西罗莫司治疗LRBA缺乏症患者肠病的新治疗方法。
New therapeutic approach by sirolimus for enteropathy treatment in patients with LRBA deficiency.
作者信息
Azizi G, Abolhassani H, Yazdani R, Mohammadikhajehdehi S, Parvaneh N, Negahdari B, Mohammadi J, Aghamohammadi A
机构信息
Department of Laboratory Medicine, Imam Hassan Mojtaba Hospital, Alborz University of Medical Sciences, Karaj, Iran. Research Center for Immunodeficiencies, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran. Primary Immunodeficiency Diseases Network (PIDNet), Universal Scientific Education and Research Network (USERN), Tehran, Iran.
Research Center for Immunodeficiencies, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran. Primary Immunodeficiency Diseases Network (PIDNet), Universal Scientific Education and Research Network (USERN), Tehran, Iran. Division of Clinical Immunology, Department of Laboratory Medicine, Karolinska Institute at Karolinska University Hospital Huddinge, Stockholm, Sweden.
出版信息
Eur Ann Allergy Clin Immunol. 2017 Sep;49(5):235-239. doi: 10.23822/EurAnnACI.1764-1489.22.
To report the successful use of sirolimus for management of enteropathy in four patients with LPS-responsive beige-like anchor protein (LRBA) deficiency. Case series. sirolimus therapy led to a complete improvement of symptoms including decrease in frequency and severity of diarrhea, as well as patients' weight gain. No signs of abdominal cramps and anorexia were also detected during the follow up period after treatment. sirolimus with its potential efficacy and immunomodulatory properties may be recommended for the treatment of severe enteropathy refractory to conventional therapy in patients with LRBA deficiency.
报告西罗莫司成功用于治疗4例脂多糖反应性米色样锚定蛋白（LRBA）缺乏患者的肠病。病例系列。西罗莫司治疗使症状完全改善，包括腹泻频率和严重程度降低以及患者体重增加。治疗后的随访期间也未发现腹部绞痛和厌食的迹象。鉴于其潜在疗效和免疫调节特性，西罗莫司可推荐用于治疗LRBA缺乏患者中对传统治疗难治的严重肠病。
Zotero 插件