loading page

Bernard-Soulier syndrome caused by a novel GP1BB variant and 22q11.2 deletion
  • +4
  • Rintaro Nagoshi,
  • Atsushi Sakamoto,
  • Tsuyoshi Imai,
  • Toru Uchiyama,
  • Tadashi Kaname,
  • Shinji Kunishima,
  • Akira Ishiguro
Rintaro Nagoshi
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Byoin
Author Profile
Atsushi Sakamoto
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Byoin

Corresponding Author:sakamoto-a@ncchd.go.jp

Author Profile
Tsuyoshi Imai
Kokuritsu Byoin Kiko Shikoku Kodomoto Otonano Iryo Center
Author Profile
Toru Uchiyama
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Seiiku Iden Kenkyubu
Author Profile
Tadashi Kaname
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Kenkyujo
Author Profile
Shinji Kunishima
Gifu Iryo Kagaku Daigaku
Author Profile
Akira Ishiguro
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Byoin
Author Profile

Abstract

Bernard-Soulier syndrome (BSS) is caused by defects in GP1BA, GP1BB, or GP9 genes. Patients with 22q11.2 deletion syndrome (22q11.2DS) are obligate carriers for BSS because GP1BB resides on chromosome 22q11.2. A 15-month-old girl without bleeding symptoms had giant platelets and thrombocytopenia. Physical findings and macrothrombocytopenia suggested 22q11.2DS, which was confirmed by fluorescence in situ hybridization. Flow-cytometry showed decreased GPIbα on the platelets. A novel variant in GP1BB, p.(Val169_Leu172del), was revealed by gene panel testing. These findings confirmed that she had BSS. This case suggests that any 22q11.2DS patient associated with macrothrombocytopenia should be further investigated for the presence of BSS.