Successful Liver Transplantation for Adolescent Patient With Pyruvate Kinase Deficiency-induced Cirrhosis

J Pediatr Hematol Oncol. 2021 May 1;43(4):e605-e607. doi: 10.1097/MPH.0000000000001876.

Abstract

We present the case of a successful liver transplant in a young adult patient with cholestasis and cirrhosis secondary to severe pyruvate kinase (PK) deficiency. Liver transplant resulted in resolution of liver dysfunction, decreased need for blood transfusions and eligibility for bone marrow transplantation. This case represents the third reported patient in the literature with severe PK deficiency who successfully underwent liver transplant as a result of profound cholestasis and liver failure. Explant pathology demonstrated a lack of significant iron deposition indicating that PK deficiency predisposes the liver to injury independent of transfusion-related iron overload.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Anemia, Hemolytic, Congenital Nonspherocytic / complications*
  • Anemia, Hemolytic, Congenital Nonspherocytic / pathology
  • Cholestasis / etiology
  • Cholestasis / pathology
  • Cholestasis / therapy
  • Female
  • Humans
  • Liver Cirrhosis / etiology*
  • Liver Cirrhosis / pathology
  • Liver Cirrhosis / therapy*
  • Liver Transplantation*
  • Pyruvate Kinase / deficiency*
  • Pyruvate Metabolism, Inborn Errors / complications*
  • Pyruvate Metabolism, Inborn Errors / pathology
  • Treatment Outcome

Substances

  • Pyruvate Kinase

Supplementary concepts

  • Pyruvate Kinase Deficiency of Red Cells