Reduced-intensity conditioning haematopoietic cell transplantation for haemophagocytic lymphohistiocytosis: an important step forward.
Reduced-intensity conditioning haematopoietic cell transplantation for haemophagocytic lymphohistiocytosis: an important step forward.
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DOI：
10.1111/j.1365-2141.2011.08785.x
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发表时间：
2011-09
影响因子：
6.5
通讯作者：
Filipovich AH
中科院分区：
文献类型：
--
作者：
Marsh RA;Jordan MB;Filipovich AH
噬血细胞性淋巴组织细胞增生症（HLH）是一种危及生命的免疫缺陷，其特征是对感染或免疫系统的其他触发物的严重全身性炎症反应。在许多患者中，HLH的根本原因是遗传缺陷，导致缺陷的CD8 + T细胞和自然杀伤细胞颗粒介导的细胞毒性。HLH的治疗主要包括免疫抑制，然后是异基因造血细胞移植（HCT），以治愈潜在的缺陷和预防HLH复发。最初的治疗方案包括类固醇与依托泊苷或抗胸腺细胞球蛋白，+/-环孢菌素。仅在50 - 75%的患者中观察到完全缓解，即使在完全缓解后，仍会发生复发和死亡。唯一明确的，长期治愈患者的遗传形式的HLH是同种异体HCT。不幸的是，HLH患者的同种异体HCT通常并发危重疾病、广泛器官受累、活动性感染或难治性HLH。由于这些原因，患者通常容易发生移植相关的毒性和并发症。近年来，在HLH患者的护理和移植方面取得了很大进展。在这里，我们回顾了HLH患者的同种异体HCT治疗的现状，强调了降低强度预处理的重要进展。
Haemophagocytic lymphohistiocytosis (HLH) is a life-threatening immunodeficiency characterized by severe systemic hyper-inflammatory responses to infectious or other triggers of the immune system. In many patients, the underlying cause of HLH is a genetic defect leading to defective CD8+ T-cell and natural killer cell granule-mediated cytotoxicity. The treatment of HLH consists principally of immune suppression followed by allogeneic haematopoietic cell transplantation (HCT) to cure the underlying defect and prevent relapse of HLH. Initial treatment regimens consist of steroids coupled with either etoposide or antithymocyte globulin, +/- cyclosporine. Complete responses are observed in only 50-75% of patients and even after a complete response, relapse and death still occur. The only definitive, long-term cure for patients with genetic forms of HLH is allogeneic HCT. Unfortunately, allogeneic HCT for patients with HLH is often complicated by critical illness, extensive organ involvement, active infections, or refractory HLH. For these reasons, patients are unusually prone to developing transplant-related toxicities and complications. In recent years, great strides have been made with regard to the care and transplantation of patients with HLH. Here we review the current state of the treatment of patients with HLH with allogeneic HCT, highlighting the important steps forward that have been made with reduced-intensity conditioning.
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