Results of the ALEGRIA study in Luxembourg. An epidemiological, observational study to describe symptom impact and control in patients with GERD and an evaluation of the GERD Impact Scale.

BACKGROUND AND STUDY AIMS: The aim of this observational study was to perform the first epidemiology study in a primary care patient population with GERD in the Grand Duchy of Luxembourg and to evaluate the added value of the GERD Impact Scale (GIS) patient questionnaire. PATIENTS AND METHODS: 152 Patients with symptoms of GERD from 20 study centers were included. At visit 1, demographic data including lifestyle factors and the patients' symptoms were recorded. GERD symptoms and their severity, treatment changes and the GIS were all assessed at baseline (visit 1), visit 2 (4-6 weeks) and visit 3 (8-14 weeks). Analyses were performed on an intent-to-treat basis. RESULTS: 142 patients were included in the analysis, which comprised 50% men and 50% women with a mean BMI of 27 kg/m2. Documented lifestyle factors included consumption of caffeine-containing beverages (87% of patients), stress (62%) and alcohol consumption (53%); 44% of patients were smokers or ex-smokers. The median duration of GERD was 2.0 years. Upon inclusion, 46% were receiving, or had received, proton pump inhibitors (PPIs), antacids (44%), H2-receptor antagonists (21%) or no treatment (21%). PPIs were prescribed at the first visit in the majority of cases (94%) with 75% of patients being prescribed esomeprazole with a median daily dose of 40 mg. The GIS score correlated well with the clinician's judgment of symptom severity and was reported to help determine the appropriate treatment and evaluate the patient's response in approximately 80% of patients. CONCLUSIONS: In this, the first epidemiological study on GERD patients in the Grand Duchy of Luxembourg, data was obtained as planned. The novel patient questionnaire was judged to be helpful by the physician and data shows that the GIS may have an added value over current assessments.

Role of observational studies in supporting extrapolation of efficacy data from adults to children with epilepsy - A systematic review of the literature using lacosamide as an example.

Extrapolation of efficacy data from adults to children is accepted for focal epilepsy - the antiepileptic drug, lacosamide, has been approved for the treatment of children ≥4 years of age on this basis. Since many small-scale, open-label studies are reported in the literature before approval, a systematic review was conducted to ascertain whether results of these could be used to support extrapolation in epilepsy in the future. In the absence of randomised trials, a second analysis was conducted for reports on lacosamide use in adults with generalized epilepsies. Twenty-seven articles were included in the paediatric qualitative synthesis, and 14 in the adult. Paediatric studies were analysed separately based on seizure type: focal, generalised and mixed. In focal epilepsy, safety and seizure-related findings mirrored those observed in the adult Phase II/III trials, supporting the feasibility of data extrapolation. Few studies reported outcomes in children with epilepsies associated with generalised seizures, and those that included children with different seizure types, mostly did not provide results separately. Lacosamide treatment appeared beneficial for children and adults experiencing tonic-clonic and myoclonic seizures. Reports of seizure aggravation were inconsistent and, in many cases, could not be clearly attributed to lacosamide. Given the absence of sufficient data, evidence for the feasibility of extrapolation was not as clear-cut as it was in focal epilepsy. These results highlight the complexities of conducting trials in the generalised epilepsy setting, and the importance of studies in the real-life setting and of analysing efficacy data per generalized seizure type and syndrome.

Evaluation of the GERD Impact Scale, an international, validated patient questionnaire, in daily practice. Results of the ALEGRIA study.

BACKGROUND AND STUDY AIMS: Gastroesophageal reflux disease (GERD) is a common chronic disease that is primarily diagnosed based on symptom severity and frequency. This study gathered epidemiological data in a population of GERD patients and evaluated the added-value of the GERD Impact Scale (GIS), a novel, validated patient questionnaire, as a tool for initial and long-term patient management. PATIENTS AND METHODS: This observational study recruited patients (296 study centers) with symptomatic GERD and a history of erosive, or reflux, esophagitis. Symptoms were assessed by GIS and physician-subject interview and recorded at baseline (visit 1), at 4-6 weeks (visit 2) and 8-14 weeks (visit 3); also recorded at each visit was the physician's assessment of GERD severity and treatment changes. Analyses were performed on an intent-to-treat basis. RESULTS: Subjects (n = 1919; mean age, 55 years) were 54% female. Lifestyle characteristics included stress (approximately 70% of subjects), mean daily consumption of five cups of caffeine-containing beverages (approximately 70%), alcohol consumption of approximately nine units per week (approximately 50%) and smoking/ex-smoker (41%). Proton pump inhibitors were prescribed in 99% of cases: mainly esomeprazole (82%), with a median dose of 40 mg. Prescribed therapy was changed (mainly dosage levels) between visits in approximately 60% of subjects. The severity of GERD symptoms and GIS scores decreased substantially throughout the study. Mean GIS scores correlated positively with increasing GERD severity and clinical judgment at all visits. Physicians reported that the GIS helped them define the appropriate treatment for the patient and to evaluate the patient's response to treatment in 81% of cases. CONCLUSIONS: This study demonstrates the added-value and usefulness of the patient self-assessment GIS as a management tool for GERD.