Badhiwala Jetan H, Karmur Brij, Elkaim Lior M, Alotaibi Naif M, Morgan Benjamin R, Lipsman Nir, De Vloo Philippe, Kalia Suneil K, Lozano Andres M, Ibrahim George M
1Division of Neurosurgery, Department of Surgery, and.
2Faculty of Medicine, University of Toronto, Toronto, Ontario.
J Neurosurg Pediatr. 2019 Jul 12;24(4):442-450. doi: 10.3171/2019.5.PEDS1973. Print 2019 Oct 1.
Although deep brain stimulation (DBS) is an accepted treatment for childhood dystonia, there is significant heterogeneity in treatment response and few data are available to identify ideal surgical candidates.
Data were derived from a systematic review and individual patient data meta-analysis of DBS for dystonia in children that was previously published. Outcomes were assessed using the Burke-Fahn-Marsden Dystonia Rating Scale for movement (BFMDRS-M) and for disability (BFMDRS-D). The authors used partial least squares, bootstrapping, and permutation statistics to extract patterns of contributions of specific preoperative characteristics to relationship with distinct outcomes, in all patients and in patients with primary and secondary dystonia separately.
Of 301 children undergoing DBS for dystonia, 167 had primary dystonia, 125 secondary dystonia, and 9 myoclonus dystonia. Three dissociable preoperative phenotypes (latent variables) were identified and associated with the following: 1) BFMDRS-M at last follow-up; 2) relative change in BFMDRS-M score; and 3) relative change in BFMDRS-D score. The phenotype of patients with secondary dystonia, with a high BFMDRS-M score and truncal involvement, undergoing DBS at a younger age, was associated with a worse postoperative BFMDRS-M score. Children with primary dystonia involving the trunk had greater improvement in BFMDRS-M and -D scores. Those with primary dystonia of shorter duration and proportion of life with disease, undergoing globus pallidus DBS, had greater improvements in BFMDRS-D scores at long-term follow-up.
In a comprehensive, data-driven, multivariate analysis of DBS for childhood dystonia, the authors identified novel and dissociable patient phenotypes associated with distinct outcomes. The findings of this report may inform surgical candidacy for DBS.
尽管深部脑刺激（DBS）是治疗儿童肌张力障碍的一种公认方法，但治疗反应存在显著异质性，且几乎没有数据可用于确定理想的手术候选者。
数据来自先前发表的关于儿童肌张力障碍DBS的系统评价和个体患者数据荟萃分析。使用伯克-法恩-马斯登肌张力障碍运动评分量表（BFMDRS-M）和残疾评分量表（BFMDRS-D）评估结果。作者使用偏最小二乘法、自抽样法和排列统计法，分别在所有患者以及原发性和继发性肌张力障碍患者中，提取特定术前特征对与不同结果关系的贡献模式。
在301例接受DBS治疗肌张力障碍的儿童中，167例为原发性肌张力障碍，125例为继发性肌张力障碍，9例为肌阵挛性肌张力障碍。确定了三种可分离的术前表型（潜在变量），并与以下情况相关：1）最后一次随访时的BFMDRS-M；2）BFMDRS-M评分的相对变化；3）BFMDRS-D评分的相对变化。继发性肌张力障碍患者的表型为BFMDRS-M评分高且有躯干受累，在较年轻时接受DBS治疗，其术后BFMDRS-M评分较差。涉及躯干的原发性肌张力障碍儿童的BFMDRS-M和-D评分有更大改善。那些原发性肌张力障碍病程较短且患病时间占生命比例较小、接受苍白球DBS治疗的儿童，在长期随访中BFMDRS-D评分有更大改善。
在对儿童肌张力障碍DBS进行的全面、数据驱动的多变量分析中，作者确定了与不同结果相关的新的可分离患者表型。本报告的研究结果可能为DBS的手术候选资格提供参考。
