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Aim: Evidence suggests low-carbohydrate diets (LCHF) may assist in treating neurodegenerative diseases such as Parkinson's disease (PD); however, gaps exist in the literature. Patients & methods: We conducted a small 24-week pilot study to investigate the effects of an LCHF diet on motor and nonmotor symptoms, health biomarkers, anxiety, and depression in seven people with PD. We also captured patient experiences during the process (quality of life [QoL]). Results: Participants reported improved biomarkers, enhanced cognition, mood, motor and nonmotor symptoms, and reduced pain and anxiety. Participants felt improvements enhanced their QoL. Conclusion: We conclude that an LCHF intervention is safe, feasible, and potentially effective in mitigating the symptoms of this disorder. However, more extensive randomized controlled studies are needed to create generalizable recommendations.
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Introducción. A pesar de la importante presencia de la población haitiana en países de América Latina, existen pocos estudios que evalúen la salud mental en esta población; una de las razones es la falta de instrumentos psicométricos en kreyol (creole haitiano). Objetivo. Adaptar la versión en español de la Escala de Depresión del Centro de Estudios Epidemiológicos (CESD-R-20) al kreyol. Métodos. Estudio transversal de muestreo bietápico con 207 migrantes haitianos viviendo en Santiago de Chile, se tradujo y retrotradujo la Escala CESD-R-20 del español al kreyol. Resultados. 132 participantes respondieron la encuesta en kreyol y 75 en español. Las técnicas de detección de la dimensionalidad recomiendan retener un único componente y tres factores, con pocas excepciones a través de las muestras. Las soluciones factoriales llevan a retener un único factor, que presenta un Alfa de Cronbach =0.92 (IC: 0.90-0.94) adecuado. Estos resultados indican que se necesita un único factor. Discusión y conclusión. La adaptación del instrumento CESD-R-20 de español al kreyol obtiene evidencias de validez favorables y puede servir para iniciar nuevos estudios en población migrante haitiana en su trayecto por América Latina, si bien es necesario seguir ampliando el proceso de validación.
Introduction. Although the Haitian migrant population has a significant presence in Latin American countries, a few studies assess this population's mental health; one of the reasons is the lack of psychometric instruments in kreyol (Haitian Creole). Objective. Adapt the Spanish version of the Depression Scale of the Center for Epidemiological Studies (CESD-R-20) into kreyol. Methods. Based on a cross-sectional two-stage cluster sampling with 207 Haitian migrants living in Santiago de Chile, we conducted translation and back translation of the CESD-R-20 Scale from Spanish to kreyol; additionally, we reached an adapted version in Spanish. Results. In total, 132 participants answered the survey in Kreyol and 75 in Spanish. Dimensionality detection techniques recommend retaining a single component and three factors, with few exceptions across samples. Exploration of factor solutions leads to the retention of a single factor, which has an adequate Cronbach's alpha=0.92 (IC: 0.90;0.94). These results indicate that only one factor is needed. Discussion and conclusion. The adaptation of the Spanish CESD-R-20 instrument to Kreyol has obtained favorable validity evidence and can be used to initiate new studies on the Haitian migrant population on their journey through Latin America, although the validation process needs to be further extended.
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The use of available treatments for Fabry disease (FD) (including enzyme replacement therapy [ERT]) may be restricted by their limited symptom improvement and mode of administration. Lucerastat is currently being investigated in the MODIFY study as oral substrate reduction therapy for the treatment of FD. By reducing the net globotriaosylceramide (Gb3) load in tissues, lucerastat has disease-modifying potential to improve symptoms and delay disease progression. MODIFY is a multicenter, double-blind, randomized, placebo-controlled, parallel-group Phase 3 study (ClinicalTrial.gov: NCT03425539); here we present the rationale and design of this study. Eligible adults with a genetically confirmed diagnosis of FD and FD-specific neuropathic pain entered screening. Patients were randomized (2:1) to receive either oral lucerastat twice daily or placebo for 6 months; treatment allocation was stratified according to sex and ERT treatment status. The main objectives of MODIFY are to assess the effects of lucerastat on neuropathic pain, gastrointestinal (GI) symptoms, FD biomarkers, and determine its safety and tolerability. Neuropathic pain and GI symptoms are key features of FD that have a significant impact on quality of life. Despite various tools available to assess pain and GI symptoms, there are currently limited tools available to assess neuropathic and GI symptoms in FD, validated according to health authority guidelines. Based on FDA recommendations, we undertook a patient-reported outcome (PRO) validation study, using a novel eDiary-based PRO tool to assess the validity of evaluating neuropathic pain as a primary efficacy endpoint in MODIFY. Results from the PRO validation study are included. To date, MODIFY is the largest Phase 3 clinical study conducted in patients with FD. Enrollment to MODIFY is now complete, with 118 patients randomized. Results will be presented in a separate publication. Long-term effects of lucerastat are being assessed in the ongoing open-label extension study (NCT03737214).