Davies S M, Wagner J E, Defor T, Blazar B R, Katsanis E, Kersey J H, Orchard P J, McGlave P B, Weisdorf D J, Ramsay N K
Department of Pediatrics, University of Minnesota, Minneapolis 55455, USA.
Br J Haematol. 1997 Mar;96(4):749-56. doi: 10.1046/j.1365-2141.1997.d01-2087.x.
Allogeneic transplantation from an HLA-matched family member has been shown to be effective in reconstituting normal haemopoiesis in young people with severe cytopenias, classified as myelodysplastic syndrome (MDS) or severe aplastic anaemia (SAA). Unrelated donor transplant is a therapeutic choice for patients without a suitable family member donor. We report the outcome of seven patients < 20 years old with SAA and 10 with MDS treated with BMT from an HLA A,B DRB1 matched (n = 8) or A or B locus mismatched (n = 9) unrelated donor at the University of Minnesota between March 1988 and August 1995. Primary graft failure occurred in two patients and secondary graft failure in one, who was subsequently successfully engrafted with a second donor marrow infusion. Grades II-IV GVHD occurred in 10/16 (63%), and grades III-IV in 6/16 (37%) evaluable patients. Nine of the 17 patients (six with MDS and three with SAA) survive with full donor chimaerism, a median of 1.2 years post-BMT (range 3 months to 7 years). We recommend early referral for consideration of unrelated donor BMT for young patients with MDS, and patients with SAA without response to immunosuppression.
对于患有严重血细胞减少症（分类为骨髓增生异常综合征（MDS）或重型再生障碍性贫血（SAA））的年轻人，来自HLA匹配家庭成员的异基因移植已被证明可有效重建正常造血功能。对于没有合适家庭成员供体的患者，非血缘供体移植是一种治疗选择。我们报告了1988年3月至1995年8月期间在明尼苏达大学接受来自HLA A、B、DRB1匹配（n = 8）或A或B位点不匹配（n = 9）的非血缘供体进行BMT治疗的7例年龄小于20岁的SAA患者和10例MDS患者的治疗结果。2例患者发生原发性移植物失败，1例发生继发性移植物失败，该患者随后通过第二次供体骨髓输注成功植入。在16例可评估患者中，10例（63%）发生II-IV级移植物抗宿主病（GVHD），6例（37%）发生III-IV级GVHD。17例患者中有9例（6例MDS患者和3例SAA患者）存活，完全为供体嵌合体，BMT后中位时间为1.2年（范围3个月至7年）。我们建议对于患有MDS的年轻患者以及对免疫抑制无反应的SAA患者，应尽早转诊考虑非血缘供体BMT。
