feasibility and Results of Bone Marrow Transplantation from an HLA-Mismatched Unrelated Donor for Children and Young Adults with Acquired Severe Aplastic Anemia
feasibility and Results of Bone Marrow Transplantation from an HLA-Mismatched Unrelated Donor for Children and Young Adults with Acquired Severe Aplastic Anemia
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DOI：
10.1532/ijh97.06229
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发表时间：
2007-06
影响因子：
2.1
通讯作者：
H. Yagasaki;Yoshiyuki Takahashi;K. Kudo;H. Ohashi;A. Hama;Tomoko Yamamoto;Makito Tanaka;N. Yoshida;Hirokazu Hidaka;N. Nishio;S. Kojima
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文献类型：
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作者：
H. Yagasaki;Yoshiyuki Takahashi;K. Kudo;H. Ohashi;A. Hama;Tomoko Yamamoto;Makito Tanaka;N. Yoshida;Hirokazu Hidaka;N. Nishio;S. Kojima
治疗对免疫抑制治疗（IST）无效且没有HLA匹配供体的重度再生障碍性贫血（SAA）患者具有挑战性。我们报告了11例从HLA不匹配的无关供体接受骨髓移植（BMT）的患者的良好结局。中位年龄为11岁（范围：3-20岁）。预处理方案包括环磷酰胺（200 mg/kg）、抗胸腺细胞球蛋白（10 mg/kg）和全身照射（5戈伊）。患者接受他克莫司和甲氨蝶呤预防移植物抗宿主病（GVHD）。供受者对不匹配的HLA-DR抗原在8例患者的血清学分型。HLA-A和HLA-B抗原分别在1例和2例患者中不匹配。10例患者实现植入。1例移植失败的患者通过其母亲的第二次移植而获救，她的母亲在2个HLA抗原上不匹配。2例发生Ⅱ ~ Ⅳ级急性GVHD。3例患者发生局限性慢性GVHD，1例患者发生肺部广泛性慢性GVHD。所有患者在移植后9至56个月存活（中位数，33个月）。考虑到我们令人鼓舞的结果，HLA不匹配的无关供体骨髓移植治疗SAA作为IST无应答者的挽救治疗是可行的。
Treating patients with severe aplastic anemia (SAA) who fail to respond to immunosuppressive therapy (IST) and do not have an HLA-matched donor is challenging. We report favorable outcomes in 11 patients who underwent bone marrow transplantation (BMT) from an HLA-mismatched unrelated donor. The median age was 11 years (range, 3–20 years). The conditioning regimen consisted of cyclophosphamide (200 mg/kg), antithymocyte globulin (10 mg/kg), and total body irradiation (5 Gy). Patients received tacrolimus and methotrexate for prophylaxis against graft-versus-host disease (GVHD). Donor-recipient pairs were mismatched for the HLA-DR antigen in 8 patients by serologic typing. HLA-A and HLA-B antigens were mismatched in 1 and 2 patients, respectively. Ten patients achieved engraftment. One patient who failed to engraft was rescued by a second transplantation from her mother, who was mismatched at 2 HLA antigens. Acute GVHD of grades II to IV occurred in 2 patients. Three patients developed limited chronic GVHD, and 1 patient developed extensive chronic GVHD of the lung. All patients are alive at 9 to 56 months after transplantation (median, 33 months). Considering our encouraging results, HLA-mismatched unrelated-donor BMT for SAA is feasible as a salvage therapy for nonresponders to IST.