Cover Image

SIOP Ependymoma I: final results, long-term follow-up, and molecular analysis of the trial cohort - a BIOMECA Consortium study

SIOP Ependymoma I was a non-randomised trial assessing event free and overall survival (EFS/OS) of non-metastatic intracranial ependymoma in children aged 3-21 years treated with a staged management strategy. A further aim was to assess the response rate (RR) of subtotally resected (STR) ependymoma...

Full description

Saved in:
Bibliographic Details
Main Authors: Ritzmann, Timothy A. (Author) , Chapman, Rebecca J (Author) , Kilday, John-Paul (Author) , Thorp, Nicola (Author) , Modena, Piergiorgio (Author) , Dineen, Robert A (Author) , Macarthur, Donald (Author) , Mallucci, Conor (Author) , Jaspan, Timothy (Author) , Pajtler, Kristian Wilfried (Author) , Giagnacovo, Marzia (Author) , Jacques, Thomas S (Author) , Paine, Simon M L (Author) , Ellison, David W (Author) , Bouffet, Eric (Author) , Grundy, Richard G (Author)
Format: Article (Journal)
Language:English
Published: 09 January 2022
In: Neuro-Oncology
Year: 2022, Volume: 24, Issue: 6, Pages: 936-948
ISSN:1523-5866
DOI:10.1093/neuonc/noac012
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1093/neuonc/noac012
Get full text
Author Notes:Timothy A. Ritzmann, Rebecca J. Chapman, John-Paul Kilday, Nicola Thorp, Piergiorgio Modena, Robert A. Dineen, Donald Macarthur, Conor Mallucci, Timothy Jaspan, Kristian W. Pajtler, Marzia Giagnacovo, Thomas S. Jacques, Simon M.L. Paine, David W. Ellison, Eric Bouffet and Richard G. Grundy, on behalf of the BIOMECA Consortium
Description
Summary:SIOP Ependymoma I was a non-randomised trial assessing event free and overall survival (EFS/OS) of non-metastatic intracranial ependymoma in children aged 3-21 years treated with a staged management strategy. A further aim was to assess the response rate (RR) of subtotally resected (STR) ependymoma to vincristine, etoposide, and cyclophosphamide (VEC). We report final results with 12-year follow-up and post hoc analyses of recently described biomarkers.Seventy-four participants were eligible. Children with gross total resection (GTR) received radiotherapy, whilst those with STR received VEC before radiotherapy. DNA methylation, 1q, hTERT, ReLA, Tenascin-C, H3K27me3, and pAKT status were evaluated.Five- and ten-year EFS was 49.5% and 46.7%, OS was 69.3% and 60.5%. GTR was achieved in 33/74 (44.6%) and associated with improved EFS (P = .003, HR = 2.6, 95% confidence interval (CI) 1.4-5.1). Grade 3 tumours were associated with worse OS (P = .005, HR = 2.8, 95%CI 1.3-5.8). 1q gain and hTERT expression were associated with poorer EFS (P = .003, HR = 2.70, 95%CI 1.49-6.10 and P = .014, HR = 5.8, 95%CI 1.2-28) and H3K27me3 loss with worse OS (P = .003, HR = 4.6, 95%CI 1.5-13.2). Methylation profiles showed expected patterns. 12 participants with STR did not receive chemotherapy; a protocol violation. However, best chemotherapy RR was 65.5% (19/29, 95%CI 45.7-82.1), exceeding the prespecified 45%.Participants with totally resected ependymoma had the best outcomes. RR of STR to VEC exceeded the pre-specified efficacy criterion. However, cases of inaccurate stratification highlighted the need for rapid central review. 1q gain, H3K27me3 loss, and hTERT expression were all associated with poorer survival outcomes.
Item Description:Gesehen am 08.07.2022
Physical Description:Online Resource
ISSN:1523-5866
DOI:10.1093/neuonc/noac012

Similar Items