儿童白营养不良:12 个病例

S. Esseddiki, I. Chahid, F. Harim, A. Abkari, A. Bousfiha
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引用次数: 0

摘要

导言:白质营养不良症是一种以髓鞘受损为特征的罕见基因性疾病。白质营养不良症是一种罕见的以髓鞘损伤为特征的基因性疾病,它代表了大量的疾病,这些疾病在临床和生理病理方面具有异质性。材料和方法:我们报告了在摩洛哥卡萨布兰卡伊本-罗赫德大学(CHU Ibn Rochd)Abderrahim Harouchi 母婴医院神经儿科收集到的 12 例白质营养不良病例。结果:平均确诊年龄为2岁零9个月,以女性为主(性别比为0.33)。5例患者为近亲结婚。发病症状以精神运动性退行为主,有 8 例,癫痫发作有 4 例。运动症状突出:锥体综合征 5 例,肌张力低下 4 例,四肢瘫痪 1 例,构音障碍 1 例。4 例患者进行了腰椎穿刺,发现 3 例患者蛋白尿过多,糖尿和细胞学检查正常。我们发现 6 例患者的 Aryllsulfatase A 水平下降。所有病例均进行了影像学检查,结果显示为弥漫性白质脱髓鞘。通过核磁共振成像,我们对病例进行了分类,结果显示7例为偏色素性白营养不良,1例为腔隙性白营养不良,1例为雷弗森病,1例为卡纳文病,1例为科凯恩综合征,1例为肾上腺白营养不良。电图显示,2 例患者的神经传导速度下降。对 1 例患者进行了分子研究,发现其存在 hyccin 突变。结论由于白质营养不良症的临床异质性,其诊断通常比较困难。临床医生与基因学家的合作可能是改善诊断和治疗管理的关键点。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Leukodytrophy in Children: 12 Cases
Introduction: Leukodystrophies are a rare geneLc disease characterized by damage of the myelin sheath. They represent a large number of diseases that are heterogeneous by their clinical and physiopathological aspects. Material and Methods: We report 12 cases of leukodystrophies collected at the Neuropediatric Unit of Abderrahim Harouchi Mother and Child Hospital CHU Ibn Rochd, Casablanca, Morocco. Results: The average age of diagnosis was 2 years and 9 months, with a predominance of females (sex raLo:0.33). Consanguinity was found in 5 cases. The onset symptomatology was dominated by psychomotor regression, found in 8 paLents, and seizures in 4 paLents. Motor signs were in the foreground: pyramidal syndrome in 5 cases, hypotonia in 4 cases, tetraparesis in 1 case, dysarthria in 1 case. The lumbar puncture, carried out in 4 paLents, revealed hyperproteinorachy in 3 cases, glycorachy and cytological study were normal. We noLced a decreased level of Aryllsulfatase A in 6 cases. Imaging was performed in all paLents and showed diffuse white ma]er demyelinaLon. MRI allowed us to classify our cases and showed 7 cases of metachromaLc leukodystrophy, 1 case of cavitary leukodystrophy, 1 case of Refsum disease, 1 case of Canavan disease, 1 case of Cockaynes syndrome and 1 case of adrenoleukodystrophy. The electroneuromyogram showed a decrease in nerve conducLon velociLes in 2 cases. Molecular study was performed in one paLent finding a hyccin mutaLon. Conclusion: The diagnosis of leukodystrophies is o_en difficult because of their clinical heterogeneity. The partnership of clinicians with geneLcists may be the key point to improve diagnosis and therapeuLc management.
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