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异基因造血干细胞移植治疗白细胞黏附缺陷症

Allogeneic Hematopoietic Stem Cell Transplantation for Leukocyte Adhesion Deficiency.

作者信息

Horikoshi Yasuo, Umeda Katsutsugu, Imai Kohsuke, Yabe Hiromasa, Sasahara Yoji, Watanabe Kenichiro, Ozawa Yukiyasu, Hashii Yoshiko, Kurosawa Hidemitsu, Nonoyama Shigeaki, Morio Tomohiro

机构信息

Department of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka.

Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto.

出版信息

J Pediatr Hematol Oncol. 2018 Mar;40(2):137-140. doi: 10.1097/MPH.0000000000001028.

Abstract

The clinical outcome of allogeneic hematopoietic stem cell transplantation (HSCT) was retrospectively analyzed in 6 patients with leukocyte adhesion deficiency. Of 3 patients transplanted with myeloablative conditioning, 2 patients had complete chimerism and 1 patient had mixed chimerism. By contrast, all 3 patients transplanted with reduced-intensity conditioning (RIC) had mixed chimerism, one of whom progressed to secondary graft failure. All patients with low-level mixed chimerism and secondary graft failure were rescued by donor lymphocyte infusion or a second HSCT. RIC-HSCT is feasible for leukocyte adhesion deficiency, although further refinement/modification of conditioning is required to achieve higher donor chimerism levels.

摘要

对6例白细胞黏附缺陷患者的异基因造血干细胞移植(HSCT)临床结果进行了回顾性分析。在3例接受清髓性预处理的患者中,2例实现了完全嵌合,1例为混合嵌合。相比之下,3例接受减低强度预处理(RIC)的患者均为混合嵌合,其中1例进展为继发性移植物失败。所有低水平混合嵌合和继发性移植物失败的患者均通过供体淋巴细胞输注或第二次HSCT得到挽救。RIC-HSCT对于白细胞黏附缺陷是可行的,尽管需要进一步优化/调整预处理以实现更高的供体嵌合水平。

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