Identification of a Novel MYH9 Mutation in a Young Adult With Inherited Thrombocytopenia and Recurrent Seizures by Targeted Exome Sequencing

J Pediatr Hematol Oncol. 2020 Apr;42(3):e188-e192. doi: 10.1097/MPH.0000000000001430.

Abstract

May-Hegglin anomaly (MHA) is a rare autosomal dominant disorder caused by a mutation in the myosin heavy chain 9 (MYH9) gene. MHA patients have variable clinical manifestations including thrombocytopenia, renal injury, hearing impairment, and cataracts. We describe a 25-year-old man with isolated thrombocytopenia initially. He experienced recurrent seizures with stable thrombocytopenia after the first seizures related to intracranial hemorrhage. He was identified a novel c.3452C>T mutation by targeted exome sequencing. If a patient with thrombocytopenia shows recurrent seizures as well as renal, hearing, visual symptoms, MHA should be suspected and the targeted exome sequencing is considered an effective diagnostic tool.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Exome Sequencing
  • Hearing Loss, Sensorineural / complications
  • Hearing Loss, Sensorineural / genetics*
  • Humans
  • Male
  • Mutation, Missense
  • Myosin Heavy Chains / genetics*
  • Seizures / genetics*
  • Thrombocytopenia / complications
  • Thrombocytopenia / congenital*
  • Thrombocytopenia / genetics

Substances

  • MYH9 protein, human
  • Myosin Heavy Chains

Supplementary concepts

  • MYH9-Related Disorders