SynthesisMuscle & nerve2022
Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: A systematic review and meta-analysis.
Synthesis in Muscle & nerve, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 25 citations in OpenAlex.
- Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: A systematic review and meta-analysis.Muscle & nerve · 2022Pooled it
- Preventing Cardiomyopathy in Duchenne Muscular Dystrophy: Long-Term Follow-Up of Patients in the Randomised, Placebo-Controlled Drug-Trial of Perindopril and Bisoprolol.European journal of neurology · 2025Trial
- A Validated Prognostic Score for Time to Loss of Ambulation in Patients With Duchenne Muscular Dystrophy.Neurology · 2026Article
- U.S. health plan coverage of Neuromuscular Disease Therapies: An assessment of policy availability and restrictions.Journal of neuromuscular diseases · 2026Article
- Change in respiratory outcomes in adults with Duchenne muscular dystrophy in the era of corticosteroids.Journal of neuromuscular diseases · 2026Article
- Unmet Needs in the Care of Patients with Duchenne Muscular Dystrophy in Brazil.Arquivos de neuro-psiquiatria · 2026Observational
- A Nomogram Integrating Clinical and Cardiac Imaging for Predicting Short-Term Left Ventricular Ejection Fraction Decline in Patients with Duchenne Muscular Dystrophy.International journal of general medicine · 2026Article
- Review
- Advances in the Regulation by Immune Cells of Skeletal Myositis Outcomes.Aging and disease · 2025Review
- Development of the accredited duchenne centers program, a global program to achieve uniform and up-to-date care for all people living with duchenne muscular dystrophy.Orphanet journal of rare diseases · 2025Article
- Duchenne muscular dystrophy patients diagnosed at the asymptomatic stage: What are the benefits of early diagnosis?Turkish journal of physical medicine and rehabilitation · 2025Article
- Surgical site infection risk in neuromuscular scoliosis patients undergoing posterior spinal fusion.Spine deformity · 2025Article
- Transforming Alzheimer's Treatment: Unveiling New Potential with Drug Repurposing Strategies.Current medicinal chemistry · 2025Review
- Current Concepts in the Orthopaedic Management of Duchenne Muscular Dystrophy.Journal of the American Academy of Orthopaedic Surgeons. Global research & reviews · 2024Review
- Evolving Role of Viltolarsen for Treatment of Duchenne Muscular Dystrophy.Advances in therapy · 2024Review
- Characteristics of Patients Receiving Novel Muscular Dystrophy Drugs in Trials vs Routine Care.JAMA network open · 2024Article
- Poor bone health in Duchenne muscular dystrophy: a multifactorial problem beyond corticosteroids and loss of ambulation.Frontiers in endocrinology · 2024Review
- Step-by-Step Double-Trouble OBAIRH and DMD Diagnosis in a One-Year-Old Boy.International journal of molecular sciences · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
INTRODUCTION/
aimsPrognostic factors in Duchenne muscular dystrophy (DMD) predict the disease course and may help individualize patient care. The aim was to summarize the evidence on prognostic factors that may support treatment decisions.
methodsWe searched six databases for prospective studies that each included ≥50 DMD patients with a minimum follow-up of 1 y. Primary outcomes were age at loss of ambulation (LoA), pulmonary function (forced vital capacity percent of predicted, FVC%p), and heart failure.
resultsOut of 5074 references, 59 studies were analyzed. Corticosteroid use was associated with a delayed LoA (pooled effect hazard ratio [HR] 0.42, 95% confidence interval [CI] 0.23-0.75, I2 94%), better pulmonary function tests (higher peak FVC%, prolonged time with FVC%p > 50%, and reduced need for assisted ventilation) and delayed cardiomyopathy. Longer corticosteroid treatment was associated with later LoA (>1 y compared to <1 y; pooled HR: 0.50, 95% CI 0.27-0.90) and early treatment start (aged <5 y) may be associated with early cardiomyopathy and higher fracture risk. Genotype appeared to be an independent driver of LoA in some studies. Higher baseline physical function tests (e.g., 6-minute walk test) were associated with delayed LoA. Left ventricular dysfunction and FVC <1 L increased and the use of angiotensin-converting enzyme (ACE) inhibitors reduced the risk of heart failure and death. Fusion surgery in scoliosis may potentially preserve pulmonary function. DISCUSSION: Prognostic factors that may inform clinical decisions include age at corticosteroid treatment initiation and treatment duration, ACE-inhibitor use, baseline physical function tests, pulmonary function, and cardiac dysfunction.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.