The Efficacy and Predictors of Using GPi-DBS to Treat Early-Onset Dystonia: An Individual Patient Analysis.

Wenxiu Chen,Guohui Lu,Houyou Fan,Fushun Wang

doi:10.1155/2021/9924639

Abstract

Objective To compare the efficacy in patients with different genotypes, identify the potential predictive factors, and summarize the complications of globus pallidus deep brain stimulation (GPi-DBS) treating early-onset dystonia. Methods Three electronic databases (PubMed, Embase, and Cochrane databases) were searched with no publication data restriction. The primary outcomes were the improvements in Burke–Fahn–Marsden Dystonia Rating Scale motor (BFMDRS-M) and disability (BFMDRS-D) score. Pearson's correlation coefficients and a metaregression analysis were used to identify the potential predictive factors. This article was registered in Prospero (CRD42020188527). Results Fifty-four studies (231 patients) were included. Patients showed significant improvement rate in BFMDRS-M (60.6%, p < 0.001) and BFMDRS-D (57.5%, p < 0.001) scores after treatment with GPi-DBS. BFMDRS-M score improved greater in the DYT-1-positive (p = 0.001) and DYT-11-positive (p = 0.008) patients compared to DYT-6-positive patients. BFMDRS-D score improved greater in the DYT-11 (+) compared to DYT-6 (+) patients (p = 0.010). The relative change of BFMDRS-M (p = 0.002) and BFMDRS-D (p = 0.010) scores was negatively correlated with preoperative BFMDRS-M score. In the metaregression analysis, the best predictive model showed that preoperative BFMDRS-M, disease duration (p = 0.047), and the age at symptom onset (p = 0.027) were important. Conclusion Patients with early-onset dystonia have a significant effect after GPi-DBS treatment, and DYT-1 (+) and DYT-11 (+) patients are better candidates for GPi-DBS. Lower preoperative score, later age of onset, and an earlier age at surgery probably predict better clinical outcomes.

Highlights

Early-onset dystonia is one type of dystonia, characterized by onset age ≤ 26, often presents with the onset of lower or upper limb symptoms, and progresses to other parts of the body [1]
A sample size of 231 patients was distributed into DYT-1 (n = 101), DYT-6 (n = 27), and DYT-11 (n = 32) mutations
The results showed that there were significant differences in the BFMDRS-M preoperative and postoperative scores between the DYT-6 (+) and DYT-11 (+) groups (49.5 vs. 23.9, p < 0:001, for preoperative scores and 24.7 vs. 7.0, p < 0:001, for postoperative scores, respectively), with significant differences in the mean improvement (52% vs. 68.7%, p = 0:008)

Summary

Introduction

Early-onset dystonia is one type of dystonia, characterized by onset age ≤ 26, often presents with the onset of lower or upper limb symptoms, and progresses to other parts of the body [1]. DYT-1 (+) patients present onset symptoms in childhood or adolescence, typically with dystonia of a single limb that often spreads within 1-3 years to involve all four limbs [3, 4]. The onset age of DYT-6 (+) patients is during childhood and adolescence, but more than half of patients started onset symptoms earlier than adolescence. 25% of DYT-6 (+) patients present with cervical dystonia [5]. Patients with DYT-11 genotype present with myoclonus, dystonia, or both, but most patients present with myoclonus in childhood or adolescence [6, 7]. The patients with DYT-28 (+) are registered as an early-onset generalized dystonia with age at onset of symptoms generally between 4 and 6 years old [8]

Methods

Discussion

Conclusion