Monoallelic loss-of-function THPO variants cause heritable thrombocytopenia

Blood Adv. 2020 Mar 10;4(5):920-924. doi: 10.1182/bloodadvances.2019001293.

Abstract

  1. We report rare monoallelic variants of THPO that alter intracellular trafficking and diminish thrombopoietin secretion.

  2. Affected cases have autosomal-dominant thrombocytopenia but no other hematological features.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Humans
  • Isoxazoles
  • Thrombocytopenia* / genetics
  • Thrombopoietin

Substances

  • Isoxazoles
  • 4,5,6,7-tetrahydroisoxazolo(4,5-c)pyridin-3-ol
  • Thrombopoietin