alexa Clinical and pathological features of paediatric malignant rhabdoid tumours.
Surgery

Surgery

Medical & Surgical Urology

Author(s): Morgenstern DA, Gibson S, Brown T, Sebire NJ, Anderson J

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Abstract BACKGROUND: Malignant rhabdoid tumours (MRT) and their central nervous system (CNS) counterparts atypical teratoid/rhabdoid tumours (ATRT) are rare, highly aggressive malignant neoplasms of childhood. Although there are isolated reports of long-term survival with intensive, multimodal therapy, outcomes are generally poor. PROCEDURE: We conducted a retrospective review of all patients diagnosed with MRT/ATRT at Great Ormond Street Hospital over the 20 years from 1989 to 2009. All cases were subjected to expert pathological review including INI-1 immunostaining. RESULTS: In a final cohort of 34 cases, overall survival was 17.4\%, with median survival 10.1 months. Outcome in patients aged <3 years was significantly worse (median survival 6.2 months vs. 19.2 months). Data demonstrated a statistically significant benefit of radiotherapy (median survival 14.9 months vs. 6.6 months), although this analysis is confounded by the impact of patient age. There were four long-term survivors (>30 months), all of whom received chemotherapy with or without surgical resection or radiotherapy. In the present study, immunohistochemistry revealed no significant staining for either c-Erb or c-Met in any case, suggesting that targeting these molecules is unlikely to be of benefit in treating MRT/ATRT. CONCLUSIONS: In view of poor outcomes, there is a clear need for new treatment strategies and the identification of novel molecular targets for MRT/ATRT. Copyright 2009 Wiley-Liss, Inc. This article was published in Pediatr Blood Cancer and referenced in Medical & Surgical Urology

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