Clinical and pathological features of paediatric malignant rhabdoid tumours.

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 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. Pediatr Blood Cancer 2010; 54:29–34. © 2009 Wiley-Liss, Inc.