深部脑刺激（DBS）联合丘脑底核（STN）作为 PKAN 患儿的治疗靶点。
Deep Brain Stimulation (DBS) with Subthalamic Nucleus (STN) as Target for Pediatric Patients with PKAN.
机构信息
Department of Pediatrics, Peking University First Hospital, Peking University, Beijing, People's Republic of China.
Department of Neurosurgery, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
出版信息
World Neurosurg. 2022 Jul;163:e317-e322. doi: 10.1016/j.wneu.2022.03.130. Epub 2022 Apr 1.
OBJECTIVE
Dystonia in pantothenate kinase-associated neurodegeneration (PKAN) is progressive despite medication. Deep brain stimulation (DBS) was reported to effectively provide symptom relief. No consensus exists in candidate and target selection for DBS. We aimed to demonstrate effectiveness of subthalamic DBS (STN-DBS) placement in pediatric patients with PKAN.
METHODS
We reviewed consecutive series of pediatric patients diagnosed with PKAN and treated with STN-DBS from 2016 to 2019 in our institution. Each case was described in detail. Preoperative and postoperative Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) were assessed to evaluate functional improvement at follow-up.
RESULTS
Seven pediatric patients were included. Mean age of initial onset was 0.6 ± 0.5 years and presentation to clinics was 6.6 ± 1.3 years. Mean preoperative BFMDRS was 73.3 ± 3.5. Following STN-DBS, for mean follow-up duration of 13.0 ± 10.7 months, mean BFMDRS was 37.3 ± 12.6, translating to score improvement of 36.0 ± 12.9 (P < 0.001), and percentage improvement of 49.0 ± 18.0%.
CONCLUSIONS
This case series demonstrated that STN-DBS is an effective symptom-based treatment for pediatric patients with PKAN.
目的
尽管药物治疗，泛酸激酶相关神经变性（PKAN）中的肌张力障碍仍呈进行性发展。深部脑刺激（DBS）已被报道能有效缓解症状。但对于 DBS 的候选靶点和目标选择尚未达成共识。我们旨在证明丘脑底核（STN）刺激（STN-DBS）在 PKAN 儿科患者中的有效性。
方法
我们回顾了 2016 年至 2019 年期间我院连续接受 STN-DBS 治疗的 PKAN 儿科患者的系列病例。详细描述了每个病例。通过术前和术后 Burke-Fahn-Marsden 肌张力障碍评定量表（BFMDRS）评估，评估随访时的功能改善情况。
结果
共纳入 7 例儿科患者。起病平均年龄为 0.6 ± 0.5 岁，就诊平均年龄为 6.6 ± 1.3 岁。平均术前 BFMDRS 为 73.3 ± 3.5。在接受 STN-DBS 后，平均随访时间为 13.0 ± 10.7 个月，平均 BFMDRS 为 37.3 ± 12.6，评分改善 36.0 ± 12.9（P < 0.001），改善率为 49.0 ± 18.0%。
结论
本病例系列研究表明，STN-DBS 是治疗 PKAN 儿科患者的一种有效的基于症状的治疗方法。
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