Conservative Iron Chelation for Neuroferritinopathy.
Parkinson's disease
iron chelation
neuroferritinopathy
neuroprotection
spinocerebellar ataxia
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
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
1948-1952Informations de copyright
© 2022 International Parkinson and Movement Disorder Society.
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