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Friedreich's ataxia: clinical pilot trial with recombinant human erythropoietin
Author(s) -
Boesch Sylvia,
Sturm Brigitte,
Hering Sascha,
Goldenberg Hans,
Poewe Werner,
ScheiberMojdehkar Barbara
Publication year - 2007
Publication title -
annals of neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.764
H-Index - 296
eISSN - 1531-8249
pISSN - 0364-5134
DOI - 10.1002/ana.21177
Subject(s) - erythropoietin , frataxin , ataxia , recombinant dna , medicine , urinary system , clinical trial , oxidative stress , gastroenterology , endocrinology , pharmacology , biology , biochemistry , iron binding proteins , gene , transferrin , psychiatry
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. Ann Neurol 2007