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[眼皮肤白化病和眼白化病]

[Oculocutaneous and ocular albinism].

作者信息

Kubasch A S, Meurer M

机构信息

UniversitätsCentrum für Seltene Erkrankungen, Universitätsklinikum Carl Gustav Carus, Technische Universität Dresden, Dresden, Deutschland.

Stiftung Hochschulmedizin Dresden, Universitätsklinikum Carl Gustav Carus, Technische Universität Dresden, Dresden, Deutschland.

出版信息

Hautarzt. 2017 Nov;68(11):867-875. doi: 10.1007/s00105-017-4061-x.

Abstract

Albinism can be divided into oculocutaneous albinism (OCA) and ocular albinism (OA). In the differential diagnostics these can be distinguished from rarer syndromes with partial albinism, which are frequently associated with susceptibility to infections and neurological symptoms. The OCA is an autosomal recessive inherited disease of melanin biosynthesis, which leads to complete or partial loss of melanin in the skin, hair follicles and eyes. Of the seven currently known subtypes (OCA 1-7), four are well-characterized (OCA 1-4). These are based on gene mutations, which code for tyrosinase, a key enzyme in melanin synthesis and for further proteins. These play an important role in the catalytic activity of tyrosinase and the structure and function of melanosomes. In the presence of these subtypes, the clinical symptoms and the course of the disease show a pronounced variability, especially in the type and extent of pigmentation of the skin and hair as well as the severity of eye involvement, which makes the phenotypic classification difficult. Treatment priorities are a consistent protection from UV light for prophylaxis against skin cancer and regular preventive investigations. The ocular alterations typical for albinism necessitate timely diagnostics and care by institutions specialized in ophthalmology. Novel strategies for systemic treatment of subtypes of albinism are in preclinical testing. The OA without skin involvement shows X‑linked inheritance, is much rarer and is characterized by reduced pigmentation of the retina and iris, nystagmus and macular hypoplasia, sometimes with substantial loss of visual acuity. The typical ocular symptoms of OA can be manifested to a varying extent in all forms of OCA.

摘要

白化病可分为眼皮肤白化病(OCA)和眼白化病(OA)。在鉴别诊断中,这些疾病可与罕见的部分白化病综合征相区分,后者常伴有易感染性和神经症状。OCA是一种常染色体隐性遗传的黑色素生物合成疾病,会导致皮肤、毛囊和眼睛中黑色素完全或部分缺失。在目前已知的七种亚型(OCA 1 - 7)中,四种已得到充分表征(OCA 1 - 4)。这些亚型基于基因突变,这些基因编码酪氨酸酶(黑色素合成中的关键酶)以及其他蛋白质。它们在酪氨酸酶的催化活性以及黑素小体的结构和功能中发挥重要作用。在这些亚型存在的情况下,临床症状和疾病进程表现出明显的变异性,尤其是在皮肤和头发色素沉着的类型和程度以及眼部受累的严重程度方面,这使得表型分类变得困难。治疗重点是持续保护免受紫外线照射以预防皮肤癌,并定期进行预防性检查。白化病典型的眼部改变需要由眼科专科医院及时进行诊断和治疗。白化病亚型的全身治疗新策略正在进行临床前测试。无皮肤受累的OA表现为X连锁遗传,更为罕见,其特征是视网膜和虹膜色素沉着减少、眼球震颤和黄斑发育不全,有时伴有视力大幅丧失。OA的典型眼部症状在所有形式的OCA中都可能有不同程度的表现。

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