Novel exon 2 α spectrin mutation and intragenic crossover: three morphological phenotypes associated with four distinct α spectrin defects

Haematologica. 2013 Dec;98(12):1972-9. doi: 10.3324/haematol.2013.086629. Epub 2013 Sep 27.

Abstract

Hereditary pyropoikilocytosis is a severe hemolytic anemia caused by spectrin deficiency and defective spectrin dimer self-association, typically found in African populations. We describe two Utah families of northern European ancestry including 2 propositi with atypical non-microcytic hereditary pyropoikilocytosis, 7 hereditary elliptocytosis members and one asymptomatic carrier. The underlying molecular defect is a novel mutation in the alpha(α) spectrin gene, SPTA(R34P) that impairs spectrin tetramer formation. It is inherited in trans to the hypomorphic SPTA(αLELY) in the 2 propositi and 5 of 7 hereditary elliptocytosis individuals indicating that SPTA(αLELY) is not the sole determinant of the variable clinical expression. α Spectrin mRNA was mildly decreased in all hereditary elliptocytosis subjects, whereas both hereditary pyropoikilocytosis propositi had a severe decrease to ~10% of normal. Genotyping identified a unique SPTA intragenic crossover and uniparental disomy in one hereditary elliptocytosis individual. Two additional crossover events demonstrated the susceptibility of SPTA gene to rearrangement and revealed a novel segregation of the two SPTA(αLELY) mutations. We conclude that the profound phenotypic heterogeneity in these families can be attributed to the SPTA(R34P) mutation in combination with: 1) inheritance in trans of either SPTA(αLELY); or 2) the wild-type SPTA; 3) a decrease of α spectrin mRNA; and 4) SPTA intragenic crossover.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Aged
  • Elliptocytosis, Hereditary / diagnosis
  • Elliptocytosis, Hereditary / genetics*
  • Exons / genetics*
  • Female
  • Humans
  • Male
  • Mutation / genetics*
  • Pedigree
  • Phenotype*
  • Protein Structure, Secondary
  • Protein Structure, Tertiary
  • Spectrin / chemistry*
  • Spectrin / genetics*

Substances

  • Spectrin

Supplementary concepts

  • Pyropoikilocytosis, Hereditary