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A randomized trial of mesenchymal stem cells in multiple system atrophy

Authors
 Phil Hyu Lee  ;  Ji E. Lee  ;  Han-Soo Kim  ;  Sook K. Song  ;  Hye Sun Lee  ;  Hyo Suk Nam  ;  June-Won Cheong  ;  Yong Jeong  ;  Hae-Jeong Park  ;  Dong Joon Kim  ;  Chung Mo Nam  ;  Jong Doo Lee  ;  Hyun Ok Kim  ;  Young H. Sohn 
Citation
 Annals of Neurology, Vol.72(1) : 32-40, 2012 
Journal Title
 Annals of Neurology 
ISSN
 0364-5134 
Issue Date
2012
Abstract
OBJECTIVE: Neuroprotective or regenerative strategies are invaluable in multiple system atrophy (MSA) due to its rapid progression with fatal prognosis. We evaluated the efficacy of autologous mesenchymal stem cells (MSC) in patients with MSA-cerebellar type (MSA-C). METHODS: Thirty-three patients with probable MSA-C and baseline unified MSA rating scale (UMSARS) scores ranging from 30 to 50 were randomly assigned to receive MSC (4 × 10(7) /injection) via intra-arterial and intravenous routes or placebo. The primary outcome was change in the total UMSARS scores from baseline throughout a 360-day follow-up period between groups. Secondary outcomes were changes in the UMSARS part II scores, cerebral glucose metabolism, gray matter density, and cognitive performance over a 360-day period. RESULTS: The mixed model analysis of neurological deficits revealed a significant interaction effect between treatment group and time, suggesting that the MSC group had a smaller increase in total and part II UMSARS scores compared with the placebo group (p = 0.047 and p = 0.008, respectively). Cerebral glucose metabolism and gray matter density at 360 days relative to the baseline were more extensively decreased in the cerebellum and the cerebral cortical areas, along with greater deterioration of frontal cognition in the placebo group compared with the MSC group. We found no serious adverse effects that were directly related to MSC treatment. However, intra-arterial infusion resulted in small ischemic lesions on magnetic resonance imaging. INTERPRETATION: MSC therapy could delay the progression of neurological deficits in patients with MSA-C, suggesting the potential of MSC therapy as a treatment candidate of MSA.
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/89525
DOI
10.1002/ana.23612
Appears in Collections:
1. Journal Papers (연구논문) > 1. College of Medicine (의과대학) > Dept. of Laboratory Medicine (진단검사의학교실)
1. Journal Papers (연구논문) > 1. College of Medicine (의과대학) > Dept. of Nuclear Medicine (핵의학교실)
1. Journal Papers (연구논문) > 1. College of Medicine (의과대학) > Dept. of Radiology (영상의학교실)
1. Journal Papers (연구논문) > 1. College of Medicine (의과대학) > Dept. of Preventive Medicine and Public Health (예방의학교실)
1. Journal Papers (연구논문) > 1. College of Medicine (의과대학) > Dept. of Internal Medicine (내과학교실)
1. Journal Papers (연구논문) > 1. College of Medicine (의과대학) > Dept. of Neurology (신경과학교실)
Yonsei Authors
김동준(Kim, Dong Joon)
김한수(Kim, Han Soo)
김현옥(Kim, Hyun Ok)
남정모(Nam, Jung Mo)
남효석(Nam, Hyo Suk)
박해정(Park, Hae Jeong)
손영호(Sohn, Young Ho)
이종두(Lee, Jong Doo)
이지은(Lee, Ji Eun)
이필휴(Lee, Phil Hyu)
정준원(Cheong, June-Won)
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Full Text
http://onlinelibrary.wiley.com/doi/10.1002/ana.23612/abstract
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