Reduced intensity transplantation for primary immunodeficiency disorders
Reduced intensity transplantation for primary immunodeficiency disorders
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DOI：
10.4081/pr.2011.s2.e11
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发表时间：
2011-01-01
影响因子：
1.1
通讯作者：
Veys, Paul
中科院分区：
文献类型：
--
作者：
Veys, Paul
迄今为止的研究表明，降低强度移植（RIT）可能在治疗原发性免疫缺陷病（PID）患者中发挥重要作用。与更标准的方法不同，这种方案可以用于患有严重肺部或肝脏疾病的患者而没有严重毒性。RIT还提供了可以避免或减少长期后遗症如不孕或生长迟缓的优点。RIT似乎最适用于有显著合并症（如T细胞缺陷）和接受无关供体造血细胞移植的患者。需要进行更多的研究，使用药代动力学监测（如白消安，曲舒凡和阿仑单抗）和不同的干细胞来源，以优化移植物与骨髓反应，并尽量减少移植物抗宿主病。在某些PID患者中，RIT将是建立供体细胞移植的“第一步”;在一些低水平供体嵌合或移植排斥的患者中，可能需要第二次输注供体干细胞、供体淋巴细胞输注或第二次清髓性HCT，这些似乎耐受良好。
Studies so far indicate that reduced intensity transplantation (RIT) may have an important role in treating patients with primary immunodeficiency disease (PID). Unlike more standard approaches, such regimens can be used without severe toxicity in patients with severe pulmonary or hepatic disease. RIT also offers the advantage that long-term sequelae such as infertility or growth retardation may be avoided or reduced. RIT appears to be most appropriate for those patients with significant co-morbidities (eg T cell deficiencies) and those undergoing unrelated donor haematopoietic cell transplantation. More studies are required using pharmacokinetic monitoring (eg busulphan, treosulfan and alemtuzumab) and varying stem cell sources to optimise graft vs marrow reactions and minimise graft vs host disease. In certain PID patients RIT will be the "first step" towards establishing donor cell engraftment; second infusions of donor stem cells, donor lymphocyte infusions, or a second myeloablative HCT, which appears to be well tolerated, may be required in some patients with low level donor chimerism or graft rejection.