Treatment of multiple system atrophy using intravenous immunoglobulin.
Treatment of multiple system atrophy using intravenous immunoglobulin.
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DOI：
10.1186/1471-2377-12-131
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发表时间：
2012-11-01
期刊：
影响因子：
2.6
通讯作者：
Novak V
中科院分区：
文献类型：
--
作者：
Novak P;Williams A;Ravin P;Zurkiya O;Abduljalil A;Novak V
多系统萎缩症（MSA）是一种病因不明的进行性神经退行性疾病，表现为帕金森综合征、小脑综合征和自主神经功能障碍。没有改善疾病的治疗方法。小胶质细胞的活化和毒性细胞因子的产生表明神经炎症在MSA发病机制中的作用。该初步临床试验评价了静脉注射免疫球蛋白（IVIG）在MSA中的安全性和耐受性。这是一项单组干预性、单中心、开放标签的初步研究。干预措施包括每月输注IVIG制剂Privigen®，剂量为0.4 g/kg，持续6个月。主要结局指标评价IVIG的安全性，次要结局指标评价IVIG的初步疗效。每月测量统一MSA评定量表（UMSRS）。治疗前后分别进行3 T MRI定量脑成像。入组了9例受试者，其中7例（2例女性和5例男性，年龄范围55-64岁）完成了方案。未发生严重不良事件。IVIG输注期间，收缩压升高（p<0.05）。两名参与者因为没有威胁的皮疹而退出研究。治疗后UMSARS-I（日常生活活动）和USMARS-II（运动功能）显着改善。所有受试者的UMSARS-I均改善（治疗前23.9 ± 6.0 vs.治疗后19.0±5.9（p=0.01））。5例受试者的UMSARS-II改善，1例无变化，1例恶化（治疗前26.1±7.5 vs.治疗后23.3±7.3（p=0.025））。磁共振成像结果与治疗前后相比无差异。IVIG治疗似乎是安全的，可行的，耐受性良好，并可能改善MSA的功能。需要进行更大规模的安慰剂对照研究。
Multiple system atrophy (MSA) is a progressive neurodegenerative disorder of unknown etiology, manifesting as combination of parkinsonism, cerebellar syndrome and dysautonomia. Disease-modifying therapies are unavailable. Activation of microglia and production of toxic cytokines suggest a role of neuroinflammation in MSA pathogenesis. This pilot clinical trial evaluated safety and tolerability of intravenous immunoglobulin (IVIG) in MSA. This was a single-arm interventional, single-center, open-label pilot study. Interventions included monthly infusions of the IVIG preparation Privigen®, dose 0.4 gram/kg, for 6 months. Primary outcome measures evaluated safety and secondary outcome measures evaluated preliminary efficacy of IVIG. Unified MSA Rating Scale (UMSARS) was measured monthly. Quantitative brain imaging using 3T MRI was performed before and after treatment. Nine subjects were enrolled, and seven (2 women and 5 men, age range 55–64 years) completed the protocol. There were no serious adverse events. Systolic blood pressure increased during IVIG infusions (p<0.05). Two participants dropped out from the study because of a non-threatening skin rash. The UMSARS-I (activities of daily living) and USMARS-II (motor functions) improved significantly post-treatment. UMSARS-I improved in all subjects (pre-treatment 23.9 ± 6.0 vs. post-treatment 19.0±5.9 (p=0.01). UMSARS-II improved in 5 subjects, was unchanged in 1 and worsened in 1 (pre-treatment 26.1±7.5 vs. post-treatment 23.3±7.3 (p=0.025). The MR imaging results were not different comparing pre- to post-treatment. Treatment with IVIG appears to be safe, feasible and well tolerated and may improve functionality in MSA. A larger, placebo-controlled study is needed.
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