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一名停用甘露糖治疗的磷酸甘露糖异构酶先天性糖基化障碍成年患者的临床结局

Clinical outcomes in an adult patient with mannose phosphate isomerase-congenital disorder of glycosylation who discontinued mannose therapy.

作者信息

Noman Kinza, Hendriksz Christian J, Radcliffe Graham, Roncaroli Federico, Moreea Sulleman, Hussain Afifah, Stepien Karolina M

机构信息

Medical School, University of Manchester, United Kingdom.

University of Pretoria, Steve Biko Academic Unit, Paediatrics and Child Health, Pretoria, South Africa.

出版信息

Mol Genet Metab Rep. 2020 Sep 7;25:100646. doi: 10.1016/j.ymgmr.2020.100646. eCollection 2020 Dec.

Abstract

The mannose phosphate isomerase-congenital disorder of glycosylation (MPI-CDG) is caused by phosphomannose isomerase deficiency. Clinical features include hyperinsulinaemic hypoglycaemia, protein losing enteropathy, hepatomegaly and hepatic fibrosis, digestive symptoms and coagulation abnormalities. The condition is treated with mannose supplementation. Long-term outcomes in adults are not well described. We present a case of an adult female patient who discontinued mannose therapy in her adolescence. In adulthood she developed gastrointestinal problems, chronic anaemia and osteophytes in her knees.

摘要

磷酸甘露糖异构酶先天性糖基化障碍(MPI-CDG)由磷酸甘露糖异构酶缺乏引起。临床特征包括高胰岛素血症性低血糖、蛋白丢失性肠病、肝肿大和肝纤维化、消化症状以及凝血异常。该病采用补充甘露糖进行治疗。关于成人的长期预后情况描述不多。我们报告一例成年女性患者,她在青春期停用了甘露糖治疗。成年后,她出现了胃肠道问题、慢性贫血以及膝关节骨赘。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2129/7490551/4bfafba7672c/gr1.jpg

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