Study of bone marrow transplantation for the patient with mucopolysaccharidosis.

1.骨髓移植（BMT）的研究为建立骨髓移植进行了两项体外基础研究。(1)大豆凝集素和羊红细胞花环法处理骨髓细胞的免疫学特性。用Reisner等人描述的方法分离骨髓细胞以预防GVH，然后研究该部分的免疫学特性。结果表明，用这种方法获得的骨髓中成熟的T细胞很少，并且具有较低的同种异体反应性。这些结果表明，通过这种方法获得的骨髓部分可以用于BMT。(2)环孢素A （CyA）和甲氧氧酯（MTX）对混合淋巴细胞反应（MLR）的影响。体内预防BVH常用CyA或MTX。MLR是GVH的参数之一。本研究探讨了CyA或MTX对MLR的抑制作用。结果表明，CyA或MTX均能显著抑制MLR。这些结果表明，CyA或MTX可用于预防骨髓中GVH。成纤维细胞移植的临床研究对1例粘多糖病（IH/S）患者和1例戈谢病患者进行成纤维细胞移植治疗。治疗后，两名患者的肝脏大小都短暂缩小，但对其他临床表现没有影响。我们得出结论，成纤维细胞移植对这些患者没有治疗作用。

1.Studies of bone marrow transplantation (BMT)Two basic studies were carried out in vitro for establishment of BMT.(1) Immunological properties of bone marrow cells treated with soybeen agglutinin and sheep red blood cell rosette methods. Bone marrow cells were bractionated by this method described by Reisner et al. for prevention of GVH, and then immunological properties of this fraction were investigated. In results, the bone marrow fraction obtained by this method had very little mature T cells and had low alloreactivity. These results suggest that the bone marrow fraction obtained by this method is able to be used for BMT.(2) Effects of cyclosporin A (CyA) and methotoxesate (MTX) for mixed lymphocyte reaction (MLR). CyA or MTX was usual used for prevention of BVH in vivo. MLR was one of the parameters for GVH. In this study, suppressive effects of CyA or MTX for MLR were investigated. In results, CyA or MTX greatly suppressed MLR. These results suggested that CyA or MTX is able to be use for prevention of GVH in BMT.2.Clinical trial of fibroblast transplantationOne patients with Mucopolysaccharidosis IH/S and one patient with Gaucher disease were treated by fibroblast transplantation. After therapy, it was recognized that both patients' liver size reduced transiently, but there was no effect on the other clinical picture. We conclude that the fibroblast transplantation was not therapeutically useful in these patients.