Alkaptonuria: Clinical Spectrum of a Diagnosed Case in Bahrain With a Literature Review

尿黑酸症:巴林一例确诊病例的临床表现及文献综述

阅读:1

Abstract

Alkaptonuria (AKU) is a rare metabolic condition caused by mutations within a gene coding for homogentisate 1,2 dioxygenase enzyme involved in the tyrosine catabolism pathway. This mutation will result in the accumulation of homogentisic acid (HGA) in the body. AKU is a multi-systemic slowly progressing disease. The onset of its clinical presentation may vary based on the extensive disposition of the HGA. Initially, it might be asymptomatic, and symptoms usually appear in the second or third decades due to the formation of HGA, melanin compounds that accumulate in the cartilage leading to ochronosis. Furthermore, by the fourth or fifth decade, ochronotic arthropathy occurs, along with other extra-articular complications such as cardiovascular manifestations (e.g., valvular heart disease), renal and prostatic stones, and hypothyroidism. Management of this condition is mainly symptomatic, focusing on the treatment of its complications. Recently, the use of nitisinone (NTBC) has shown stabilization of disease manifestations. In this report, we present in detail the first AKU-diagnosed patient, including the clinical presentations, radiological findings, genetic results, and clinical outcomes, from the main tertiary hospital in Bahrain. Moreover, we conducted a thorough literature review on this rare condition.

特别声明

1、本页面内容包含部分的内容是基于公开信息的合理引用;引用内容仅为补充信息,不代表本站立场。

2、若认为本页面引用内容涉及侵权,请及时与本站联系,我们将第一时间处理。

3、其他媒体/个人如需使用本页面原创内容,需注明“来源:[生知库]”并获得授权;使用引用内容的,需自行联系原作者获得许可。

4、投稿及合作请联系:info@biocloudy.com。