Biology and therapy of pediatric rhabdomyosarcoma.

Abstract

PURPOSETo review key developments in biology and therapy of rhabdomyosarcoma (RMS) since the early 1970s.PATIENTS AND METHODSThe literature regarding biology, therapy, and late effects of therapy through March 1995 was reviewed.RESULTSThe two major histiotypes, embryonal and alveolar, are characterized by specific genetic abnormalities that provide clues to mechanisms of tumor induction. Alveolar tumors, for example, often possess a chromosomal translocation [t(2;13)(q35;q14)] that fuses the PAX3 gene in band 2q35 with the FKHR gene in band 13q14, creating a novel chimeric protein that could inappropriately activate normal targets of the PAX3 gene product, thereby contributing to tumorigenesis. Recognition of prognostically important patient groups primarily identified by tumor extent, site, and histology, and development of effective risk-based multimodal therapy in randomized trials, have increased long-term survival in RMS from 25% in 1970 to more than 70% in current studies. The most significant recen...