Department of Dermatology, Euregional Porphyria Center Maastricht, GROW-School for Oncology and Developmental Biology, Maastricht University Medical Center (MUMC), Maastricht, The Netherlands.
Best Pract Res Clin Gastroenterol. 2010 Oct;24(5):735-45. doi: 10.1016/j.bpg.2010.07.002.
Porphyria cutanea tarda (PCT) is the most frequent type of porphyria worldwide and results from a catalytic deficiency of uroporphyrinogen decarboxylase (UROD), the fifth enzyme in heme biosynthesis. At least two different types of PCT are currently distinguished: an acquired variant, also referred to as sporadic or type I PCT, in which the enzymatic deficiency is limited to the liver; and an autosomal dominantly inherited form, also known as familial or type II PCT, in which there is a decrease of enzymatic activity in all tissues. The cutaneous findings include increased photosensitivity, skin fragility, blistering, erosions, crusts, and miliae on the sun-exposed areas of the body. Additionally, hyperpigmentation, hypertrichosis, sclerodermoid plaques, and scarring alopecia might be observed. In patients with type I PCT, there is a significant association with liver disease that can be triggered by genetic and environmental factors, such as alcohol abuse, iron overload, haemochromatosis, polychlorinated hydrocarbons, and hepatitis C virus infection. The diagnosis of PCT can be made based on the skin symptoms, a characteristic urinary porphyrin excretion profile, and the detection of isocoproporphyrin in the feces. In red blood cells of individuals with type II PCT, UROD activity is decreased by approximately 50% due to heterozygous mutations in the UROD gene. Here we provide an update on clinical, diagnostic and therapeutic aspects of PCT, a disorder that affects both skin and liver.
迟发性皮肤卟啉病(PCT)是全球最常见的卟啉病类型,由尿卟啉原脱羧酶(UROD)的催化缺陷引起,UROD 是血红素生物合成的第五种酶。目前至少区分了两种不同类型的 PCT:一种是获得性变异型,也称为散发性或 I 型 PCT,其中酶缺乏仅限于肝脏;另一种是常染色体显性遗传形式,也称为家族性或 II 型 PCT,其中所有组织的酶活性均降低。皮肤表现包括光敏性增加、皮肤脆弱、水疱、糜烂、结痂和身体暴露部位的粟粒疹。此外,还可能观察到色素沉着过度、多毛症、硬皮病样斑块和瘢痕性脱发。在 I 型 PCT 患者中,与肝脏疾病有显著关联,可能由遗传和环境因素触发,如酗酒、铁过载、血色病、多氯联苯和丙型肝炎病毒感染。根据皮肤症状、特征性尿卟啉排泄谱以及粪便中异粪卟啉的检测,可诊断 PCT。在 II 型 PCT 个体的红细胞中,由于 UROD 基因的杂合突变,UROD 活性降低约 50%。本文就 PCT 的临床、诊断和治疗方面提供最新进展,PCT 影响皮肤和肝脏。