RESUMEN
We evaluated if budesonide inhalation suspension (BIS) reduces the immunogenicity of the varicella vaccine in paediatric patients with asthma. This open-label, parallel-group, cohort study included varicella-naïve (disease and vaccine) children aged 12 months to 8 years with asthma requiring therapy. Patients who received > or = 4 weeks of asthma treatment with BIS 0.25-1 mg daily or non-steroidal conventional asthma therapy (NSCAT) daily or as needed and met eligibility requirements received the varicella vaccine (Varivax) and continued the same asthma treatment for > or = 8 weeks postvaccination. Varicella-zoster virus (VZV) antibody levels were assessed before and 6 weeks after vaccination using a glycoprotein enzyme-linked immunosorbent assay (gpELISA). Adverse events (AEs) were assessed throughout the study. Antibody levels were analysed in 243 of 274 patients who were vaccinated and received treatment. After immunisation, the percentage of patients in each group achieving a 'protective' level of VZV antibody (> or = 5 gpELISA units/ml) was similar: 85% (129/151) in the BIS group and 90% (83/92) in the NSCAT group (relative risk = 0.95; 95% confidence interval 0.86-1.04). Eight patients in each group reported AEs related to varicella vaccination (primarily pyrexia, agitation and injection-site reactions). There were no cases of severe varicella in either group; one case of mild varicella-like rash was reported in a 12-month-old child in the NSCAT group 11 days after vaccination. VZV antibody responses and tolerability to the live varicella vaccine in paediatric asthma patients treated with BIS vs. NSCAT were comparable, demonstrating that young children with asthma receiving nebulised BIS can be immunised effectively with Varivax.
Asunto(s)
Antiasmáticos/farmacología , Antiinflamatorios no Esteroideos/farmacología , Asma/inmunología , Budesonida/farmacología , Vacuna contra la Varicela/inmunología , Administración por Inhalación , Antiasmáticos/administración & dosificación , Antiinflamatorios no Esteroideos/administración & dosificación , Formación de Anticuerpos , Asma/tratamiento farmacológico , Budesonida/administración & dosificación , Varicela/inmunología , Varicela/prevención & control , Vacuna contra la Varicela/efectos adversos , Niño , Preescolar , Estudios de Cohortes , Humanos , Lactante , Cooperación del PacienteRESUMEN
BACKGROUND/OBJECTIVE: Studies in animal models of Parkinson's disease (PD) suggest that GM1 ganglioside treatment can restore neurologic and dopaminergic function. In view of positive preclinical findings and the results of a previous open-label study demonstrating efficacy of GM1 in PD patients, this study compared effects of GM1 ganglioside and placebo on motor functions in PD patients. METHODS: Forty-five patients with mild to moderate PD were studied. The primary efficacy measure was change in the Unified Parkinson's Disease Rating Scale (UPDRS) motor score. After three independent baseline assessments, patients received IV infusion of the test drug (1,000 mg GM1 or placebo) and then self-administered either GM1 or placebo twice daily (200 mg/day, subcutaneously) for 16 weeks. Patients were examined during monthly follow-up visits. RESULTS: There was a significant difference between groups in UPDRS motor scores at 16 weeks (p=0.0001). The activities of daily living portion of the UPDRS (off-period assessment) also showed a significant effect in favor of the GM1-treated patients (p=0.04). GM1-treated patients also had significantly greater mean improvements than placebo-treated patients in performance of timed motor tests including tests of arm, hand, and foot movements, and walking. GM1 was well tolerated and no serious adverse events were reported. CONCLUSIONS: This study demonstrates that GM1 ganglioside treatment enhances neurologic function significantly in PD patients. Further study is warranted to evaluate long-term effects of GM1 in PD patients and to elucidate further the mechanisms underlying patient improvements.
Asunto(s)
Gangliósido G(M1)/uso terapéutico , Enfermedad de Parkinson/tratamiento farmacológico , Enfermedad de Parkinson/fisiopatología , Actividades Cotidianas , Adulto , Anciano , Método Doble Ciego , Femenino , Humanos , Infusiones Intravenosas , Masculino , Persona de Mediana Edad , Movimiento/fisiología , Placebos , Estudios Prospectivos , Desempeño Psicomotor/fisiología , Índice de Severidad de la Enfermedad , Caminata/fisiologíaRESUMEN
We performed an open-label study testing the effects of GM1 ganglioside on 10 Parkinson's disease (PD) patients. Patients received 1,000 mg of GM1 by IV infusion after the last of three baseline functional assessments. Patients then self-administered GM1 at a dose of 200 mg/d, by subcutaneous injection, for 18 weeks. Under these conditions, GM1 ganglioside proved to be safe and well tolerated. There were no serious adverse events and none of the patients developed elevated anti-GM1 antibody titers. Improvements on at least some functional measures were present in most patients, beginning after 4 to 8 weeks of GM1 treatment. When functional improvements occurred, they lasted for the duration of the study. These results suggest that GM1 ganglioside is well tolerated and may be a useful adjunct to the treatment of PD, and that a double-blind, placebo-controlled study is now warranted.