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Case Report: α-Spectrin Mutation Associated with αLELY Polymorphism Responsible for Hereditary Pyropoikilocytosis.

María Sánchez VillalobosEduardo Salido FiérrezJorge Martínez NietoMª Carmen García GarayAsunción Beltrán VidelaAna Belen Perez OlivaMiguel BlanquerJosé María Moraleda Jiménez
Published in: Hematology reports (2022)
Hereditary pyropoikilocytosis (HPP) is characterised by severe hemolytic anemia due to membrane instability. We report the case of a 13-day-old boy with neonatal jaundice and severe hemolytic anemia. A peripheral smear examination showed severe anisopoikylocytosis. DNA sequencing revealed compound double heterozygous for mutant α-spectrin SPTA1 (Arg28His) and homozygous αLELY polymorphism (low expression α-spectrin allele), compatible with diagnosis of HPP.The patient required a blood transfusion initially, but spontaneously improved after two years. Our case illustrates that, despite the presence of the allele αLELY in homozygous, the clinical phenotype is similar to cases with a mutation in SPTA1 associated with αLELY in trans.
Keyphrases
  • case report
  • early onset
  • chronic kidney disease
  • single cell
  • poor prognosis
  • iron deficiency
  • single molecule
  • long non coding rna
  • binding protein
  • nucleic acid