Clinical burden and healthcare resource utilization associated with managing transfusion-dependent ß-thalassemia in France.

Objective: This retrospective, real-world claims database analysis aimed to describe the clinical burden and healthcare resource utilization associated with managing transfusion-dependent ß-thalassemia (TDT) in France.Methods: We used the French National Health Data System (système national des données de santé) to identify eligible patients from January 1, 2012, to March 1, 2019. Inclusion criteria were a diagnosis of ß-thalassemia, ≥8 red blood cell (RBC) transfusion episodes per year in ≥2 consecutive years following the diagnosis, and ≥1 year of follow-up data. Patients were excluded if their medical records showed evidence of sickle cell disease, α-thalassemia, hereditary persistence of fetal hemoglobin, or hematopoietic stem cell transplant. Clinical complications, mortality, treatment use, and healthcare resource utilization were evaluated in the follow-up period.Results: Overall, 331 eligible patients with TDT were identified. The mean age was 26.1 (standard deviation [SD]: 18.0) years, and 50.5% were male. Common clinical complications were endocrine (26.0%), hepatobiliary (22.7%), and cardiopulmonary (18.7%). Fifteen (4.5%) patients died during follow-up, with a mortality rate of 1.16 deaths per 100 person-years (mean age of death: 52.5 years [SD: 22]). Patients had a mean of 13.5 (SD: 5.2) RBC transfusion episodes and 11.2 (SD: 5.3) iron chelation therapy treatments per patient per year. healthcare resource utilization was substantial, with a mean of 14.8 inpatient hospitalizations (including 13.8 mean inpatient day cases) and 16.9 outpatient prescriptions per patient per year.Conclusions: Patients with TDT in France experience significant clinical complications, elevated mortality, and substantial healthcare resource utilization driven by frequent RBC transfusion episodes and inpatient hospitalizations. These results reinforce the need for disease-modifying therapies for this patient population.

Associations between the neighbourhood social environment and preschool children's physical activity and screen time.

BACKGROUND: The neighbourhood social environment (NSE) has been associated with physical activity and screen time behaviours in adults and youth however less is known about this relationship in preschool-aged children (2-5 years). This study seeks to explore associations between the NSE and the physical activity and screen time behaviours of preschool-aged children. METHOD: Cross-sectional data was collected in 2019. Parents (n = 214) of preschool-aged children (m = 3.8 ± 0.8 years), from 187 different Australian postcodes representing all states and territories were invited to complete an online survey where they answered questions about their NSE (perceived social cohesion, social interaction, sense of community, social norms and neighbourhood crime) and proxy-reported their child's usual physical activity and screen time (minutes/day). Two hierarchical linear regressions were run separately to assess relationships between NSE predictor variables and physical activity and screen time. Three logistic regressions were run to determine associations between NSE constructs and the likelihood of meeting: 1) physical activity (≥ 180 min/day including ≥ 60 min of moderate-to-vigorous-intensity), 2) screen time (≤ 60 min/day) and 3) both physical activity and screen time guidelines. Child age, gender, childcare attendance, and neighbourhood level socioeconomic status (SES) were controlled for in all analyses. RESULTS: Social interaction was associated with increased daily physical activity (b = 17.76, 95%CI = 0.81, 34.71), decreased daily screen time (b = -12.77, 95%CI = -23.23, -2.23) and improved the likelihood of meeting physical activity (OR = 1.81, 95%CI = 1.20, 2.75) and combined physical activity and screen time guidelines (OR = 1.51, 95%CI = 1.03, 2.21). Higher neighbourhood crime was associated with a lower likelihood of meeting screen time guidelines (OR = 0.47, 95%CI = 0.47, 0.99). Social cohesion, sense of community and social norms were not statistically significant predictors of daily physical activity, screen time or meeting guidelines. CONCLUSION: Social interaction showed the most consistent associations with physical activity and screen time. Future research should consider potential mediators of this relationship, including parental facilitation of children's outdoor time. Improving understanding of the relationship between the NSE and physical activity and screen time in young children can help to guide community-based initiatives striving to optimise behavioural, health and social outcomes.

Comparison of Physical Activity Between Children With and Without Autism Spectrum Disorder: A Systematic Review and Meta-Analysis.

Lower levels of physical activity (PA) are often observed among children with autism spectrum disorder (ASD) compared to children without ASD; however, some studies have demonstrated few to no PA differences between the two groups. The purpose of this systematic review with meta-analysis was to compare the differences in PA between children (2-18 years) with and without ASD. An exhaustive search of five online databases was completed, and 31 studies met the inclusion criteria. A pooled random-effects Hedges's g model was used to determine differences in PA between children with and without ASD. Children with ASD were found to be significantly less physically active than children without ASD (Δ = -0.62, p < .001). Subgroup analyses revealed significant moderate to large differences in PA by intensity level, age, setting, and measurement methods. Future studies are needed to further explore the underlying mechanisms associated with lower levels of PA among children with ASD.

Technology-Based Closed-Loop Tracking for Improving Communication and Follow-up of Pathology Results.

OBJECTIVE: Failure to follow-up on laboratory test results can lead to missed diagnoses, diagnostic delays, patient harm, and potential malpractice claims against providers. State-of-the-art tracking technologies such as the radio frequency identification (RFID) can potentially improve laboratory order processing and test result communication. We conducted a comparative evaluation of differences in completion rates for 5 testing process milestones and time to reach these process milestones, with and without RFID order tracking for skin biopsy orders. METHODS: This observational study analyzed 48,515 orders from 20 dermatology providers, sent to 8 pathology laboratories in 2016 to 2017. Descriptive t tests and multiple Cox proportional hazard regressions were used to examine the differences in completion rates and times to the 5 testing process milestones, namely, (1) provider receipt of results, (2) provider review of results, (3) patient notification, (4) follow-up scheduling, and (5) order case closure, for orders processed with and without RFID order tracking. RESULTS: Descriptive statistics illustrated that all 5 testing process milestone completion rates were statistically higher for RFID tracked orders compared with non-RFID tracked orders, and RFID tracked orders took 3 to 5 days lesser than non-RFID tracked orders to reach the 5 testing process milestones. Multiple cox proportional hazard regressions showed that the process milestones were achieved faster if orders were RFID tracked versus not (hazard ratios ranged from 1.3 to 4.9). CONCLUSIONS: The RFID tracking technology considerably improved test result communication timeliness and reliability. Such technologies can be beneficial for laboratory order processing, and their effectiveness should be explored in other practice settings.

Primary Hyperoxaluria Type 1 Disease Manifestations and Healthcare Utilization: A Multi-Country, Online, Chart Review Study.

Background: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that can result in irreversible damage to the kidneys and, eventually, extrarenal organs. While kidney failure is a known consequence of PH1, few studies to date have characterized clinical consequences of PH1 prior to kidney failure, and data on healthcare resource use outcomes across different stages of disease severity in PH1 are also limited. To help fill this knowledge gap, this study characterized the clinical and healthcare resource use (HRU) burden in patients with PH1 with varying stages of kidney disease. Methods: Nephrologists in the United States, Canada, United Kingdom, France, Germany, and Italy abstracted chart data from patients with PH1 under their care via an online questionnaire. Eligible patients had confirmed PH1 and ≥2 office visits from 2016 to 2019. Results: A total of 120 patients were analyzed (median age at diagnosis, 17.4 years old, median age at index 19.5 years old, median eGFR at index 45 ml/min/1.73 m2; median follow-up 1.7 years). During follow-up, the most common PH1 manifestations were kidney stones and urinary tract infections (UTIs, both 56.8%), and the most common symptoms were fatigue/weakness (71.7%) and pain (64.6%). With regard to HRU during follow-up, 37.4% required lithotripsy, 31.3% required ureteroscopy, and 9.6% required nephrolithotomy. PH1-related hospitalizations and emergency/urgent care visits were noted for 84.0 and 81.6% of patients, respectively. Conclusions: The current study demonstrated that patients with PH1 across various stages of kidney disease exhibited a substantial clinical burden, including kidney stones, UTIs, fatigue/weakness, and pain, and required frequent HRU, including kidney stone procedures, hospitalizations, and emergency visits. These findings highlight the significant morbidity and HRU burden in patients with PH1.

Methods for Patient-Centered Interface Design of Test Result Display in Online Portals.

OBJECTIVES: Patients have unique information needs to help them interpret and make decisions about laboratory test results they receive on web-based portals. However, current portals are not designed in a patient-centered way and little is known on how best to harness patients' information needs to inform user-centered interface design of portals. We designed a patient-facing laboratory test result interface prototype based on requirement elicitation research and used a mixed-methods approach to evaluate this interface. METHODS: After designing an initial test result display prototype, we used multiple evaluation methods, including focus group review sessions, expert consultation, and user testing, to make iterative design changes. For the user testing component, we recruited 14 patient-users to collect and analyze three types of data: comments made during testing sessions, responses to post-session questionnaires, and system usability scores. RESULTS: Our initial patient-centered interface design included visual ranges of laboratory values, nontechnical descriptions of the test and result, and access to features to help patients interpret and make decisions about their results. Findings from our evaluation resulted in 6 design iterations of the interface. Results from user testing indicate that the later versions of the interface fulfilled patient's information needs, were perceived as usable, and provided access to information and techniques that facilitated patient's ability to derive meaning from each test result. CONCLUSIONS: Requirement elicitation studies can inform the design of a patient-facing test result interface, but considerable user-centered design efforts are necessary to create an interface that patients find useful. To promote patient engagement, health information technology designers and developers can use similar approaches to enhance user-centered software design in patient portals.

Beyond Dr. Google: the evidence on consumer-facing digital tools for diagnosis.

Over a third of adults go online to diagnose their health condition. Direct-to-consumer (DTC), interactive, diagnostic apps with information personalization capabilities beyond those of static search engines are rapidly proliferating. While these apps promise faster, more convenient and more accurate information to improve diagnosis, little is known about the state of the evidence on their performance or the methods used to evaluate them. We conducted a scoping review of the peer-reviewed and gray literature for the period January 1, 2014­June 30, 2017. We found that the largest category of evaluations involved symptom checkers that applied algorithms to user-answered questions, followed by sensor-driven apps that applied algorithms to smartphone photos, with a handful of evaluations examining crowdsourcing. The most common clinical areas evaluated were dermatology and general diagnostic and triage advice for a range of conditions. Evaluations were highly variable in methodology and conclusions, with about half describing app characteristics and half examining actual performance. Apps were found to vary widely in functionality, accuracy, safety and effectiveness, although the usefulness of this evidence was limited by a frequent failure to provide results by named individual app. Overall, the current evidence base on DTC, interactive diagnostic apps is sparse in scope, uneven in the information provided and inconclusive with respect to safety and effectiveness, with no studies of clinical risks and benefits involving real-world consumer use. Given that DTC diagnostic apps are rapidly evolving, rigorous and standardized evaluations are essential to inform decisions by clinicians, patients, policymakers and other stakeholders.

An electronic trigger based on care escalation to identify preventable adverse events in hospitalised patients.

BACKGROUND: Methods to identify preventable adverse events typically have low yield and efficiency. We refined the methods of Institute of Healthcare Improvement's Global Trigger Tool (GTT) application and leveraged electronic health record (EHR) data to improve detection of preventable adverse events, including diagnostic errors. METHODS: We queried the EHR data repository of a large health system to identify an 'index hospitalization' associated with care escalation (defined as transfer to the intensive care unit (ICU) or initiation of rapid response team (RRT) within 15 days of admission) between March 2010 and August 2015. To enrich the record review sample with unexpected events, we used EHR clinical data to modify the GTT algorithm and limited eligible patients to those at lower risk for care escalation based on younger age and presence of minimal comorbid conditions. We modified the GTT review methodology; two physicians independently reviewed eligible 'e-trigger' positive records to identify preventable diagnostic and care management events. RESULTS: Of 88 428 hospitalisations, 887 were associated with care escalation (712 ICU transfers and 175 RRTs), of which 92 were flagged as trigger-positive and reviewed. Preventable adverse events were detected in 41 cases, yielding a trigger positive predictive value of 44.6% (reviewer agreement 79.35%; Cohen's kappa 0.573). We identified 7 (7.6%) diagnostic errors and 34 (37.0%) care management-related events: 24 (26.1%) adverse drug events, 4 (4.3%) patient falls, 4 (4.3%) procedure-related complications and 2 (2.2%) hospital-associated infections. In most events (73.1%), there was potential for temporary harm. CONCLUSION: We developed an approach using an EHR data-based trigger and modified review process to efficiently identify hospitalised patients with preventable adverse events, including diagnostic errors. Such e-triggers can help overcome limitations of currently available methods to detect preventable harm in hospitalised patients.

Patient perceptions of receiving test results via online portals: a mixed-methods study.

Objective: Online portals provide patients with access to their test results, but it is unknown how patients use these tools to manage results and what information is available to promote understanding. We conducted a mixed-methods study to explore patients' experiences and preferences when accessing their test results via portals. Materials and Methods: We conducted 95 interviews (13 semistructured and 82 structured) with adults who viewed a test result in their portal between April 2015 and September 2016 at 4 large outpatient clinics in Houston, Texas. Semistructured interviews were coded using content analysis and transformed into quantitative data and integrated with the structured interview data. Descriptive statistics were used to summarize the structured data. Results: Nearly two-thirds (63%) did not receive any explanatory information or test result interpretation at the time they received the result, and 46% conducted online searches for further information about their result. Patients who received an abnormal result were more likely to experience negative emotions (56% vs 21%; P = .003) and more likely to call their physician (44% vs 15%; P = .002) compared with those who received normal results. Discussion: Study findings suggest that online portals are not currently designed to present test results to patients in a meaningful way. Patients experienced negative emotions often with abnormal results, but sometimes even with normal results. Simply providing access via portals is insufficient; additional strategies are needed to help patients interpret and manage their online test results. Conclusion: Given the absence of national guidance, our findings could help strengthen policy and practice in this area and inform innovations that promote patient understanding of test results.

Patient portals and health apps: Pitfalls, promises, and what one might learn from the other.

Widespread use of health information technology (IT) could potentially increase patients' access to their health information and facilitate future goals of advancing patient-centered care. Despite having increased access to their health data, patients do not always understand this information or its implications, and digital health data can be difficult to navigate when displayed in a small-format, complex interface. In this paper, we discuss two forms of patient-facing health IT tools-patient portals and applications (apps)-and highlight how, despite several limitations of each, combining high-yield features of mobile health (mHealth) apps with portals could increase patient engagement and self-management and be more effective than either of them alone. Patient portal adoption is variable, and due to design and interface limitations and health literacy issues, many people find the portal difficult to use. Conversely, apps have experienced rapid adoption and traditionally have more consumer-friendly features with easy log-in access, real-time tracking, and simplified data display. These features make the applications more intuitive and easy-to-use than patient portals. While apps have their own limitations and might serve different purposes, patient portals could adopt some high-yield features and functions of apps that lead to engagement success with patients. We thus suggest that to improve user experience with future portals, developers could look towards mHealth apps in design, function, and user interface. Adding new features to portals may improve their use and empower patients to track their overall health and disease states. Nevertheless, both these health IT tools should be subjected to rigorous evaluation to ensure they meet their potential in improving patient outcomes.