RESUMO
BACKGROUND: Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is endogenously reduced in these patients. 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.
Assuntos
Eritropoetina , Ataxias Espinocerebelares , Método Duplo-Cego , Epoetina alfa , Eritropoetina/uso terapêutico , Estudos de Viabilidade , Humanos , Proteínas Recombinantes/uso terapêutico , Ataxias Espinocerebelares/tratamento farmacológicoRESUMO
Se describe el caso clínico de una paciente de 18 años de edad, con antecedentes de otitis media crónica supurada. Fue ingresada en el Servicio de Otorrinolaringología del Hospital Provincial Docente Clinicoquirúrgico "V.I. Lenin de Holguín" por sospecha clínica de complicación de su enfermedad de base. Se le indicaron estudios según se requieren en estos casos y antibioticoterapia endovenosa. Se comprobó el diagnóstico de otitis media crónica supurada con complicación endocraneana siendo sometida de urgencia a tratamiento quirúrgico (antroaticotomía), teniendo evolución favorable y egresando a los 19 días fuera de peligro.
Describes the clinical case of a patient of 18-year-old with a history of otitis media Chronic suppurative. Clinical and surgical teaching "V.I. Lenin de Holguín" was entered into the service of Otolaryngology of the Provincial Hospital by clinical suspicion of complication of their underlying disease. Studies were identified as required in these cases and intravenous antibiotic therapy. Found the diagnosis of otitis media chronic definition with complication endocranial still undergoing emergency surgical treatment (Antroaticotomy), least favourable and graduating at 19 days out of danger.
