期刊
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
卷 136, 期 4, 页码 1018-1024出版社
MOSBY-ELSEVIER
DOI: 10.1016/j.jaci.2015.02.020
关键词
CD40 ligand; hematopoietic stem cell transplantation; Pneumocystis jirovecii; Cryptosporidium parvum; Cryptococcus neoformans; primary immunodeficiency; hyper-IgM syndrome; combined immunodeficiency; class-switch recombination
资金
- Grants-in-Aid for Scientific Research [15K09639, 24591541] Funding Source: KAKEN
Background: The long-term outcome of X-linked hyper-IgM syndrome (XHIM) caused by mutations in CD40LG is poor, and the only curative treatment is hematopoietic stem cell transplantation (HSCT). Objective: We sought to determine the clinical features and factors affecting outcomes in patients with XHIM. Methods: We enrolled and retrospectively analyzed data from 56 Japanese patients with XHIM, including 29 patients who received HSCT. Results: The long-term survival rate was poor in those not undergoing HSCT (overall survival rate at 40 years of age, 28.2%). The overall survival rate of patients undergoing HSCT (n = 29) was significantly higher than that of those not undergoing HSCT (n = 27, P = .0231). Moreover, event-free and disease-free survival rates were significantly greater in patients 5 years old or younger at the time of transplantation (n = 14) than in older patients (n = 15). Conclusion: On the basis of these results, we concluded that HSCT improved the outcomes of patients with XHIM and that an age of 5 years or younger was optimal for the timing of HSCT because persistent infections and severe organ damage were frequently observed in patients older than 6 years.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据