Abstract
Chemotherapy plays a prominent role in the treatment of paediatric malignancies. Several pharmacokinetic and pharmacodynamic mechanisms must be taken into account when administering chemotherapy. These mechanisms may contribute to the clearance, toxicity, and/or efficacy of cytotoxic drugs. Examples are age, sex, body composition, genetic polymorphisms, and co-administered drugs. A new era of childhood cancer treatment is emerging, characterized by the development of new agents resulting from advances in molecular biology. Directing drugs against the abnormalities identified only in tumour cells is known as targeted therapy. Such compounds ought to have better anti-tumour activity and improved safety profiles. Monoclonal antibodies are becoming more important, especially in the treatment of haematological malignancies, but also in neuroblastoma. Moreover, other advances in immunotherapy, with bispecific T-cell engaging (BITE) antibodies and chimeric-antigen receptor T-cells for example, are rapidly changing the landscape of available therapies to treat childhood cancer. After a long period during which there were very few registrations of new drugs for use in children, the regulatory incentives introduced in both North America and Europe now stimulate research directed towards the identification and evaluation of new drugs in paediatric oncology.
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