Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA; Neurology Service, Department of Pediatrics, KK Women's and Children's Hospital, Singapore.
Child Health Research Institute, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL.
J Pediatr. 2016 Nov;178:227-232. doi: 10.1016/j.jpeds.2016.07.040. Epub 2016 Aug 24.
To characterize the longitudinal clinical and electrophysiological patterns of thalidomide neuropathy in children and adolescents.
Retrospective analysis of clinical records at a tertiary care children's hospital, including serial electrophysiological studies.
Sixteen patients aged 6-24 years received thalidomide to treat Crohn's disease from 2002 to 2012. Nine subjects had electrophysiological evidence of sensorimotor axonal polyneuropathy, 8 of whom had sensory and/or motor symptoms. The patients with polyneuropathy received thalidomide for 5 weeks to 52 months, with cumulative doses ranging from 1.4 to 207.7 g. All subjects with cumulative doses greater than 60 g developed polyneuropathy, and 4 of the 5 subjects who received thalidomide for more than 20 months developed polyneuropathy. The 7 subjects who had normal neurophysiological studies received therapy for 1 week to 25 months, with cumulative doses ranging from 0.7 to 47 g. In contrast to some previous reports, several patients had sensorimotor polyneuropathies, rather than pure sensory neuropathies. In patients with neuropathy who received therapy for more than 24 months and had 3 or more electromyography studies, the severity of the neuropathy plateaued.
Factors in addition to the total dose may contribute to the risk profile for thalidomide neuropathy, including pharmacogenetic susceptibilities. The severity of the neuropathy does not worsen relentlessly. Children, adolescents, and young adults receiving thalidomide should undergo regular neurophysiological studies to monitor for neuropathy.
描述儿童和青少年沙利度胺神经病的纵向临床和电生理模式。
对一家三级儿童保健医院的临床记录进行回顾性分析，包括连续的电生理研究。
2002 年至 2012 年期间，16 名年龄在 6 至 24 岁的克罗恩病患儿接受沙利度胺治疗。9 名患者有感觉运动轴索性多发性神经病的电生理证据，其中 8 名有感觉和/或运动症状。患有多发性神经病的患者接受沙利度胺治疗的时间为 5 周至 52 个月，累积剂量为 1.4 至 207.7g。所有累积剂量大于 60g 的患者均发生多发性神经病，5 名接受沙利度胺治疗超过 20 个月的患者中有 4 名发生多发性神经病。7 名神经生理学检查正常的患者接受治疗的时间为 1 周至 25 个月，累积剂量为 0.7 至 47g。与一些先前的报告不同，一些患者有感觉运动性多发性神经病，而不是单纯的感觉神经病。在接受治疗超过 24 个月且进行了 3 次或更多次肌电图研究的多发性神经病患者中，神经病的严重程度趋于稳定。
除总剂量外，其他因素（包括遗传易感性）可能会增加沙利度胺神经病的风险。神经病的严重程度不会持续恶化。接受沙利度胺治疗的儿童、青少年和年轻成年人应定期进行神经生理学检查，以监测神经病。
