PK-LR gene mutations in pyruvate kinase deficient Portuguese patients

Br J Haematol. 1999 Jun;105(3):591-5. doi: 10.1046/j.1365-2141.1999.01387.x.

Abstract

In nine unrelated Portuguese patients with pyruvate kinase (PK) deficient anaemia, whose symptoms ranged from a mild chronic haemolytic anaemia to a severe anaemia presenting at birth and requiring multiple transfusions, the PK-LR gene mutations were identified and correlated with their phenotypes. Five different mutations were identified, three of them for the first time: a missense mutation 1670G --> C on exon 12 and two 5' splice donor site (GT) mutations on intron 8 [IVS8(+2)T --> G] and intron 10 [IVS10(+1)G --> C]. Two previously described missense mutations, 1456C --> T and 993C --> A, were also found. The genotype/phenotype correlation showed that patients with two missense mutations or with a missense mutation and a splicing mutation had a mild haemolytic anaemia. The three patients with severe anaemia, who were transfusion dependent until splenectomy, were homozygous for the splicing site mutations IVS10(+1)G --> C or IVS8(+2)T --> G.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Child
  • Female
  • Homozygote
  • Humans
  • Infant, Newborn
  • Male
  • Mutation / genetics*
  • Polymorphism, Single-Stranded Conformational
  • Portugal
  • Pyruvate Kinase / deficiency*
  • Pyruvate Kinase / genetics

Substances

  • Pyruvate Kinase