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科斯特夫综合征的非典型表现——慢波睡眠期严重精神运动障碍及癫痫性电持续状态。

Atypical presentation of Costeff syndrome-severe psychomotor involvement and electrical status epilepticus during slow wave sleep.

作者信息

Carmi Nirit, Lev Dorit, Leshinsky-Silver Esther, Anikster Yair, Blumkin Lubov, Kivity Sara, Lerman-Sagie Tally, Zerem Ayelet

机构信息

Pediatric Neurology Unit, Wolfson Medical Center, Holon, Israel; Metabolic-Neurogenetic Service, Wolfson Medical Center, Holon, Israel.

Metabolic-Neurogenetic Service, Wolfson Medical Center, Holon, Israel; Institute of Medical Genetics, Wolfson Medical Center, Holon, Israel; Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel.

出版信息

Eur J Paediatr Neurol. 2015 Nov;19(6):733-6. doi: 10.1016/j.ejpn.2015.06.006. Epub 2015 Jul 9.

Abstract

BACKGROUND

Costeff syndrome or OPA3-related 3-methylglutaconic aciduria is an autosomal recessive neurodegenerative disorder characterized by early onset optic atrophy and choreoathetosis with later onset of ataxia and spasticity. Costeff syndrome is prevalent among Iraqi Jews.

METHODS

We describe a 5 year old girl from Syrian Jewish origin with an atypical presentation of Costeff syndrome.

RESULTS

The patient presented with asymmetric optic atrophy, severe dystonia and choreoathetosis and global developmental regression at the age of 7 months; no achievement of independent walking and only minimal speech; and appearance of electrical status epilepticus during slow wave sleep in the second year of life with further deterioration. She harbors the classic mutation (c.143-1G > C) in the OPA3 gene.

CONCLUSION

Costeff syndrome may present in an atypical manner regarding the ethnic origin, clinical manifestations and co-occurrence of epilepsy. Mutations in OPA3 should be evaluated in all cases presenting with the core features of typical Costeff syndrome.

摘要

背景

科斯特夫综合征或OPA3相关的3-甲基戊二酸尿症是一种常染色体隐性神经退行性疾病,其特征为早发性视神经萎缩和舞蹈手足徐动症,后期出现共济失调和痉挛。科斯特夫综合征在伊拉克犹太人中很常见。

方法

我们描述了一名来自叙利亚犹太裔的5岁女孩,其表现为非典型的科斯特夫综合征。

结果

该患者在7个月大时出现不对称性视神经萎缩、严重肌张力障碍和舞蹈手足徐动症以及全面发育迟缓;未能实现独立行走,仅能说极少的话;在1岁时慢波睡眠期间出现癫痫持续状态,病情进一步恶化。她携带OPA3基因的经典突变(c.143-1G>C)。

结论

科斯特夫综合征在种族起源、临床表现和癫痫共发方面可能表现为非典型。对于所有具有典型科斯特夫综合征核心特征的病例,均应评估OPA3基因的突变情况。

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