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أعرض في EDS

Abatacept is effective in Chinese patients with LRBA and CTLA4 deficiency

التفاصيل البيبلوغرافية
العنوان: Abatacept is effective in Chinese patients with LRBA and CTLA4 deficiency
المؤلفون: Lu Yang, Xiuhong Xue, Xuemei Chen, Junfeng Wu, Xi Yang, Li Xu, Xuemei Tang, Mo Wang, Huawei Mao, Xiaodong Zhao
المصدر: Genes and Diseases, Vol 8, Iss 5, Pp 662-668 (2021)
بيانات النشر: KeAi Communications Co., Ltd., 2021.
سنة النشر: 2021
المجموعة: LCC:Medicine (General)
LCC:Genetics
مصطلحات موضوعية: LRBA deficiency, CTLA4 deficiency, Target therapy, Abatacept, Efficacy, Medicine (General), R5-920, Genetics, QH426-470
الوصف: CTLA4 deficiency and LRBA deficiency are a group disorders of immune dysregulation that affect CTLA4 pathway. The patients mainly present with autoimmunity, antibody deficiency and recurrent infections. Here we reported three Chinese patients with LRBA and CTLA4 mutations. They all presented with chronic diarrhea, hypokalemia, organomegaly, recurrent infections, and hypogammaglobulinemia. Reduced Treg cells and increased percentage of circulating follicular helper T (cTfh) cells were revealed in these patients. Although steroid and immunoglobulin therapy were given, the enteropathy was persistent. Therefore, abatacept treatment was provided to these patients. They showed a marked improvement of enteropathy and gastrointestinal endoscopy showed alleviated inflammatory lesion and follicular hyperplasia. Furthermore, the frequency of cTfh cells was reduced after abatacept therapy. Taken together, targeted therapy with abatacept is a promising treatment modality for patients with LRBA and CTLA4 deficiency. The findings also suggest that the frequency of cTfh cells could serve as a marker for tracking disease activity and the response to abatacept therapy.
نوع الوثيقة: article
وصف الملف: electronic resource
اللغة: English
تدمد: 2352-3042
العلاقة: http://www.sciencedirect.com/science/article/pii/S2352304220300350Test; https://doaj.org/toc/2352-3042Test
DOI: 10.1016/j.gendis.2020.03.001
الوصول الحر: https://doaj.org/article/d7e3ebccd78f46e0b47f1562d1402bedTest
رقم الانضمام: edsdoj.7e3ebccd78f46e0b47f1562d1402bed
قاعدة البيانات: Directory of Open Access Journals
الوصف
تدمد:23523042
DOI:10.1016/j.gendis.2020.03.001