Friedreich's ataxia: Clinical pilot trial with recombinant human erythropoietin

被引：91

作者：

Boesch, Sylvia

Sturm, Brigitte

Hering, Sascha

Goldenberg, Hans

Poewe, Werner

Scheiber-Mojdehkar, Barbara

机构：

[1] Med Univ Vienna, Dept Med Chem, A-1090 Vienna, Austria

[2] Innsbruck Med Univ, Dept Neurol, Innsbruck, Austria

来源：

ANNALS OF NEUROLOGY | 2007年 / 62卷 / 05期

关键词：

D O I：

10.1002/ana.21177

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

To determine the role of recombinant human erythropoietin as a possible treatment option in Friedreich's ataxia, we performed an open-label clinical pilot study. Primary outcome measure was the change of frataxin levels at week 8 versus baseline. Twelve Friedreich's ataxia patients received 5,000 units recombinant human erythropoietin three times weekly subcutaneously. Frataxin levels were measured in isolated lymphocytes by enzyme-linked immunosorbent assay. In addition, urinary 8-hydroxydeoxyguanosine and serum peroxides, were measured. Treatment with recombinant human erythropoietin showed a persistent and significant increase in frataxin levels after 8 weeks (p < 0.01). All patients showed a reduction of oxidative stress markers.

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页码：521 / 524

页数：4

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