Title :

Trial of Deferiprone in Parkinson's Disease

Author(s) :

Devos, David [Auteur]
Département de Pharmacologie Médicale [Lille] [Pôle Recherche]
Labreuche, Julien [Auteur]
Lille Neurosciences & Cognition - U 1172 [LilNCog]
Rascol, Olivier [Auteur]
Centre d'investigation clinique de Toulouse [CIC 1436]
Corvol, Jean-Christophe [Auteur]
542029|||Institut du Cerveau = Paris Brain Institute [ICM] (VALID)
Duhamel, Alain [Auteur]
Lille Neurosciences & Cognition - U 1172 [LilNCog]
Guyon Delannoy, Pauline [Auteur]
CHU Lille - Direction de la recherche et de l’innovation
Poewe, Werner [Auteur]
Innsbruck Medical University = Medizinische Universität Innsbruck [IMU]
Compta, Yaroslau [Auteur]
Universitat de Barcelona [UB]
Institut d'Investigacions Biomèdiques August Pi i Sunyer [IDIBAPS]
Centro de Investigacion Biomédica en Red sobre Enfermedades Neurodegenerativas [CIBERNED]
Pavese, Nicola [Auteur]
Newcastle University [Newcastle]
Růžička, Evžen [Auteur]
1. lékařská fakulta, Univerzita Karlova [Praha, Česká republika] = First Faculty of Medicine, Charles University [Prague, Czech Republic] [1. LF UK / 1st Faculty of Medicine]
Dušek, Petr [Auteur]
1. lékařská fakulta, Univerzita Karlova [Praha, Česká republika] = First Faculty of Medicine, Charles University [Prague, Czech Republic] [1. LF UK / 1st Faculty of Medicine]
Post, Bart [Auteur]
Donders Institute for Brain, Cognition and Behaviour
Radboud University Medical Center [Nijmegen]
Bloem, Bastiaan R. [Auteur]
Donders Institute for Brain, Cognition and Behaviour
Radboud University Medical Center [Nijmegen]
Berg, Daniela [Auteur]
Christian-Albrechts-Universität zu Kiel = Christian-Albrechts University of Kiel = Université Christian-Albrechts de Kiel [CAU]
Maetzler, Walter [Auteur]
Christian-Albrechts-Universität zu Kiel = Christian-Albrechts University of Kiel = Université Christian-Albrechts de Kiel [CAU]
Otto, Markus [Auteur]
Universität Ulm - Ulm University [Ulm, Allemagne]
Habert, Marie-Odile [Auteur]
Sorbonne Université [SU]
Centre d'Acquisition et de Traitement des Images [Paris] = CATI Multicenter Neuroimaging Platform [CATI]
Lehericy, Stéphane [Auteur]
Institut du Cerveau = Paris Brain Institute [ICM]
Ferreira, Joaquim [Auteur]
Universidade de Lisboa = University of Lisbon = Université de Lisbonne [ULISBOA]
Dodel, Richard [Auteur]
Universität Duisburg-Essen = University of Duisburg-Essen [Essen]
Tranchant, Christine [Auteur]
Fédération de Médecine Translationnelle de Strasbourg [FMTS]
Eusebio, Alexandre [Auteur]
Institut de Neurosciences de la Timone [INT]
Aix Marseille Université [AMU]
Thobois, Stéphane [Auteur]
Institut des sciences cognitives Marc Jeannerod - Centre de neuroscience cognitive - UMR5229 [ISC-MJ]
Université Claude Bernard Lyon 1 [UCBL]
Marques, Ana-Raquel [Auteur]
Université Clermont Auvergne [UCA]
Meissner, Wassilios G. [Auteur]
University of Otago [Dunedin, Nouvelle-Zélande]
Institut des Maladies Neurodégénératives [Bordeaux] [IMN]
Ory-Magne, F. [Auteur]
Centre d'investigation clinique de Toulouse [CIC 1436]
Walter, Uwe [Auteur]
University of Rostock
De Bie, Rob M.A. [Auteur]
Amsterdam University Medical Centers [Amsterdam UMC]
Gago, Miguel [Auteur]
Universidade do Minho = University of Minho [Braga]
Vilas, Dolores [Auteur]
Germans Trias i Pujol University Hospital [Badalona, Barcelona, Spain] [GTPUH]
Kulisevsky, Jaime [Auteur]
Universitat Autònoma de Barcelona = Autonomous University of Barcelona = Universidad Autónoma de Barcelona [UAB]
Januario, Cristina [Auteur]
Hospitais da Universidade de Coimbra [H.U.C.]
Coelho, Miguel V.S. [Auteur]
Hospital de Santa Maria [Lisboa]
Behnke, Stefanie [Auteur]
Saarland University Hospital [UKS]
Worth, Paul [Auteur]
Addenbrooke's Hospital
Seppi, Klaus [Auteur]
Innsbruck Medical University = Medizinische Universität Innsbruck [IMU]
Ouk, Thavarak [Auteur]
CHU Lille - Direction de la recherche et de l’innovation
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Potey, Camille [Auteur]
CHU Lille - Direction de la recherche et de l’innovation
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Leclercq, Céline [Auteur]
CHU Lille - Direction de la recherche et de l’innovation
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Viard, Romain [Auteur]
Services de neuroradiologie [Lille]
Kuchcinski, Gregory [Auteur]
Services de neuroradiologie [Lille]
Lopes, Renaud [Auteur]
Services de neuroradiologie [Lille]
Pruvo, Jean-Pierre [Auteur]
Service de neuroradiologie [Lille]
Pigny, Pascal [Auteur]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Garçon, Guillaume [Auteur]
IMPact de l'Environnement Chimique sur la Santé humaine (IMPECS) - ULR 4483
Simonin, Ophelie [Auteur]
Impact de l'environnement chimique sur la santé humaine - ULR 4483 [IMPECS]
Carpentier, Jessica [Auteur]
Impact de l'environnement chimique sur la santé humaine - ULR 4483 [IMPECS]
Rolland, Anne-Sophie [Auteur]
Département de Pharmacologie Médicale [Lille] [Pôle Recherche]
Nyholm, Dag [Auteur]
Uppsala University
Scherfler, Christoph [Auteur]
Innsbruck Medical University = Medizinische Universität Innsbruck [IMU]
Mangin, Jean-François [Auteur]
Building large instruments for neuroimaging: from population imaging to ultra-high magnetic fields [BAOBAB]
542029|||Institut du Cerveau = Paris Brain Institute [ICM] (VALID)
444929|||Centre d'Acquisition et de Traitement des Images [Paris] = CATI Multicenter Neuroimaging Platform [CATI] (VALID)
Chupin, Marie [Auteur]
Building large instruments for neuroimaging: from population imaging to ultra-high magnetic fields [BAOBAB]
542029|||Institut du Cerveau = Paris Brain Institute [ICM] (VALID)
444929|||Centre d'Acquisition et de Traitement des Images [Paris] = CATI Multicenter Neuroimaging Platform [CATI] (VALID)
Bordet, Regis [Auteur]
Département de Pharmacologie Médicale [Lille] [Pôle Recherche]
Dexter, David T. [Auteur]
Fradette, Caroline [Auteur]
University of Toronto
Chiesi SAS
Spino, Michael [Auteur]
University of Toronto
Chiesi SAS
Tricta, Fernando [Auteur]
University of Toronto
Chiesi SAS
Ayton, Scott [Auteur]
University of Melbourne
Bush, Ashley I. [Auteur]
University of Melbourne
Devedjian, Jean-Christophe [Auteur]
Département de Pharmacologie Médicale [Lille] [Pôle Recherche]
Duce, James A. [Auteur]
The Florey Institute of Neuroscience and Mental Health
Cabantchik, Ioav [Auteur]
The Hebrew University of Jerusalem [HUJ]
Defebvre, Luc [Auteur]
Département de neurologie [Lille]
Deplanque, Dominique [Auteur]
Lille Neurosciences & Cognition (LilNCog) - U 1172
Moreau, caroline [Auteur]
Département de neurologie [Lille]

Journal title :

New England Journal of Medicine

Abbreviated title :

N Engl J Med

Volume number :

387

Pages :

2045-2055

Publisher :

Massachusetts Medical Society

Publication date :

2022-12-01

ISSN :

1533-4406

English keyword(s) :

Parkinson Disease
Hematology/Oncology
Neuroscience
Geriatrics/​Aging

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

Background Iron content is increased in the substantia nigra of persons with Parkinson’s disease and may contribute to the pathophysiology of the disorder. Early research suggests that the iron chelator deferiprone can ...
Show more >Background Iron content is increased in the substantia nigra of persons with Parkinson’s disease and may contribute to the pathophysiology of the disorder. Early research suggests that the iron chelator deferiprone can reduce nigrostriatal iron content in persons with Parkinson’s disease, but its effects on disease progression are unclear. METHODS We conducted a multicenter, phase 2, randomized, double-blind trial involving participants with newly diagnosed Parkinson’s disease who had never received levodopa. Participants were assigned (in a 1:1 ratio) to receive oral deferiprone at a dose of 15 mg per kilogram of body weight twice daily or matched placebo for 36 weeks. Dopaminergic therapy was withheld unless deemed necessary for symptom control. The primary outcome was the change in the total score on the Movement Disorder Society–sponsored revision of the Unified Parkinson’s Disease Rating Scale (MDS-UPDRS; range, 0 to 260, with higher scores indicating more severe impairment) at 36 weeks. Secondary and exploratory clinical outcomes at up to 40 weeks included measures of motor and nonmotor disability. Brain iron content measured with the use of magnetic resonance imaging was also an exploratory outcome. Results A total of 372 participants were enrolled; 186 were assigned to receive deferiprone and 186 to receive placebo. Progression of symptoms led to the initiation of dopaminergic therapy in 22.0% of the participants in the deferiprone group and 2.7% of those in the placebo group. The mean MDS-UPDRS total score at baseline was 34.3 in the deferiprone group and 33.2 in the placebo group and increased (worsened) by 15.6 points and 6.3 points, respectively (difference, 9.3 points; 95% confidence interval, 6.3 to 12.2; P<0.001). Nigrostriatal iron content decreased more in the deferiprone group than in the placebo group. The main serious adverse events with deferiprone were agranulocytosis in 2 participants and neutropenia in 3 participants. Conclusions In participants with early Parkinson’s disease who had never received levodopa and in whom treatment with dopaminergic medications was not planned, deferiprone was associated with worse scores in measures of parkinsonism than those with placebo over a period of 36 weeks. (Funded by the European Union Horizon 2020 program; FAIRPARK-II ClinicalTrials.gov number, NCT02655315. opens in new tab.)Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

European Project :

Conservative iron chelation as a disease-modifying strategy in Parkinson’s disease: a multicentric, parallel-group, placebo-controlled, randomized clinical trial of deferiprone

Administrative institution(s) :

Université de Lille
CHU Lille
Institut Pasteur de Lille

Collections :

• IMPact de l'Environnement Chimique sur la Santé humaine (IMPECS) - ULR 4483
• Lille Neurosciences & Cognition (LilNCog) - U 1172

Research team(s) :

Troubles cognitifs dégénératifs et vasculaires

Submission date :

2023-10-20T05:56:48Z
2024-02-26T14:37:49Z