Experiencia de uso de fampridina en pacientes con nistagmo vertical inferior / Clinical use of 4-aminopyridine in patients with downbeat nystagmus

OBJETIVO: Presentar los resultados del tratamiento con fampridina de liberación prolongada (4-AP-SR) en pacientes con nistagmo vertical inferior. MATERIAL Y MÉTODO: Serie de casos de nistagmo vertical inferior tratados con 10 mg de 4-AP-SR cada 12h. Se valoraron la mejoría clínica y la variación del nistagmo antes y después del tratamiento, registrando la puntuación en el Dizziness Handicap Inventory, la velocidad de la fase lenta del nistagmo y su frecuencia. RESULTADOS: Se trataron 3 pacientes, ninguno de los cuales notó cambios durante el tratamiento en relación con los síntomas visuales, aunque 2 pacientes notaron mejoría subjetiva de la marcha. No se detectaron diferencias significativas en la puntuación del Dizziness Handicap Inventory ni en las variables videonistagmográficas estudiadas. CONCLUSIÓN: No se han detectado cambios en los síntomas visuales con el tratamiento con 4-AP-SR. La contradicción con estudios previos puede estar producida por un bajo tamaño muestral, la etiología de los casos o la forma de medir los resultado

OBJECTIVE: To present the results of treatment with sustained-release fampridine (4-AP-SR) in patients with downbeat nystagmus. MATERIAL AND METHOD: Series of cases with downbeat nystagmus treated with 10 milligrams of 4-AP-SR every 12 hours. The clinical improvement and the variation of the nystagmus before and after the treatment were evaluated, recording the score in the Dizziness Handicap Inventory, the velocity of the slow phase of the nystagmus and its frequency. RESULTS: Three patients were treated, none of whom reported significant subjective changes during the treatment. No significant differences were detected in the Dizziness Handicap Inventory score or in the studied videonystagmographic variables. CONCLUSION: This communication does not present positive results on the use of 4-AP-SR. The contradiction with previous studies may be caused by a small sample size, by the etiology of the cases or by the way in which the results were measured

Clinical use of 4-aminopyridine in patients with downbeat nystagmus. / Experiencia de uso de fampridina en pacientes con nistagmo vertical inferior.

OBJECTIVE: To present the results of treatment with sustained-release fampridine (4-AP-SR) in patients with downbeat nystagmus. MATERIAL AND METHOD: Series of cases with downbeat nystagmus treated with 10 milligrams of 4-AP-SR every 12hours. The clinical improvement and the variation of the nystagmus before and after the treatment were evaluated, recording the score in the Dizziness Handicap Inventory, the velocity of the slow phase of the nystagmus and its frequency. RESULTS: Three patients were treated, none of whom reported significant subjective changes during the treatment. No significant differences were detected in the Dizziness Handicap Inventory score or in the studied videonystagmographic variables. CONCLUSION: This communication does not present positive results on the use of 4-AP-SR. The contradiction with previous studies may be caused by a small sample size, by the etiology of the cases or by the way in which the results were measured.

A multicentre observational study of the effectiveness, safety and economic impact of nivolumab on non-small-cell lung cancer in real clinical practice.

Background Immunotherapy has become a standard treatment for lung cancer; however, the high cost makes it necessary to assess health outcomes. Objective The aim of this study was to evaluate the effectiveness, safety and economic cost of nivolumab in real-world clinical practice. Setting Fifteen regional and academic hospitals from Spain participated in this study. Methods This study was a retrospective, multicentre and observational study involving patients who experienced progression after first-line therapy for non-small-cell lung cancer and were treated with nivolumab between January 2016 and July 2017. Effectiveness and safety were evaluated by the oncologist, and the data from the electronic clinical records of the patients were collected by the research team. Economic cost was calculated using the cost of acquiring nivolumab for the public health system. Main outcome measures Effectiveness variables were overall survival (OS) and progression-free survival (PFS). The safety variable was the incidence of adverse events (AEs), and the cost per life-year gained (LYG) was the economic variable. Results A total of 221 patients were enrolled (83.7% men). The mean age was 64.5 years, and 84.6% of the patients had an Eastern Cooperative Oncology Group (ECOG) performance-status score of 0-1. Squamous tumours accounted for 59.7% of the total, and 78.7% of the patients presented a time since platinum therapy (TPT) > 6 months. The mean nivolumab dose was 216 mg (SD 211), and the treatment duration was 7.0 months (95% CI 5.8-8.1). The median PFS was 5.3 months (95% CI 3.2-7.3), and OS was 9.7 months (95% CI 7.6-11.8). The median PFS and OS values were statistically significantly superior for patients with an ECOG score of 0-1 and for patients with a TPT > 6 months. The median OS was also statistically significantly superior for patients with non-squamous histology. Regarding safety, 71% of the patients presented AEs of any grade, and in 18.6%, the nivolumab treatment had to be delayed or discontinued. The cost of nivolumab per patient was €19,910.00 (SD 19,369), and the cost per LYG was €110,026.00 (€77,557.00-€231,171.00). Conclusions This study confirms that the efficacy and safety of nivolumab treatment in a real population are comparable to the results obtained in clinical trials. A greater clinical benefit of nivolumab therapy was observed in patients with an ECOG score of 0-1, a TPT > 6 months or non-squamous histology. Despite the benefit observed, the cost per LYG is above the threshold of efficiency established by public health institutes.