Hussain Shaun A, Navarro Mario, Heesch Jaeden, Ji Matthew, Asilnejad Brenda, Peters Haley, Rajaraman Rajsekar R, Sankar Raman
Department of Pediatrics (Division of Pediatric Neurology) David Geffen School of Medicine and UCLA Mattel Children's Hospital Los Angeles California.
Children's Discovery and Innovation Institute David Geffen School of Medicine and UCLA Mattel Children's Hospital Los Angeles California.
Epilepsia Open. 2020 Jan 24;5(1):121-126. doi: 10.1002/epi4.12381. eCollection 2020 Mar.
A series of relatively small studies collectively suggest that zonisamide may be effective in the treatment of infantile spasms. Using a large single-center cohort of children with infantile spasms, we set out to evaluate the efficacy and safety of zonisamide. We retrospectively identified all patients with infantile spasms who were treated with zonisamide at our center. For each patient, we recorded dates of birth, infantile spasms onset, response (if any), and most recent follow-up. To quantify zonisamide exposure, we recorded daily dosage and patient weight at each sequential encounter so as to allow calculation of peak and weighted-average weight-based dosage. We identified 87 children who were treated with zonisamide, of whom 78 had previously been treated with hormonal therapy or vigabatrin. Peak and weighted-average zonisamide dosage were 7.1 (interquartile range 3.6, 10.2) and 5.4 (interquartile range 3.0, 8.9) mg/kg/day, respectively. Whereas five (6%) patients exhibited resolution of epileptic spasms, only two (2%) patients exhibited video-EEG confirmed resolution of both epileptic spasms and hypsarrhythmia (electroclinical response). Importantly, both electroclinical responders had not previously been treated with hormonal therapy or vigabatrin; in contrast, none of the 78 children with prior failure of hormonal therapy or vigabatrin subsequently responded to zonisamide. Zonisamide was well tolerated, and there were no deaths. This study suggests that zonisamide exhibits favorable tolerability but very limited efficacy among patients who do not respond to first-line therapy.
一系列相对较小规模的研究共同表明，唑尼沙胺可能对婴儿痉挛症的治疗有效。我们利用一个大型单中心婴儿痉挛症患儿队列，着手评估唑尼沙胺的疗效和安全性。我们回顾性地确定了在我们中心接受唑尼沙胺治疗的所有婴儿痉挛症患者。对于每位患者，我们记录了出生日期、婴儿痉挛症发作时间、反应（如有）以及最近的随访情况。为了量化唑尼沙胺的暴露量，我们在每次连续就诊时记录每日剂量和患者体重，以便计算基于体重的峰值剂量和加权平均剂量。我们确定了87名接受唑尼沙胺治疗的儿童，其中78名此前曾接受过激素治疗或氨己烯酸治疗。唑尼沙胺的峰值剂量和加权平均剂量分别为7.1（四分位间距3.6，10.2）和5.4（四分位间距3.0，8.9）mg/kg/天。虽然有5名（6%）患者的癫痫痉挛症状得到缓解，但只有2名（2%）患者通过视频脑电图证实癫痫痉挛和高峰失律（电临床反应）均得到缓解。重要的是，这两名电临床反应者此前均未接受过激素治疗或氨己烯酸治疗；相比之下，78名此前激素治疗或氨己烯酸治疗失败的儿童随后均未对唑尼沙胺产生反应。唑尼沙胺耐受性良好，且无死亡病例。这项研究表明，唑尼沙胺在对一线治疗无反应的患者中耐受性良好，但疗效非常有限。
