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The causes of individual relapses in children with acute lymphoblastic leukemia (ALL) remain incompletely understood. We evaluated the contribution of germline genetic factors to relapse in 2225 children treated on Children's Oncology Group trial AALL0232. We identified 302 germline single-nucleotide polymorphisms (SNPs) associated with relapse after adjusting for treatment and ancestry and 715 additional SNPs associated with relapse in an ancestry-specific manner. We tested for replication of these relapse-associated SNPs in external data sets of antileukemic drug pharmacokinetics and pharmacodynamics and an independent clinical cohort. 224 SNPs were associated with rapid drug clearance or drug resistance, and 32 were replicated in the independent cohort. The adverse risk associated with black and Hispanic ancestries was attenuated by addition of the 4 SNPs most strongly associated with relapse in these populations (for blacks: model without SNPs hazard ratio (HR)=2.32, P=2.27 × 10-4, model with SNPs HR=1.07, P=0.79; for Hispanics: model without SNPs HR=1.7, P=8.23 × 10-5, model with SNPs HR=1.31, P=0.065). Relapse SNPs associated with asparaginase resistance or allergy were overrepresented among SNPs associated with relapse in the more asparaginase intensive treatment arm (20/54 in Capizzi-methorexate arm vs 8/54 in high-dose methotrexate arm, P=0.015). Inherited genetic variation contributes to race-specific and treatment-specific relapse risk.

BackgroundPersonal safety is a widespread public health issue that affects people of all demographics. There is a growing interest in the use of mobile apps for enhancing personal safety, particularly for children and youth at risk, who are among the most vulnerable groups in society. ObjectiveThis study aims to explore what is known about the use of mobile apps for personal safety among children and youth identified to be “at risk.” MethodsA scoping review following published methodological guidelines was conducted. In total, 5 databases (Scopus, SocINDEX, PsycINFO, Compendex, and Inspec Archive) were searched for relevant scholarly articles published between January 2005 and October 2023. The gray literature was searched using Google and Google Scholar search engines. The results were reported using the PRISMA-ScR (Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews) guidelines. For summarizing the features and users’ experiences of the apps, a published framework for evaluating the quality of mobile health apps for youth was used. ResultsA total of 1986 articles were identified, and 41 (2.1%) were included in the review. Nine personal safety apps were captured and categorized into 4 groups based on the goals of the apps, as follows: dating and sexual violence prevention (n=4, 44% of apps), bullying and school violence prevention (n=2, 22% of apps), self-harm and suicide prevention (n=2, 22% of apps), and homeless youth support (n=1, 11% of apps). Of the 41 articles, 25 (61%) provided data solely on app descriptions and features, while the remaining 16 (39%) articles provided data on app evaluations and descriptions. Outcomes focused on app engagement, users’ experiences, and effectiveness. Four articles reported on app use, 3 (75%) of which reported relatively high app use. Data on users’ experience were obtained from 13 studies. In general, participants found the app features to be easy to use and useful as educational resources and personal safety tools. Most of the views were positive. Negative perceptions included redundancy of app features and a lack of usefulness. Five apps were evaluated for effectiveness (n=2, 40% dating and sexual violence prevention; n=2, 40% self-harm and suicide prevention; and n=1, 20% bullying and school violence prevention) and were all associated with a statistically significant reduction (P=.001 to .048) in harm or risk to participants at the 95% CI. ConclusionsAlthough many personal safety apps are available, few studies have specifically evaluated those designed for youth. However, the evidence suggests that mobile safety apps generally appear to be beneficial for reducing harm to at-risk children and youth without any associated adverse events. Recommendations for future research have been made to strengthen the evidence and increase the availability of effective personal safety apps for children and youth.

BackgroundHealth care organizations implement electronic health record (EHR) systems with the expectation of improved patient care and enhanced provider performance. However, while these technologies hold the potential to create improved care and system efficiencies, they can also lead to unintended negative consequences, such as patient safety issues, communication problems, and provider burnout. ObjectiveThis study aims to document metrics related to the In Basket communication hub (time in In Basket per day, time in In Basket per appointment, In Basket messages received per day, and turnaround time) of the EHR system implemented by Alberta Health Services, the province-wide health delivery system called Connect Care (Epic Systems). The objective was to identify how a newly implemented EHR system was used, the timing of its use, and the duration of use specifically related to In Basket activities. MethodsA descriptive study was conducted. Due to the diversity of specialties, the providers were grouped into medical and surgical based on previous similar studies. The participants were further subgrouped based on their self-reported clinical full-time equivalent (FTE ) measure. This resulted in 3 subgroups for analysis: medical FTE 0.5, and surgical (all of whom reported FTE >0.5). The analysis was limited to outpatient clinical interactions and explicitly excluded inpatient activities. ResultsA total of 72 participants from 19 different specialties enrolled in this study. The providers had, on average, 8.31 appointments per day during the reporting periods. The providers received, on average, 21.93 messages per day, and they spent 7.61 minutes on average in the time in In Basket per day metric and 1.84 minutes on average in the time in In Basket per appointment metric. The time for the providers to mark messages as done (turnaround time) was on average 11.45 days during the reporting period. Although the surgical group had, on average, approximately twice as many appointments per scheduled day, they spent considerably less connected time (based on almost all time metrics) than the medical group. However, the surgical group took much longer than the medical group to mark messages as done (turnaround time). ConclusionsWe observed a range of patterns with no consistent direction. There does not seem to be evidence of a “learning curve,” which would have shown a consistent reduction in time spent on the system over time due to familiarity and experience. While this study does not show how the included metrics could be used as predictors of providers’ satisfaction or feelings of burnout, the use trends could be used to start discussions about future Canadian studies needed in this area.

Abstract Introduction Drugs for rare diseases (DRDs) offer important health benefits, but challenge traditional health technology assessment, reimbursement, and pricing processes due to limited effectiveness evidence. Recently, modified processes to address these challenges while improving patient access have been proposed in Canada. This review examined processes in 12 jurisdictions to develop recommendations for consideration during formal government-led multi-sectoral discussions currently taking place in Canada. Methods (i) A scoping review of DRD reimbursement processes, (ii) key informant interviews, (iii) a case study of evaluations for and the reimbursement status of a set of 7 DRDs, and (iv) a virtual, multi-stakeholder consultation retreat were conducted. Results Only NHS England has a process specifically for DRDs, while Italy, Scotland, and Australia have modified processes for eligible DRDs. Almost all consider economic evaluations, budget impact analyses, and patient-reported outcomes; but less than half accept surrogate measures. Disease severity, lack of alternatives, therapeutic value, quality of evidence, and value for money are factors used in all decision-making process; only NICE England uses a cost-effectiveness threshold. Budget impact is considered in all jurisdictions except Sweden. In Italy, France, Germany, Spain, and the United Kingdom, specific factors are considered for DRDs. However, in all jurisdictions opportunities for clinician/patient input are the same as those for other drugs. Of the 7 DRDs included in the case study, the number that received a positive reimbursement recommendation was highest in Germany and France, followed by Spain and Italy. No relationship between recommendation type and specific elements of the pricing and reimbursement process was found. Conclusions Based on the collective findings from all components of the project, seven recommendations for possible action in Canada are proposed. These focus on defining “appropriate access”, determining when a “full” HTA may not be needed, improving coordination among stakeholder groups, developing a Canadian framework for Managed Access Plans, creating a pan-Canadian DRD/rare disease data infrastructure, genuine and continued engagement of patient groups and clinicians, and further research on different decision and financing options, including MAPs.

Abstract Background Although remote home monitoring (RHM) has the capacity to prevent exacerbations in patients with chronic obstructive pulmonary disease (COPD), evidence regarding its effectiveness remains unclear. The objective of this study was to determine the effectiveness of RHM in patients with COPD. Methods A systematic review of the scholarly literature published within the last 10 years was conducted using internationally recognized guidelines. Search strategies were applied to several electronic databases and clinical trial registries through March 2020 to identify studies comparing RHM to ‘no remote home monitoring’ (no RHM) or comparing RHM with provider’s feedback to RHM without feedback. To critically appraise the included randomized studies, the Cochrane Collaboration risk of bias tool (ROB) was used. The quality of included non-randomized interventional and comparative observational studies was evaluated using the ACROBAT-NRSI tool from the Cochrane Collaboration. The quality of evidence relating to key outcomes was assessed using Grading of Recommendations, Assessment, Development and Evaluations (GRADE) on the following: health-related quality of life (HRQoL), patient experience and number of exacerbations, number of emergency room (ER) visits, COPD-related hospital admissions, and adherence as the proportion of patients who completed the study. Three independent reviewers assessed methodologic quality and reviewed the studies. Results Seventeen randomized controlled trials (RCTs) and two comparative observational studies were included in the review. The primary finding of this systematic review is that a considerable amount of evidence relating to the efficacy/effectiveness of RHM exists, but its quality is low. Although RHM is safe, it does not appear to improve HRQoL (regardless of the type of RHM), lung function or self-efficacy, or to reduce depression, anxiety, or healthcare resource utilization. The inclusion of regular feedback from providers may reduce COPD-related hospital admissions. Though adherence RHM remains unclear, both patient and provider satisfaction were high with the intervention. Conclusions Although a considerable amount of evidence to the effectiveness of RHM exists, due to heterogeneity of care settings and the low-quality evidence, they should be interpreted with caution.

Abstract Background Although pulmonary rehabilitation (PR) is considered a key component in managing chronic obstructive pulmonary disease (COPD) patients, uptake remains suboptimal. This systematic review aimed to determine the effectiveness of home-based PR (HBPR) programs for COPD patients. Methods A systematic review of scholarly literature published within the last 10 years from the conception of this project was conducted using internationally recognized guidelines. Search strategies were applied to electronic databases and clinical trial registries through March 2020 and updated in November 2021 to identify studies comparing HBPR with ‘usual care’ or outpatient pulmonary rehabilitation (OPR). To critically appraise randomized studies, the Cochrane Collaboration risk of bias tool (ROB) was used. The quality of non-randomized studies was evaluated using the ACROBAT-NRSI tool. The quality of evidence relating to key outcomes was assessed using Grading of Recommendations, Assessment, Development and Evaluations (GRADE) on health-related quality of life (HRQoL), exacerbation frequencies, COPD-related hospital admissions, and program adherence. Three independent reviewers assessed methodologic quality and reviewed the studies. Results Twelve randomized controlled trials (RCTs) and 2 comparative observational studies were included. While considerable evidence relating to the effectiveness of HBPR programs for COPD patients exist, overall quality is low. There were no differences between HBPR and OPR in terms of safety, HRQoL, functional exercise capacity and health care resource utilization. Compared to usual care, functional exercise capacity seemed to significantly improve after HBPR. While patient compliance with HBPR is good, two factors appeared to increase the ‘risk’ of non-compliance: expectations of patients to 1) complete daily diaries/activity logs and 2) engage in solely unsupervised exercise sessions. Conclusion The overall quality for most outcomes was low to very low; however, HBPR seems to offer comparable short-term benefits to OPR.

BackgroundDiabetes—a high-burden chronic disease—requires lifetime active management involving the use of different tools and health care resources to improve patient health outcomes. Recent studies have demonstrated promising results regarding the impact of the use of virtual care technology on the treatment of chronic diseases, such as diabetes. However, it is unclear whether the use of technologies, such as secure messaging, improves the quality of care and reduces diabetes-related costs to the health care system. ObjectiveThe purpose of our scoping review is to explore what is known about the use of secure messaging in the treatment of diabetes within the primary care setting and how its impact has been assessed from the patient and health system perspectives. Our review aims to understand to what extent secure messaging improves the quality of diabetes care. MethodsOur scoping review will follow the 6-step Arksey and O’Malley methodological framework, as well as the Joanna Briggs Institute methodology for scoping reviews and their recommended tools. The tools to guide the development and reporting of the review in a structured way will include the Population, Concept, and Context framework and the PRISMA-ScR (Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews) guidelines and checklist. The search strategy was developed iteratively in collaboration with a professional information specialist. Furthermore, a peer review of electronic search strategies was also conducted by an independent, third-party, professional information specialist. A systematic literature search will be conducted against databases, including Ovid MEDLINE ALL, Embase, APA PsycINFO, Cochrane Library on Wiley, CINAHL on EBSCO, and PubMed. Grey literature sources will also be searched for relevant literature. Literature on the use of secure messaging in the treatment of diabetes (types 1 and 2) within a primary care setting will be included. Two reviewers will review the literature based on the inclusion criteria in the following two steps: (1) title and abstract review and (2) full-text review. Discrepancies will be discussed to reach consensus where possible; otherwise, a third reviewer will resolve the dispute. ResultsThe results and a final report are expected to be completed and submitted to a peer-reviewed journal in 6 months. ConclusionsThe review will examine existing literature to identify the impact of secure messaging in diabetes treatment within primary care settings. Research gaps will also be identified to determine if there is a need for further studies. International Registered Report Identifier (IRRID)DERR1-10.2196/42339