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Forschungsdatenbank PMU-SQQUID

Neurological effects of recombinant human erythropoietin in Friedreich"s ataxia: a clinical pilot trial.
Boesch, S; Sturm, B; Hering, S; Scheiber-Mojdehkar, B; Steinkellner, H; Goldenberg, H; Poewe, W;
Mov Disord. 2008; 23(13):1940-1944
Originalarbeiten (Zeitschrift)

PMU-Autor/inn/en

Hering Sascha

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.


Useful keywords (using NLM MeSH Indexing)

Adult

Deoxyguanosine/analogs*

derivatives

Deoxyguanosine/urine

Enzyme-Linked Immunosorbent Assay/methods

Erythropoietin/therapeutic use*

Female

Follow-Up Studies

Friedreich Ataxia/drug therapy*

Friedreich Ataxia/physiopathology*

Friedreich Ataxia/urine

Humans

Iron-Binding Proteins/metabolism

Lymphocytes/metabolism

Male

Middle Aged

Pilot Projects

Recombinant Proteins

Severity of Illness Index

Treatment Outcome


Find related publications in this database (Keywords)

Friedreichxxxs ataxia
erythropoietin
frataxin
oxidative stress