Dupuy Sheena L, Tauhid Shahamat, Hurwitz Shelley, Chu Renxin, Yousuf Fawad, Bakshi Rohit
Department of Neurology, Laboratory for Neuroimaging Research, Partners MS Center, Harvard Medical School, Brigham and Women's Hospital, Boston, MA, USA.
Department of Medicine, Harvard Medical School, Brigham and Women's Hospital, Boston, MA, USA.
Neurol Ther. 2016 Dec;5(2):215-229. doi: 10.1007/s40120-016-0054-4. Epub 2016 Oct 15.
The objective of this pilot study was to compare cerebral gray matter (GM) atrophy over 1 year in patients starting dimethyl fumarate (DMF) for multiple sclerosis (MS) to that of patients on no disease-modifying treatment (noDMT). DMF is an established therapy for relapsing-remitting (RR) MS.
We retrospectively analyzed 20 patients with RRMS at the start of DMF [age (mean ± SD) 46.1 ± 10.2 years, Expanded Disability Status Scale (EDSS) score 1.1 ± 1.2, timed 25-foot walk (T25FW) 4.6 ± 0.8 s] and eight patients on noDMT (age 42.5 ± 6.6 years, EDSS 1.7 ± 1.1, T25FW 4.4 ± 0.6 s). Baseline and 1-year 3D T1-weighted 3T MRI was processed with automated pipelines (SIENA, FSL-FIRST) to assess percentage whole brain volume change (PBVC) and deep GM (DGM) atrophy. Group differences were assessed by analysis of covariance, with time between MRI scans as a covariate.
Over 1 year, the DMF group showed a lower rate of whole brain atrophy than the noDMT group (PBVC: -0.37 ± 0.49% vs. -1.04 ± 0.67%, p = 0.005). The DMF group also had less change in putamen volume (-0.06 ± 0.22 vs. -0.32 ± 0.28 ml, p = 0.02). There were no significant on-study differences between groups in caudate, globus pallidus, thalamus, total DGM volume, T2 lesion volume, EDSS, or T25FW (all p > 0.20).
These results suggest a treatment effect of DMF on GM atrophy appearing at 1 year after starting therapy. However, due to the retrospective study design and sample size, these findings should be considered preliminary, and require confirmation in future investigations.
Biogen.
本初步研究的目的是比较开始使用富马酸二甲酯(DMF)治疗多发性硬化症(MS)的患者与未接受疾病修饰治疗(noDMT)的患者在1年内脑灰质(GM)萎缩情况。DMF是复发缓解型(RR)MS的既定疗法。
我们回顾性分析了20例开始使用DMF治疗的RRMS患者[年龄(均值±标准差)46.1±10.2岁,扩展残疾状态量表(EDSS)评分1.1±1.2,25英尺步行时间(T25FW)4.6±0.8秒]以及8例接受noDMT治疗的患者(年龄42.5±6.6岁,EDSS 1.7±1.1,T25FW 4.4±0.6秒)。使用自动化流程(SIENA,FSL - FIRST)处理基线和1年时的3D T1加权3T MRI,以评估全脑体积变化百分比(PBVC)和深部GM(DGM)萎缩情况。通过协方差分析评估组间差异,将MRI扫描之间的时间作为协变量。
在1年期间,DMF组全脑萎缩率低于noDMT组(PBVC:-0.37±0.49%对-1.04±0.67%,p = 0.005)。DMF组壳核体积变化也较小(-0.06±0.22对-0.32±0.28 ml,p = 0.02)。在尾状核、苍白球、丘脑、总DGM体积、T2病变体积、EDSS或T25FW方面,组间在研究期间无显著差异(所有p>0.20)。
这些结果表明DMF对开始治疗1年后出现的GM萎缩有治疗效果。然而,由于回顾性研究设计和样本量,这些发现应被视为初步结果,需要在未来研究中得到证实。
百健公司。