Dyslipidemia in Children Treated with a BRAF Inhibitor for Low-Grade Gliomas: A New Side Effect?

Simple Summary The use of targeted therapies is revolutionizing the prognosis of many cancers; however, there is still limited knowledge of their side effects. Dyslipidemia is often present in cancer patients due to mechanisms that are directly or indirectly related to cancer or therapies. The aim of our study is to investigate the effects of vemurafenib on lipid metabolism in a cohort of pediatric patients treated for brain tumors. For the first time, we describe dyslipidemia as a possible side effect of the BRAF inhibitors. A better understanding of the pathways that are involved in dyslipidemia could also help with a better understanding of the drug-resistance mechanisms in cancer cells. BRAF inhibitors, in recent years, have played a central role in the disease control of unresectable BRAF-mutated pediatric low-grade gliomas (LGGs). The aim of the study was to investigate the acute and long-term effects of vemurafenib on the lipid metabolism in children treated for an LGG. In our cohort, children treated with vemurafenib (n = 6) exhibited alterations in lipid metabolism a few weeks after starting, as was demonstrated after 1 month (n = 4) by the high plasma levels of the total cholesterol (TC = 221.5 +/- 42.1 mg/dL), triglycerides (TG = 107.8 +/- 44.4 mg/dL), and low-density lipoprotein (LDL = 139.5 +/- 51.5 mg/dL). Despite dietary recommendations, the dyslipidemia persisted over time. The mean lipid levels of the TC (222.3 +/- 34.7 mg/dL), TG (134.8 +/- 83.6 mg/dL), and LDL (139.8 +/- 46.9 mg/dL) were confirmed abnormal at the last follow-up (45 +/- 27 months, n = 6). Vemurafenib could be associated with an increased risk of dyslipidemia. An accurate screening strategy in new clinical trials, and a multidisciplinary team, are required for the optimal management of unexpected adverse events, including dyslipidemia.