Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof-of-Principle Issues from a Randomized Controlled Study.
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Rodriguez-Labrada Roberto, Ortega-Sanchez Ricardo, Hernández Casaña Patricia, Santos Morales Orestes, Padrón-Estupiñan Maria Del Carmen, Batista-Nuñez Maricela, Jiménez Rodríguez Daise, Canales-Ochoa Nalia, Peña Acosta Arnoy, Medrano Montero Jacqueline, Labrada Aguilera Pedro Enrique, Estupiñán Rodriguez Annelie, Vazquez-Mojena Yaimee, Almaguer Gotay Dennis, Aymed-García Judey, García-García Idrian, Torres Vega Reydenis, Viada González Carmen, Valenzuela Silva Carmen M, Silva Ricardo Yanelis, Columbié Ximelis Jorge, Tribin Rivero Kenia, Valle Cabrera Roselin, García-Rodriguez Julio Cesar, Crombet Ramos Tania, Amaro-González Daniel, Rodriguez-Obaya Teresita, Velázquez-Pérez Luis
Аннотация
OBJECTIVE: The study aims to investigate the safety, tolerability, and clinical effects of a nasally administered recombinant EPO in SCA2 patients.
METHODS: Thirty-four patients were enrolled in this double-blind, randomized, placebo-controlled, phase I-II clinical trial of the nasally administered human-recombinant EPO (NeuroEPO) for 6 months. The primary outcome was the change in the spinocerebellar ataxia functional index (SCAFI), while other motor, neuropsychological, and oculomotor measures were assessed.
RESULTS: The 6-month changes in SCAFI score were slightly higher in the patients allocated to NeuroEPO treatment than placebo in spite of the important placebo effect observed for this parameter. However, saccade latency was significantly decreased in the NeuroEPO group but not in placebo. The frequency and severity of adverse events were similar between both groups, without evidences of hematopoietic activity of the drug.
CONCLUSIONS: This study demonstrated the safety and tolerability of NeuroEPO in SCA2 patients after 6 months of treatments and suggested a small clinical effect of this drug on motor and cognitive abnormalities, but confirmatory studies are warranted. © 2022 International Parkinson and Movement Disorder Society.