchester Children's Hospital, Manchester, UK. Electronic address: Bernadette.brennan@cmft.nhs.uk.
2
Clinical Trials and Biostatistics Unit, IRCCS Istituto Oncologico Veneto, Padova, Italy.
3
Pediatric, Adolescent, Young Adult Department, Institut Curie, Paris, France.
4
Department of Diagnostic Paediatric Histopathology, Royal Manchester Children's Hospital, Manchester, UK.
5
Department of Pediatric Oncology, University Children's Hospital, Brno, Czech Republic.
6
Department of Pediatrics, CHC-Clinique Esperance, Montegnée, Belgium.
7
Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
8
Pediatric Hematology and Oncology Division, Padova University, Padova, Italy.
9
Fondazione IRCCS Istituto Nazionale Tumori Milano, Milan, Italy.

Abstract

BACKGROUND:

Extracranial malignant rhabdoid tumours (MRT) are rare lethal childhood cancers that often occur in infants and have a characteristic genetic mutation in the SMARCB1 gene. The European Paediatric Soft Tissue Sarcoma Study Group (EpSSG) conducted a multinational prospective study of registered cases of extracranial MRT to test an intensive multimodal approach of treatment for children with newly diagnosed extracranial MRT.

METHODS:

Between December 2005 and June 2014, we prospectively registered 100 patients from 12 countries with a diagnosis of MRT tumour at an extracranial site on the EpSSG Non-Rhabdomyosarcoma Soft Tissue Sarcoma 2005 Study (NRSTS 2005). They were all treated on a standard multimodal protocol of surgery, radiotherapy, and chemotherapy over 30 weeks as follows: vincristine, cyclophosphamide, and doxorubicin (VDCy) at weeks 1, 10, 13, 22, and 28; vincristine was also given alone on weeks 2, 3, 11, 12, 14, 15, 23, 24, 29, and 30. Cyclophosphamide, carboplatin, and etoposide (Cy*CE) was given at weeks 4, 7, 16, 19, and 25. Radiotherapy was recommended for all primary tumour sites and all sites of metastatic disease.

RESULTS:

Forty-three patients completed the protocol treatment. Median follow-up for alive patients of the complete cohort was 44.6 months (range 11.5-84.6). For the whole cohort, the 3-year event-free survival (EFS) was 32.3% (95% confidence interval [CI] 23.2-41.6%) with a 3-year overall survival (OS) of 38.4% (95% CI 28.8-47.9%). For localised disease, the 4-year EFS was 39.3% (95% CI 28.2-50.1%) with a 4-year OS of 40.1% (95% CI 28.4-51.5%). For metastatic disease, the 2-year EFS was 8.7% (95% CI 1.5-24.2%) with a 2-year OS of 13.0% (95% CI 3.3-29.7%). Multivariable analysis disclosed that all patients ≤1 year of age were associated with at higher risk of death (hazard ratio [HR]: 2.6; 95% CI 1.0-6.8; p-value = 0.0094). Risk of death was also related with gender in metastatic patients (HR for males: 2.9, 95% CI 1.0-8.0; p-value = 0.0077).

CONCLUSIONS:

The EpSSG NRSTS 2005 protocol of intensive therapy can be delivered to extracranial MRT patients, with a possible improvement in outcome. The outcome, however, remains poor for patients who progress or with metastatic disease.

KEYWORDS:

Malignant rhabdoid tumour; Paediatric; Prognostic factors; Prospective registry; Survival

PMID:
27082136
DOI:
10.1016/j.ejca.2016.02.027
[Indexed for MEDLINE]

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