Erythropoietin in amyotrophic lateral sclerosis: a multicentre, randomised, double blind, placebo controlled, phase III study. Issue 8 (16th January 2015)

• Record Type: Journal Article
• Authors: Lauria, Giuseppe; Dalla Bella, Eleonora; Antonini, Giovanni; Borghero, Giuseppe; Capasso, Margherita; Caponnetto, Claudia; Chiò, Adriano; Corbo, Massimo; Eleopra, Roberto; Fazio, Raffaella; Filosto, Massimiliano; Giannini, Fabio; Granieri, Enrico; La Bella, Vincenzo; Logroscino, Giancarlo; Mandrioli, Jessica; Mazzini, Letizia; Monsurrò, Maria Rosaria; Mora, Gabriele; Pietrini, Vladimiro; Quatrale, Rocco; Rizzi, Romana; Salvi, Fabrizio; Siciliano, Gabriele; Sorarù, Gianni; Volanti, Paolo; Tramacere, Irene; Filippini, Graziella
• Other Names: author non-byline.; Cazzato Daniele author non-byline.; Cesnik Edward author non-byline.; Groppo Elisabetta author non-byline.; Sette Elisabetta author non-byline.; Pani Carla author non-byline.; Costantino Emanuela author non-byline.; Orlandini Francesco author non-byline.; Boi Daniela author non-byline.; Querin Giorgia author non-byline.; D'Ascenzo Carla author non-byline.; Sagnelli Anna author non-byline.; Piccirillo Giovanni author non-byline.; Aiello Marina author non-byline.; Chetta Alfredo author non-byline.; Grassi Andrea author non-byline.; Lunetta Christian author non-byline.; Maestri Eleonora author non-byline.; Padovani Alessandro author non-byline.; Cotelli Mariasofia author non-byline.; Todeschini Alice author non-byline.; Morino Stefania author non-byline.; Di Pasquale Antonella author non-byline.; Latino Pamela author non-byline.; Casali Stefania author non-byline.; Battistini Stefania author non-byline.; Pirrelli Marilena author non-byline.; Cantello Roberto author non-byline.; Nasuelli Nicola author non-byline.; Servo Serena author non-byline.; De Gennaro Riccardo author non-byline.; Gastaldo Ernesto author non-byline.; Georgoulopoulou Eleni author non-byline.; Fini Nicola author non-byline.; Taiello Alfonsa C. author non-byline.; Colletti Tiziana author non-byline.; Calvo Andrea author non-byline.; Moglia Cristina author non-byline.; Fuda Giuseppe author non-byline.; Marinou Kalliopi author non-byline.; Riva Nilo author non-byline.; Cerri Federica author non-byline.; Lopez Ignazio D. author non-byline.; De Cicco Domenico author non-byline.; Battaglia Gianluca author non-byline.; Marcello Norina author non-byline.; Rinaldi Manuela author non-byline.; Scialò Carlo author non-byline.; Mantero Vittorio author non-byline.; Mascolo Maria author non-byline.; Carlesi Cecilia author non-byline.; Caldarazzo Ienco Elena author non-byline.; Di Muzio Antonio author non-byline.; Verriello Lorenzo author non-byline.; D'Amico Delia author non-byline.; Simone Isabella L. author non-byline.; Tortelli Rosanna author non-byline.; Cortese Rosa author non-byline.; Bartolomei Ilaria author non-byline.;
• Is Part Of: Journal of neurology, neurosurgery and psychiatry. Volume 86:Issue 8(2015)
• Page Start: 879
• Page End: 886
• Publication Date: 2015-01-16
• Subjects: ALS -- MOTOR NEURON DISEASE; Neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; Psychiatry -- Periodicals; 616.8
• DOI: 10.1136/jnnp-2014-308996
• Languages: English
• ISSNs: 0022-3050

Abstract

Abstract : Objective: To assess the efficacy of recombinant human erythropoietin (rhEPO) in amyotrophic lateral sclerosis (ALS). Methods: Patients with probable laboratory-supported, probable or definite ALS were enrolled by 25 Italian centres and randomly assigned (1:1) to receive intravenous rhEPO 40 000 IU or placebo fortnightly as add-on treatment to riluzole 100 mg daily for 12 months. The primary composite outcome was survival, tracheotomy or >23 h non-invasive ventilation (NIV). Secondary outcomes were ALSFRS-R, slow vital capacity (sVC) and quality of life (ALSAQ-40) decline. Tolerability was evaluated analysing adverse events (AEs) causing withdrawal. The randomisation sequence was computer-generated by blocks, stratified by centre, disease severity (ALSFRS-R cut-off score of 33) and onset (spinal or bulbar). The main outcome analysis was performed in all randomised patients and by intention-to-treat for the entire population and patients stratified by severity and onset. The study is registered, EudraCT 2009-016066-91. Results: We randomly assigned 208 patients, of whom 5 (1 rhEPO and 4 placebo) withdrew consent and 3 (placebo) became ineligible (retinal thrombosis, respiratory insufficiency, SOD1 mutation) before receiving treatment; 103 receiving rhEPO and 97 placebo were eligible for analysis. At 12 months, the annualised rate of death (rhEPO 0.11, 95% CI 0.06 to 0.20; placebo: 0.08, CI 0.04 to 0.17), tracheotomy or >23 h NIV (rhEPO 0.16, CI 0.10 to 0.27; placebo 0.18, CI 0.11 to 0.30) did not differ between groups, also after stratification by onset and ALSFRS-R at baseline. Withdrawal due to AE was 16.5% in rhEPO and 8.3% in placebo. No differences were found for secondary outcomes. Conclusions: RhEPO 40 000 IU fortnightly did not change the course of ALS.