Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Eur Rev Med Pharmacol Sci. 2020 Oct;24(19):10036-10044. doi: 10.26355/eurrev_202010_23218.
Glycogen storage disease type Ia (GSDIa) is a glucose metabolic disorder. GSDIa patients are characterized by hypoglycemia, hepatomegaly, hyperlipidemia, and hyperlactacidemia. This retrospective study aimed to review the lipid status, explore lipid treatment targets, and assess preferable lipid-lowering drugs.
Clinical data on GSDIa patients' characteristics were collected. Most patients were followed-up once a year. Diet control and raw cornstarch treatment were used to maintain normal blood glucose and lipid levels. Some patients were given lipid-lowering drugs. We compared the lipid levels before and after each treatment.
A total of 163 GSDIa patients were enrolled in this study. After treatment with raw cornstarch, the total triglycerides (TG) level has significantly decreased by 30±50% (8.37±7.23 to 5.39±5.29 mmol/L, p<0.001). There was no change in the total cholesterol (TC) level. Fifteen patients regularly took atorvastatin, and 15 took fibrates for more than one year. The therapeutic effect of atorvastatin was better than fibrates. The TC was positively correlated with TG after treatment, resulting in the following linear equation: TG=1.63×TC-2.86. Using this equation and Chinese children's normal TC level of 5.18 mmol/L, we aimed to maintain the patients at TG < 5.58 mmol/L.
Patients with GSDIa have significant abnormalities in lipid metabolism. Because the complications of hyperlipidemia are caused mainly by TC, thereby, by maintaining it at a normal level, we could set a TG target by the linear equation that allowed a certain degree of hypertriglyceridemia. This study found that the therapeutic effect of atorvastatin was better than fibrates.
糖原贮积病 Ia 型（GSDIa）是一种葡萄糖代谢紊乱。GSDIa 患者的特征是低血糖、肝肿大、高血脂和高乳酸血症。本回顾性研究旨在回顾脂质状况，探讨脂质治疗目标，并评估更优的降脂药物。
收集 GSDIa 患者特征的临床数据。大多数患者每年随访一次。通过饮食控制和生玉米淀粉治疗来维持正常血糖和血脂水平。一些患者给予降脂药物。我们比较了每种治疗前后的血脂水平。
本研究共纳入 163 例 GSDIa 患者。生玉米淀粉治疗后，总甘油三酯（TG）水平显著下降 30±50%（8.37±7.23 至 5.39±5.29 mmol/L，p<0.001）。总胆固醇（TC）水平无变化。15 例患者定期服用阿托伐他汀，15 例患者服用贝特类药物超过 1 年。阿托伐他汀的治疗效果优于贝特类药物。治疗后 TC 与 TG 呈正相关，得出以下线性方程：TG=1.63×TC-2.86。使用该方程和中国儿童正常 TC 水平 5.18mmol/L，我们旨在将患者的 TG 维持在<5.58mmol/L。
GSDIa 患者的脂质代谢存在明显异常。由于高血脂的并发症主要由 TC 引起，因此通过将其维持在正常水平，可以通过线性方程设定 TG 目标，允许一定程度的高甘油三酯血症。本研究发现，阿托伐他汀的治疗效果优于贝特类药物。
