© 2019, Fondazione Società Italiana di Neurologia. Background: Superficial siderosis (SS) of the central nervous system is a rare and heterogeneous condition due to deposition of hemosiderin on the surface of the brain and spinal cord. The usually progressive clinical course is characterized by a combination of hearing loss, cerebellar ataxia, and myelopathy. There is no known treatment for SS, but the iron chelator deferiprone (DFP) has been proposed as a potentially useful treatment. Methods: We present a long-term (average 3.7 years) evaluation of four cases of SS treated with DFP (15 mg/kg po bid). Results: Treatment with DFP proved safe and well tolerated. Two out of the four subjects were unchanged while the other two presented a clinical improvement with reduction of postural instability and cerebellar signs. Blinded evaluation of magnetic resonance imaging (performed every 6 months during follow-up) showed a reduction of the abnormal iron deposition for all patients. Conclusions: This long-term observational study suggests that DFP may be effective in the management of the neurological manifestations associated with iron accumulation in SS. Clinicaltrials.gov identifier: NTC00907283.
- Iron accumulation
- Magnetic resonance imaging
- Superficial siderosis
Cossu, G., Abbruzzese, G., Forni, G. L., Matta, G., Pinto, V., Ruffinengo, U., Oppo, V., & Marchese, R. (2019). Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study. Neurological Sciences, 40(7), 1357-1361. https://doi.org/10.1007/s10072-019-03847-x