Trial of Deferiprone in Parkinson’s Disease

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MARC

Devos, David (author)

Trial of Deferiprone in Parkinson’s Disease

Article/chapterEnglish2022

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Massachusetts Medical Society,2022
printrdacarrier

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LIBRIS-ID:oai:DiVA.org:uu-491089
https://urn.kb.se/resolve?urn=urn:nbn:se:uu:diva-491089URI
https://doi.org/10.1056/nejmoa2209254DOI

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Language:English
Summary in:English

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SwePubSwePub

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Subject category:ref swepub-contenttype
Subject category:art swepub-publicationtype

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BACKGROUNDIron 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.METHODSWe 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.RESULTSA 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.CONCLUSIONSIn 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.

Subject headings and genre

MEDICIN OCH HÄLSOVETENSKAP Klinisk medicin Neurologi hsv//swe
MEDICAL AND HEALTH SCIENCES Clinical Medicine Neurology hsv//eng

Added entries (persons, corporate bodies, meetings, titles ...)

Labreuche, Julien (author)
Rascol, Olivier (author)
Corvol, Jean-Christophe (author)
Duhamel, Alain (author)
Guyon Delannoy, Pauline (author)
Poewe, Werner (author)
Compta, Yaroslau (author)
Pavese, Nicola (author)
Růžička, Evžen (author)
Dušek, Petr (author)
Post, Bart (author)
Bloem, Bastiaan R. (author)
Berg, Daniela (author)
Maetzler, Walter (author)
Otto, Markus (author)
Habert, Marie-Odile (author)
Lehericy, Stéphane (author)
Ferreira, Joaquim (author)
Dodel, Richard (author)
Tranchant, Christine (author)
Eusebio, Alexandre (author)
Thobois, Stéphane (author)
Marques, Ana-Raquel (author)
Meissner, Wassilios G. (author)
Ory-Magne, Fabienne (author)
Walter, Uwe (author)
de Bie, Rob M.A. (author)
Gago, Miguel (author)
Vilas, Dolores (author)
Kulisevsky, Jaime (author)
Januario, Cristina (author)
Coelho, Miguel V.S. (author)
Behnke, Stefanie (author)
Worth, Paul (author)
Seppi, Klaus (author)
Ouk, Thavarak (author)
Potey, Camille (author)
Leclercq, Céline (author)
Viard, Romain (author)
Kuchcinski, Gregory (author)
Lopes, Renaud (author)
Pruvo, Jean-Pierre (author)
Pigny, Pascal (author)
Garçon, Guillaume (author)
Simonin, Ophélie (author)
Carpentier, Jessica (author)
Rolland, Anne-Sophie (author)
Nyholm, Dag,Professor,1974-Uppsala universitet,Neurologi(Swepub:uu)danyh856 (author)
Scherfler, Christoph (author)
Mangin, Jean-François (author)
Chupin, Marie (author)
Bordet, Régis (author)
Dexter, David T. (author)
Fradette, Caroline (author)
Spino, Michael (author)
Tricta, Fernando (author)
Ayton, Scott (author)
Bush, Ashley I. (author)
Devedjian, Jean-Christophe (author)
Duce, James A. (author)
Cabantchik, Ioav (author)
Defebvre, Luc (author)
Deplanque, Dominique (author)
Moreau, Caroline (author)
Uppsala universitetNeurologi (creator_code:org_t)

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In:New England Journal of Medicine: Massachusetts Medical Society387:22, s. 2045-20550028-47931533-4406

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By the author/editor: Devos, David; Labreuche, Julie ...; Rascol, Olivier; Corvol, Jean-Chr ...; Duhamel, Alain; Guyon Delannoy, ...; show more...; Poewe, Werner; Compta, Yaroslau; Pavese, Nicola; Růžička, Evžen; Dušek, Petr; Post, Bart; Bloem, Bastiaan ...; Berg, Daniela; Maetzler, Walter; Otto, Markus; Habert, Marie-Od ...; Lehericy, Stépha ...; Ferreira, Joaqui ...; Dodel, Richard; Tranchant, Chris ...; Eusebio, Alexand ...; Thobois, Stéphan ...; Marques, Ana-Raq ...; Meissner, Wassil ...; Ory-Magne, Fabie ...; Walter, Uwe; de Bie, Rob M.A.; Gago, Miguel; Vilas, Dolores; Kulisevsky, Jaim ...; Januario, Cristi ...; Coelho, Miguel V ...; Behnke, Stefanie; Worth, Paul; Seppi, Klaus; Ouk, Thavarak; Potey, Camille; Leclercq, Céline; Viard, Romain; Kuchcinski, Greg ...; Lopes, Renaud; Pruvo, Jean-Pier ...; Pigny, Pascal; Garçon, Guillaum ...; Simonin, Ophélie; Carpentier, Jess ...; Rolland, Anne-So ...; Nyholm, Dag, Pro ...; Scherfler, Chris ...; Mangin, Jean-Fra ...; Chupin, Marie; Bordet, Régis; Dexter, David T.; Fradette, Caroli ...; Spino, Michael; Tricta, Fernando; Ayton, Scott; Bush, Ashley I.; Devedjian, Jean- ...; Duce, James A.; Cabantchik, Ioav; Defebvre, Luc; Deplanque, Domin ...; Moreau, Caroline; show less...

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MEDICAL AND HEALTH SCIENCES: MEDICAL AND HEAL ...; and Clinical Medicin ...; and Neurology

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By the university: Uppsala University

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