Efficacy of Sirolimus in Treating Refractory Lymphatic Malformation in Noonan Syndrome: A Case Study

西罗莫司治疗努南综合征难治性淋巴管畸形的疗效:病例研究

阅读:1

Abstract

A female patient with Noonan syndrome, attributed to a pathogenic RIT1 variant, presented with white perineal discharge at 8 years and 5 months of age. She had a history of chylothorax and recurrent respiratory infections requiring hospitalization. Discharge analysis revealed 99.5% lymphocytes and elevated triglyceride levels (1939 mg/dL; 21.9 mmol/L). Magnetic resonance imaging and contrast-enhanced computed tomography identified lymphatic abnormalities extending from the thoracic to the pelvic region. We suspected that the chylous ascites was being discharged through the genitals. Administration of sirolimus, an mTOR inhibitor, was initiated, leading to a significant reduction in perineal chyle discharge and improved respiratory function with no adverse events. Sirolimus shows promise as a therapeutic intervention for lymphatic abnormalities in patients with Noonan syndrome; however, long-term follow-up is necessary to evaluate its efficacy and safety.

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。