This multicentre, blinded, sham-controlled study was performed to assess the safety and effectiveness of bronchial valve therapy using a bilateral upper lobe treatment approach without the goal of lobar atelectasis.Patients with upper lobe predominant severe emphysema were randomised to bronchoscopy with (n537) or without (n536) IBV Valves for a 3-month blinded phase. A positive responder was defined as having both a o4-point improvement in St George's Respiratory Questionnaire (SGRQ) and a lobar volume shift as measured by quantitative computed tomography.At 3 months, there were eight (24%) positive responders in the treated group versus none (0%) in the control group (p50.002). Also, there was a significant shift in volume in the treated group from the upper lobes (mean¡SD -7.3¡9.0%) to the non-treated lobes (6.7¡14.5%), with minimal change in the control group (p,0.05). Mean SGRQ total score improved in both groups (treatment: -4.3¡16.2; control: -3.6¡10.7). The procedure and devices were well tolerated and there were no differences in adverse events reported in the treatment and control groups.Treatment with bronchial valves without complete lobar occlusion in both upper lobes was safe, but not effective in the majority of patients.

Background Pulmonary rehabilitation is one of the main interventions to reduce the use of health resources, and it promotes a reduction in chronic obstructive pulmonary disease (COPD) costs. mHealth systems in COPD aim to improve adherence to maintenance programs after pulmonary rehabilitation by promoting the change in attitude and behavior necessary for patient involvement in the management of the disease. Objective This study aimed to assess the effects of an integrated care plan based on an mHealth web-based platform (HappyAir) on adherence to a 1-year maintenance program applied after pulmonary rehabilitation in COPD patients. Methods COPD patients from three hospitals were randomized to a control group or an intervention group (HappyAir group). Patients from both groups received an 8-week program of pulmonary rehabilitation and educational sessions about their illness. After completion of the process, only the HappyAir group completed an integrated care plan for 10 months, supervised by an mHealth system and therapeutic educator. The control group only underwent the scheduled check-ups. Adherence to the program was rated using a respiratory physiotherapy adherence self-report (CAP FISIO) questionnaire. Other variables analyzed were adherence to physical activity (Morisky-Green Test), quality of life (Chronic Obstructive Pulmonary Disease Assessment Test, St. George’s Respiratory Questionnaire, and EuroQOL-5D), exercise capacity (6-Minute Walk Test), and lung function. Results In total, 44 patients were recruited and randomized in the control group (n=24) and HappyAir group (n=20). Eight patients dropped out for various reasons. The CAP FISIO questionnaire results showed an improvement in adherence during follow-up period for the HappyAir group, which was statistically different compared with the control group at 12 months (56.1 [SD 4.0] vs 44.0 [SD 13.6]; P=.004) after pulmonary rehabilitation. Conclusions mHealth systems designed for COPD patients improve adherence to maintenance programs as long as they are accompanied by disease awareness and patient involvement in management. Trial Registration ClinicalTrials.gov NCT04479930; https://clinicaltrials.gov/ct2/show/NCT04479930

MDSGene is an online database on movement disorders that collates genetic and clinical knowledge using a standardized published literature abstraction strategy. This review is dedicated to X-linked dystonia-parkinsonism (XDP). We screened 233 citations and curated phenotypic and genotypic data for 414 cases. To reduce data missingness, we (1) contacted authors and engaged the research community to provide additional clinical and genetic information, and (2) revisited previously unpublished data from a cohort of XDP patients seen at our institution. Using these approaches, we expanded the cohort to 577 cases and increased information available for important clinical and genetic features such as age at onset, initial manifestation, predominant motor symptoms, functional impairments, and repeat size information. We established the use of mining unpublished data to expand the MDSGene workflow and present an up-to-date description of the phenomenology of XDP using an extensive collection of previously reported and unreported data.

BACKGROUND Pulmonary rehabilitation is the most successful intervention to reduce the use of health resources and it promotes a reduction in COPD costs. m-Health systems in COPD aim to improve adherence to maintenance programs after PR by promoting the change in attitude and behaviour necessary for patient involvement in the management of the disease. OBJECTIVE This study aimed to assess the effectivity to promote adherence of an m-health system designed specifically for COPD patients and their needs. METHODS COPD patients from three different hospitals were randomized to a control group (CG) or an intervention group (Happyair group: HG). They developed an 8-week program of rehabilitation and educational sessions about their illness. After completion of the process, only HG performed a comprehensive maintenance program for 10 months, supervised by an m-health system and therapeutic educator. The control group only underwent the scheduled check-ups. Adherence to the program was rated using CAP FISIO questionnaire. Other variables as adherence to physical activity, quality of life or exercise capacity were analysed by Morisky-Green questionnaire, CAT questionnaire and 6MWT, respectively. RESULTS In total, 44 patients were recruited and randomized in CG(n=20) and HG(n=24). 8 patients dropped out for different reasons. CAP FISIO questionnaire results showed an improvement in adherence during follow up period, for HG, observed at 6 and 12 months after pulmonary rehabilitation: 6 months (53.6±5.4; P<0.05) and 12 months (56.1±4; P<0.04) CONCLUSIONS m-health systems designed for COPD patients improve adherence to maintenance programs, as long as they are accompanied by disease awareness and patient involvement in management.