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Forschungsdatenbank PMU-SQQUID

Friedreich"s ataxia: clinical pilot trial with recombinant human erythropoietin.
Boesch, S; Sturm, B; Hering, S; Goldenberg, H; Poewe, W; Scheiber-Mojdehkar, B;
Ann Neurol. 2007; 62(5):521-524
Originalarbeiten (Zeitschrift)

PMU-Autor/inn/en

Hering Sascha

Abstract


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.


Useful keywords (using NLM MeSH Indexing)

Adolescent

Adult

Erythropoietin/therapeutic use*

Female

Friedreich Ataxia/blood

Friedreich Ataxia/drug therapy*

Humans

Iron-Binding Proteins/blood

Male

Middle Aged

Pilot Projects

Recombinant Proteins