X-linked recessive nephrogenic diabetes insipidus: a clinico-genetic study

J Pediatr Endocrinol Metab. 2014 Jan;27(1-2):93-9. doi: 10.1515/jpem-2013-0231.

Abstract

A retrospective genotype and phenotype analysis of X-linked congenital nephrogenic diabetes insipidus (NDI) was conducted on a nationwide cohort of 25 (24 male, 1 female) Korean children with AVPR2 gene mutations, comparing non-truncating and truncating mutations. In an analysis of male patients, the median age at diagnosis was 0.9 years old. At a median follow-up of 5.4 years, urinary tract dilatations were evident in 62% of patients and their median glomerular filtration rate was 72 mL/min/1.73 m2. Weights and heights were under the 3rd percentile in 22% and 33% of patients, respectively. One patient had low intelligence quotient and another developed end-stage renal disease. No statistically significant genotype-phenotype correlation was found between non-truncating and truncating mutations. One patient was female; she was analyzed separately because inactivation and mosaicism of the X chromosome may influence clinical manifestations in female patients. Current unsatisfactory long-term outcome of congenital NDI necessitates a novel therapeutic strategy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child
  • Child, Preschool
  • Diabetes Insipidus, Nephrogenic / genetics*
  • Diabetes Insipidus, Nephrogenic / pathology
  • Diabetes Insipidus, Nephrogenic / physiopathology
  • Female
  • Genes, Recessive*
  • Genetic Diseases, X-Linked / genetics*
  • Genetic Diseases, X-Linked / pathology
  • Genetic Diseases, X-Linked / physiopathology
  • Glomerular Filtration Rate
  • Growth
  • Humans
  • Infant
  • Male
  • Receptors, Vasopressin / genetics
  • Republic of Korea

Substances

  • AVPR2 protein, human
  • Receptors, Vasopressin