25595151
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 100mg daily for 12months .
METHODS	The primary composite outcome was survival , tracheotomy or > 23h non-invasive ventilation ( NIV ) .
METHODS	Secondary outcomes were ALSFRS-R , slow vital capacity ( sVC ) and quality of life ( ALSAQ-40 ) decline .
METHODS	Tolerability was evaluated analysing adverse events ( AEs ) causing withdrawal .
METHODS	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 ) .
METHODS	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 .
METHODS	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 .
RESULTS	At 12months , 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 > 23h 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 .
RESULTS	Withdrawal due to AE was 16.5 % in rhEPO and 8.3 % in placebo .
RESULTS	No differences were found for secondary outcomes .
CONCLUSIONS	RhEPO 40,000 IU fortnightly did not change the course of ALS .

