Tyrosinemia type 1 in pediatric nephrology: Not always straightforward

Arch Pediatr. 2021 May;28(4):338-341. doi: 10.1016/j.arcped.2021.03.002. Epub 2021 Apr 12.

Abstract

The main clinical features of tyrosinemia type 1 usually appear in the first months of life, including fever, diarrhea, vomiting, liver involvement, growth failure, and renal proximal tubulopathy with subsequent hypophosphatemic rickets. An early diagnosis is crucial in order to provide specific management and to prevent complications. Here, we report on two cases referred primarily to pediatric nephrologists for the diagnosis of "neonatal tubulopathy" and management of "X-linked hypophosphatemia (XLH)," respectively. Our aim is to emphasize that (1) even a mixed tubulopathy can reveal tyrosinemia, and (2) tyrosinemia is a classic differential diagnosis of XLH that should not be forgotten, especially in the era of the anti-FGF23 burosumab.

Keywords: Alkalosis; Hypophosphatemic rickets; Neonatal tubulopathy; Proximal tubulopathy; Tyrosinemia.

Publication types

  • Case Reports

MeSH terms

  • Antibodies, Monoclonal / therapeutic use
  • Antibodies, Monoclonal, Humanized
  • Child, Preschool
  • Disease Management
  • Familial Hypophosphatemic Rickets / diagnosis*
  • Fanconi Syndrome
  • Female
  • Genetic Diseases, Inborn*
  • Humans
  • Hydrolases / deficiency*
  • Infant, Newborn
  • Kidney Tubules, Proximal / physiopathology*
  • Tyrosinemias / diagnosis*

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Hydrolases
  • fumarylacetoacetase