Speck B, Gluckman E, Haak H L, van Rood J J
Clin Haematol. 1978 Oct;7(3):611-21.
Forty-one patients, suffering from severe aplastic anaemia were treated either with ALG alone (27 patients) or ALG followed by infusion of allogeneic bone marrow (14 patients). Eighteen patients (67 per cent) are presently alive after ALG alone at over 100 to over 550 days. Fourteen (52 per cent) showed sustained improvement of haematopoiesis, two are alive without change, one recovered autologous haematopoiesis after cyclophosphamide conditioning and transfusion of HLA identical marrow and one is lost to follow-up. Eight patients (57 per cent) are currently alive after ALG and transfusion of haplotype identical marrow with self-sustaining autologous haematopoiesis at over 200 days to over four and a half years. No lethal complications occurred and none of the bone marrow infusions led to permanent engraftment or graft-versus-host disease. The mechanism of action is not known, but our results support the hypothesis that unspecified autoimmune reactions block the normal outgrowth of haematopoietic precursor cells in a substantial number of patients with aplastic anaemia. This therapeutic approach seems to offer good chances of survival, especially for those patients who do not have an HLA identical sibling. Its value should be further investigated.
41例严重再生障碍性贫血患者接受了治疗，其中27例单独使用抗淋巴细胞球蛋白（ALG）治疗，14例先使用ALG治疗，随后输注同种异体骨髓。单独使用ALG治疗后，18例患者（67%）目前存活，存活时间超过100天至550天以上。14例（52%）患者造血功能持续改善，2例患者存活且病情无变化，1例患者在环磷酰胺预处理及输注HLA相同的骨髓后恢复了自身造血功能，1例患者失访。8例患者（57%）在接受ALG及输注单倍型相同的骨髓后，自身造血功能持续维持，目前存活，存活时间超过200天至四年半以上。未发生致命并发症，且所有骨髓输注均未导致永久性植入或移植物抗宿主病。其作用机制尚不清楚，但我们的结果支持这样一种假说，即在大量再生障碍性贫血患者中，未明确的自身免疫反应会阻碍造血前体细胞的正常生长。这种治疗方法似乎提供了良好的生存机会，尤其是对于那些没有HLA相同同胞的患者。其价值应进一步研究。
