Kohut Taisa, Tou Andrea, Carr Emily, Xanthakos Stavra, Arce-Clachar Ana Catalina, Fawaz Rima, Valentino Pamela L, Panganiban Jennifer, Mouzaki Marialena
Division of Pediatric Gastroenterology, Hepatology, and Nutrition, University of Miami Miller School of Medicine, Miami, Florida, USA.
Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
JPEN J Parenter Enteral Nutr. 2025 Apr;49(3):308-313. doi: 10.1002/jpen.2722. Epub 2024 Dec 25.
Metabolic dysfunction-associated steatotic liver disease (MASLD) is a common disease in children. Lifestyle modification is the primary treatment but difficult to achieve and maintain. Topiramate is a component of an approved weight loss medication (topiramate-phentermine) in children aged 12 years and older but is more commonly used as a single agent, off-label, for pediatric obesity. Our aim is to describe change in anthropometrics and laboratory values while providing topiramate treatment of pediatric MASLD.
Descriptive cohort study including patients aged <18 years with MASLD and body mass index (BMI) >95th percentile treated with topiramate for weight loss for ≥3 months from January 1, 2010, to December 30, 2023. The primary outcome was change in serum alanine aminotransferase (ALT) levels from baseline to 3-6 months. Secondary outcomes were changes in BMI z score, glycated hemoglobin, and lipid profile.
Of 43 patients prescribed topiramate, 11 were excluded for nonadherence, leaving 32 (56% boys, 72% non-Hispanic) for further analyses. With topiramate, ALT levels improved (76 vs 50 U/L, p = 0.001). Further, 43% of patients had either ALT normalization or reduction by >50% from baseline. BMI z score decreased by 0.1 from baseline to 3-6 months. There were no improvements in glycated hemoglobin or lipids. Eight patients (25%) reported mild side effects.
Topiramate, as an adjunct to lifestyle intervention, may be considered in the treatment of pediatric MASLD, specifically in the context of failed lifestyle modification and inability to tolerate or qualify for other obesity pharmacotherapy.
代谢功能障碍相关脂肪性肝病（MASLD）在儿童中是一种常见疾病。生活方式改变是主要治疗方法，但难以实现和维持。托吡酯是一种已获批用于12岁及以上儿童减肥药物（托吡酯-苯丁胺）的成分，但更常用于作为单一药物非标签用于儿童肥胖症治疗。我们的目的是描述在为儿童MASLD提供托吡酯治疗时人体测量学和实验室指标的变化。
描述性队列研究，纳入2010年1月1日至2023年12月30日期间年龄小于18岁、患有MASLD且体重指数（BMI）高于第95百分位数、接受托吡酯治疗减肥≥3个月的患者。主要结局是从基线到3至6个月时血清丙氨酸氨基转移酶（ALT）水平的变化。次要结局是BMI z评分、糖化血红蛋白和血脂谱的变化。
在43例开具托吡酯处方的患者中，11例因未坚持治疗被排除，剩余32例（56%为男孩，72%为非西班牙裔）进行进一步分析。使用托吡酯后，ALT水平有所改善（76 vs 50 U/L，p = 0.001）。此外，43%的患者ALT恢复正常或较基线降低>50%。从基线到3至6个月，BMI z评分下降了0.1。糖化血红蛋白或血脂没有改善。8例患者（25%）报告有轻微副作用。
托吡酯作为生活方式干预的辅助手段，可考虑用于儿童MASLD的治疗，特别是在生活方式改变失败且无法耐受或不符合其他肥胖症药物治疗条件的情况下。
