Maleknia Pedram, McWilliams Timothy D, Barkley Ariana, Estevez-Ordonez Dagoberto, Rozzelle Curtis, Blount Jeffrey P
1School of Medicine, University of Alabama at Birmingham; and.
2Division of Neurosurgery, University of Alabama at Birmingham, Children's of Alabama, Birmingham, Alabama.
J Neurosurg Pediatr. 2023 Jan 20;31(4):329-332. doi: 10.3171/2022.12.PEDS22302. Print 2023 Apr 1.
Food and Drug Administration (FDA) approval for vagus nerve stimulator (VNS) implantation is limited to patients older than 4 years of age with medically refractory partial-onset seizures. In younger children with severe generalized epilepsy, however, VNS implantation remains off-label. In this study, the authors followed up on their previously reported cohort to review the longer-term safety and efficacy of VNS placement in children younger than 6 years with generalized medically refractory epilepsy (MRE), providing the largest cohort with > 2 years of follow-up to date in this age group.
This was a retrospective observational cohort study of patients younger than 6 years of age with generalized MRE who underwent VNS implantation at a single institution between 2010 and 2020. Inclusion criteria encompassed failure of more than two antiepileptic drugs alone or in combination, neurologist referral for vagus nerve stimulation, informed consent with knowledge of the off-label status in young children, and > 1 year of follow-up. Outcome measures included seizure reduction rate ≥ 50% and postoperative morbidity defined nominally. Statistical analysis was conducted with Stata/SE.
Forty-five patients were included: 11 patients younger than 4 years of age and 34 between 4 and 6 years of age. There were no intraoperative complications. Perioperative complications within 1 year occurred in 11% (n = 5) of the patients and included two wound infections, a mild cough, hyperactivity, hoarseness, and 1 patient with persistent surgical site pain. A seizure reduction ≥ 50% was observed in 36.4% (n = 4) of the patients younger than 4 years of age at the 6-month and 1-, 2-, and 5-year follow-ups. In the 4- to 6-year-old cohort, this was observed in 32.4% (n = 11) of the patients at 6 months, 41.2% (n = 14) at 1 year, 38.2% (n = 13) at 2 years, and 41.2% (n = 14) at 5 years.
VNS implantation for patients younger than 4 years of age with generalized onset MRE has not been approved by the FDA. This retrospective study establishes feasibility, illustrates an acceptable safety profile in children younger than 6 years, and demonstrates efficacy comparable to that reported in older patients.
美国食品药品监督管理局（FDA）批准迷走神经刺激器（VNS）植入仅适用于年龄大于4岁、患有药物难治性局灶性发作癫痫的患者。然而，对于患有严重全身性癫痫的年幼儿童，VNS植入仍属于未获批准的应用。在本研究中，作者对其先前报告的队列进行随访，以评估VNS植入在6岁以下全身性药物难治性癫痫（MRE）患儿中的长期安全性和有效性，这是该年龄组迄今为止随访时间超过2年的最大队列研究。
这是一项回顾性观察性队列研究，研究对象为2010年至2020年间在单一机构接受VNS植入的6岁以下全身性MRE患儿。纳入标准包括单独或联合使用两种以上抗癫痫药物治疗失败、经神经科医生转诊接受迷走神经刺激、知晓年幼儿童未获批准应用情况后签署知情同意书以及随访时间超过1年。观察指标包括癫痫发作减少率≥50%以及名义上定义的术后发病率。使用Stata/SE进行统计分析。
共纳入45例患者：11例年龄小于4岁，34例年龄在4至6岁之间。术中无并发症发生。1年内围手术期并发症发生率为11%（n = 5），包括2例伤口感染、1例轻度咳嗽、多动、声音嘶哑，以及1例手术部位持续疼痛患者。在年龄小于4岁的患者中，6个月及1年、2年和5年随访时癫痫发作减少≥50%的比例为36.4%（n = 4）。在4至6岁队列中，6个月时为32.4%（n = 11），1年时为41.2%（n = 14），2年时为38.2%（n = 13），5年时为41.2%（n = 14）。
FDA未批准年龄小于4岁的全身性发作MRE患者植入VNS。这项回顾性研究证实了其可行性，表明6岁以下儿童具有可接受的安全性，并证明其疗效与年长患者报告的疗效相当。
