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以8 Gy全身照射、氟达拉滨和环磷酰胺为预处理方案用于小儿血液恶性肿瘤异基因造血干细胞移植时毒性较低。

Low toxicity of a conditioning with 8-Gy total body irradiation, fludarabine and cyclophosphamide as preparative regimen for allogeneic hematopoietic stem cell transplantation in pediatric hematological malignancies.

作者信息

Yanagisawa Ryu, Nakazawa Yozo, Sakashita Kazuo, Tanaka Miyuki, Shikama Naoto, Kamijo Takehiko, Shiohara Masaaki, Koike Kenichi

机构信息

Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.

出版信息

Pediatr Transplant. 2009 Sep;13(6):737-45. doi: 10.1111/j.1399-3046.2008.01065.x. Epub 2008 Dec 16.

Abstract

We here report the efficacy and toxicity of a conditioning regimen with fractionated 8-Gy TBI, fludarabine, and cyclophosphamide in allogeneic HSCT for pediatric hematological malignancies. Among 22 children who received related or unrelated HSCT, nine were transplanted with refractory disease and/or from HLA two or more loci-mismatched family donors. None of the patients developed graft failure. The Seattle grading system revealed that 18 patients had no RRT, and the remaining patients had grade I gastrointestinal toxicity alone. The estimated overall survival and leukemia-free survival at two yr were 57.1% and 48.0%, respectively, in 10 patients with acute lymphoblastic leukemia; 91.7% and 71.3%, respectively, in 12 patients with myeloid leukemia. The incidence of TRM was 4.8% at two yr. The rates of RRT above grade II and TRM in an 8-Gy TBI-containing regimen were significantly lower than the data of historical control patients who underwent 12-Gy TBI and cyclophosphamide with or without etoposide. The intermediate-dose TBI-based conditioning regimen may confer successful engraftment combined with minimized RRT, although its efficacy should be further evaluated.

摘要

我们在此报告在儿科血液系统恶性肿瘤的异基因造血干细胞移植中,采用分次8 Gy全身照射、氟达拉滨和环磷酰胺的预处理方案的疗效和毒性。在22例接受相关或无关造血干细胞移植的儿童中,9例移植时患有难治性疾病和/或来自HLA两个或更多位点不匹配的家族供者。所有患者均未发生移植物失败。西雅图分级系统显示,18例患者无重度放射性损伤,其余患者仅出现I级胃肠道毒性。10例急性淋巴细胞白血病患者2年时的估计总生存率和无白血病生存率分别为57.1%和48.0%;12例髓系白血病患者分别为91.7%和71.3%。2年时移植相关死亡率为4.8%。含8 Gy全身照射方案中II级以上重度放射性损伤和移植相关死亡率显著低于接受12 Gy全身照射和环磷酰胺(含或不含依托泊苷)的历史对照患者的数据。基于中等剂量全身照射的预处理方案可能实现成功植入,同时使重度放射性损伤最小化,尽管其疗效仍需进一步评估。

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