Suppr超能文献

Ⅰ 型戊二酸尿症的诊断和治疗——修订建议。

Diagnosis and management of glutaric aciduria type I--revised recommendations.

机构信息

Department of General Pediatrics, Division of Inborn Metabolic Diseases, University Children's Hospital, Im Neuenheimer Feld 430, 69120 Heidelberg, Germany.

出版信息

J Inherit Metab Dis. 2011 Jun;34(3):677-94. doi: 10.1007/s10545-011-9289-5. Epub 2011 Mar 23.

Abstract

Glutaric aciduria type I (synonym, glutaric acidemia type I) is a rare organic aciduria. Untreated patients characteristically develop dystonia during infancy resulting in a high morbidity and mortality. The neuropathological correlate is striatal injury which results from encephalopathic crises precipitated by infectious diseases, immunizations and surgery during a finite period of brain development, or develops insidiously without clinically apparent crises. Glutaric aciduria type I is caused by inherited deficiency of glutaryl-CoA dehydrogenase which is involved in the catabolic pathways of L-lysine, L-hydroxylysine and L-tryptophan. This defect gives rise to elevated glutaric acid, 3-hydroxyglutaric acid, glutaconic acid, and glutarylcarnitine which can be detected by gas chromatography/mass spectrometry (organic acids) or tandem mass spectrometry (acylcarnitines). Glutaric aciduria type I is included in the panel of diseases that are identified by expanded newborn screening in some countries. It has been shown that in the majority of neonatally diagnosed patients striatal injury can be prevented by combined metabolic treatment. Metabolic treatment that includes a low lysine diet, carnitine supplementation and intensified emergency treatment during acute episodes of intercurrent illness should be introduced and monitored by an experienced interdisciplinary team. However, initiation of treatment after the onset of symptoms is generally not effective in preventing permanent damage. Secondary dystonia is often difficult to treat, and the efficacy of available drugs cannot be predicted precisely in individual patients. The major aim of this revision is to re-evaluate the previous diagnostic and therapeutic recommendations for patients with this disease and incorporate new research findings into the guideline.

摘要

I 型戊二酸血症(同义词:I 型戊二酸血症)是一种罕见的有机酸血症。未经治疗的患者在婴儿期通常会出现肌张力障碍,导致发病率和死亡率较高。神经病理学的相关表现是纹状体损伤,这是由脑发育有限期内的感染性疾病、免疫接种和手术引起的脑病性危象,或在无明显临床危象的情况下进行性发展引起的。I 型戊二酸血症是由于谷氨酸酰辅酶 A 脱氢酶的遗传性缺乏引起的,该酶参与 L-赖氨酸、L-羟赖氨酸和 L-色氨酸的分解代谢途径。这种缺陷导致戊二酸、3-羟基戊二酸、戊二酸酸和戊二酰肉碱升高,这些物质可以通过气相色谱/质谱(有机酸)或串联质谱(酰基肉碱)检测到。在一些国家,I 型戊二酸血症已被纳入扩展新生儿筛查所识别的疾病之列。研究表明,在大多数新生儿确诊的患者中,通过联合代谢治疗可以预防纹状体损伤。代谢治疗包括低赖氨酸饮食、肉碱补充和在急性并发疾病期间加强急救治疗,应由经验丰富的跨学科团队来实施和监测。然而,在症状出现后开始治疗通常无法有效预防永久性损伤。继发性肌张力障碍通常难以治疗,并且无法在个体患者中精确预测现有药物的疗效。本次修订的主要目的是重新评估该疾病患者的先前诊断和治疗建议,并将新的研究结果纳入指南。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/675a/3109243/5bab79154b51/10545_2011_9289_Fig1_HTML.jpg

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验