Observational Study of Deferiprone (Ferriprox®) in the Treatment of Superficial Siderosis

Plain-language summary

Superficial siderosis is a progressive neurological disease caused by iron deposition in the central nervous system (CNS) from chronic subarachnoid bleeding. Until 2011, there has been no effective treatment for this progressive condition that leads to hearing loss, spasticity, weakness, loss of bowel/bladder function, incoordination, dementia and ultimately death. Last year, the investigators demonstrated that a lipid soluble iron chelator, deferiprone, can reduce hemosiderin deposition in patients with superficial siderosis by MRI in as little as 3 months. As the only therapy that can improve this condition, chelation with deferiprone is the standard of care for treatment of superficial siderosis. Now that the FDA has approved deferiprone in the United States for thalassemia, the investigators propose documenting the clinical effect of deferiprone over 2 years in superficial siderosis patients. The investigators' goal is to understand how the clinical course of this disease is altered using standard of care chelation therapy with deferiprone. Patients with superficial siderosis who are taking deferiprone for chelation therapy at doses consistent with the standard of care will be offered enrollment into this observational study. Patients will be treated and monitored locally by participating neurologists who have agreed to help the investigators collect information for this study. The investigators are interested in documenting the clinical effect of deferiprone on hearing, ataxia and myelopathy using standardized scales performed and documenting the effect of deferiprone on hemosiderin deposition in the CNS by MRI, all performed according to standard of care.

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Trial details