Allogeneic stem cell transplantation in Fanconi anemia patients presenting with myelodysplasia and/or clonal abnormality: update on the Saudi experience
Allogeneic stem cell transplantation in Fanconi anemia patients presenting with myelodysplasia and/or clonal abnormality: update on the Saudi experience
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DOI：
10.1038/sj.bmt.1705903
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发表时间：
2008-02-01
影响因子：
4.8
通讯作者：
El-Solh, H.
中科院分区：
文献类型：
--
作者：
Ayas, M.;Al-Jefri, A.;El-Solh, H.
在文献中，有大量关于异基因干细胞移植（SCT）治疗范可尼贫血（FA）患者的结果的有希望的数据;然而，关于出现骨髓增生异常和/或异常克隆的FA患者的结果的数据是粗略的，因为实体本身是罕见的，尽管，据信，这些因素中的任何一种的存在都会使移植结果的预后更差。这是我们对11例在费萨尔国王专科医院和研究中心接受SCT的此类患者的经验的更新; 10例来自匹配和相关供体，1例来自部分匹配的无关脐带血单位;预处理采用相同的方案，包括环磷酰胺（总剂量为20 mg/kg）、抗胸腺细胞球蛋白（总剂量为160 mg/kg的马产品或52 mg/kg的兔产品）和450 cGy的全身照射。10名患者目前仍然活着，身体健康，没有疾病的证据，中位随访时间近4年。
In the literature, there is an abundance of promising data on the outcome of allogeneic stem cell transplantation (SCT) in patients with Fanconi anemia (FA); however, the data on the outcome of FA patients who present with myelodysplasia and/or abnormal clone are sketchy as the entity itself is a rare one, although, it is believed that the presence of any of these factors confers a worse prognosis on the outcome of the transplant. This is an update of our experience in 11 such patients who underwent SCT at King Faisal Specialist Hospital and Research Center; 10 from the matched and related donors and 1 from a partially matched unrelated cord blood unit; the conditioning was with the same regimen consisting of cyclophosphamide (total of 20 mg/kg), anti-thymocyte globulin (total dose 160 mg/kg of the equine product or 52 mg/kg of the rabbit product) and total-body irradiation at 450 cGy. Ten patients remain currently alive, well and with no evidence of disease, with a median follow-up of almost 4 years.