Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2019
Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study.
СтатьяНаблюдательное исследованиеНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Cossu Giovanni, Abbruzzese Giovanni, Forni Gian Luca, Matta Gildo, Pinto Valeria, Ruffinengo Uberto, Oppo Valentina, Marchese Roberta
Аннотация
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.
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.