Successful bone marrow transplantation for children with aplastic anemia based on a best-available evidence strategy
Successful bone marrow transplantation for children with aplastic anemia based on a best-available evidence strategy
复制标题
DOI：
10.1111/j.1399-3046.2010.01388.x
复制
发表时间：
2010-12-01
影响因子：
1.3
通讯作者：
Kawano, Yoshifumi
中科院分区：
文献类型：
--
作者：
Okamoto, Yasuhiro;Kodama, Yuichi;Kawano, Yoshifumi
回顾性分析了最佳可用证据策略，即在 AA 的 BMT 时选择最佳可用供体、预处理方案和 GVHD 预防。对 18 名接受同种异体 BMT 的 AA 儿童的结果进行了分析。平均年龄为 11 岁（范围 4-16 岁），其中 9 名男性。作为预处理方案，7 个为低剂量照射 + CY，6 个为 ATG + CY + Flu，5 个为 ATG + CY。捐献者中有 10 人是 HLA 匹配的兄弟姐妹，1 人是 HLA 不匹配的家庭，3 人是 HLA 匹配的无血缘关系的人，4 人是 HLA 不匹配的无血缘关系的人。作为 GVHD 预防，3 名患者单独接受 CsA，9 名患者接受 CsA + MTX，6 名患者接受他克莫司 + MTX。所有 18 名患者均显示植入。移植后中性粒细胞计数超过 500/μL 的中位天数为 16 天（范围 11-21）。 5 名患者出现了 2 级以上的急性 GVHD，3 名患者出现了广泛的 cGVHD。 1例患者因间质性肺炎并发cGVHD死亡。五年 OS 为 94% (95% CI: 83-105)。这些结果表明，基于现有最佳证据的治疗患者策略是可以接受的。
A best-available evidence strategy, i.e., the best-available donors, conditioning regimens and GVHD prophylaxis were chosen at the time of BMT for AA, was analyzed retrospectively. The outcomes for 18 children with AA who underwent allogeneic BMT were analyzed. The median age was 11 yr (range 4-16), and nine were men. As conditioning regimens, seven had low-dose irradiation + CY, six had ATG + CY + Flu, and five had ATG + CY. Donors were HLA-matched siblings in 10, HLA-mismatched family in one, HLA-matched unrelated in three, and HLA-mismatched unrelated in four. As GVHD prophylaxis, three received CsA alone, nine received CsA + MTX, and six received tacrolimus + MTX. All 18 patients showed engraftment. The median number of days until the neutrophil count exceeded 500/mu L was 16 (range 11-21) post-transplant. Five developed more than grade 2 acute GVHD, and three developed extensive cGVHD. One patient died because of interstitial pneumonia complicated with cGVHD. Five-yr OS was 94% (95% CI: 83-105). These results suggest that a strategy of treating patients based on the best-available evidence is acceptable.