title: Epoetin alfa increases frataxin production in Friedreich's ataxia without affecting hematocrit.
abstract: 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.
authors.affiliation: Department of Neurological Sciences, University Federico II, Napoli, NA, Italy. francesco.sacca@unina.it
authors.forename: Francesco
authors.initials: F
authors.lastname: Saccà
authors.forename: Raffaele
authors.initials: R
authors.lastname: Piro
authors.forename: Giuseppe
authors.initials: G
authors.lastname: De Michele
authors.forename: Fabio
authors.initials: F
authors.lastname: Acquaviva
authors.forename: Antonella
authors.initials: A
authors.lastname: Antenora
authors.forename: Guido
authors.initials: G
authors.lastname: Carlomagno
authors.forename: Sergio
authors.initials: S
authors.lastname: Cocozza
authors.forename: Alessandra
authors.initials: A
authors.lastname: Denaro
authors.forename: Anna
authors.initials: A
authors.lastname: Guacci
authors.forename: Angela
authors.initials: A
authors.lastname: Marsili
authors.forename: Gaetano
authors.initials: G
authors.lastname: Perrotta
authors.forename: Giorgia
authors.initials: G
authors.lastname: Puorro
authors.forename: Antonio
authors.initials: A
authors.lastname: Cittadini
authors.forename: Alessandro
authors.initials: A
authors.lastname: Filla
mesh_terms: D000328:Adult; D000704:Analysis of Variance; D004305:Dose-Response Relationship, Drug; D000068817:Epoetin Alfa; D004921:Erythropoietin / Q000097:blood / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D005621:Friedreich Ataxia / Q000097:blood* / Q000188:drug therapy*; D006397:Hematinics / Q000627:therapeutic use*; D006400:Hematocrit; D006801:Humans; D007501:Iron / Q000097:blood; D033862:Iron-Binding Proteins / Q000097:blood*; D008297:Male; D011994:Recombinant Proteins; D013997:Time Factors; D000096923:Frataxin
chemical_list: D000068817:Epoetin Alfa; D004921:Erythropoietin; D006397:Hematinics; D007501:Iron; D033862:Iron-Binding Proteins; D011994:Recombinant Proteins; D000096923:Frataxin