Fachin Alice, De Carlo Chiara, Maestro Alessandra, Zanon Davide, Barbi Egidio, Maximova Natalia
Department of Medicine, Surgery and Health Sciences, University of Trieste, 34127 Trieste, Italy.
Department of Medicine, Surgery and Health Sciences, University of Udine, 33100 Udine, Italy.
Pharmaceuticals (Basel). 2023 Jul 27;16(8):1069. doi: 10.3390/ph16081069.
Evinacumab, a human monoclonal antibody against angiopoietin-like protein 3 (ANGPTL3), has recently been approved by the U.S. Food and Drug Administration as an add-on therapy for homozygous familial hypercholesterolemia (HoFH) in patients of 12 years and older. Its role as a triglyceride-lowering drug is also emerging in the literature. However, it has not been approved for this indication yet, neither in the adult nor in the pediatric population. We describe the case of a 10-year-old boy who underwent an allogeneic hematopoietic stem cell transplant for acute lymphoblastic leukemia complicated by chronic graft-versus-host disease (GVHD) and presented life-threatening refractory hypertriglyceridemia due to the concomitant use of ruxolitinib and sirolimus. After the failure of the insulin treatment and due to the technical impossibility of performing lipid apheresis, the child underwent evinacumab treatment, obtaining a dramatic rapid reduction in triglyceride and cholesterol levels. This is the first report of a pediatric patient younger than 12 years in Europe receiving evinacumab to treat severe hypertriglyceridemia. The therapy with angiopoietin-like proteins inhibitors has been effective, safe, and well-tolerated in our patient, suggesting that evinacumab may be used in the pediatric population when other therapeutic strategies are ineffective or contraindicated.
evinacumab是一种针对血管生成素样蛋白3（ANGPTL3）的人单克隆抗体，最近已被美国食品药品监督管理局批准作为12岁及以上纯合子家族性高胆固醇血症（HoFH）患者的附加疗法。其作为降甘油三酯药物的作用在文献中也逐渐显现。然而，它尚未被批准用于这一适应症，无论是在成人还是儿科人群中。我们描述了一名10岁男孩的病例，他因急性淋巴细胞白血病接受了异基因造血干细胞移植，并发慢性移植物抗宿主病（GVHD），由于同时使用鲁索替尼和西罗莫司，出现了危及生命的难治性高甘油三酯血症。在胰岛素治疗失败且由于技术上无法进行血脂分离术之后，该患儿接受了evinacumab治疗，甘油三酯和胆固醇水平迅速显著降低。这是欧洲首例12岁以下儿科患者接受evinacumab治疗严重高甘油三酯血症的报告。血管生成素样蛋白抑制剂治疗在我们的患者中有效、安全且耐受性良好，这表明当其他治疗策略无效或禁忌时，evinacumab可用于儿科人群。
