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RESEARCH PRODUCT
Erythropoietin in amyotrophic lateral sclerosis: a multicentre, randomized, double blind, placebo controlled, phase III study.
G LauriaE Dalla BellaG AntoniniG BorgheroM CapassoC CaponnettoA ChiòM CorboR EleopraR FazioM FilostoF GianniniE GranieriV La BellaG LogroscinoJ MandrioliL MazziniMr MonsurròG MoraV PietriniR QuatraleR RizziF SalviG SicilianoG SorarùP VolantiI TramacereG FilippiniD CazzatoE CesnikE GroppoE SetteC PaniE CostantinoF OrlandiniD BoiG QuerinC D'ascenzoA SagnelliG PiccirilloM AielloA ChettaA GrassiC LunettaE MaestriA PadovaniM CotelliA TodeschiniS MorinoA Di PasqualeP LatinoS CasaliS BattistiniM PirrelliR CantelloN NasuelliS ServoR De GennaroE GastaldoE GeorgoulopoulouN FiniAc TaielloT CollettiA CalvoC MogliaG FudaK MarinouN RivaF CerriId LopezD De CiccoG BattagliaN MarcelloM RinaldiC ScialòV ManteroM MascoloC CarlesiE Caldarazzo IencoA Di MuzioL VerrielloD D'amicoIl SimoneR TortelliR CorteseI Bartolomeisubject
MaleGastroenterologylaw.inventionRandomized controlled triallaw1506Amyotrophic lateral sclerosisMOTOR NEURON DISEASEeducation.field_of_studyRecombinant ProteinMiddle AgedRecombinant ProteinsTreatment OutcomePsychiatry and Mental HealthNeuromuscularSettore MED/26 - NeurologiaFemaleerythropoietyn clinical trialmedicine.drugHumanALS; MOTOR NEURON DISEASE; Adult; Aged; Amyotrophic Lateral Sclerosis; Double-Blind Method; Erythropoietin; Female; Humans; Male; Middle Aged; Recombinant Proteins; Treatment OutcomeAdultmedicine.medical_specialtyPopulationSocio-culturalePlaceboDouble blindALS; erythropoietyn clinical trialDouble-Blind MethodArts and Humanities (miscellaneous)ALS; MOTOR NEURON DISEASE; Adult; Aged; Amyotrophic Lateral Sclerosis; Double-Blind Method; Epoetin Alfa; Erythropoietin; Female; Humans; Male; Middle Aged; Recombinant Proteins; Treatment Outcome; Neurology (clinical); Psychiatry and Mental Health; Surgery; Arts and Humanities (miscellaneous)Internal medicinemedicineALS; MOTOR NEURON DISEASEHumanseducationErythropoietinAgedbusiness.industryAmyotrophic Lateral SclerosisEpoetin alfamedicine.diseaseSurgeryClinical trialEpoetin AlfaErythropoietinSurgeryNeurology (clinical)ALSbusinessAmyotrophic Lateral Sclerosidescription
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 intentionto-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.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2015-01-01 |