Schwinger W, Urban C, Lackner H, Kerbl R, Benesch M, Dornbusch H J, Sovinz P, Schauenstein K, Schumm M, Handgretinger R
Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Graz, Austria.
Bone Marrow Transplant. 2000 Mar;25(5):513-7. doi: 10.1038/sj.bmt.1702202.
Three children with refractory severe aplastic anemia were transfused with high numbers of unrelated matched (n = 2) or C-locus haploidentical mismatched (n = 1) CD34-selected peripheral blood stem cells in the absence of an HLA-identical family donor. Two leukaphereses of the donors yielded a median number of 10.1 x 10(10) nucleated cells (range 9.7-15.4) with a median number of 9.89 x 10(8) CD34+ cells (range 7.46-26.1) and a median percentage of CD34+cells of 0.98% (range 0.77-1.7). After positive selection by magnetic cell sorting the patients received a median of 14.3 x 10(6) CD34+ cells/kg (range 11.7-24.3) and of 1.3 x 10(4) CD3+ cells/kg (range 0.57-5.8). Median time to ANC >/=0.5 x 10(9)/l was 7 days (range 7-12) and to platelets >/=20 x 10(9)/l 13 days (range 13-27). Chimerism analysis of peripheral blood after transplantation revealed permanent 100% donor hematopoiesis in all patients. The patient with the C-locus haploidentical mismatch presented with acute GVHD (grade III-IV) of the skin, liver and lower gastrointestinal tract (onset day +40) and died despite intensive immunosuppressive treatment on day +238. The two survivors developed lymphopoietic recovery of B and T lymphocytes within 3 months after transplantation. To our knowledge this experience represents the first report of transplantation with unrelated CD34+ enriched peripheral blood stem cell in children with refractory severe aplastic anemia. Bone Marrow Transplantation (2000) 25, 513-517.
三名难治性严重再生障碍性贫血患儿在没有 HLA 相合同胞供者的情况下，输注了大量不相关配型相合（n = 2）或 C 位点单倍型半相合不相合（n = 1）的经 CD34 选择的外周血干细胞。供者的两次白细胞分离术获得的有核细胞中位数为 10.1×10¹⁰ 个（范围 9.7 - 15.4），CD34⁺细胞中位数为 9.89×10⁸ 个（范围 7.46 - 26.1），CD34⁺细胞百分比中位数为 0.98%（范围 0.77 - 1.7）。经磁性细胞分选阳性选择后，患者接受的 CD34⁺细胞中位数为 14.3×10⁶ 个/kg（范围 11.7 - 24.3），CD3⁺细胞中位数为 1.3×10⁴ 个/kg（范围 0.57 - 5.8）。中性粒细胞绝对值≥0.5×10⁹/L 的中位时间为 7 天（范围 7 - 12），血小板≥20×10⁹/L 的中位时间为 13 天（范围 13 - 27）。移植后外周血嵌合分析显示所有患者均出现永久性 100%供者造血。C 位点单倍型半相合不相合的患者出现皮肤、肝脏和下消化道的急性移植物抗宿主病（III - IV 级，发病于 +40 天），尽管进行了强化免疫抑制治疗，仍于 +238 天死亡。两名幸存者在移植后 3 个月内出现 B 和 T 淋巴细胞的淋巴细胞生成恢复。据我们所知，这一经验是关于难治性严重再生障碍性贫血患儿接受不相关 CD³⁴⁺富集外周血干细胞移植的首例报告。《骨髓移植》（2000 年）25卷，513 - 517 页 。
