Kitao Ruriko, Honma Yutaka, Hashiguchi Akihiro, Mizoguchi Kouichi, Takashima Hiroshi, Komori Tetsuo
Department of Neurology, National Hospital Organization Hakone Hospital.
Department of Neurology, National Hospital Organization Shizuoka Fuji Hospital (Present address: Department of Neurology, National Hospital Organization Shizuoka Medical Center).
Rinsho Shinkeigaku. 2020 Jul 31;60(7):466-472. doi: 10.5692/clinicalneurol.60.cn-001415. Epub 2020 Jun 13.
The patient was a 29-year-old male. He took his first steps at two-and-a-half years old, but his physical strength deteriorated and he became non-ambulatory at 12 years old. He had respiratory failure at the age of 20, and finally underwent tracheostomy with invasive positive-pressure ventilation (TPPV). He showed distal dominant muscle weakness and atrophy, including the face. Spinal scoliosis was recognized. He had peripheral predominance of sensory disorders. Nerve conduction studies showed a decrease of compound muscle action potential and a reduction of motor nerve conduction velocity. Sensory nerve action potential was not evoked. In genetic analysis, c.23 C> T (p. T8M) heterozygous mutation was found in the senataxin gene (SETX). Although SETX is a causative gene of familial amyotrophic lateral sclerosis type 4 (ALS4), this case suggests that SETX mutation can also cause motor and sensory polyneuropathy.
该患者为一名29岁男性。他在两岁半时开始走路,但体力逐渐下降,12岁时无法行走。20岁时出现呼吸衰竭,最终接受了气管切开术并进行有创正压通气(TPPV)。他表现出以远端为主的肌肉无力和萎缩,包括面部。发现有脊柱侧弯。他的感觉障碍以周围为主。神经传导研究显示复合肌肉动作电位降低,运动神经传导速度减慢。未引出感觉神经动作电位。基因分析发现,senataxin基因(SETX)存在c.23 C>T(p.T8M)杂合突变。虽然SETX是4型家族性肌萎缩侧索硬化症(ALS4)的致病基因,但该病例表明SETX突变也可导致运动和感觉性多发性神经病。