臨床神経学

<シンポジウム04―4>神経内科領域における前方向的コホート研究から見えてきたもの

MSAに対するdisease-modifying therapy実現のための臨床治験デザイン

市川 弥生子 ,後藤 順 ,中原 康雄 ,三井 純 ,辻 省次

JAMSAC(Japan MSA research consortium)
東京大学神経内科〔〒113―8655 東京都文京区本郷7―3―1〕

Multiple system atrophy (MSA) is an adult-onset, progressive neurodegenerative disorder which is clinically characterized by various combinations of cerebellar ataxia, Parkinsonism, autonomic dysfunction and pyramidal signs. MSA is known as a sporadic disease, however, multiplex families with MSA suggest a genetic predisposition to MSA. The advanced genome research will clarify the pathogenetic mechanisms of MSA, and the diseasemodifying therapy of MSA may be available in the future. To clarify the natural history of MSA for the future therapeutic trials, and to elucidate the molecular pathogenetic mechanisms of MSA, JAMSAC (Japan MSA research consortium), a nationwide consortium, was established in 2003. In the view of the future therapeutic trial for MSA, it is essential to design appropriate end point, sample size, duration of the trial. And inclusion criteria are also important for effective therapeutic trial. We conducted a cross-sectional study on 225 MSA patients using unified multiple system atrophy rating scale (UMSARS). As inclusion criteria, we employed additional criteria based on specific MRI findings to recruit earlier stage patients. Sample size estimation from the longitudinal study suggested we need sensitive outcome measures beside UMSARS. JAMSAC is planning to a longitudinal study for natural history of MSA in Japan.
Full Text of this Article in Japanese PDF (211K)

(臨床神経, 51:910−913, 2011)
key words:多系統萎縮症,自然史前向き研究,JAMSAC,臨床治験,必要症例数

(受付日:2011年5月19日)