Nivolumab in paediatric cancer: children are not little adults

Mené sur 85 enfants et des jeunes adultes (âge : 1-30 ans) atteints d’une tumeur solide ou d’un lymphome réfractaire ou récidivant, cet essai de phase I/II évalue la dose maximale tolérée du nivolumab et analyse ses caractéristiques pharmacocinétiques (durée médiane de suivi : 30 jours)

Résumé en anglais

The anti-PD-1 monoclonal antibody nivolumab is one of 33 US Food and Drug Administration approved antibodies or conjugates for the treatment of cancer. Nivolumab is approved in adults with high microsatellite instability or mismatch repair-deficient metastatic colon cancer, and as second-line therapy in advanced melanoma, several carcinomas, and relapsed Hodgkin lymphoma. With such a broad profile of anticancer activity, testing its activity in paediatric malignancies seems reasonable. In doing so, there is a potential benefit for all stakeholders involved in novel anticancer drug development for children. The pharmaceutical industry is incentivised to test such drugs in children and is encouraged by potential regulatory and economic benefits. Paediatric oncologists and patients welcome new options for recurrent and refractory cancer because they have fewer anticancer drugs available than do adult patients. However, this approach to clinical trials that tests drugs from adult oncology yields results that are mostly negative. And they are negative because we continue to forget that paediatric tumours are disparate from those in adults.