Beyond Cystic Fibrosis: Recognising Shwachman-Diamond Syndrome in the Respiratory Clinic

超越囊性纤维化:在呼吸科门诊识别施瓦赫曼-戴蒙德综合征

阅读:1

Abstract

Cystic fibrosis (CF) and Shwachman-Diamond Syndrome (SDS) share overlapping features, including recurrent respiratory infections and pancreatic insufficiency, which can complicate diagnosis. We report a case of a six-year-old girl who presented with productive cough, steatorrhea and recurrent infections. Initial evaluation showed abnormal liver enzymes, elevated immunoreactive trypsin, neutrophil dysfunction and skeletal anomalies. Sweat test was equivocal and CFTR genetic panel was negative. Further genomic analysis identified compound heterozygous mutations in the SBDS gene: c.258+2T>C (known pathogenic variant) and c.284T>A (novel variant), confirming SDS. Over a 42-year follow-up, she experienced intermittent neutropenia, recurrent respiratory infections and pregnancy-related complications. This case emphasises the importance of considering SDS in CF-like presentations with atypical features and equivocal CF testing. Milder SDS phenotypes can survive into adulthood. Further work is needed to refine genotype-phenotype correlations and guide long-term management.

特别声明

1、本页面内容包含部分的内容是基于公开信息的合理引用;引用内容仅为补充信息,不代表本站立场。

2、若认为本页面引用内容涉及侵权,请及时与本站联系,我们将第一时间处理。

3、其他媒体/个人如需使用本页面原创内容,需注明“来源:[生知库]”并获得授权;使用引用内容的,需自行联系原作者获得许可。

4、投稿及合作请联系:info@biocloudy.com。