Ma Yuelin, Teng Qing, Zhang Yiran, Zhang Songyun
Department of Endocrinology, The second Hospital of Hebei Medical University, Shijiazhuang, Hebei, China.
School of First Clinical Medical College, Southern Medical University, Guangzhou, Guangdong, China.
Intractable Rare Dis Res. 2020 Nov;9(4):187-195. doi: 10.5582/irdr.2020.03054.
Porphyrias are a group of inherited metabolic diseases that include eight types, each of which is caused by a mutation that affects an enzyme of the heme biosynthetic pathway. When an enzyme defect has physiological significance, it leads to overproduction of pathway precursors prior to the defective step. The partial absence of the third enzyme in the heme biosynthetic pathway, porphobilinogen deaminase (PBGD) also known as hydroxymethylbilane synthase (HMBS), results in acute intermittent porphyria (AIP), which affects mainly women. Subjects who had AIP symptoms were deemed to have manifest AIP (MAIP). Clinical manifestations are usually diverse and non-specific. Acute AIP episodes may present with abdominal pain, nausea, and vomiting, and repeated episodes may result in a series of chronic injuries. Therefore, studying the mechanisms of acute and chronic manifestations of AIP is of great significance. This review aims to summarize the possible mechanisms of acute and chronic manifestations in patients with AIP.
卟啉病是一组遗传性代谢疾病,包括八种类型,每种类型都由影响血红素生物合成途径中一种酶的突变引起。当酶缺陷具有生理意义时,它会导致缺陷步骤之前的途径前体过量产生。血红素生物合成途径中第三种酶卟胆原脱氨酶(PBGD)也称为羟甲基胆色素原合酶(HMBS)部分缺乏,会导致急性间歇性卟啉病(AIP),主要影响女性。有AIP症状的受试者被认为患有显性AIP(MAIP)。临床表现通常多样且不具特异性。急性AIP发作可能表现为腹痛、恶心和呕吐,反复发可能导致一系列慢性损伤。因此,研究AIP急性和慢性表现的机制具有重要意义。本综述旨在总结AIP患者急性和慢性表现的可能机制。