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A longitudinal VBM study monitoring treatment with erythropoietin in patients with Friedreich ataxia

机译:一项纵向VBM研究监测弗里德赖希共济失调患者接受促红细胞生成素的治疗

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Recombinant human erythropoietin (rhuEPO) has received considerable attention because of its neuroprotective properties. It has recently been reported that rhuEPO increases frataxin levels in combination with clinical improvement in rhuEPO treated patients with Friedreich ataxia (FRDA). To determine possible therapy dependent intracranial volume changes after treatment with rhuEPO using voxel-based morphometry (VBM). Nine FRDA patients were scanned on the same 1.5-Tesla MRI scanner before and after treatment with rhuEPO. FRDA patients received 5000?IU rhuEPO thrice weekly subcutaneously for a time period of 8 weeks followed by 2000?IU thrice weekly over 6 months. To test for re-test reliability a control group of 12 healthy volunteers were scanned twice on the same scanner without rhuEPO treatment. Neurological state was defined by the Friedreich Ataxia Rating Scale (FARS) and the Scale for the Assessment and Rating of Ataxia (SARA). Statistical parametric mapping software was used for image processing and statistical analysis. When comparing follow-up scans after rhuEPO treatment with baseline scans (P Given the limitation of a small sample size, our study confirms previous findings that MRI may serve as reliable biomarker in neurodegenerative diseases as well as in monitoring of microstructural changes representing disease progression and/or therapy effects.
机译:重组人促红细胞生成素(rhuEPO)由于其神经保护特性而受到了广泛的关注。最近有报道说,rhuEPO增加frataxin水平并结合rhuEPO治疗的Friedreich共济失调(FRDA)患者的临床改善。为了确定可能的依赖于颅内体积变化的可能的疗法,在基于体素的形态计量学(VBM)的rhuEPO治疗后。在用rhuEPO治疗之前和之后,在相同的1.5-Tesla MRI扫描仪上扫描了9名FRDA患者。 FRDA患者每周皮下注射5000?IU rhuEPO,持续8周,然后在6个月内每周两次皮下2000?IU。为了测试重新测试的可靠性,对照组的12名健康志愿者在未经rhuEPO治疗的同一台扫描仪上扫描了两次。神经状态由弗里德里希共济失调评定量表(FARS)和共济失调评定和评定量表(SARA)定义。统计参数映射软件用于图像处理和统计分析。当将rhuEPO治疗后的后续扫描与基线扫描进行比较时(P鉴于样本量有限,我们的研究证实了先前的发现,MRI可作为神经退行性疾病以及监测代表疾病进展和疾病的微观结构变化的可靠生物标志物/或治疗效果。

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