In osteosarcoma, large variation is observed in the efficacy and toxicity of chemotherapeutic drugs among similarly treated patients. Treatment optimization using predictive factors or algorithms is of importance, because there has been a lack of improvement of treatment outcome and survival for decades. The outcome of cancer treatment is influenced by the genome, thus studying genetic variants involved in the efficacy and toxicity of the chemotherapeutic drugs used in the treatment of osteosarcoma could be an opportunity to optimize current treatments and improve our understanding of the individual's drug response in osteosarcoma patients. This review discusses the current insights in the pharmacogenetics of the treatment response of osteosarcoma patients regarding efficacy and toxicity, and implications for future research and treatment.
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