Chronic granulomatous disease: overview and hematopoietic stem cell transplantation.

Chronic granulomatous disease: overview and hematopoietic stem cell transplantation.
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DOI:
10.1016/j.jaci.2011.03.028
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发表时间:
2011-06
期刊:
The Journal of allergy and clinical immunology
影响因子:
--
通讯作者:
Malech HL
Malech HL
中科院分区:
其他
文献类型:
--
作者:
Kang EM;Marciano BE;DeRavin S;Zarember KA;Holland SM;Malech HL

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慢性肉芽肿病(CGD)仍然导致显著的发病率和死亡率。考虑高风险但治愈性治疗的困难,如异基因骨髓移植,在于CGD和骨髓移植在不同患者中的不可预测的过程。一些CGD患者可能频繁感染和/或患有肉芽肿性或自身免疫性疾病,需要免疫抑制治疗,但也经历了长期的相对良好的健康状况。然而,所有类型的CGD的死亡风险明显更高,并且CGD的并发症(死亡除外)仍然可以导致显著的发病率。因此,随着最近的发展和改进,骨髓移植,以前被认为是实验性或高风险的程序,已成为CGD患者的一个重要选择。我们将讨论CGD的并发症,导致显着的发病率和死亡率,特别是最常见的感染和自身免疫/炎症并发症,以及他们的典型管理。我们将讨论骨髓移植的现状。
Chronic Granulomatous Disease (CGD) still causes significant morbidity and mortality. The difficulty in considering high-risk yet curative treatments, such as allogeneic bone marrow transplantation, lies in the unpredictable courses of both CGD and bone marrow transplantation in different patients. Some CGD patients may have frequent infections and/or suffer from granulomatous or autoimmune disorders necessitating immunosuppressive therapy, but also experience long periods of relative good health. However, the risk of death is clearly higher in CGD of all types, and the complications of CGD short of death can still cause significant morbidity. Therefore, with recent developments and improvements, bone marrow transplantation, previously considered an experimental or high-risk procedure, has emerged as an important option for patients with CGD. We will discuss the complications of CGD that result in significant morbidity and mortality, in particular the most common infections and autoimmune/inflammatory complications as well as their typical management. We will then discuss the status of bone marrow transplantation.
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