Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: A clinical pilot trial |
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| Authors: | Sylvia Boesch MD Brigitte Sturm PhD Sascha Hering MD Barbara Scheiber‐Mojdehkar PhD Hannes Steinkellner Mag Hans Goldenberg PhD Werner Poewe MD |
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| Institution: | 1. Department of Neurology, Innsbruck Medical University, Innsbruck, Austria;2. The first two authors contributed equally to this work.;3. Department of Medical Chemistry, Medical University of Vienna, Vienna, Austria |
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| Abstract: | In a “proof‐of‐concept” study, we demonstrated that recombinant human erythropoietin (rhuEPO) increases frataxin levels in Friedreich's ataxia (FRDA) patients. We now report a 6‐month open‐label clinical pilot study of safety and efficacy of rhuEPO treatment in FRDA. Eight adult FRDA patients received 2.000 IU rhuEPO thrice a week subcutaneously. Clinical outcome measures included Ataxia Rating Scales. Frataxin levels and indicators for oxidative stress were assessed. Hematological parameters were monitored biweekly. Scores in Ataxia Rating Scales such as FARS (P = 0.0063) and SARA (P = 0.0045) improved significantly. Frataxin levels increased (P = 0.017) while indicators of oxidative stress such as urine 8‐OHdG (P = 0.012) and peroxide levels decreased (P = 0.028). Increases in hematocrit requiring phlebotomies occurred in 4 of 8 patients. In this explorative open‐label clinical pilot study, we found an evidence for clinical improvement together with a persistent increase of frataxin levels and a reduction of oxidative stress parameters in patients with FRDA receiving chronic treatment with rhuEPO. Safety monitoring with regular blood cell counts and parameters of iron metabolism is a potential limitation of this approach. © 2008 Movement Disorder Society |
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| Keywords: | Friedreich's ataxia erythropoietin frataxin oxidative stress |
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