Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia.
Department of Neurology, The Royal Children's Hospital, Melbourne, Victoria, Australia.
J Paediatr Child Health. 2022 Dec;58(12):2197-2202. doi: 10.1111/jpc.16181. Epub 2022 Aug 24.
To report response to first treatment in infants with infantile spasms (IS), including incremental benefit of prednisolone 60 mg/day and vigabatrin following prednisolone 40 mg/day failure in infants commenced on the United Kingdom Infantile Spasms Study (UKISS) treatment sequence.
In this retrospective analysis, we compared effectiveness of prednisolone, vigabatrin and nonstandard treatments as first treatment for IS. In infants who commenced the UKISS treatment sequence, we evaluated response to each step. Primary outcome was spasm cessation after 42 days. Secondary outcomes were severe side effects and spasm relapse after 42 days.
Treatment response data were available for 151 infants. First treatment was prednisolone in 99 infants, vigabatrin in 18 and nonstandard treatment in 34. The rate of spasm cessation with first treatment was significantly higher with prednisolone (62/99, 63%) than vigabatrin (5/18, 28%, P = 0.01) or nonstandard treatment (2/34, 5.9%, P < 0.01). Of 112 infants who commenced the UKISS treatment sequence, 71/112 (63%) responded to prednisolone 40 mg/day. Among non-responders, 12/29 (41%) subsequently responded to prednisolone 60 mg/day, and 10/22 (45%) to vigabatrin. Severe side effects and spasm relapse were not significantly different between each treatment.
We confirm higher rates of spasm cessation with initial treatment with prednisolone than vigabatrin and nonstandard therapy. Non-use of prednisolone as first treatment in over one third of infants highlights a concerning treatment gap. The UKISS treatment sequence has high overall treatment response (total 93/112; 83%), with similar benefit of subsequent prednisolone 60 mg/day and vigabatrin in prednisolone 40 mg/day non-responders.
报告婴儿痉挛症（IS）患儿首次治疗的反应，包括在接受英国婴儿痉挛症研究（UKISS）治疗方案中起始予泼尼松龙 40mg/日后治疗失败的患儿加用泼尼松龙 60mg/日和氨己烯酸的增量获益。
在这项回顾性分析中，我们比较了泼尼松龙、氨己烯酸和非标准治疗作为 IS 一线治疗的疗效。在起始予 UKISS 治疗方案的患儿中，我们评估了每一步的反应。主要结局为 42 天内痉挛停止。次要结局为 42 天后严重不良反应和痉挛复发。
共 151 例患儿有治疗反应数据。99 例患儿接受泼尼松龙治疗，18 例患儿接受氨己烯酸治疗，34 例患儿接受非标准治疗。首次治疗后痉挛停止率，泼尼松龙组（62/99，63%）显著高于氨己烯酸组（5/18，28%，P=0.01）和非标准治疗组（2/34，5.9%，P<0.01）。在起始予 UKISS 治疗方案的 112 例患儿中，71/112（63%）对泼尼松龙 40mg/日有反应。在无反应者中，29 例中有 12 例（41%）随后对泼尼松龙 60mg/日有反应，22 例中有 10 例（45%）对氨己烯酸有反应。各治疗组之间严重不良反应和痉挛复发的发生率无显著差异。
我们证实初始治疗应用泼尼松龙较氨己烯酸和非标准治疗可使痉挛停止率更高。超过三分之一的患儿未应用泼尼松龙作为一线治疗，这突出了令人担忧的治疗差距。UKISS 治疗方案总体治疗反应率高（总有效率为 93/112，83%），在对泼尼松龙 40mg/日无反应的患儿中，随后应用泼尼松龙 60mg/日和氨己烯酸同样获益。
