Delaporta Polyxeni, Sofocleous Christalena, Economou Marina, Makis Alexandros, Kostaridou Stavroula, Kattamis Antonis
First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece.
Department of Medical Genetics, National and Kapodistrian University of Athens, Athens, Greece.
Pediatr Blood Cancer. 2017 Nov;64(11). doi: 10.1002/pbc.26630. Epub 2017 May 16.
This study presents the clinical phenotype and molecular analysis findings from 11 patients recorded in the Greek Shwachman-Diamond syndrome (SDS) Registry. The most severely affected patient in our registry was diagnosed at birth and is the first patient reported to require bone marrow transplantation so early in life. Severe psoriasis, a feature not previously reported in SDS, was observed in one patient. Mutations in the Shwachman-Bodian-Diamond syndrome gene (SBDS) were found in all patients. Cytogenetic analyses revealed clonal abnormalities, one novel, in two patients.
本研究展示了希腊舒-戴二氏综合征(SDS)登记处记录的11例患者的临床表型和分子分析结果。我们登记处受影响最严重的患者在出生时就被诊断出来,是有记录以来生命早期就需要进行骨髓移植的首例患者。在一名患者中观察到严重银屑病,这是先前SDS中未报道的特征。所有患者均发现舒-博-戴三氏综合征基因(SBDS)突变。细胞遗传学分析在两名患者中发现了克隆异常,其中一种是新发现的。