Unrelated cord blood transplantation in children with idiopathic severe aplastic anemia
Unrelated cord blood transplantation in children with idiopathic severe aplastic anemia
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DOI：
10.1038/bmt.2008.227
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发表时间：
2008-11-01
影响因子：
4.8
通讯作者：
Wall, D. A.
中科院分区：
文献类型：
--
作者：
Chan, K. W.;McDonald, L.;Wall, D. A.
非亲属脐带血移植（UCBT）治疗严重再生障碍性贫血（SAA）的早期结果较差，移植失败率高。这与移植物细胞剂量较低和宿主免疫系统完整有关。我们对9名患有难治性SAA的儿童（中位年龄9岁）进行了UCBT，使用的是免疫抑制增强的准备方案。从诊断到UCBT的时间为3.4-20个月（中位年龄7.2岁），所有儿童至少有一个疗程的免疫抑制失败。供体/受体HLA配型为6人中6人（n = 1）， 6人中5人（n = 2）， 6人中4人（n = 6）。灌注有核细胞的中位剂量为5.7 × 10(7)个细胞/kg（范围为3.5-20 × 10(7)个细胞/kg）。6例患者在第一次UCBT后移植。3例无造血重建的患者中有2例在第二次UCBT后移植。所有在预备方案中接受>= 120mg /kg CY的患儿均被植入。骨髓移植的中位时间为25天（17-59天）。2例患者出现急性GVHD， 5例患者出现慢性GVHD。5例患者移植后出现EBV病毒血症（3例为淋巴增生性疾病）。中位随访时间为34个月，7例患者存活且无需输血。UCBT对于难治性SAA患儿缺乏匹配的成人供体是一种可行的治疗策略。
Early results of unrelated cord blood transplantation (UCBT) for severe aplastic anemia (SAA) were poor with a high rate of engraftment failure. This was attributed to the combination of lower graft cell dose and intact host immune system. We performed UCBT in nine children (median age 9 years) with refractory SAA using increasingly immunosuppressive preparative regimens. The time from diagnosis to UCBT was 3.4-20 months (median age 7.2 years), with all children having failed at least one course of immunosuppression. Donor/recipient HLA matching was six of six (n = 1), five of six (n = 2) and four of six ( n = 6). The median nucleated cell dose infused was 5.7 x 10(7) cells/kg (range 3.5-20 x 10(7) cells/kg). Six patients were engrafted after the first UCBT. Two of the three patients without hematopoietic reconstitution were engrafted after a second UCBT. All children receiving >= 120 mg/kg of CY in the preparative regimen were engrafted. The median time to myeloid engraftment was 25 (17-59 days) days. Acute GVHD developed in two, and chronic GVHD in five patients. Five patients developed EBV viremia post transplant (lymphoproliferative disorder in three patients). At a median follow-up of 34 months, seven patients are alive and transfusion-independent. UCBT is a feasible treatment strategy for children with refractory SAA lacking a well-matched adult donor.