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Conservative Iron Chelation for Neuroferritinopathy
Author(s) -
Marchand Felix,
Moreau Caroline,
Kuchcinski Gregory,
Huin Vincent,
Defebvre Luc,
Devos David
Publication year - 2022
Publication title -
movement disorders
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.352
H-Index - 198
eISSN - 1531-8257
pISSN - 0885-3185
DOI - 10.1002/mds.29145
Subject(s) - deferiprone , discontinuation , medicine , chelation therapy , disease , conservative treatment , deferoxamine , chelation , movement disorders , pediatrics , surgery , thalassemia , chemistry , organic chemistry
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 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.