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Management and outcome of children and adolescents with non-medulloblastoma CNS embryonal tumors in Spain: room for improvement in standards of care

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Abstract

Non-medulloblastoma CNS embryonal tumors (former PNET/Pineoblastomas) are aggressive malignancies with poor outcome that have been historically treated with medulloblastoma protocols. The purpose of this study is to present a tumor-specific, real-world data cohort of patients with CNS-PNET/PB to analyze quality indicators that can be implemented to improve the outcome of these patients. Patients 0–21 years with CNS-PNET treated in eight large institutions were included. Baseline characteristics, treatment and outcome [progression-free and overall survival (PFS and OS respectively)] were analyzed. From 2005 to 2014, 43 patients fulfilled entry criteria. Median age at diagnosis was 3.6 years (range 0.0–14.7). Histology was pineoblastoma (9%), ependymoblastoma (5%), ETANTR (7%) and PNET (77%). Median duration of the main symptom was 2 weeks (range 0–12). At diagnosis, 28% presented with metastatic disease. Seventeen different protocols were used on frontline treatment; 44% had gross total resection, 42% craniospinal radiotherapy, 86% chemotherapy, and 33% autologous hematopoietic stem cell transplantation (aHSCT). Median follow-up for survivors was 3.5 years (range 1.7–9.3). 3-year PFS was 31.9% (95% CI 17–47%) and OS 35.1% (95% CI 20–50%). Age, extent of resection and radiotherapy were prognostic of PFS and OS in univariate analysis (p < 0.05). Our series shows a dismal outcome for CNS-PNET, especially when compared to patients included in clinical trials. Establishing a common national strategy, implementing referral circuits and collaboration networks, and incorporating new molecular knowledge into routine clinical practice are accessible measures that can improve the outcome of these patients.

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Abbreviations

aHSCT:

Autologous hematopoietic stem cell transplantation

CNS:

Central nervous system

CT:

Chemotherapy

CTCAE:

Common terminology criteria for adverse events

CI:

Confidence interval

CSI:

Craniospinal irradiation

ETANTR:

Embryonal tumor with abundant neuropil and true rosettes

ETMR:

Embryonal tumor with multilayered rosettes

GTR:

Gross total resection

MRI:

Magnetic resonance imaging

NCI:

National Cancer Institute

OS:

Overall survival

PB:

Pineoblastomas

PNET:

Primitive neuroectodermal tumors

PFS:

Progression free survival

RT:

Radiotherapy

SEHOP:

Spanish society of pediatric hematology and oncology

STR:

Subtotal resection

WHO:

World Health Organization

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Correspondence to Lucas Moreno.

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The authors declare that they have no conflict of interest.

Ethical approval

The study protocol was approved by the Institutional Review Board of Hospital Niño Jesús, and approval for retrospective chart review was sought at all participating hospitals. All procedures performed in this study were in accordance with the declaration of Helsinki (1964) and its later amendments and comparable ethical standards.

Additional information

On behalf of the CNS Tumors Group of the Spanish Society of Pediatric Hematology and Oncology (SEHOP).

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de Rojas, T., Bautista, F., Flores, M. et al. Management and outcome of children and adolescents with non-medulloblastoma CNS embryonal tumors in Spain: room for improvement in standards of care. J Neurooncol 137, 205–213 (2018). https://doi.org/10.1007/s11060-017-2713-4

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  • DOI: https://doi.org/10.1007/s11060-017-2713-4

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