Albers James W, Fink John K
Department of Neurology, 1C325/0032 University Hospital, University of Michigan Health System, 1500 East Medical Center Drive, Ann Arbor 48109-0032, USA.
Muscle Nerve. 2004 Oct;30(4):410-22. doi: 10.1002/mus.20137.
The hepatic porphyrias are a group of rare metabolic disorders characterized by enzymatic defects in the biosynthesis of heme, a metalloporphyrin that is the principal product of porphyrin metabolism. The hepatic porphyrias are genetically transmitted as autosomal-dominant disorders with variable expression that produce a particularly severe form of neuropathy. Most medical students readily recognize acute attacks of porphyria when the classic triad of abdominal pain, psychosis, and neuropathy is present. Yet, porphyric neuropathy is a source of confusion in practice, and patients with porphyria rarely receive the correct diagnosis early in the course of the illness. Porphyric neuropathy is manifest by symptoms, signs, and cerebrospinal fluid abnormalities resembling acute Guillain-Barré syndrome. However, accompanying psychological features, a proximal predilection of asymmetric weakness, and electrodiagnostic findings indicative of an axonal polyradiculopathy or neuronopathy all suggest the diagnosis of porphyria. Confirmation of the diagnosis depends on use of appropriate laboratory studies. The underlying pathophysiology of porphyric neuropathy has not been established, but it may be related to direct neurotoxicity of elevated levels of delta-aminolevulinic acid. The severity of the neuropathy and the availability of potential treatments, including avoidance of provocative factors, make identification important.
肝性卟啉病是一组罕见的代谢紊乱疾病,其特征是血红素生物合成中的酶缺陷,血红素是一种金属卟啉,是卟啉代谢的主要产物。肝性卟啉病以常染色体显性疾病的形式遗传,表现多样,可导致一种特别严重的神经病变。当出现腹痛、精神病和神经病变这一典型三联征时,大多数医学生都能轻易识别出急性卟啉病发作。然而,卟啉性神经病变在实际中容易造成混淆,卟啉病患者在疾病早期很少能得到正确诊断。卟啉性神经病变的症状、体征和脑脊液异常类似于急性吉兰 - 巴雷综合征。然而,伴随的心理特征、不对称性肌无力的近端倾向以及提示轴索性多神经根病或神经元病的电诊断结果均提示卟啉病的诊断。诊断的确立依赖于适当的实验室检查。卟啉性神经病变的潜在病理生理学尚未明确,但可能与δ-氨基乙酰丙酸水平升高的直接神经毒性有关。鉴于神经病变的严重性以及包括避免诱发因素在内的潜在治疗方法的可获得性,进行鉴别诊断很重要。