Finsterer Josef
Neurology, Neurology and Neurophysiology Center, Vienna, AUT.
Cureus. 2025 Aug 17;17(8):e90315. doi: 10.7759/cureus.90315. eCollection 2025 Aug.
To the best of our knowledge, autosomal dominant Charcot-Marie-Tooth disease type 1B (CMT1B) due to the c.1A>G variant in myelin protein zero (MPZ) has not yet been reported. The patient was a 56-year-old man with slowly progressive atrophy of the distal muscles of the lower limbs that started in his forties. He later developed transient numbness in both feet. One year before presentation, he noticed paresthesia in the distal lower limbs and easy fatigability when walking uphill. His family history revealed hereditary neuropathy in his mother and her two sisters. Genetic testing of his mother and an aunt revealed the new variant c.1A>G in MPZ (p.Met1Val). Clinical neurological examination revealed myopia, hypoacusis, absent tendon reflexes on the upper and lower limbs, mild weakening of the left thumb and right foot extensors, hypoesthesia and hypoalgesia on both soles and dorsum of the feet, and mild pallhypesthesia on the lower limbs. Nerve conduction studies (NCS) revealed severe sensorimotor axonal neuropathy. The index patient also carried the mutation identified in his mother and aunt. The MPZ variant c.1A>G manifests phenotypically with late-onset CMT1B. Neurologists should be aware that hereditary neuropathy can have a late onset and a slowly progressive course over a number of years, with the ability to walk unimpaired into old age.
据我们所知,尚未有因髓磷脂蛋白零(MPZ)基因c.1A>G变异导致的常染色体显性遗传性1B型夏科-马里-图思病(CMT1B)的相关报道。该患者为一名56岁男性,40多岁时开始出现双下肢远端肌肉缓慢进行性萎缩。后来他双脚出现短暂麻木。就诊前一年,他注意到下肢远端感觉异常,上坡行走时容易疲劳。他的家族史显示其母亲和她的两个姐妹患有遗传性神经病变。对他的母亲和一位阿姨进行基因检测,发现MPZ基因存在新的c.1A>G变异(p.Met1Val)。临床神经学检查发现患者有近视、听力减退、上下肢腱反射消失、左拇指和右足背伸肌轻度减弱、双足底和足背感觉减退和痛觉减退,以及下肢轻度触觉减退。神经传导研究(NCS)显示为严重的感觉运动轴索性神经病变。该索引患者也携带了在其母亲和阿姨身上发现的突变。MPZ基因c.1A>G变异在表型上表现为迟发性CMT1B。神经科医生应意识到,遗传性神经病变可能起病较晚,且在数年中呈缓慢进展过程,患者有可能在步入老年时仍能正常行走。