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☐ ☆ ✇ BMJ Open

Exploring the evidence for the use of protein biomarkers of muscular damage and disease progression in Duchenne and Becker muscular dystrophy: a systematic review and meta-analysis protocol

Por: Mondello · S. · McGill · A. · Lu · A. · Spitali · P. · Russell · A. J. · Belfiore-Oshan · R. · Martin · P. B. · on behalf of the Duchenne Regulatory Science Consortium — Noviembre 5th 2025 at 06:23
Introduction

Duchenne and Becker muscular dystrophies (DMD and BMD) are devastating conditions characterised by progressive muscle degeneration and weakness. Despite advances in understanding their pathogenetic processes, there is a critical need for reliable biomarkers to aid in patient stratification and inform clinical decision-making, predict disease progression and evaluate therapeutic responses. Several promising protein biomarkers have been investigated as potential diagnostic/prognostic tools, but, to date, this evidence has not been systematically synthesised. We aim to comprehensively and critically review and summarise published studies reporting the use of protein signatures of muscular damage in DMD and BMD.

Methods and analysis

We will systematically search Ovid MEDLINE (PubMed), OVID Embase, OVID Evidence-Based Medicine Reviews and Cochrane Library to retrieve all relevant articles. For ongoing trials, we will search WHO International clinical trials registry and ClinicalTrials.gov registry. We will include studies that measure circulating and urine levels of established and/or promising protein biomarkers associated with skeletal muscular damage and disease progression, such as creatine kinase, myoglobin, skeletal troponin I fast-twitch (type II), myostatin, creatine/creatinine ratio, creatinine and titin. We will consider randomised controlled trials, observational studies and longitudinal cohort studies with serial sampling, without restrictions on sample size, geographic location or language, while excluding animal and in vitro studies. Two independent reviewers will screen articles for inclusion using predefined eligibility criteria and extract data of retained articles. A third author will be consulted in case of disagreement. The approach recommended by the Agency for Healthcare Research and Quality’s Methods Guide for Effectiveness and Comparative Effectiveness Reviews will be used. The risk of bias and reporting quality will be assessed with standardised scales. The analysis will involve a structured narrative synthesis and evidentiary tables. If a meta-analysis is possible, biomarker data for each outcome will be pooled using random effects models. Subgroup analyses have been planned as a function of age, genetic mutation, disease severity, imaging and clinical assessment, length of the observation and risk of bias.

Ethics and dissemination

Ethics approval is not required for this study as no original data will be collected. The findings will be shared through peer-reviewed publications and conference presentations. Additionally, this systematic review will guide the recommendations of the Duchenne Regulatory Science Consortium. This work will provide a rigorous, exhaustive and accessible evidence synthesis to identify candidate biomarkers of potential clinical value. Furthermore, it is expected that these results could be used to facilitate the development of future research strategies and guidelines, inform resource allocation decisions and accelerate the route towards clinical implementation of biomarkers for DMD and BMD.

PROSPERO registration number

CRD42024549471. Available from: https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42024549471

☐ ☆ ✇ BMJ Open

Insights and interventions for improving cultural humility towards Asian American, Native Hawaiian and Pacific Islander populations in medical curricula: a qualitative case study

Por: Cai · C. · Kong · S. · Im · C. · Mondell · E. · Le · T. K. · Irvin · N. · Lawson · S. M. — Julio 8th 2025 at 02:04
Objectives

The Asian American, Native Hawaiian and Pacific Islander (AANHPI) community is the fastest-growing racial/ethnic population in the USA. Previous research identified that medical students perceived a lack of exposure to AANHPI patients and topics in medical school curricula; however, there remains a lack of potential interventions to address this need. The goal of our study is to present a case study for identifying interventions in medical school curricula that improve cultural humility-based training for providing medical care for AANHPI populations.

Design

In this qualitative study, authors conducted four virtual focus groups with 15 medical students at a single institution to identify curricular interventions. The authors then conducted virtual semistructured interviews with eight medical educators one-on-one to explore the feasibility of the proposed interventions. Data were analysed using qualitative thematic analysis, and analysis was performed with ATLAS.ti.

Setting

Medical students and medical educators based at medical institutions in the USA.

Participants

15 medical students and eight medical educators participated in the study.

Results

All medical students (n=15) and educators (n=8) noted that there is limited engagement of AANHPI communities in current medical curricula and limited curricular components that address the diversity within the AANHPI umbrella. Medical student focus groups identified three interventions to improve cultural humility-based training for treating AANHPI patients: reflection spaces, community engagement and clinical training on documenting cultural needs. Educators supported the feasibility and importance of these interventions to prepare students to work with not only AANHPI patients but also with patients of other diverse backgrounds.

Conclusion

AANHPIs represent a heterogeneous population consisting of unique cultural heritages. Our research demonstrates the importance of highlighting this community in cultural humility curricula to provide an example of how to consider and appreciate diversity in patient populations. In this paper, we present student and medical educator-supported curricular interventions that not only increase awareness of issues impacting AANHPI communities, but also emphasise building skills of self-reflection, lifelong learning and empathy that are applicable to patients of all backgrounds.

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