Long-term results of deep brain stimulation in a cohort of eight children with isolated dystonia
Long-term results of deep brain stimulation in a cohort of eight children with isolated dystonia
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DOI：
10.1007/s00415-016-8253-6
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发表时间：
2016-11-01
影响因子：
6
通讯作者：
Kuehn, A. A.
中科院分区：
文献类型：
--
作者：
Krause, P.;Lauritsch, K.;Kuehn, A. A.
苍白球脑深部电刺激（DBS）是一种治疗严重孤立性肌张力障碍患者的既定疗法。然而，儿童长期使用DBS的临床证据有限，尚未进行对照试验。在这里，我们提供了8例全身性特发性或遗传性孤立性肌张力障碍儿童患者（5例男性，手术时平均年龄12.5 +/-A3.5岁）长达13年的苍白球DBS的长期结果，通过回顾性视频评级进行评估。在3个时间点进行视频评级：术前、1年短期随访（1年-FU）和长期末次随访（LT-FU，长达13年）。使用Burke-Fahn-Marsden肌张力障碍评定量表（BFMDRS）评估症状严重程度和残疾。从临床图表和末次随访期间获得残疾评分。1年FU时BFMDRS运动评分的平均改善为54.4 +/- A 8.9%，LT-FU时为42.9 +/- A 11.6%;残疾评分分别改善59.8 +/- A 10.3和63.3 +/- A 7.8%。在1例患者中观察到电极脱位，在另1例患者中观察到植入式脉冲发生器脱位，均需要手术干预;未发生进一步严重不良事件。我们的研究首次对苍白球DBS治疗特发性或遗传性孤立性肌张力障碍儿童的短期和长期效果进行了盲法视频评级评估。结果证实，苍白球DBS是一种安全有效的儿童长期治疗方法，总体运动改善与成人对照试验中描述的相似。
Pallidal deep brain stimulation (DBS) is an established treatment for patients with severe isolated dystonia. However, clinical evidence for the long-term use of DBS in children is limited and controlled trials have not yet been conducted. Here, we provide the long-term results of up to 13 years of pallidal DBS in eight pediatric patients with generalized idiopathic or hereditary isolated dystonia (five males, mean age at surgery 12.5 +/- A 3.5 years), as assessed by retrospective video rating. Video rating was performed at three time points: pre-operative, 1-year short-term follow-up (1y-FU) and long-term last FU (LT-FU, up to 13 years). Symptom severity and disability were assessed using the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS). Disability scores were obtained from clinical charts and during the last FU. The mean improvement in BFMDRS motor score was 54.4 +/- A 8.9 % at 1y-FU and 42.9 +/- A 11.6 % at LT-FU; the disability scores improved by 59.8 +/- A 10.3 and 63.3 +/- A 7.8 %, respectively. Electrode dislocation was noted in one patient and implantable pulse generator dislocation in another, both requiring surgical intervention; no further serious adverse events occurred. Our study presents the first blinded video rating assessment of the short- and long-term effects of pallidal DBS in children with idiopathic or hereditary isolated dystonia. Results confirm that pallidal DBS is a safe and efficacious long-term treatment in children, with overall motor improvement similar to that described in controlled trials in adults.