Estudio longitudinal de doentes com doença de Parkinson (ELEP): objectivos e metodologia

La enfermedad de Parkinson (EP) es crónica y progresiva. Desde la perspectiva sociosanitaria, representa una fuente de sufrimiento para el paciente y sus cuidadores, así como una importante carga para la sociedad. La información actual sobre la EP es limitada en cuanto al conocimiento del curso evolutivo relacionado con: 1) el desarrollo y la evolución de los aspectos no motores de la enfermedad; 2) el impacto de estas manifestaciones sobre la discapacidad y la calidad de vida relacionada con la salud (CVRS); 3) los determinantes de la discapacidad y de la pérdida de CVRS; 4) los factores relacionados con la velocidad de progresión de la enfermedad; 5) las pautas de aplicación y la repercusión diferencial a largo plazo (sobre complicaciones, discapacidad, CVRS) de las medidas terapéuticas disponibles; y 6) el impacto de la EP sobre los cuidadores. Además, en la información existente se detecta heterogeneidad en la calidad de las propiedades métricas de los instrumentos de medida aplicados y de los sesgos de selección.Parkinson’s disease (PD) is a chronic and progressive disorder. It produces a significant burden not only for patients, but also for their family and caregivers, with a major socio-economic impact on society. Current knowledge on PD is characterized by scarce information about the evolutionary course of: 1) the non-motor PD features; 2) impact of non-motor PD features on disability and health related quality of life (HRQL) impairment; 3) factors related to disability and HRQL determinants; 4) factors that speed or slow the progression of PD; 5) differential long-term effect of available PD therapeutic schedules and their relationships with disability, complications, and HRQL; and 6) impact of the disease on patients’ caregivers. In addition, heterogeneity in the metric quality of the applied measures and selection bias are frequently foundA doença de Parkinson (DP) é crónica e progresiva. De uma perspectiva socio-sanitária, representa uma fonte de sufrimento para o paciente e seus cuidadores, assim como uma carga importante para a sociedade. A informação actual sobre a DP é limitada em quanto ao conhecimento do curso evolutivo relacionado com: 1) o desenvolvimento e a evolução dos aspectos não motores da doença; 2) o impacto destas manifestações sobre a discapacidade e a qualidade de vida relacionada com a saúde (QVRS); 3) os determinantes da discapacidade e da diminuição de QVRS; 4) os factores relacionados com a velocidade de progressão da doença; 5) as pautas de aplicação e a repercursão diferencial a longo prazo (sobre complicações, discapacidade, QVRS) das medidas terapêuticas disponíveis; e 6) o impacto da DP sobre os cuidadores. Além disso, na informação disponível há uma heterogeneidade na qualidade das propriedades métricas dos instrumentos de medida aplicados e dos enviesamentos de selecção

Criação e protocolo de seguimento longitudinal de uma coorte multipropósito de doentes com doença de Parkinson de diagnóstico recente: projecto VIP

La enfermedad de Parkinson (EP) es una enfermedad neurodegenerativa muy heterogénea desde el punto de vista etiológico, clínico y terapéutico, lo que dificulta la interpretación de resultados de estudios transversales. Son necesarios los registros de pacientes y los estudios longitudinales de cohortes bien caracterizadas desde el punto de vista clínico y terapéutico.Parkinson’s disease (PD) is a quite heterogeneous disorder, thus difficulting the interpretation of transversal studies. Patients’ registries and longitudinal studies can be considered as a priority in order to understand many still unknown aspects of the disease.A doença de Parkinson (DP) é uma doença neurodegenerativa muito heterogénea do ponto de vista etiológico, clínico e terapêutico, o que dificulta a interpretação de resultados de estudos transversais. São necessários os registos de doentes e os estudos longitudinais de coortes bem caracterizadas do ponto de vista clínico e terapêutico

A Phase II Study to Evaluate the Safety and Efficacy of Prasinezumab in Early Parkinson's Disease (PASADENA) : Rationale, Design, and Baseline Data

Altres ajuts: F. Hoffmann-La Roche Ltd.Background: Currently available treatments for Parkinson's disease (PD) do not slow clinical progression nor target alpha-synuclein, a key protein associated with the disease. Objective: The study objective was to evaluate the efficacy and safety of prasinezumab, a humanized monoclonal antibody that binds aggregated alpha-synuclein, in individuals with early PD. Methods: The PASADENA study is a multicenter, randomized, double-blind, placebo-controlled treatment study. Individuals with early PD, recruited across the US and Europe, received monthly intravenous doses of prasinezumab (1,500 or 4,500 mg) or placebo for a 52-week period (Part 1), followed by a 52-week extension (Part 2) in which all participants received active treatment. Key inclusion criteria were: aged 40-80 years; Hoehn & Yahr (H&Y) Stage I or II; time from diagnosis ≤2 years; having bradykinesia plus one other cardinal sign of PD (e.g., resting tremor, rigidity); DAT-SPECT imaging consistent with PD; and either treatment naïve or on a stable monoamine oxidase B (MAO-B) inhibitor dose. Study design assumptions for sample size and study duration were built using a patient cohort from the Parkinson's Progression Marker Initiative (PPMI). In this report, baseline characteristics are compared between the treatment-naïve and MAO-B inhibitor-treated PASADENA cohorts and between the PASADENA and PPMI populations. Results: Of the 443 patients screened, 316 were enrolled into the PASADENA study between June 2017 and November 2018, with an average age of 59.9 years and 67.4% being male. Mean time from diagnosis at baseline was 10.11 months, with 75.3% in H&Y Stage II. Baseline motor and non-motor symptoms (assessed using Movement Disorder Society-Unified Parkinson's Disease Rating Scale [MDS-UPDRS]) were similar in severity between the MAO-B inhibitor-treated and treatment-naïve PASADENA cohorts (MDS-UPDRS sum of Parts I + II + III [standard deviation (SD)]; 30.21 [11.96], 32.10 [13.20], respectively). The overall PASADENA population (63.6% treatment naïve and 36.4% on MAO-B inhibitor) showed a similar severity in MDS-UPDRS scores (e.g., MDS-UPDRS sum of Parts I + II + III [SD]; 31.41 [12.78], 32.63 [13.04], respectively) to the PPMI cohort (all treatment naïve). Conclusions: The PASADENA study population is suitable to investigate the potential of prasinezumab to slow disease progression in individuals with early PD. Trial Registration: NCT03100149