Minimal exposure intra-arterial chemotherapy for children with retinoblastoma and 13q syndrome.

Two infants with retinoblastoma and 13q syndrome with multiorgan system anomalies were treated with targeted intra-arterial chemotherapy (IAC) using one-to-three cycles of melphalan 5 mg to avoid systemic chemotherapeutic side effects. Both patients showed good response, with tumor control and no systemic chemotherapy side effects. Of the treatment modalities currently available, IAC may represent an optimal balance between tumor extermination and adverse drug reactions in this patient population with classically reduced multiorgan reserve.