Morimoto T, Shikada M, Yabe H, Yabe M, Hattori K, Shimizu T, Inokuchi S, Tsuji K, Iwasaki K, Banba M, Kato S
Department of Pediatrics, Tokai University School of Medicine.
Rinsho Ketsueki. 1997 Jul;38(7):610-5.
A five-year-old girl with Diamond-Blackfan syndrome received cord blood transplantation from an HLA-identical sibling. The patient showed pale face at birth, and was diagnosed to have Diamond-Blackfan syndrome. She had been treated with prednisolone (PSL), high dose of methylprednisolone, erythropoietin, and anti-lymphocyte globulin. Despite of these intensive therapies, erythropoiesis did not entirely improve, and transfusion of red blood cells had been required every third or fourth week until cord blood transplantation. Conditioning regimen consisted of thoraco-abdominal irradiation (TAI; 8 Gy), cyclophosphamide (CY; 50 mg/kg x 4), and anti-thymocyte globulin (ATG; 2.5 mg/kg x 4), Cyclosporin (CyA 3 mg/kg) was administered for the prophylaxis of graft-versus-host disease (GVHD). 4.14 x 10 (7)/kg of cord blood mononuclear cells were infused to the patient. White blood cell (WBC) and reticulocyte counts increased promptly, but recovery of platelet count was delayed. Skin GVHD (grade I) appeared on day +9, which responded to the administration of PSL (2 mg/kg). Chromosomal analyses of bone marrow cells for sex mismatch revealed complete chimerism on day +14, on day +28 and thereafter. Umbilical cord blood cells can be an alternative source of hematopoietic stem cells for allogeneic transplantation.
一名患有先天性纯红细胞再生障碍性贫血（Diamond-Blackfan综合征）的5岁女孩接受了来自 HLA 匹配同胞的脐血移植。该患者出生时面色苍白，被诊断为先天性纯红细胞再生障碍性贫血。她曾接受过泼尼松龙（PSL）、大剂量甲泼尼龙、促红细胞生成素和抗淋巴细胞球蛋白治疗。尽管进行了这些强化治疗，红细胞生成并未完全改善，在脐血移植前每隔三到四周仍需要输注红细胞。预处理方案包括胸腹部照射（TAI；8 Gy）、环磷酰胺（CY；50 mg/kg×4）和抗胸腺细胞球蛋白（ATG；2.5 mg/kg×4），给予环孢素（CyA 3 mg/kg）预防移植物抗宿主病（GVHD）。向患者输注了4.14×10⁷/kg 的脐血单个核细胞。白细胞（WBC）和网织红细胞计数迅速增加，但血小板计数的恢复延迟。在+9天出现了皮肤GVHD（I级），对给予PSL（2 mg/kg）有反应。对骨髓细胞进行性别不匹配的染色体分析显示，在+14天、+28天及之后均为完全嵌合体。脐血细胞可作为异基因移植的造血干细胞替代来源。
