Journal
MOVEMENT DISORDERS
Volume 26, Issue 4, Pages 739-742Publisher
WILEY-BLACKWELL
DOI: 10.1002/mds.23435
Keywords
Friedreich's ataxia; Epoetin alfa; erythropoietin; frataxin; iron
Categories
Funding
- Associazione il cuore in un dono'' Cardito, Naples, Italy
- Associazione Italiana per la lotta alle Sindromi Atassiche (AISA) sez. Campania'', Napoli, Italy
Ask authors/readers for more resources
Objective of the study was to test the efficacy, safety, and tolerability of two single doses of Epoetin alfa in patients with Friedreich's ataxia. Ten patients were treated subcutaneously with 600 IU/kg for the first dose, and 3 months later with 1200 IU/kg. Epoetin alfa had no acute effect on frataxin, whereas a delayed and sustained increase in frataxin was evident at 3 months after the first dose (+35%; P < 0.05), and up to 6 months after the second dose (+54%; P < 0.001). The treatment was well tolerated and did not affect hematocrit, cardiac function, and neurological scale. Single high dose of Epoetin alfa can produce a considerably larger and sustained effect when compared with low doses and repeated administration schemes previously adopted. In addition, no hemoglobin increase was observed, and none of our patients required phlebotomy, indicating lack of erythropoietic effect of single high dose of erythropoietin. (C) 2010 Movement Disorder Society
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available