August C S, King E, Githens J H, McIntosh K, Humbert J R, Greensheer A, Johnson R B
Blood. 1976 Oct;48(4):491-8.
Marrow transplantation was attempted in a 13-yr-old boy with congenital hypoplastic anemia who had never responded to corticosteroid therapy. Prior to the transplant, he had received 238 transfusions, at least 12 of which were from his father. He was prepared for grafting with antilymphocyte globulin, procarbazine, and total body irradiation (1000 rads). The patient, whose red cells were Group B, then received marrow cells from his Group O, histocompatible, sister. Thereafter, reticulocytes, Group O erythrocytes, and female leukocytes appeared in the peripheral blood. Erythroid precursors were seen in the patient's marrow for the first time in his life, and all lacked fluorescent Y chromosomes. Dividing cells were all female. After initially progressing well, the patient developed interstitial pneumonia and died 55 days after the transplant. The successful erythroid graft suggested that this patient's failure to produce red blood cells was due to a defective stem cell rather than to a humoral defect, plasma inhibitor, or abnormal marrow microenvironment. It suggested further that sibling marrow may be engrafted in patients who have received multiple transfusions, even from a parent.
对一名13岁先天性再生障碍性贫血男孩尝试进行骨髓移植，该男孩对皮质类固醇治疗从未有过反应。移植前，他接受了238次输血，其中至少12次来自他的父亲。他接受了抗淋巴细胞球蛋白、丙卡巴肼和全身照射（1000拉德）以准备移植。该患者红细胞为B型，随后接受了来自其O型、组织相容性匹配的姐姐的骨髓细胞。此后，外周血中出现了网织红细胞、O型红细胞和女性白细胞。患者骨髓中首次出现红系前体细胞，且均缺乏荧光Y染色体。正在分裂的细胞均为女性。起初病情进展良好，但患者随后出现间质性肺炎，并在移植后55天死亡。红系移植的成功表明，该患者无法产生红细胞是由于干细胞缺陷，而非体液缺陷、血浆抑制剂或异常的骨髓微环境所致。这进一步表明，即使患者接受过多次输血，甚至是来自父母的输血，同胞骨髓仍可能成功植入。
