RESUMEN
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.
Asunto(s)
Eritropoyetina , Ataxias Espinocerebelosas , Método Doble Ciego , Epoetina alfa , Eritropoyetina/uso terapéutico , Estudios de Factibilidad , Humanos , Proteínas Recombinantes/uso terapéutico , Ataxias Espinocerebelosas/tratamiento farmacológicoRESUMEN
La celulitis orbitaria se define como la inflamación de los tejidos de la órbita, es una infección con riesgo vital de las partes blandas por detrás del tejido orbitario. En la patogenia más frecuente se cita la sinusitis etmoidal. En el lactante, el seno maxilar no está neumatizado. El Streptococcus pneumoniae, Staphylococcus aureus e Haemophilus influenzae, son microorganismos frecuentes. La tomografía axial computarizada y la resonancia magnética son necesarios para detectar alteraciones de las estructuras orbitarias, los senos paranasales y el cerebro. Se presentó un cuadro de celulitis orbitaria y osteomielitis, secundaria a sinusitis etmoidal y maxilar en una lactante femenina de dos meses de edad, dolencia infrecuente que fue atendida en el Hospital Pediátrico Provincial Octavio de la Concepción de la Pedraja, de Holguín. Evolucionó satisfactoriamente, por la intervención precoz y adecuada de un equipo multidisciplinario.
Orbital cellulitis, is the inflammation of the orbital tissue, it is an infection with vital risk of the soft parts behind the orbital tissue. Ethmoidal sinusitis is reported as the more frequent pathogenic factor. The maxillary sinus is not pneumatizaised in nursling. Streptococcus pneumoniae, Staphylococcus aureus and Haemophilus influenzae are frequent microorganism. Computer axial tomography and magnetic resonance are necessary to detect orbital, paranasal sinus, and brain structures changes. A female patient of two months of age was reported in this paper, the patient presented an orbital cellulite picture, caused by ethmoidal and maxillary sinusitis, who was attended at Octavio de la Concepción de la Pedraja pediatric hospital. She had a satisfactory evolution due to the early and adequate care of a multidisciplinary team.
RESUMEN
La celulitis orbitaria se define como la inflamación de los tejidos de la órbita, es una infección con riesgo vital de las partes blandas por detrás del tejido orbitario. En la patogenia más frecuente se cita la sinusitis etmoidal. En el lactante, el seno maxilar no está neumatizado. El Streptococcus pneumoniae, Staphylococcus aureus e Haemophilus influenzae, son microorganismos frecuentes. La tomografía axial computarizada y la resonancia magnética son necesarios para detectar alteraciones de las estructuras orbitarias, los senos paranasales y el cerebro. Se presentó un cuadro de celulitis orbitaria y osteomielitis, secundaria a sinusitis etmoidal y maxilar en una lactante femenina de dos meses de edad, dolencia infrecuente que fue atendida en el Hospital Pediátrico Provincial Octavio de la Concepción de la Pedraja, de Holguín. Evolucionó satisfactoriamente, por la intervención precoz y adecuada de un equipo multidisciplinario.(AU)
Orbital cellulitis, is the inflammation of the orbital tissue, it is an infection with vital risk of the soft parts behind the orbital tissue. Ethmoidal sinusitis is reported as the more frequentpathogenic factor. The maxillary sinus is not pneumatizaised in nursling. Streptococcus pneumoniae, Staphylococcus aureus and Haemophilus influenzae are frequent microorganism. Computer axial tomography and magnetic resonance are necessary to detect orbital, paranasal sinus, and brain structures changes. A female patient of two months of age was reported in this paper, the patient presented an orbital cellulite picture, caused by ethmoidal and maxillary sinusitis, who was attended at Octavio de la Concepción de la Pedraja pediatric hospital. She had a satisfactory evolution due to the early and adequate care of a multidisciplinary team.
