Conservative Iron Chelation for Neuroferritinopathy.


Journal

Movement disorders : official journal of the Movement Disorder Society
ISSN: 1531-8257
Titre abrégé: Mov Disord
Pays: United States
ID NLM: 8610688

Informations de publication

Date de publication:
09 2022
Historique:
revised: 03 06 2022
received: 21 04 2022
accepted: 15 06 2022
pubmed: 24 8 2022
medline: 16 9 2022
entrez: 23 8 2022
Statut: ppublish

Résumé

Neuroferritinopathy is a rare inherited neurodegenerative disease with brain iron accumulation characterized by brain iron overload resulting in progressive movement disorders. No treatment is currently available. We assessed conservative iron chelation with deferiprone at 30 mg/kg/day on the disease progression with controlled periods of discontinuation. Four patients with confirmed molecular diagnosis of neuroferritinopathy were given deferiprone at different stages of disease progression and with clinical and biological monitoring to control benefit and risk. The four patients showed slight to high improvement. In one case, we managed to stabilize disease progression for more than 11 years. In another case, we were able to reverse symptoms after a few months of treatment. The earliest the treatment was started, the most efficient it was on disease progression. Conservative iron chelation should be further assessed in neuroferritinopathy. © 2022 International Parkinson and Movement Disorder Society.

Sections du résumé

BACKGROUND
Neuroferritinopathy is a rare inherited neurodegenerative disease with brain iron accumulation characterized by brain iron overload resulting in progressive movement disorders. No treatment is currently available.
OBJECTIVE
We assessed conservative iron chelation with deferiprone at 30 mg/kg/day on the disease progression with controlled periods of discontinuation.
METHODS
Four patients with confirmed molecular diagnosis of neuroferritinopathy were given deferiprone at different stages of disease progression and with clinical and biological monitoring to control benefit and risk.
RESULTS
The four patients showed slight to high improvement. In one case, we managed to stabilize disease progression for more than 11 years. In another case, we were able to reverse symptoms after a few months of treatment. The earliest the treatment was started, the most efficient it was on disease progression.
CONCLUSIONS
Conservative iron chelation should be further assessed in neuroferritinopathy. © 2022 International Parkinson and Movement Disorder Society.

Identifiants

pubmed: 35996824
doi: 10.1002/mds.29145
pmc: PMC10360136
doi:

Substances chimiques

Iron Chelating Agents 0
Pyridones 0
Deferiprone 2BTY8KH53L

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

1948-1952

Informations de copyright

© 2022 International Parkinson and Movement Disorder Society.

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Auteurs

Felix Marchand (F)

Department of Neurology, Neurogenetic Center, Univ. Lille, CHU Lille, Lille Neurosciences and Cognition Inserm UMR-S-U1172, Lille, France.
Univ. Lille, Inserm, CHU Lille, U1172 - LilNCog - Lille Neurosciences and Cognition, Lille, France.

Caroline Moreau (C)

Department of Neurology, Neurogenetic Center, Univ. Lille, CHU Lille, Lille Neurosciences and Cognition Inserm UMR-S-U1172, Lille, France.
Univ. Lille, Inserm, CHU Lille, U1172 - LilNCog - Lille Neurosciences and Cognition, Lille, France.

Gregory Kuchcinski (G)

Department of Neuroradiology, CHU Lille, Lille, France.

Vincent Huin (V)

Univ. Lille, Inserm, CHU Lille, U1172 - LilNCog - Lille Neurosciences and Cognition, Lille, France.
Department of Molecular Biology, CHU Lille, Lille, France.

Luc Defebvre (L)

Department of Neurology, Neurogenetic Center, Univ. Lille, CHU Lille, Lille Neurosciences and Cognition Inserm UMR-S-U1172, Lille, France.
Univ. Lille, Inserm, CHU Lille, U1172 - LilNCog - Lille Neurosciences and Cognition, Lille, France.

David Devos (D)

Department of Neurology, Neurogenetic Center, Univ. Lille, CHU Lille, Lille Neurosciences and Cognition Inserm UMR-S-U1172, Lille, France.
Univ. Lille, Inserm, CHU Lille, U1172 - LilNCog - Lille Neurosciences and Cognition, Lille, France.
Department of Medical Pharmacology, CHU Lille, Lille, France.

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Classifications MeSH