Treatment of multiple system atrophy using intravenous immunoglobulin

摘要: Multiple system atrophy (MSA) is a progressive neurodegenerative disorder of unknown etiology, manifesting as combination parkinsonism, cerebellar syndrome and dysautonomia. Disease-modifying therapies are unavailable. Activation microglia production toxic cytokines suggest role neuroinflammation in MSA pathogenesis. This pilot clinical trial evaluated safety tolerability intravenous immunoglobulin (IVIG) MSA. was single-arm interventional, single-center, open-label study. Interventions included monthly infusions the IVIG preparation Privigen®, dose 0.4 gram/kg, for 6 months. Primary outcome measures secondary preliminary efficacy IVIG. Unified Rating Scale (UMSARS) measured monthly. Quantitative brain imaging using 3T MRI performed before after treatment. Nine subjects were enrolled, seven (2 women 5 men, age range 55–64 years) completed protocol. There no serious adverse events. Systolic blood pressure increased during (p<0.05). Two participants dropped out from study because non-threatening skin rash. The UMSARS-I (activities daily living) USMARS-II (motor functions) improved significantly post-treatment. all (pre-treatment 23.9 ± 6.0 vs. post-treatment 19.0±5.9 (p=0.01). UMSARS-II subjects, unchanged 1 worsened 26.1±7.5 23.3±7.3 (p=0.025). MR results not different comparing pre- to Treatment with appears be safe, feasible well tolerated may improve functionality A larger, placebo-controlled needed.