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首页> 外文期刊>Neurological sciences >Autologous hematopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: comparison with secondary progressive multiple sclerosis
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Autologous hematopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: comparison with secondary progressive multiple sclerosis

机译：复发 - 延缓多发性硬化症的自体造血干细胞移植：与二次进步多发性硬化的比较

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Abstract The main objective of our work is to describe the long-term results of myeloablative autologous hematopoietic stem cell transplant (AHSCT) in multiple sclerosis patients. Patients that failed to conventional therapies for multiple sclerosis (MS) underwent an approved protocol for AHSCT, which consisted of peripheral blood stem cell mobilization with cyclophosphamide and granulocyte colony-stimulating factor (G-CSF), followed by a conditioning regimen of BCNU, Etoposide, Ara-C, Melphalan IV, plus Rabbit Thymoglobulin. Thirty-eight MS patients have been transplanted since 1999. Thirty-one patients have been followed for more than 2?years (mean 8.4?years). There were 22 relapsing-remitting multiple sclerosis (RRMS) patients and 9 secondary progressive multiple sclerosis (SPMS) patients. No death related to AHSCT. A total of 10 patients (32.3%) had at least one relapse during post-AHSCT evolution, 6 patients in the RRMS group (27.2%) and 4 in the SPMS group (44.4%). After AHSCT, 7 patients (22.6%) experienced progression of disability, all within SP form. By contrast, no patients with RRMS experienced worsening of disability after a median follow-up of 5.4?years, 60% of them showed a sustained reduction in disability (SRD), defined as the improvement of 1.0 point in the expanded disability status scale (EDSS) sustains for 6?months (0.5 in cases of EDSS?≥?5.5). The only clinical variable that predicted a poor response to AHSCT was a high EDSS in the year before transplant. AHSCT using the BEAM-ATG scheme is safe and efficacious to control the aggressive forms of RRMS.
机译：摘要我们工作的主要目标是描述多发性硬化症患者中髓鞘自体造血干细胞移植（AHSCT）的长期结果。未对多发性硬化症（MS）进行常规疗法的患者进行了AHSCT的批准方案，其由环磷酰胺和粒细胞菌落刺激因子（G-CSF）组成的外周血液干细胞动员，其次是BCNU，依托泊苷的调理方案，Ara-C，Melphalan IV，加兔胸腺蛋白。自1999年以来，已经移植了38例患者。三十一名患者已被遵循超过2年（平均8.4岁）。有22例重复延迟多发性硬化症（RRMS）患者和9例继发性多发性硬化症（SPMS）患者。没有与AHSCT相关的死亡。在AHSCT进化后，共有10名患者（32.3％）在RRMS组（27.2％）和4名患者中有6名患者（44.4％）。在AHSCT后，7名患者（22.6％）经历了残疾进展，全部内在SP表格中。相比之下，在5.4岁的中位随访后，没有RRM的患者在5.4岁以下的时间后，60％的人表现出残疾（SRD）的持续减少，定义为扩大残疾状态规模中的1.0点（ EDSS）维持6个月（EDSS的情况下为0.5？≥？5.5）。唯一预测对AHSCT良差较差的临床变量是移植前一年的高EDS。使用光束ATG方案的AHSCT是安全而有效地控制侵略性的RRMS形式。

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