Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Biol Blood Marrow Transplant. 2012 Dec;18(12):1911-20. doi: 10.1016/j.bbmt.2012.07.014. Epub 2012 Jul 27.
This study analyzes the hematopoietic cell transplantation experience in patients with immune deficiency at a single institution. The objective is to comprehensively evaluate the short-term and long-term outcomes with various preparative regimens, donor grafts, and ex vivo manipulations to identify transplantation approaches that most likely favor early donor immune competency without generating excessive toxicity. Clinical outcomes were evaluated in 52 consecutive patients with immune deficiencies. Thirty-seven of the 52 patients (71%) survived with attenuation of their underlying disease. The use of a melphalan-based reduced-intensity conditioning preparative regimen and immunomagnetic CD3(+) T cell depletion techniques (when T cell depletion was indicated) were associated with improved event-free survival. Survivors who received a preparative regimen other than a melphalan-based reduced-intensity regimen suffered from therapy-related morbidities or chronic/recurrent infections. Our findings indicate that melphalan-based reduced-intensity conditioning regimens and immunomagnetic CD3(+) T cell depletion limit therapy-related toxicity, and demonstrate promising results for the early establishment of donor immune competency.
本研究分析了单中心免疫缺陷患者的造血细胞移植经验。目的是全面评估各种预处理方案、供体移植物和体外操作的短期和长期结果，以确定最有可能有利于早期供体免疫能力而不产生过度毒性的移植方法。临床结果评估了 52 例连续免疫缺陷患者。52 例患者中有 37 例（71%）存活，基础疾病得到缓解。使用基于马法兰的减低强度预处理方案和免疫磁珠 CD3(+)T 细胞清除技术（当需要 T 细胞清除时）与无事件生存相关。接受非马法兰为基础的减低强度方案预处理的幸存者患有与治疗相关的发病率或慢性/复发性感染。我们的研究结果表明，基于马法兰的减低强度预处理方案和免疫磁珠 CD3(+)T 细胞清除可以限制治疗相关的毒性，并为早期建立供体免疫能力提供了有希望的结果。
