The Value of Patient-Reported Outcome (PRO) Data in Digital Healthcare: Using the How-R-you App as a PRO Instrument

This paper evaluates the value of using a mobile digital technology for the purpose of collecting Patient-Reported Outcome (PRO) data. We compare the use of a paper-based questionnaire and a mobile application as two different PRO instruments. Based on analysis, we conclude that use of the mobile application allows for a more nuanced picture of patients\u27 health to be established. Implications for diagnosis and medical treatment are discussed along with the need for future research

Rationale and Design of the First Double-Blind, Placebo-Controlled Trial with Allogeneic Adipose Tissue-Derived Stromal Cell Therapy in Patients with Ischemic Heart Failure:A Phase II Danish Multicentre Study

Background. Ischemic heart failure (IHF) has a poor prognosis in spite of optimal therapy. We have established a new allogeneic Cardiology Stem Cell Centre adipose-derived stromal cell (CSCC_ASC) product from healthy donors. It is produced without animal products, in closed bioreactor systems and cryopreserved as an off-the-shelf product ready to use. Study Design. A multicentre, double-blind, placebo-controlled phase II study with direct intramyocardial injections of allogeneic CSCC_ASC in patients with chronic IHF. A total of 81 patients will be randomised at 2 : 1 to CSCC_ASC or placebo. There is no HLA tissue type matching needed between the patients and the donors. Methods. The treatment will be delivered by direct injections into the myocardium. The primary endpoint is change in the left ventricle endsystolic volume at 6-month follow-up. Secondary endpoints are safety and changes in left ventricle ejection fraction, myocardial mass, stroke volume, and cardiac output. Other secondary endpoints are change in clinical symptoms, 6-minute walking test, and the quality of life after 6 and 12 months. Conclusion. The aim of the present study is to demonstrate safety and the regenerative efficacy of the allogeneic CSCC_ASC product from healthy donors in a double-blind, placebo-controlled, multicentre study in patients with IHF

Cerebellar mutism syndrome in children with brain tumours of the posterior fossa

Background: Central nervous system tumours constitute 25% of all childhood cancers; more than half are located in the posterior fossa and surgery is usually part of therapy. One of the most disabling late effects of posterior fossa tumour surgery is the cerebellar mutism syndrome (CMS) which has been reported in up to 39% of the patients but the exact incidence is uncertain since milder cases may be unrecognized. Recovery is usually incomplete. Reported risk factors are tumour type, midline location and brainstem involvement, but the exact aetiology, surgical and other risk factors, the clinical course and strategies for prevention and treatment are yet to be determined. Methods: This observational, prospective, multicentre study will include 500 children with posterior fossa tumours. It opened late 2014 with participation from 20 Nordic and Baltic centres. From 2016, five British centres and four Dutch centres will join with a total annual accrual of 130 patients. Three other major European centres are invited to join from 2016/17. Follow-up will run for 12 months after inclusion of the last patient. All patients are treated according to local practice. Clinical data are collected through standardized online registration at pre-determined time points pre- and postoperatively. Neurological status and speech functions are examined pre- operatively and postoperatively at 1-4 weeks, 2 and 12 months. Pre- and postoperative speech samples are recorded and analysed. Imaging will be reviewed centrally. Pathology is classified according to the 2007 WHO system. Germline DNA will be collected from all patients for associations between CMS characteristics and host genome variants including pathway profiles. Discussion: Through prospective and detailed collection of information on 1) differences in incidence and clinical course of CMS for different patient and tumour characteristics, 2) standardized surgical data and their association with CMS, 3) diversities and results of other therapeutic interventions, and 4) the role of host genome variants, we aim to achieve a better understanding of risk factors for and the clinical course of CMS - with the ultimate goal of defining strategies for prevention and treatment of this severely disabling condition.Peer reviewe

Cognitive Abilities in the Wild: Population-scale game-based cognitive assessment

Psychology and the social sciences are undergoing a revolution: It has become increasingly clear that traditional lab-based experiments fail to capture the full range of differences in cognitive abilities and behaviours across the general population. Some progress has been made toward devising measures that can be applied at scale across individuals and populations. What has been missing is a broad battery of validated tasks that can be easily deployed, used across different age ranges and social backgrounds, and employed in practical, clinical, and research contexts. Here, we present Skill Lab, a game-based approach allowing the efficient assessment of a suite of cognitive abilities. Skill Lab has been validated outside the lab in a crowdsourced population-size sample recruited in collaboration with the Danish Broadcast Company (Danmarks Radio, DR). Our game-based measures are five times faster to complete than the equivalent traditional measures and replicate previous findings on the decline of cognitive abilities with age in a large population sample. Furthermore, by combining the game data with an in-game survey, we demonstrate that this unique dataset has implication for key questions in social science, challenging the Jack-of-all-Trades theory of entrepreneurship and provide evidence for risk preference being independent of executive functioning.Comment: 11 pages, 4 figures, and 2 table