Hamano Shin-ichiro, Yamashita Shintaro, Tanaka Manabu, Yoshinari Satoshi, Minamitani Motoyuki, Eto Yoshikatsu
Division of Neurology, Saitama Children's Medical Center, and Department of Pediatrics, Jikei University School of Medicine, Japan.
J Pediatr. 2006 Apr;148(4):485-8. doi: 10.1016/j.jpeds.2005.11.041.
To determine the dosage and factors influencing efficacy of adrenocorticotropic hormone (ACTH) for West syndrome.
A retrospective study of 135 patients receiving ACTH therapy with a synthetic analogue for initial effect, seizure outcome 1 year after therapy, and adverse effects. Efficacy and adverse effects were compared among the groups divided by clinical factors: dosage, treatment lag, onset age, and cause.
One hundred thirteen patients had seizure control with ACTH. For more than 1 year after ACTH, 59 remained seizure free. Adverse effects were observed in 57, and ACTH therapy was discontinued in 23. The lowest dosage group (0.0125 mg/kg/d) had fewer episodes of discontinuation (P<.05), whereas differences in efficacy between different dosages were insignificant. None of the clinical factors correlated with initial effect. The earlier-onset group (<4 months) showed unfavorable seizure outcome 1 year after ACTH (P<.01). The cryptogenic patients showed better seizure outcome (P<.05) compared with the symptomatic.
Synthetic ACTH therapy at a lower dosage is as effective as natural ACTH therapy at a higher dosage. Considering the adverse effects and the benefits for seizure control, the ACTH dosage of 0.0125 mg/kg/d (synthetic analogue) is more favorable than larger dosage.
确定促肾上腺皮质激素（ACTH）治疗韦斯特综合征的剂量及影响疗效的因素。
一项回顾性研究，纳入135例接受ACTH合成类似物治疗的患者，观察初始疗效、治疗1年后的癫痫发作结局及不良反应。根据临床因素（剂量、治疗延迟、发病年龄和病因）将患者分组，比较各组的疗效和不良反应。
113例患者经ACTH治疗后癫痫得到控制。ACTH治疗1年多后，59例患者无癫痫发作。57例出现不良反应，23例停止ACTH治疗。最低剂量组（0.0125mg/kg/d）的停药次数较少（P<0.05），而不同剂量之间的疗效差异不显著。临床因素均与初始疗效无关。发病较早组（<4个月）在ACTH治疗1年后的癫痫发作结局较差（P<0.01）。与症状性患者相比，隐源性患者的癫痫发作结局更好（P<0.05）。
低剂量的合成ACTH治疗与高剂量的天然ACTH治疗效果相当。考虑到不良反应及对癫痫控制的益处，0.0125mg/kg/d（合成类似物）的ACTH剂量比更大剂量更有利。
