Outcomes from a Spanish Expanded Access Program on cannabidiol treatment in pediatric and adult patients with epilepsy.

AIM: To evaluate the effectiveness and tolerability of cannabidiol (CBD) in patients with developmental and epileptic encephalopathies, including Dravet syndrome (DS), and Lennox-Gastaut syndrome (LGS), in a Spanish Expanded Access Program (EAP). METHODS: This was a multicenter, retrospective, observational study of patients treated with purified CBD in 14 hospitals across Spain. Patients with (1) written informed consent and (2) at least 6 months follow-up before the closure of the database were included. Primary effectiveness endpoints included reductions (100 %, ≥75 %, ≥50 %, ≥25 %, or 0 %) or worsening in seizure frequency (all seizure types and most disabling seizures) at 1-, 3-, 6-, and 12-month visits and at the last visit, and median relative seizure reduction between baseline and last visit. Secondary effectiveness endpoints included retention rate, reduction in seizure severity, status epilepticus, healthcare utilization, and quality of life. Primary safety endpoints included rates of adverse events (AEs) and AEs leading to discontinuation. RESULTS: One hundred and two patients (DS 12 %; LGS 59 %; other epilepsy syndromes 29 %) with a mean age of 15.9 years were enrolled. Patients were highly refractory to antiseizure medications (ASMs); mean number of prior failed ASMs was 7.5 (SD 3.7). The mean CBD dose was 13.0 mg/kg/day at the last visit. The proportion of patients with ≥50 % reduction in the total number of seizures from baseline was 44.9 % at 6 months and 38.9 % at 12 months. The median number of total seizures per month reduced by 47.6 % from baseline to the last visit. At 12 months, seizure severity was lower in 33/54 patients (61.1 %) and unchanged in 17/54 patients (31.5 %). Quality of life, based on the CAVE scale, increased from a mean score of 17.9 ± 4.7 (n = 54) at baseline to 21.7 ± 5.5 (n = 51) at the last patient visit (21.2 % improvement). The mean treatment retention time was 10.3 months. There were no statistically significant changes in the number of status epilepticus episodes, but lower healthcare utilization was observed. Adverse events occurred in sixty-eight patients (66.7 %), and the most common were somnolence (34.3 %) and diarrhea (12.7 %). Cannabidiol was discontinued exclusively due to AEs in 7.8 % of patients, increasing to 25.5 % when both lack of efficacy and AEs were considered together. CONCLUSIONS: Cannabidiol demonstrated promising effectiveness and tolerability in patients with developmental and epileptic encephalopathies taking part in a Spanish EAP.

Tratamiento con estimulación del nervio vago en pacientes con epilepsia resistente a los fármacos: experiencia en el Hospital Universitario Son Espases / Vagal nerve stimulation treatment in patients with drug resistant epilepsy: Son Espases University Hospital experience

Objetivo Presentar la experiencia en el tratamiento de la epilepsia resistente a los fármacos mediante estimulación del nervio vago (ENV) en nuestro centro, valorando el impacto de este tratamiento sobre el control de la enfermedad, así como sobre aspectos relacionados con la calidad de vida de los pacientes y de sus cuidadores principales. Material y métodos Se realizó un análisis retrospectivo de los pacientes implantados desde enero de 2004 hasta diciembre de 2012. Se evaluaron los resultados de encuestas y test completados por los pacientes y sus cuidadores principales. Resultados Se incluyeron 15 pacientes con un seguimiento medio tras la cirugía de 4,41 (0,5-8) años. La edad media en el momento de la cirugía fue de 25 (10-50) años. Más del 66% de los pacientes experimentaron una disminución en la intensidad de las crisis > 25%. El 47% presentó una reducción > 50% en la frecuencia de las mismas. Como efectos adversos indeseables un paciente presentó disfonía persistente, otro tos y molestias cervicales autolimitados, y otro molestias cervicales. En 2 pacientes fue necesaria la explantación por cefalea refractaria al tratamiento médico. No hubo complicaciones derivadas del acto (AU)

OBJECTIVE: To present our experience in treating drug-resistant epilepsy with vagal nerve stimulation in our centre, evaluating its impact on disease control and on different aspects related to the patients and main caretakers' quality of life. MATERIALS AND METHODS: This was a retrospective analysis of patients operated from January 2004 until December 2012. Interviews and tests completed by outpatients and principle caretakers were evaluated. RESULTS: Fifteen patients were included, with a mean postoperative follow-up of 4.41 (0.5-8) years. Mean age at implantation was 25 (10-50) years. Over 66% of the patients perceived a reduction greater than 25% of their crisis intensity. Forty-seven percent of the patients experienced a decrease greater than 50% in the number of crises. As undesired adverse events, one patient presented persistent dysphonia, another self-limited cough and cervical discomfort and another, persistent cervical discomfort. The device had to be removed in 2 patients due to refractory headaches. There were no complications derived from the surgical procedure. CONCLUSIONS: Vagal nerve stimulation is an effective treatment for reducing crisis frequency and intensity. The patients as well as their caretakers experience a subjective improvement in their quality of life. Despite its economic cost, it seems to reduce their care needs to a certain degree and its use may therefore be justified (AU)

[Vagal nerve stimulation treatment in patients with drug resistant epilepsy: Son Espases University Hospital experience]. / Tratamiento con estimulación del nervio vago en pacientes con epilepsia resistente a los fármacos: experiencia en el Hospital Universitario Son Espases.

OBJECTIVE: To present our experience in treating drug-resistant epilepsy with vagal nerve stimulation in our centre, evaluating its impact on disease control and on different aspects related to the patients and main caretakers' quality of life. MATERIALS AND METHODS: This was a retrospective analysis of patients operated from January 2004 until December 2012. Interviews and tests completed by outpatients and principle caretakers were evaluated. RESULTS: Fifteen patients were included, with a mean postoperative follow-up of 4.41 (0.5-8) years. Mean age at implantation was 25 (10-50) years. Over 66% of the patients perceived a reduction greater than 25% of their crisis intensity. Forty-seven percent of the patients experienced a decrease greater than 50% in the number of crises. As undesired adverse events, one patient presented persistent dysphonia, another self-limited cough and cervical discomfort and another, persistent cervical discomfort. The device had to be removed in 2 patients due to refractory headaches. There were no complications derived from the surgical procedure. CONCLUSIONS: Vagal nerve stimulation is an effective treatment for reducing crisis frequency and intensity. The patients as well as their caretakers experience a subjective improvement in their quality of life. Despite its economic cost, it seems to reduce their care needs to a certain degree and its use may therefore be justified.

Patterns of care and outcome for patients with glioblastoma diagnosed during 2008-2010 in Spain.

BACKGROUND: To assess management patterns and outcome in patients with glioblastoma multiforme (GBM) treated during 2008-2010 in Spain. METHODS: Retrospective analysis of clinical, therapeutic, and survival data collected through filled questionnaires from patients with histologically confirmed GBM diagnosed in 19 Spanish hospitals. RESULTS: We identified 834 patients (23% aged >70 years). Surgical resection was achieved in 66% of patients, although the extent of surgery was confirmed by postoperative MRI in only 41%. There were major postoperative complications in 14% of patients, and age was the only independent predictor (Odds ratio [OR], 1.03; 95% confidence interval [CI],1.01-1.05; P = .006). After surgery, 57% received radiotherapy (RT) with concomitant and adjuvant temozolomide, 21% received other regimens, and 22% were not further treated. In patients treated with surgical resection, RT, and chemotherapy (n = 396), initiation of RT ≤42 days was associated with longer progression-free survival (hazard ratio [HR], 0.8; 95% CI, 0.64-0.99; P = .042) but not with overall survival (HR, 0.79; 95% CI, 0.62-1.00; P = .055). Only 32% of patients older than 70 years received RT with concomitant and adjuvant temozolomide. The median survival in this group was 10.8 months (95% CI, 6.8-14.9 months), compared with 17.0 months (95% CI, 15.5-18.4 months; P = .034) among younger patients with GBM treated with the same regimen. CONCLUSIONS: In a community setting, 57% of all patients with GBM and only 32% of older patients received RT with concomitant and adjuvant temozolomide. In patients with surgical resection who were eligible for chemoradiation, initiation of RT ≤42 days was associated with better progression-free survival.