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[先天性代谢缺陷的临床疑似情况]

[Clinical suspicion of inborn errors of metabolism].

作者信息

Sperl W

机构信息

Universitäts-Kinderklinik, Innsbruck.

出版信息

Wien Klin Wochenschr. 1992;104(16):497-502.

PMID:1413807
Abstract

A number of inherited metabolic disorders are diagnosed by means of the nationwide newborn screening programme, usually before the first clinical signs occur. As for the rest of the varied metabolic disorders, knowledge and intuition of the paediatrician is a prerequisite for selection of patients for further metabolic investigation (selective screening procedure). Clinical symptoms of the most important metabolic diseases can be classified according to their pathophysiological background as: "intoxication type, energy deficiency type, storage type, neurodegenerative type". Especially in the first year of life, clinical features are unspecific: psychomotoric retardation, muscular hypotonia, cerebral convulsions, recurrent vomiting, sepsis-like conditions. In these cases indication for metabolic screening is broad. Especially in older children some clinical symptoms can be specific for a metabolic disorder: distinctive odour of urine, changes in hair, skin or eyes, organomegaly, skeletal changes. Recently, Reye-like syndrome, stridor, macrocephaly and vague, cerebral ischaemic episodes have been described in association with a metabolic defect. In conclusion, experience has shown that only a small number of metabolic disorders will be diagnosed from the typical clinical picture alone. In most cases a selective screening procedure leads to diagnosis because initial symptoms are unspecific.

摘要

许多遗传性代谢紊乱是通过全国性的新生儿筛查计划来诊断的,通常在出现首个临床症状之前。至于其他各种代谢紊乱,儿科医生的知识和直觉是选择患者进行进一步代谢检查(选择性筛查程序)的先决条件。最重要的代谢疾病的临床症状可根据其病理生理背景分类为:“中毒型、能量缺乏型、储存型、神经退行性型”。特别是在生命的第一年,临床特征不具特异性:精神运动发育迟缓、肌张力低下、惊厥、反复呕吐、类似败血症的情况。在这些情况下,代谢筛查的指征很广泛。特别是在大龄儿童中,一些临床症状可能对某种代谢紊乱具有特异性:尿液有特殊气味、毛发、皮肤或眼睛变化、器官肿大、骨骼改变。最近,已描述了与代谢缺陷相关的类瑞氏综合征、喘鸣、巨头畸形和模糊的脑缺血发作。总之,经验表明,仅从典型的临床症状中只能诊断出少数代谢紊乱。在大多数情况下,由于初始症状不具特异性,选择性筛查程序可导致诊断。

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