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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.
Full Text
http://onlinelibrary.wiley.com/doi/10.1002/ana.23612/abstract
DOI
10.1002/ana.23612
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Laboratory Medicine (진단검사의학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Nuclear Medicine (핵의학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Radiology (영상의학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Preventive Medicine and Public Health (예방의학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Internal Medicine (내과학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Neurology (신경과학교실) > 1. Journal Papers
Yonsei Authors
Kim, Dong Joon(김동준) ORCID logo https://orcid.org/0000-0002-7035-087X
Kim, Han Soo(김한수)
Kim, Hyun Ok(김현옥) ORCID logo https://orcid.org/0000-0002-4964-1963
Nam, Jung Mo(남정모) ORCID logo https://orcid.org/0000-0003-0985-0928
Nam, Hyo Suk(남효석) ORCID logo https://orcid.org/0000-0002-4415-3995
Park, Hae Jeong(박해정) ORCID logo https://orcid.org/0000-0002-4633-0756
Sohn, Young Ho(손영호) ORCID logo https://orcid.org/0000-0001-6533-2610
Lee, Jong Doo(이종두)
Lee, Ji Eun(이지은)
Lee, Phil Hyu(이필휴) ORCID logo https://orcid.org/0000-0001-9931-8462
Cheong, June-Won(정준원) ORCID logo https://orcid.org/0000-0002-1744-0921
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URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/89525
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