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.