Tan Poh-Lin, Wagner John E, Auerbach Arleen D, Defor Todd E, Slungaard Arne, Macmillan Margaret L
Department of Pediatrics, Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota 55455, USA.
Pediatr Blood Cancer. 2006 May 1;46(5):630-6. doi: 10.1002/pbc.20538.
To potentially reduce late effects of malignancy, chronic graft-versus-host disease (GVHD), endocrinopathy, and infertility in patients with Fanconi anemia (FA) undergoing HLA-matched related donor hematopoietic cell transplantation (HCT), we developed a regimen using fludarabine (FLU), cyclophosphamide (CY), and anti-thymocyte globulin (ATG) followed by infusion of T-cell depleted (TCD) bone marrow (BM) or unmanipulated umbilical cord blood (UCB). GVHD prophylaxis consisted of cyclosporine and short course methylprednisolone.
Between April 2000 and June 2003, 11 patients (10 aplastic anemia (AA), 1 myelodysplastic syndrome (MDS)) underwent HCT using this regimen. Stem cell sources were BM and UCB in eight and three patients, respectively.
All patients demonstrated primary engraftment. Median days to neutrophil and platelet engraftment were 11 days (range 9-21) and 38 days (range 19-381), respectively. No patient developed GVHD after primary HCT. The patient with MDS relapsed with AML and a maternal donor recipient experienced secondary graft failure. For the nine FA patients with AA who underwent HLA-identical sibling donor HCT, the Kaplan-Meier estimates of overall survival and event-free survival (EFS) at 2 years are 100% and 82%, respectively, at a median follow-up of 2.9 years (range 1.9-4.8).
In summary, a FLU-based, non-irradiation approach is effective for FA patients with AA undergoing HLA-identical sibling donor HCT.
为了潜在降低接受人类白细胞抗原（HLA）匹配的相关供体造血细胞移植（HCT）的范可尼贫血（FA）患者发生恶性肿瘤的晚期效应、慢性移植物抗宿主病（GVHD）、内分泌病和不育症，我们制定了一种方案，即使用氟达拉滨（FLU）、环磷酰胺（CY）和抗胸腺细胞球蛋白（ATG），随后输注去除T细胞（TCD）的骨髓（BM）或未处理的脐带血（UCB）。移植物抗宿主病的预防措施包括环孢素和短疗程甲基强的松龙。
2000年4月至2003年6月期间，11例患者（10例再生障碍性贫血（AA），1例骨髓增生异常综合征（MDS））采用该方案进行了HCT。干细胞来源分别为8例患者的BM和3例患者的UCB。
所有患者均实现了初次植入。中性粒细胞和血小板植入的中位天数分别为11天（范围9 - 21天）和38天（范围19 - 381天）。初次HCT后无患者发生GVHD。MDS患者复发为急性髓系白血病（AML），一名接受母亲供体移植的患者出现了继发性移植失败。对于9例接受HLA相同同胞供体HCT的AA型FA患者，在中位随访2.9年（范围1.9 - 4.8年）时，2年总生存率和无事件生存率（EFS）的Kaplan - Meier估计值分别为100%和82%。
总之，基于氟达拉滨的非照射方法对接受HLA相同同胞供体HCT的AA型FA患者有效。
