Effectiveness of chemotherapy in rhabdomyosarcoma: example of orbital primary

The survival of patients with rhabdomyosarcoma has been progressively improved with successive protocols due to the development of multidisciplinary management and the data accumulated by international groups. Orbital rhabdomyosarcoma represents 10% of all cases and affects young children (median age: 6.8 years). It is a chemosensitive and radiosensitive tumour. Chemotherapy is designed to decrease the indications for local therapy (mainly radiotherapy) responsible for a high rate of sequelae (cosmetic, functional or secondary cancer). According to the International Society of Paediatric Oncology guidelines, local therapy is not indicated as first-line treatment in case of complete remission after chemotherapy. The 10-year survival of children with non-parameningeal orbital rhabdomyosarcoma is currently 87% and identical survivals are reported by the various collaborative groups despite the use of different treatments. Despite clinical trials demonstrating the efficacy of many types of chemotherapy (cisplatin, etoposide, doxorubicin, dacarbazine), the value of adding these drugs to combination chemotherapy comprising of an alkylating agent (cyclophosphamide or ifosfamide), vincristine and dactinomycin has not been formally demonstrated in terms of survival benefit for children with rhabdomyosarcoma. The authors review these various results and compare the current guidelines for the management of orbital rhabdomyosarcoma recommended by North American and European groups.