Clinical Study Clinical trial participation and outcome for patients with glioblastoma: Multivariate analysis from a comprehensive dataset Kathryn M. Field a,b,c,⇑ , Katharine J. Drummond d,e , Merve Yilmaz f , Mark Tacey g , Daniel Compston c , Peter Gibbs a,b,c,h , Mark A. Rosenthal a,h a Department of Medical Oncology, Royal Melbourne Hospital, Victoria, Australia b Ludwig Institute for Cancer Research (Parkville Branch), Victoria, Australia c BioGrid Australia, Level 6 North, Royal Melbourne Hospital, Grattan Street, Parkville, Victoria 3050, Australia d Department of Neurosurgery, Royal Melbourne Hospital, Victoria, Australia e Department of Surgery, University of Melbourne, Victoria, Australia f Melbourne Medical School, University of Melbourne, Victoria, Australia g Melbourne EpiCentre, Royal Melbourne Hospital, Victoria, Australia h Department of Medicine, University of Melbourne, Victoria, Australia article info Article history: Keywords: Clinical trial Glioblastoma Outcomes research Prognosis Survival abstract Glioblastoma multiforme (GBM) is the most common malignant brain tumor in adults. Although multiple clinical and tumor-related variables affect survival outcomes, the effect of clinical trial participation has not been explored. The aim of this study was to determine whether clinical trial participation improves outcome for patients with GBM. Data from patients with GBM were accessed from a dataset collected over 12 years (1998–2010) at two institutions. Univariable and multivariate logistic regression analyses were performed to look for relationships between clinical trial participation, other baseline clinical and sociodemographic variables and overall survival (OS). In total, 542 patients were identified and included in the analysis; median age was 62 years. Sixty-one patients (11%) were enrolled in a clinical trial. Clinical trial enrollment was associated with improved median survival (14.5 months compared to 6.3 months, p < 0.001) and this difference remained significant in multivariate analysis (hazard ratio 0.67, p = 0.046). Age, poor performance status and operation type were also independent predictors for OS in multivariate analysis. Disease site, socioeconomic status and co-morbidity did not affect survival out- come. This is the first study in patients with GBM to suggest a survival benefit from clinical trial partic- ipation, independent of age and performance status; while also confirming the importance of other previously reported prognostic factors. This should encourage clinicians to offer trial therapies to patients with GBM and encourage patients to participate in available studies. Ó 2012 Elsevier Ltd. All rights reserved. 1. Introduction Glioblastoma multiforme (GBM) is the most common and most aggressive malignant glial tumor, accounting for 60% to 70% of malignant gliomas. It carries a high mortality and a high social bur- den to both cancer sufferers and their carers. For patients treated on the landmark European Organisation for Research and Treat- ment of Cancer (EORTC) trial, the 2-year and 5-year overall survival (OS) rate was 27% and 9% respectively; with a median OS of 14.6 months. 1 Population studies suggest worse survival outcomes, with a median survival of 7 months and 5-year survival of only 3% in a large retrospective study of patients with GBM. 2 It would be ideal to understand why there appears to be a spectrum of survival outcomes ranging from months to years to be able to identify prognostic factors at the outset of a patient’s diagnosis, to detect inferior treatment strategies that could be modified, and to individualise management strategies. With more treatment options available, identifying those who may be most (or least) likely to benefit from a treatment becomes of para- mount importance. Clinical trials are the benchmark by which new treatments be- come established. Despite their importance, only a small percent- age of patients with cancer are enrolled in clinical trials. 3 Participation in trials, for many with cancer, appears to improve outcomes regardless of the treatment arm allocated. 4 Surprisingly, whether this is true for patients with GBM has never been investi- gated to our knowledge. This is particularly pertinent as treatment options and clinical trials for patients with GBM have expanded in recent years, reflecting recent developments in targeted therapies and the increasing understanding of biological mechanisms behind malignant disease processes. 0967-5868/$ - see front matter Ó 2012 Elsevier Ltd. All rights reserved. http://dx.doi.org/10.1016/j.jocn.2012.09.013 ⇑ Corresponding author. Tel.: +61 3 9342 7000, fax: +61 3 9342 8548. E-mail address: Kathryn.field@mh.org.au (K.M. Field). Journal of Clinical Neuroscience 20 (2013) 783–789 Contents lists available at SciVerse ScienceDirect Journal of Clinical Neuroscience journal homepage: www.elsevier.com/locate/jocn