Crocco Marco, Verrico Antonio, Milanaccio Claudia, Piccolo Gianluca, De Marco Patrizia, Gaggero Gabriele, Iurilli Valentina, Di Profio Sonia, Malerba Federica, Panciroli Marta, Giordano Paolo, Calevo Maria Grazia, Casalini Emilio, Di Iorgi Natascia, Garrè Maria Luisa
Neuroncology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genova, 16132 Genova, Italy.
Cancers (Basel). 2022 May 29;14(11):2693. doi: 10.3390/cancers14112693.
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 ( = 6) exhibited alterations in lipid metabolism a few weeks after starting, as was demonstrated after 1 month ( = 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, = 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.
近年来，BRAF抑制剂在不可切除的BRAF突变型儿童低级别胶质瘤（LGG）的疾病控制中发挥了核心作用。本研究的目的是调查维莫非尼对接受LGG治疗的儿童脂质代谢的急性和长期影响。在我们的队列中，接受维莫非尼治疗的儿童（n = 6）在开始治疗几周后就出现了脂质代谢改变，1个月后（n = 4）总胆固醇（TC = 221.5 ± 42.1 mg/dL）、甘油三酯（TG = 107.8 ± 44.4 mg/dL）和低密度脂蛋白（LDL = 139.5 ± 51.5 mg/dL）的血浆水平升高就证明了这一点。尽管有饮食建议，但血脂异常仍持续存在。在最后一次随访时（45 ± 27个月，n = 6），TC（222.3 ± 34.7 mg/dL）、TG（134.8 ± 83.6 mg/dL）和LDL（139.8 ± 46.9 mg/dL）的平均脂质水平仍被确认为异常。维莫非尼可能与血脂异常风险增加有关。在新的临床试验中，需要一种准确的筛查策略和一个多学科团队，以优化对包括血脂异常在内的意外不良事件的管理。
