Titre :

Therapeutic modalities of deferiprone in Parkinson's disease: SKY and EMBARK studies.

Auteur(s) :

Devos, David [Auteur]
Lille Neurosciences & Cognition (LilNCog) - U 1172
Rascol, O. [Auteur]
Université de Toulouse [UT]
Meissner, W. G. [Auteur]
Institut des Maladies Neurodégénératives [Bordeaux] [IMN]
Foubert-Samier, A. [Auteur]
Service de neurologie [Bordeaux]
Lewis, S. [Auteur]
The University of Sydney
Tranchant, C. [Auteur]
Les Hôpitaux Universitaires de Strasbourg [HUS]
Anheim, M. [Auteur]
Les Hôpitaux Universitaires de Strasbourg [HUS]
Maltête, D. [Auteur]
CHU Rouen
Remy, P. [Auteur]
Université Paris-Est Créteil Val-de-Marne - Paris 12 [UPEC UP12]
Eggert, K. [Auteur]
Philipps Universität Marburg = Philipps University of Marburg
Pape, H. [Auteur]
Philipps Universität Marburg = Philipps University of Marburg
Geny, C. [Auteur]
CHU Montpellier = Montpellier University Hospital
Couratier, P. [Auteur]
Carroll, C. [Auteur]
Plymouth University
Sheridan, R. [Auteur]
Royal Devon and Exeter Hospital [Exeter, UK] [RDEH]
Burn, D. [Auteur]
Newcastle University [Newcastle]
Pavese, N. [Auteur]
Newcastle University [Newcastle]
Raw, J. [Auteur]
Berg, D. [Auteur]
University Medical Center of Schleswig–Holstein = Universitätsklinikum Schleswig-Holstein [UKSH]
Suchowersky, O. [Auteur]
University of Alberta
Kalia, L. V. [Auteur]
University Health Network [Toronto, ON, Canada]
Evans, A. [Auteur]
The Royal Melbourne Hospital
Drapier, S. [Auteur]
Service de neurologie [Rennes]
Danaila, T. [Auteur]
Hospices Civils de Lyon [HCL]
Schnitzler, A. [Auteur]
Heinrich Heine Universität Düsseldorf = Heinrich Heine University [Düsseldorf]
Corvol, J. C. [Auteur]
CHU Pitié-Salpêtrière [AP-HP]
Defer, G. [Auteur]
CHU Caen
Temin, N. T. [Auteur]
Fradette, C. [Auteur]
Tricta, F. [Auteur]
Moreau, caroline [Auteur]
Lille Neurosciences & Cognition (LilNCog) - U 1172

Titre de la revue :

J Parkinsons Dis

Nom court de la revue :

J Parkinsons Dis

Pagination :

1877718X241300295

Date de publication :

2025-02-23

ISSN :

1877-718X

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

Background Reducing nigrostriatal iron overload reduces neuronal loss in Parkinson's disease (PD) models. Objective Examine the safety and efficacy of deferiprone in reducing motor disability progression in dopaminergic-treated ...
Lire la suite >Background Reducing nigrostriatal iron overload reduces neuronal loss in Parkinson's disease (PD) models. Objective Examine the safety and efficacy of deferiprone in reducing motor disability progression in dopaminergic-treated and treatment-naïve patients with early-stage PD. Methods Two phase II, multicenter studies, SKY and EMBARK, enrolled patients diagnosed with early PD (<3 years from screening). In SKY, patients on stable dopaminergic therapy were randomized 1:1 to one of four dosage (or placebo-matching) cohorts (300, 600, 900, 1200 mg twice daily [BID]) for 9 months. EMBARK enrolled patients on stable dopaminergic therapy or treatment-naïve patients and received 15 mg/kg BID. For both studies, the primary outcome was the change from baseline to month 9 in motor examination score (Movement Disorder Society-Unified Parkinson's Disease Rating Scale [MDS-UPDRS] Part III). ClinicalTrials.gov: NCT02728843; ANZCTR: ACTRN12617001578392. Results Overall, 140 patients were randomized in SKY (28 per cohort). Thirty-six patients enrolled in EMBARK (27 dopaminergic-treated; 9 treatment-naïve). In the SKY study, all doses showed the same worsening as the placebo group, with the exception of the 600 mg dose, which was associated with non-significant reductions in MDS-UPDRS Part III least-squares mean (LSM) between baseline and 9 months (−2–8 points versus placebo). In EMBARK, LSM (SE) changes from baseline in MDS-UPDRS Part III were nonsignificant (–1.6 [1.7]) and significant (8.3 [3.9]) for dopaminergic-treated and treatment-naïve patients, respectively, the latter indicating disease worsening. Adverse events possibly related to deferiprone were reported in 35.7%–88.9% across all deferiprone groups vs. 42.9% for placebo. Conclusions SKY and EMBARK studies indicate that deferiprone combined with L-dopa does not provide significant motor function benefit, while the absence of L-dopa treatment worsens symptoms.Lire moins >

Langue :

Anglais

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Lille Neurosciences & Cognition (LilNCog) - U 1172

Date de dépôt :

2025-02-25T22:02:42Z
2025-03-12T09:13:27Z