A Mild Clinical Phenotype with Myopathic and Hemolytic Forms of Phosphoglycerate Kinase Deficiency (PGK Osaka): A Case Report and Literature Review

Intern Med. 2022 Dec 1;61(23):3589-3594. doi: 10.2169/internalmedicine.9221-21. Epub 2022 May 7.

Abstract

Phosphoglycerate kinase (PGK) deficiency is an X-linked disorder characterized by a combination of hemolytic anemia, myopathy, and brain involvement. We herein report a Japanese man who had several episodes of rhabdomyolysis but was training strenuously to be a professional boxer. Mild hemolytic anemia was noted. The enzymatic activity of PGK was significantly reduced, and a novel missense mutation, p.S62N, was identified in the PGK1 gene. A literature review revealed only one case with a mixed hemolytic and myopathic phenotype like ours. This mild phenotype indicates the complex pathophysiology of PGK deficiency and suggests the benefits of dietary control and exercise.

Keywords: PGK Osaka; dietary intake; glycogen storage disease; hemolysis; ischemic exercise test; myopathy.

Publication types

  • Review
  • Case Reports

MeSH terms

  • Genetic Diseases, X-Linked* / genetics
  • Hemolysis
  • Humans
  • Metabolism, Inborn Errors* / complications
  • Metabolism, Inborn Errors* / diagnosis
  • Metabolism, Inborn Errors* / genetics
  • Phenotype
  • Phosphoglycerate Kinase / genetics

Substances

  • Phosphoglycerate Kinase

Supplementary concepts

  • Phosphoglycerate Kinase 1 Deficiency