Dataset Information

Prospective observational study in patients with obstructive lung disease: NOVELTY design.

ABSTRACT: Asthma and chronic obstructive pulmonary disease (COPD) have overlapping clinical features and share pathobiological mechanisms but are often considered distinct disorders. Prospective, observational studies across asthma, COPD and asthma-COPD overlap are limited. NOVELTY is a global, prospective observational 3-year study enrolling ?12?000 patients ?12 years of age from primary and specialist clinical practices in 19 countries (ClinicalTrials.gov identifier: NCT02760329). NOVELTY's primary objectives are to describe patient characteristics, treatment patterns and disease burden over time, and to identify phenotypes and molecular endotypes associated with differential outcomes over time in patients with a diagnosis/suspected diagnosis of asthma and/or COPD. NOVELTY aims to recruit real-world patients, unlike clinical studies with restrictive inclusion/exclusion criteria. Data collected at yearly intervals include clinical assessments, spirometry, biospecimens, patient-reported outcomes (PROs) and healthcare utilisation (HCU). PROs and HCU will also be collected 3-monthly via internet/telephone. Data will be used to identify phenotypes and endotypes associated with different trajectories for symptom burden, clinical progression or remission and HCU. Results may allow patient classification across obstructive lung disease by clinical outcomes and biomarker profile, rather than by conventional diagnostic labels and severity categories. NOVELTY will provide a rich data source on obstructive lung disease, to help improve patient outcomes and aid novel drug development.

SUBMITTER: Reddel HK

PROVIDER: S-EPMC6355976 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature

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Prospective observational study in patients with obstructive lung disease: NOVELTY design.

Reddel Helen K HK Gerhardsson de Verdier Maria M Agustí Alvar A Anderson Gary G Beasley Richard R Bel Elisabeth H EH Janson Christer C Make Barry B Martin Richard J RJ Pavord Ian I Price David D Keen Christina C Gardev Asparuh A Rennard Stephen S Sveréus Alecka A Bansal Aruna T AT Brannman Lance L Karlsson Niklas N Nuevo Javier J Nyberg Fredrik F Young Simon S SS Vestbo Jørgen J

ERJ open research 20190201 1

Asthma and chronic obstructive pulmonary disease (COPD) have overlapping clinical features and share pathobiological mechanisms but are often considered distinct disorders. Prospective, observational studies across asthma, COPD and asthma-COPD overlap are limited. NOVELTY is a global, prospective observational 3-year study enrolling ∼12 000 patients ≥12 years of age from primary and specialist clinical practices in 19 countries (ClinicalTrials.gov identifier: NCT02760329). NOVELTY's primary obje ...[more]

PMID: 30723727

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Project description:BackgroundChronic obstructive pulmonary disease (COPD) affects millions of Americans and has a high economic impact partially due to frequent emergency room visits and hospitalizations. Advances in digital health have made it possible to collect data remotely from multiple devices to assist in managing chronic diseases such as COPD.ObjectiveIn this pilot study, we evaluated the ability of patients with COPD to use the Wellinks mHealth platform to collect information from multiple modalities important to the management of COPD. We also assessed patient satisfaction and engagement with the platform.MethodsA single-site, observational, prospective pilot study (N=19) was conducted using the Wellinks platform in adults with COPD. All patients were aged over 30 years at screening, owned an iPhone, and were currently undergoing a treatment regimen that included nebulized therapy. Enrolled patients received a study kit consisting of the Flyp nebulizer, Smart One spirometer, the Nonin pulse oximeter, plus the Wellinks mHealth app, and training for all devices. For 8 weeks, participants were to enter daily symptoms and medication use manually; spirometry, nebulizer, and pulse oximeter data were automatically recorded. Data were sent to the attending physician in a monthly report. Patient satisfaction was measured via a 5-point scale and the Net Promoter Score (NPS) captured in interviews at the end of the observation period.ResultsAverage age of the patients was 79.6 (range 65-95) years. Participants (10 female; 9 male) had an average FEV1% (forced expiratory volume in 1 second as % of predicted for the patient) of 56.2% of predicted (range 23%-113%) and FEV1/forced vital capacity of 65%. COPD severity, as assessed by the Global Initiative for Chronic Obstructive Lung Disease (GOLD) classification, was mild in 2 patients, moderate in 6, and severe/very severe in 11; 9 patients were on home oxygen. During this 8-week study, average use of the spirometer was 2.5 times/week, and the pulse oximeter 4.2 times/week. Medication use was manually documented 9.0 times/week, nebulizer use 1.9 times/week, and symptoms recorded 1.2 times/week on average. The correlation coefficients of home to office measurements for peak flow and FEV1 were high (r=0.94 and 0.96, respectively). Patients found the app valuable (13/16, 81%) and easy to use (15/16, 94%). The NPS was 59.ConclusionsThis study demonstrates that our cohort of patients with COPD engaged with the Wellinks mHealth platform avidly and consistently over the 8-week period, and that patient satisfaction was high, as indicated by the satisfaction survey and the NPS of 59. In this small, selected sample, patients were both willing to use the technology and capable of doing so successfully regardless of disease severity, age, or gender. The Wellinks mHealth platform was considered useful and valuable by patients, and can assist clinicians in improved, timely decision making for better COPD management.

Project description:RATIONALE & OBJECTIVES:Glomerular diseases, including minimal change disease, focal segmental glomerulosclerosis, membranous nephropathy, and immunoglobulin A (IgA) nephropathy, share clinical presentations, yet result from multiple biological mechanisms. Challenges to identifying underlying mechanisms, biomarkers, and new therapies include the rarity of each diagnosis and slow progression, often requiring decades to measure the effectiveness of interventions to prevent end-stage kidney disease (ESKD) or death. STUDY DESIGN:Multicenter prospective cohort study. SETTING & PARTICIPANTS:Cure Glomerulonephropathy (CureGN) will enroll 2,400 children and adults with minimal change disease, focal segmental glomerulosclerosis, membranous nephropathy, or IgA nephropathy (including IgA vasculitis) and a first diagnostic kidney biopsy within 5 years. Patients with ESKD and those with secondary causes of glomerular disease are excluded. EXPOSURES:Clinical data, including medical history, medications, family history, and patient-reported outcomes, are obtained, along with a digital archive of kidney biopsy images and blood and urine specimens at study visits aligned with clinical care 1 to 4 times per year. OUTCOMES:Patients are followed up for changes in estimated glomerular filtration rate, disease activity, ESKD, and death and for nonrenal complications of disease and treatment, including infection, malignancy, cardiovascular, and thromboembolic events. ANALYTICAL APPROACH:The study design supports multiple longitudinal analyses leveraging the diverse data domains of CureGN and its ancillary program. At 2,400 patients and an average of 2 years' initial follow-up, CureGN has 80% power to detect an HR of 1.4 to 1.9 for proteinuria remission and a mean difference of 2.1 to 3.0mL/min/1.73m2 in estimated glomerular filtration rate per year. LIMITATIONS:Current follow-up can only detect large differences in ESKD and death outcomes. CONCLUSIONS:Study infrastructure will support a broad range of scientific approaches to identify mechanistically distinct subgroups, identify accurate biomarkers of disease activity and progression, delineate disease-specific treatment targets, and inform future therapeutic trials. CureGN is expected to be among the largest prospective studies of children and adults with glomerular disease, with a broad goal to lessen disease burden and improve outcomes.

Project description:BackgroundThe demographic changes in Brazil as a result of population aging is one of the fastest in the world. The far-reaching new challenges that come with a large older population are particularly disquieting in low- and middle-income countries (LMICs). Longitudinal studies must be completed in LMICs to investigate the social and biological determinants of aging and the consequences of such demographic changes in their context. Therefore, we designed the Prospective GERiatric Observational (ProGERO) study, a longitudinal study of outpatient older adults in São Paulo, Brazil, to collect data both on aging and chronic diseases, and investigate characteristics associated with adverse outcomes in this population.MethodsThe ProGERO study takes place in a geriatric outpatient clinic in the largest academic medical center in Latin America. We performed baseline health examinations in 2017 and will complete subsequent in-person visits every 3 years when new participants will also be recruited. We will use periodic telephone interviews to collect information on the outcomes of interest between in-person visits. The baseline evaluation included data on demographics, medical history, physical examination, and comprehensive geriatric assessment (CGA; including multimorbidity, medications, social support, functional status, cognition, depressive symptoms, nutritional status, pain assessment, frailty, gait speed, handgrip strength, and chair-stands test). We used a previously validated CGA-based model to rank participants according to mortality risk (low, medium, high). Our selected outcomes were falls, disability, health services utilization (emergency room visits and hospital admissions), institutionalization, and death. We will follow participants for at least 10 years.ResultsWe included 1336 participants with a mean age of 82 ± 8 years old. Overall, 70% were women, 31% were frail, and 43% had a Charlson comorbidity index score ≥ 3. According to our CGA-based model, the incidence of death in 1 year varied significantly across categories (low-risk = 0.6%; medium-risk = 7.4%; high-risk = 17.5%; P < 0.001).ConclusionThe ProGERO study will provide detailed clinical data and explore the late-life trajectories of outpatient older patients during a follow-up period of at least 10 years. Moreover, the study will substantially contribute to new information on the predictors of aging, senescence, and senility, particularly in frail and pre-frail outpatients from an LMIC city.

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Prospective observational study in patients with obstructive lung disease: NOVELTY design.

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Prospective observational study in patients with obstructive lung disease: NOVELTY design.

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