Departments of Medicine, Biochemistry and Molecular Biology, University of Miami Miller School of Medicine, Miami, FL, United States.
Department of Medicine, University of Washington, Seattle, WA, United States.
Front Endocrinol (Lausanne). 2020 Oct 23;11:593931. doi: 10.3389/fendo.2020.593931. eCollection 2020.
The chylomicronemia syndrome is characterized by severe hypertriglyceridemia and fasting chylomicronemia and predisposes affected individuals to acute pancreatitis. When due to very rare monogenic mutations in the genes encoding the enzyme, lipoprotein lipase, or its regulators, APOC2, APOA5, GPIHBP1, and LMF1, it is referred to as the familial chylomicronemia syndrome. Much more frequently, the chylomicronemia syndrome results from a cluster of minor genetic variants causing polygenic hypertriglyceridemia, which is exacerbated by conditions or medications which increase triglyceride levels beyond the saturation point of triglyceride removal systems. This situation is termed the multifactorial chylomicronemia syndrome. These aggravating factors include common conditions such as uncontrolled diabetes, overweight and obesity, alcohol excess, chronic kidney disease and pregnancy and several medications, including diuretics, non-selective beta blockers, estrogenic compounds, corticosteroids, protease inhibitors, immunosuppressives, antipsychotics, antidepressants, retinoids, L-asparaginase, and propofol. A third uncommon cause of the chylomicronemia syndrome is familial forms of partial lipodystrophy. Development of pancreatitis is the most feared complication of the chylomicronemia syndrome, but the risk of cardiovascular disease as well as non-alcoholic steatohepatitis is also increased. Treatment consists of dietary fat restriction and weight reduction combined with the use of triglyceride lowering medications such as fibrates, omega 3 fatty acids and niacin. Effective management of aggravating factors such as improving diabetes control, discontinuing alcohol and replacing or reducing the dose of medications that raise triglyceride levels is essential. Importantly, many if not most cases of the chylomicronemia syndrome can be prevented by effective identification of polygenic hypertriglyceridemia in people with conditions that increase its likelihood or before starting medications that may increase triglyceride levels. Several new pharmacotherapeutic agents are being tested that are likely to considerably improve treatment of hypertriglyceridemia in people at risk.
乳糜微粒血症综合征的特征是严重的高甘油三酯血症和空腹乳糜微粒血症,使受影响的个体易患急性胰腺炎。当由于编码酶脂蛋白脂酶或其调节剂 APOC2、APOA5、GPIHBP1 和 LMF1 的非常罕见的单基因突变引起时,它被称为家族性乳糜微粒血症综合征。更为常见的是,乳糜微粒血症综合征是由一系列导致多基因高甘油三酯血症的轻微遗传变异引起的,这些变异会因增加甘油三酯水平的情况或药物而加剧,超过甘油三酯清除系统的饱和度点。这种情况被称为多因素乳糜微粒血症综合征。这些加重因素包括常见的情况,如未控制的糖尿病、超重和肥胖、过量饮酒、慢性肾病和怀孕以及几种药物,包括利尿剂、非选择性β受体阻滞剂、雌激素化合物、皮质类固醇、蛋白酶抑制剂、免疫抑制剂、抗精神病药、抗抑郁药、类视黄醇、L-天冬酰胺酶和丙泊酚。乳糜微粒血症综合征的第三个不常见原因是家族性部分脂肪营养不良。胰腺炎的发展是乳糜微粒血症综合征最可怕的并发症,但心血管疾病和非酒精性脂肪性肝炎的风险也增加了。治疗包括限制饮食中的脂肪和减轻体重,同时使用降低甘油三酯的药物,如贝特类、ω-3 脂肪酸和烟酸。有效管理加重因素,如改善糖尿病控制、停止饮酒以及更换或减少可能增加甘油三酯水平的药物的剂量,是至关重要的。重要的是,通过在增加其可能性的情况下或在开始可能增加甘油三酯水平的药物之前,对具有增加甘油三酯水平可能性的人群进行多基因高甘油三酯血症的有效识别,可以预防许多(如果不是大多数)乳糜微粒血症综合征病例。正在测试几种新的药物治疗方法,这可能会极大地改善高危人群的高甘油三酯血症的治疗。