Wilson R C, Dave-Sharma S, Wei J Q, Obeyesekere V R, Li K, Ferrari P, Krozowski Z S, Shackleton C H, Bradlow L, Wiens T, New M I
Pediatric Endocrinology, The New York Hospital-Cornell Medical Center, 525 East 68th Street, New York, NY 10021, USA.
Proc Natl Acad Sci U S A. 1998 Aug 18;95(17):10200-5. doi: 10.1073/pnas.95.17.10200.
Severe low-renin hypertension has few known causes. Apparent mineralocorticoid excess (AME) is a genetic disorder that results in severe juvenile low-renin hypertension, hyporeninemia, hypoaldosteronemia, hypokalemic alkalosis, low birth weight, failure to thrive, poor growth, and in many cases nephrocalcinosis. In 1995, it was shown that mutations in the gene (HSD11B2) encoding the 11beta-hydroxysteroid dehydrogenase type 2 enzyme (11beta-HSD2) cause AME. Typical patients with AME have defective 11beta-HSD2 activity, as evidenced by an abnormal ratio of cortisol to cortisone metabolites and by an exceedingly diminished ability to convert [11-3H]cortisol to cortisone. Recently, we have studied an unusual patient with mild low-renin hypertension and a homozygous mutation in the HSD11B2 gene. The patient came from an inbred Mennonite family, and though the mutation identified her as a patient with AME, she did not demonstrate the typical features of AME. Biochemical analysis in this patient revealed a moderately elevated cortisol to cortisone metabolite ratio. The conversion of cortisol to cortisone was 58% compared with 0-6% in typical patients with AME whereas the normal conversion is 90-95%. Molecular analysis of the HSD11B2 gene of this patient showed a homozygous C-->T transition in the second nucleotide of codon 227, resulting in a substitution of proline with leucine (P227L). The parents and sibs were heterozygous for this mutation. In vitro expression studies showed an increase in the Km (300 nM) over normal (54 nM). Because approximately 40% of patients with essential hypertension demonstrate low renin, we suggest that such patients should undergo genetic analysis of the HSD11B2 gene.
重度低肾素性高血压的已知病因很少。表观盐皮质激素增多症(AME)是一种遗传性疾病,可导致严重的青少年低肾素性高血压、低肾素血症、低醛固酮血症、低钾性碱中毒、低出生体重、发育不良、生长迟缓,且在许多情况下会出现肾钙质沉着症。1995年,研究表明编码2型11β-羟类固醇脱氢酶(11β-HSD2)的基因(HSD11B2)发生突变会导致AME。AME的典型患者具有缺陷的11β-HSD2活性,这可通过皮质醇与可的松代谢物的异常比值以及将[11-3H]皮质醇转化为可的松的能力极度降低来证明。最近,我们研究了一名患有轻度低肾素性高血压且HSD11B2基因纯合突变的不寻常患者。该患者来自一个近亲结婚的门诺派家庭,尽管该突变确定她为AME患者,但她并未表现出AME的典型特征。对该患者的生化分析显示皮质醇与可的松代谢物的比值适度升高。皮质醇向可的松的转化率为58%,而AME典型患者的转化率为0-6%,正常转化率为90-95%。对该患者的HSD11B2基因进行分子分析显示,密码子227的第二个核苷酸发生了纯合的C→T转换,导致脯氨酸被亮氨酸替代(P227L)。其父母和兄弟姐妹为该突变的杂合子。体外表达研究显示Km(300 nM)高于正常水平(54 nM)。由于大约40%的原发性高血压患者表现为低肾素,我们建议此类患者应进行HSD11B2基因的遗传分析。