Duchenne muscular dystrophy is a devastating neuromuscular disorder characterized by the loss of dystrophin, inevitably leading to cardiomyopathy. Despite publications on prophylaxis and treatment with cardiac medications to mitigate cardiomyopathy progression, gaps remain in the specifics of medication initiation and optimization.

This document is an expert opinion statement, addressing a critical gap in cardiac care for Duchenne muscular dystrophy. It provides thorough recommendations for the initiation and titration of cardiac medications based on disease progression and patient response. Recommendations are derived from the expertise of the Advance Cardiac Therapies Improving Outcomes Network and are informed by established guidelines from the American Heart Association, American College of Cardiology, and Duchenne Muscular Dystrophy Care Considerations. These expert-derived recommendations aim to navigate the complexities of Duchenne muscular dystrophy-related cardiac care.

Comprehensive recommendations for initiation, titration, and optimization of critical cardiac medications are provided to address Duchenne muscular dystrophy-associated cardiomyopathy.

The management of Duchenne muscular dystrophy requires a multidisciplinary approach. However, the diversity of healthcare providers involved in Duchenne muscular dystrophy can result in variations in cardiac care, complicating treatment standardization and patient outcomes. The aim of this report is to provide a roadmap for managing Duchenne muscular dystrophy-associated cardiomyopathy, by elucidating timing and dosage nuances crucial for optimal therapeutic efficacy, ultimately improving cardiac outcomes, and improving the quality of life for individuals with Duchenne muscular dystrophy.

This document seeks to establish a standardized framework for cardiac care in Duchenne muscular dystrophy, aiming to improve cardiac prognosis.

To examine the effectiveness of Self-Help Plus (SH+) as an intervention for alleviating stress levels and mental health problems among healthcare workers.

This was a prospective, two-arm, unblinded, parallel-designed randomised controlled trial. Participants were recruited at all levels of medical facilities within all municipal districts of Guangzhou. Eligible participants were adult healthcare workers experiencing psychological stress (10-item Perceived Stress Scale scores of ≥15) but without serious mental health problems or active suicidal ideation. A self-help psychological intervention developed by the World Health Organization in alleviating psychological stress and preventing the development of mental health problems. The primary outcome was psychological stress, assessed at the 3-month follow-up. Secondary outcomes were depression symptoms, anxiety symptoms, insomnia, positive affect (PA) and self-kindness assessed at the 3-month follow-up.

Between November 2021 and April 2022, 270 participants were enrolled and randomly assigned to either SH+ (n = 135) or the control group (n = 135). The SH+ group had significantly lower stress at the 3-month follow-up (b = −1.23, 95% CI = −2.36, −0.10, p = 0.033) compared to the control group. The interaction effect indicated that the intervention effect in reducing stress differed over time (b = −0.89, 95% CI = −1.50, −0.27, p = 0.005). Analysis of the secondary outcomes suggested that SH+ led to statistically significant improvements in most of the secondary outcomes, including depression, insomnia, PA and self-kindness.

This is the first known randomised controlled trial ever conducted to improve stress and mental health problems among healthcare workers experiencing psychological stress in a low-resource setting. SH+ was found to be an effective strategy for alleviating psychological stress and reducing symptoms of common mental problems. SH+ has the potential to be scaled-up as a public health strategy to reduce the burden of mental health problems in healthcare workers exposed to high levels of stress.

During the COVID-19 pandemic, people with mental disorders were exposed to a common and prolonged source of stress. Studies focusing on the consequences of the pandemic on individuals with a history of mental disorder are scarce, but they suggest a higher vulnerability as compared to the general population.

We aimed at identifying predictors of stress resilience maintained over time among these people during the first two years of the pandemic.

The presented study is part of a larger 2-year, 5-wave international longitudinal online survey.

The Patient Health Questionnaire, the Generalized Anxiety Disorder scale and the PTSD Checklist DSM-5 were used as latent class indicators for a proxy measure of distress. Specifically, a Latent-Class Analysis was performed to identify a group that showed resilient outcomes across all waves.

We investigated socio-demographic characteristics, economic and housing status, lifestyle and habits, pandemic-related issues, and chronic disease. Adherence to and approval of the restrictions imposed, trust in governments and the scientific community during the pandemic were also assessed. Social support, fear of contamination and personal values were investigated respectively through the Oslo Social Support Scale, the Padua Inventory, and the Portrait Values Questionnaire. The aforementioned characteristics were used to predict sustained resilience through a logistic regression.

A total of 1711 participants out of the total sample (8011 participants from 13 different countries) reported a diagnosis of mental disorder before the pandemic. Nine hundred forty-three participants completed at least three of the five versions of the survey and were included in the analysis. A latent class of participants with resilience maintained over time (sustained resilience) was identified, with an estimated probability of 24.8%. The demographic and clinical variables associated with a higher chance of sustained resilience were older age, maintaining a job during the pandemic, and having a larger number of people in the household. In contrast, female gender, losing job during the pandemic, having difficulty meeting basic needs, greater fear of contamination, a stronger focus on hedonism, less social support and feeling lonely resulted in a lower likelihood of being sustained resilient.

This study identified a number of factors that may help predict resilient outcomes maintained over time in people with mental disorders. COVID-19 related predictors of sustained resilience are new findings which might inform resilience-building interventions during pandemics.

None Declared

Many clinical trials leverage real-world data. Typically, these data are manually abstracted from electronic health records (EHRs) and entered into electronic case report forms (CRFs), a time and labor-intensive process that is also error-prone and may miss information. Automated transfer of data from EHRs to eCRFs has the potential to reduce data abstraction and entry burden as well as improve data quality and safety.

We conducted a test of automated EHR-to-CRF data transfer for 40 participants in a clinical trial of hospitalized COVID-19 patients. We determined which coordinator-entered data could be automated from the EHR (coverage), and the frequency with which the values from the automated EHR feed and values entered by study personnel for the actual study matched exactly (concordance).

The automated EHR feed populated 10,081/11,952 (84%) coordinator-completed values. For fields where both the automation and study personnel provided data, the values matched exactly 89% of the time. Highest concordance was for daily lab results (94%), which also required the most personnel resources (30 minutes per participant). In a detailed analysis of 196 instances where personnel and automation entered values differed, both a study coordinator and a data analyst agreed that 152 (78%) instances were a result of data entry error.

An automated EHR feed has the potential to significantly decrease study personnel effort while improving the accuracy of CRF data.

To examine differences in surgical practices between salaried and fee-for-service (FFS) surgeons for two common degenerative spine conditions. Surgeons may offer different treatments for similar conditions on the basis of their compensation mechanism.

The study assessed the practices of 63 spine surgeons across eight Canadian provinces (39 FFS surgeons and 24 salaried) who performed surgery for two lumbar conditions: stable spinal stenosis and degenerative spondylolisthesis. The study included a multicenter, ambispective review of consecutive spine surgery patients enrolled in the Canadian Spine Outcomes and Research Network registry between October 2012 and July 2018. The primary outcome was the difference in type of procedures performed between the two groups. Secondary study variables included surgical characteristics, baseline patient factors, and patient-reported outcome.

For stable spinal stenosis (n = 2234), salaried surgeons performed statistically fewer uninstrumented fusion (p < 0.05) than FFS surgeons. For degenerative spondylolisthesis (n = 1292), salaried surgeons performed significantly more instrumentation plus interbody fusions (p < 0.05). There were no statistical differences in patient-reported outcomes between the two groups.

Surgeon compensation was associated with different approaches to stable lumbar spinal stenosis and degenerative lumbar spondylolisthesis. Salaried surgeons chose a more conservative approach to spinal stenosis and a more aggressive approach to degenerative spondylolisthesis, which highlights that remuneration is likely a minor determinant in the differences in practice of spinal surgery in Canada. Further research is needed to further elucidate which variables, other than patient demographics and financial incentives, influence surgical decision-making.

Data from randomized controlled trials (RCTs) are the primary source for health technology assessment (HTA) however these are limited by strict patient inclusion criteria, leading to concerns about whether treatment benefit estimates are accurate for all patients (generalizability). Real-World Data (RWD) have been proposed as a solution however as these are observational data there is additional potential for bias when estimating treatment effectiveness. To maximize the utility of RWD it is useful to consider the whole process of evidence generation and robustly address issues of feasibility and validity.

A series of complementary studies investigated whether population-based routinely collected health data from Scotland are suitable for estimating the effectiveness of chemotherapy for early breast cancer. Firstly, a prognostic score was validated in this population. Secondly, a comparison of RWD and randomized trial effectiveness estimates was made to investigate feasibility and validity of several methods – Propensity Score Matching (PSM), Instrumental variables (IV) and Regression Discontinuity. Finally, effectiveness estimates in trial underrepresented groups were produced.

PSM and IV were feasible and produced results in relatively close agreement with randomized data. Effectiveness estimates in trial underrepresented groups (women over 70 years and women with high comorbidity) were consistent with an approximate one-third reduction in the risk of death from breast cancer. This is equivalent to approximately a 3–4 percentage point difference in all cause mortality over 10 years in these groups.

RWD are a feasible for generating estimates of effectiveness of adjuvant chemotherapy in early stage breast cancer. The process of using RWD for this purpose should include careful assessment of data quality and comparison of alternative strategies for causal identification in the context of available randomized data.

Schizotypy is a putative risk phenotype for psychosis liability, but the overlap of its genetic architecture with schizophrenia is poorly understood.

We tested the hypothesis that dimensions of schizotypy (assessed with the SPQ-B) are associated with a polygenic risk score (PRS) for schizophrenia in a sample of 623 psychiatrically healthy, non-clinical subjects from the FOR2107 multi-centre study and a second sample of 1133 blood donors.

We did not find correlations of schizophrenia PRS with either overall SPQ or specific dimension scores, nor with adjusted schizotypy scores derived from the SPQ (addressing inter-scale variance). Also, PRS for affective disorders (bipolar disorder and major depression) were not significantly associated with schizotypy.

This important negative finding demonstrates that despite the hypothesised continuum of schizotypy and schizophrenia, schizotypy might share less genetic risk with schizophrenia than previously assumed (and possibly less compared to psychotic-like experiences).

Quality improvement and patient safety (QIPS) competencies are increasingly important in emergency medicine (EM) and are now included in the CanMEDS framework. We conducted a survey aimed at determining the Canadian EM residents’ perspectives on the level of QIPS education and support available to them.

An electronic survey was distributed to all Canadian EM residents from the Royal College and Family Medicine training streams. The survey consisted of multiple-choice, Likert, and free-text entry questions aimed at understanding familiarity with QIPS, local opportunities for QIPS projects and mentorship, and the desire for further QIPS education and involvement.

Of 535 EM residents, 189 (35.3%) completed the survey, representing all 17 medical schools; 77.2% of respondents were from the Royal College stream; 17.5% of respondents reported that QIPS methodologies were formally taught in their residency program; 54.7% of respondents reported being “somewhat” or “very” familiar with QIPS; 47.2% and 51.5% of respondents reported either “not knowing” or “not having readily available” opportunities for QIPS projects and QIPS mentorship, respectively; 66.9% of respondents indicated a desire for increased QIPS teaching; and 70.4% were interested in becoming involved with QIPS training and initiatives.

Many Canadian EM residents perceive a lack of QIPS educational opportunities and support in their local setting. They are interested in receiving more QIPS education, as well as project and mentorship opportunities. Supporting residents with a robust QIPS educational and mentorship framework may build a cohort of providers who can enhance the local delivery of care.

Item 9 of the Patient Health Questionnaire-9 (PHQ-9) queries about thoughts of death and self-harm, but not suicidality. Although it is sometimes used to assess suicide risk, most positive responses are not associated with suicidality. The PHQ-8, which omits Item 9, is thus increasingly used in research. We assessed equivalency of total score correlations and the diagnostic accuracy to detect major depression of the PHQ-8 and PHQ-9.

We conducted an individual patient data meta-analysis. We fit bivariate random-effects models to assess diagnostic accuracy.

16 742 participants (2097 major depression cases) from 54 studies were included. The correlation between PHQ-8 and PHQ-9 scores was 0.996 (95% confidence interval 0.996 to 0.996). The standard cutoff score of 10 for the PHQ-9 maximized sensitivity + specificity for the PHQ-8 among studies that used a semi-structured diagnostic interview reference standard (N = 27). At cutoff 10, the PHQ-8 was less sensitive by 0.02 (−0.06 to 0.00) and more specific by 0.01 (0.00 to 0.01) among those studies (N = 27), with similar results for studies that used other types of interviews (N = 27). For all 54 primary studies combined, across all cutoffs, the PHQ-8 was less sensitive than the PHQ-9 by 0.00 to 0.05 (0.03 at cutoff 10), and specificity was within 0.01 for all cutoffs (0.00 to 0.01).

PHQ-8 and PHQ-9 total scores were similar. Sensitivity may be minimally reduced with the PHQ-8, but specificity is similar.

Different diagnostic interviews are used as reference standards for major depression classification in research. Semi-structured interviews involve clinical judgement, whereas fully structured interviews are completely scripted. The Mini International Neuropsychiatric Interview (MINI), a brief fully structured interview, is also sometimes used. It is not known whether interview method is associated with probability of major depression classification.

To evaluate the association between interview method and odds of major depression classification, controlling for depressive symptom scores and participant characteristics.

Data collected for an individual participant data meta-analysis of Patient Health Questionnaire-9 (PHQ-9) diagnostic accuracy were analysed and binomial generalised linear mixed models were fit.

A total of 17 158 participants (2287 with major depression) from 57 primary studies were analysed. Among fully structured interviews, odds of major depression were higher for the MINI compared with the Composite International Diagnostic Interview (CIDI) (odds ratio (OR) = 2.10; 95% CI = 1.15–3.87). Compared with semi-structured interviews, fully structured interviews (MINI excluded) were non-significantly more likely to classify participants with low-level depressive symptoms (PHQ-9 scores ≤6) as having major depression (OR = 3.13; 95% CI = 0.98–10.00), similarly likely for moderate-level symptoms (PHQ-9 scores 7–15) (OR = 0.96; 95% CI = 0.56–1.66) and significantly less likely for high-level symptoms (PHQ-9 scores ≥16) (OR = 0.50; 95% CI = 0.26–0.97).

The MINI may identify more people as depressed than the CIDI, and semi-structured and fully structured interviews may not be interchangeable methods, but these results should be replicated.

Drs Jetté and Patten declare that they received a grant, outside the submitted work, from the Hotchkiss Brain Institute, which was jointly funded by the Institute and Pfizer. Pfizer was the original sponsor of the development of the PHQ-9, which is now in the public domain. Dr Chan is a steering committee member or consultant of Astra Zeneca, Bayer, Lilly, MSD and Pfizer. She has received sponsorships and honorarium for giving lectures and providing consultancy and her affiliated institution has received research grants from these companies. Dr Hegerl declares that within the past 3 years, he was an advisory board member for Lundbeck, Servier and Otsuka Pharma; a consultant for Bayer Pharma; and a speaker for Medice Arzneimittel, Novartis, and Roche Pharma, all outside the submitted work. Dr Inagaki declares that he has received grants from Novartis Pharma, lecture fees from Pfizer, Mochida, Shionogi, Sumitomo Dainippon Pharma, Daiichi-Sankyo, Meiji Seika and Takeda, and royalties from Nippon Hyoron Sha, Nanzando, Seiwa Shoten, Igaku-shoin and Technomics, all outside of the submitted work. Dr Yamada reports personal fees from Meiji Seika Pharma Co., Ltd., MSD K.K., Asahi Kasei Pharma Corporation, Seishin Shobo, Seiwa Shoten Co., Ltd., Igaku-shoin Ltd., Chugai Igakusha and Sentan Igakusha, all outside the submitted work. All other authors declare no competing interests. No funder had any role in the design and conduct of the study; collection, management, analysis and interpretation of the data; preparation, review or approval of the manuscript; and decision to submit the manuscript for publication.