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.


METHODS
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.
Morgenstern
Daniel A
DA
Unit of Molecular Haematology and Cancer Biology, UCL Institute of Child Health, London, UK. j.anderson@ich.ucl.ac.uk
Gibson
Sian
S
Brown
Tanya
T
Sebire
Neil J
NJ
Anderson
John
J
D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D014408:Biomarkers, Tumor / Q000032:analysis; D002648:Child; D003131:Combined Modality Therapy; D005260:Female; D006801:Humans; D007124:Immunoenzyme Techniques; D007223:Infant; D008297:Male; D009367:Neoplasm Staging; D011379:Prognosis; D011878:Radiotherapy; D012189:Retrospective Studies; D018335:Rhabdoid Tumor / Q000401:mortality / Q000473:pathology* / Q000628:therapy; D015996:Survival Rate; D013724:Teratoma / Q000401:mortality / Q000473:pathology* / Q000628:therapy; D016896:Treatment Outcome
D014408:Biomarkers, Tumor