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Improved Stratification of Standard Risk Medulloblastoma Patients by Clinical and Biological Prognostic Factors

Lookup NU author(s): Professor Steven CliffordORCiD, Professor David Ellison, Kieran O'Toole


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Event-free survival rates of standard risk medulloblastoma (MB) average 80% in current studies. We aim to improve the current risk stratification of standard MB patients based on clinical risk factors by addition of biological markers. MB patients at age between 3 and 22 years, absence of metastasis and groß total or near groß total surgical excision are considered as standard risk patients and were treated in the prospective randomised HIT-SIOP PNET4 trial by conventional or hyperfractionated radiotherapy between 2001 and 2006. Residual tumor was assessed by radio-imaging, histopathological diagnosis was confirmed and MB subtype determined by microscopy, β-catenin accumulation by immunohistochemistry (IHC), c-MYC or N-MYC gene amplification was tested by fluorescence in situ hybridization (FISH) and semi-quantitative PCR of genomic DNA. Two risk groups were identified in 97 patients from the HIT-group with completely analyzed parameters. Favourable risk group: Twelve patients with β-catenin accumulation and absence of significant postoperative residual tumor, and without anaplastic-large cell histology, c-MYC or N-MYC DNA amplification remained relapse-free (5-year event-free and overall survival, 100%). The EFS/OS of the remaining 85 patients was 70±5.5% and 84.6±4.7%. In addition to the already used clinical factors, this preliminary subgroup analysis suggests that stratification may be improved by molecular markers in standard medulloblastoma patients. The next European standard risk medulloblastoma trial, using biological and clinical factors for treatment stratification, is under preparation.

Publication metadata

Author(s): Rutkowski S, von Bueren AO, Gerber NU, Clifford SC, Ellison DW, Grotzer MA, Hartmann W, zur Muhlen A, O'Toole KT, Nicholson S, Kortmann RD, Lannering B, Pietsch T

Publication type: Conference Proceedings (inc. Abstract)

Publication status: Published

Conference Name: Klinische Paediatrie: 22nd Annual Meeting of the Kind-Philipp Foundation for Leukemia Research

Year of Conference: 2009

Pages: 206 abstract no. A56

ISSN: 0300-8630

Publisher: Georg Thieme Verlag


DOI: 10.1055/s-0029-1222676

Library holdings: Search Newcastle University Library for this item

ISBN: 14393824