Mehta J, Singhal S, Gee A P, Chiang K-Y, Godder K, Rhee Fv F van, DeRienzo S, O'Neal W, Lamb L, Henslee-Downey P J
Division of Transplantation Medicine, South Carolina Cancer Center, University of South Carolina, Columbia, SC, USA.
Bone Marrow Transplant. 2004 Feb;33(4):389-96. doi: 10.1038/sj.bmt.1704391.
Between February 1993 and December 1999, 201 patients (1-59 years old, median 23) with acute leukemia (67% not in remission) underwent ex vivo T-cell-depleted (TCD) bone marrow transplants (BMT) from partially mismatched related donors (PMRD; 92% mismatched for 2-3 HLA A, B, DR antigens). Conditioning comprised total body irradiation, cyclophosphamide, cytarabine, etoposide, anti-thymocyte globulin (ATG), and methylprednisolone. Graft-versus-host disease (GVHD) prophylaxis comprised partial TCD with OKT3 (n=143) or T10B9 (n=58), steroids, ATG, and cyclosporine. The engraftment rate was 98%. The cumulative incidences of grades II-IV acute GVHD and chronic GVHD were 13 and 15%, respectively. The 5-year cumulative incidences of relapse and transplant-related mortality (TRM) were 31 and 51%, respectively. The actuarial 5-year overall survival (OS) and disease-free survival (DFS) probabilities were 19 and 18%, respectively. Patient age >15 years, active disease at transplant, donor age >25 years, and 3-antigen donor mismatch (host-versus-graft) affected the outcome adversely. The actuarial 5-year OS of four groups of patients identified based upon these risk factors was 39, 20, 13, and 0%, respectively (P<0.0001). We conclude that PMRD BMT is a potential treatment option for patients with high-risk acute leukemia who require an alternative donor transplant and fall into a group with a reasonable expected outcome.
1993年2月至1999年12月期间，201例急性白血病患者（年龄1 - 59岁，中位年龄23岁）（67%未缓解）接受了来自部分不匹配相关供者（PMRD；92%在2 - 3个HLA A、B、DR抗原上不匹配）的体外去除T细胞（TCD）的骨髓移植（BMT）。预处理包括全身照射、环磷酰胺、阿糖胞苷、依托泊苷、抗胸腺细胞球蛋白（ATG）和甲泼尼龙。移植物抗宿主病（GVHD）预防包括用OKT3（n = 143）或T10B9（n = 58）进行部分TCD、类固醇、ATG和环孢素。植入率为98%。II - IV级急性GVHD和慢性GVHD的累积发生率分别为13%和15%。复发和移植相关死亡率（TRM）的5年累积发生率分别为31%和51%。精算的5年总生存率（OS）和无病生存率（DFS）概率分别为19%和18%。患者年龄>15岁、移植时疾病处于活动期、供者年龄>25岁以及3抗原供者不匹配（宿主抗移植物）对结局有不利影响。根据这些危险因素确定的四组患者的精算5年OS分别为39%、20%、13%和0%（P<0.0001）。我们得出结论，PMRD BMT是需要替代供者移植且预期结局合理的高危急性白血病患者的一种潜在治疗选择。
