结节性硬化
西罗莫司
医学
癫痫
不利影响
耐火材料(行星科学)
前瞻性队列研究
队列
抗惊厥药
内科学
队列研究
麻醉
胃肠病学
儿科
天体生物学
精神科
物理
作者
Wen He,Jian Chen,Yangyang Wang,Mengna Zhang,Qian-Lu,Qiuhong Wang,Xiaomei Luo,Xiao-Qiao Chen,Li‐Ping Zou
标识
DOI:10.1016/j.seizure.2020.03.018
摘要
PurposeThis study aimed to analyze the therapeutic effect of sirolimus on seizures in pediatric patients with tuberous sclerosis.MethodsWe first compared the efficacy of controlling seizures in all patients after they had taken sirolimus for one year, and then we performed a subgroup analysis based on whether the administered antiepileptic drugs were changed to determine whether the efficacy was associated with changes of antiepileptic drugs.ResultsA total of 91 eligible children were enrolled. The response rate was 78.0 % (71/91), and 47.2 % (43/91) of all patients were became seizure-free. The improvement in seizure control before and after treatment with sirolimus was significant (p < 0.001). In the AEDs unaltered group, 34 were responders (34/45, 75.6 %, 95 % CI 17.4–88.3), of which 24 were seizure-free (24/34, 70.6 %). In the AEDs-altered group, 37 were responders (37/46, 80.4 %, 95 % CI 56.7–88.1), of which 19 were seizure-free (19/37, 51.4 %). There was no significant difference between the two groups for reductions in rate of seizure frequency (p = 0.308). In the patients with refractory epilepsy, treatment with sirolimus was also effective (p = 0.01). Logistic regression analysis showed that age was an important factor affecting outcome of epilepsy (p = 0.003, 95 % CI 2.05–38.31). No Grade 3 or 4 adverse events were noted during the follow-up.ConclusionsSirolimus has a significant effect on seizures associated with tuberous sclerosis complex (TSC), with no or only moderate adverse events after long-term administration. Sirolimus could be used as the first-line medication for pediatric patients with TSC-associated epilepsy.
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