Miller Melissa L, Wright Chanin C, Browne Barry
Rocky Mountain Hospital for Children, Presbyterian/St. Luke's Medical Center, Denver, Colorado.
McLane Children's Baylor Scott & White Health, Temple, Texas.
J Clin Lipidol. 2015 Sep-Oct;9(5 Suppl):S67-76. doi: 10.1016/j.jacl.2015.06.013. Epub 2015 Jul 2.
As demonstrated by the 2011 publication of the National Heart, Lung, and Blood Institute Integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents, the information available regarding the treatment of pediatric lipid disorders has greatly expanded. HMG-CoA reductase inhibitor, or statin, therapy is now considered a first-line pharmacologic intervention for pediatric patients with severe dyslipidemias failing treatment with diet and exercise alone. Despite their ability to effectively reduce cholesterol levels, bile acid sequestrants continue to pose challenges for pediatric patients because of their unpalatability and are typically used as adjunctive therapy or for patients not able to tolerate statins. Fibric acid derivatives, as a class of medications, not only lack a Food and Drug Administration (FDA)-approved agent, but also continue to lack significant pediatric safety and efficacy data. Niacin, a potential adjunct therapy, lacks FDA approval for pediatric patients and is plagued by significant adverse effects making it an unlikely therapy option for pediatric patients. Ezetimibe provides clinicians with an alternative adjunct therapy option when synergistically paired with an HMG-CoA reductase inhibitor or it can be used as monotherapy for patients intolerant to statins and bile acid sequestrants. Finally, despite several marketed formulations, omega-3 fish oils currently lack FDA approval in pediatric patients and have failed to demonstrate statistically significant lipid lowering in pediatric and adolescent patients. Although recent years have witnessed a dramatic increase in data available for the use of lipid-lowering medications for pediatric patients, long-term study data are still generally lacking and continues to present an active focus of research.
美国国立心肺血液研究所于2011年发布的《儿童和青少年心血管健康与风险降低综合指南》表明,有关儿童脂质异常治疗的可用信息已大幅扩展。HMG-CoA还原酶抑制剂即他汀类药物治疗,现在被认为是对仅通过饮食和运动治疗无效的重度血脂异常儿科患者的一线药物干预措施。尽管胆汁酸螯合剂有有效降低胆固醇水平的能力,但由于其难吃的味道,对儿科患者来说仍然存在挑战,通常用作辅助治疗或用于无法耐受他汀类药物的患者。作为一类药物,纤维酸衍生物不仅缺乏美国食品药品监督管理局(FDA)批准的药物,而且仍然缺乏重要的儿科安全性和有效性数据。烟酸作为一种潜在的辅助治疗药物,未获得FDA对儿科患者的批准,且存在严重不良反应,使其不太可能成为儿科患者的治疗选择。依泽替米贝与HMG-CoA还原酶抑制剂协同使用时,为临床医生提供了另一种辅助治疗选择,或者它可以用作对他汀类药物和胆汁酸螯合剂不耐受患者的单一疗法。最后,尽管有几种上市制剂,但ω-3鱼油目前尚未获得FDA对儿科患者的批准,并且在儿科和青少年患者中未能显示出具有统计学意义的降脂效果。尽管近年来用于儿科患者的降脂药物可用数据急剧增加,但长期研究数据仍然普遍缺乏,并且仍然是一个积极的研究重点。