School of Medicine, Louisiana State University Health Sciences Center at Shreveport, Shreveport, LA, 71103, USA.
Department of Behavioral Neuroscience, Quinnipiac University, 275 Mount Carmel Ave, Hamden, CT, 06518, USA.
Neurol Sci. 2024 Feb;45(2):507-514. doi: 10.1007/s10072-023-07078-z. Epub 2023 Sep 22.
Infantile spasms, newly classified as infantile epileptic spasm syndrome (IESS), occur in children under 2 years of age and present as an occur as brief, symmetrical, contractions of the musculature of the neck, trunk, and extremities. When infantile spasms occur with a concomitant hypsarrhythmia on electroencephalogram (EEG) and developmental regression, it is known as West Syndrome. There is no universally accepted mainstay of treatment for this condition, but some options include synthetic adrenocorticotropic hormone (ACTH), repository corticotropin injection (RCI/Acthar Gel), corticosteroids, valproic acid, vigabatrin, and surgery. Without effective treatment, infantile spasms can cause an impairment of psychomotor development and/or cognitive and behavioral functions. The first-line treatment in the USA is ACTH related to high efficacy for cessation of infantile spasms long-term and low-cost profile. Acthar Gel is a repository corticotropin intramuscular injection that became FDA-approved for the treatment of IESS in 2010. Though it is believed that ACTH, Acthar Gel, and corticosteroids all work via a negative feedback pathway to decrease corticotropin-releasing hormone (CRH) release, their safety and efficacy profiles all vary. Vigabatrin and valproic acid are both anti-seizure medications that work by increasing GABA concentrations in the CNS and decreasing excitatory activity. Acthar Gel has been shown to have superior efficacy and a diminished side effect profile when compared with other treatment modalities.
婴儿痉挛症,新分类为婴儿癫痫痉挛综合征(IESS),发生于 2 岁以下儿童,表现为短暂、对称的颈部、躯干和四肢肌肉收缩。当婴儿痉挛症伴有脑电图(EEG)上的高度失律和发育退化时,称为 West 综合征。目前尚无被广泛接受的该病治疗方法,但一些治疗选择包括合成促肾上腺皮质激素(ACTH)、长效促皮质素(RCI/Acthar Gel)、皮质类固醇、丙戊酸、氨己烯酸和手术。如果没有有效的治疗,婴儿痉挛症可能导致精神运动发育和/或认知和行为功能受损。在美国,一线治疗是 ACTH,因为其对停止婴儿痉挛症具有高效性,且费用低廉。Acthar Gel 是一种长效促皮质素肌肉注射剂,于 2010 年获得 FDA 批准用于治疗 IESS。尽管人们认为 ACTH、Acthar Gel 和皮质类固醇都是通过负反馈途径来减少促肾上腺皮质激素释放激素(CRH)的释放而起作用的,但它们的安全性和疗效特征都有所不同。氨己烯酸和丙戊酸都是抗癫痫药物,通过增加中枢神经系统中的 GABA 浓度和减少兴奋性活动来发挥作用。与其他治疗方式相比,Acthar Gel 已被证明具有更好的疗效和更少的副作用。