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Current Opinion in Neurology Oct 2022To discuss recent developments in our understanding of epidemiology, diagnostics, biomarkers, pathology, pathogenesis, outcome measures, and therapeutics in inclusion... (Review)
Review
PURPOSE OF REVIEW
To discuss recent developments in our understanding of epidemiology, diagnostics, biomarkers, pathology, pathogenesis, outcome measures, and therapeutics in inclusion body myositis (IBM).
RECENT FINDINGS
Recent epidemiology data confirms a relatively higher prevalence in the population aged above 50 years and the reduced life expectancy. Association with cancer and other systemic disorders is better defined. The role of magnetic resonance imaging (MRI) and ultrasound in diagnosis as well as in following disease progression has been elucidated. There are new blood and imaging biomarkers that show tremendous promise for diagnosis and as outcome measures in therapeutic trials. Improved understanding of the pathogenesis of the disease will lead to better therapeutic interventions, but also highlights the importance to have sensitive and responsive outcome measures that accurately quantitate change.
SUMMARY
There are exciting new developments in our understanding of IBM which should lead to improved management and therapeutic options.
Topics: Aged; Biomarkers; Disease Progression; Humans; Magnetic Resonance Imaging; Myositis; Myositis, Inclusion Body; Ultrasonography
PubMed: 36069417
DOI: 10.1097/WCO.0000000000001095 -
Best Practice & Research. Clinical... Feb 2020Idiopathic inflammatory myopathies (IIM) are a heterogeneous group of inflammatory myopathies whose common feature is immune-mediated muscle injury. There are distinct... (Review)
Review
Idiopathic inflammatory myopathies (IIM) are a heterogeneous group of inflammatory myopathies whose common feature is immune-mediated muscle injury. There are distinct subgroups including dermatomyositis (DM), polymyositis (PM), inclusion body myositis, and immune-mediated necrotizing myopathy. Antisynthetase syndrome is also emerging as a distinct subgroup with its unique muscle histopathological characteristic of perifascicular necrosis. While the newly updated EULAR/ACR Classification Criteria for IIM have brought advancements in diagnosis and the exclusion of mimickers, the use of only one autoantibody in the derivation of the schema limits its use. Similarly, while the advent of multiple novel therapeutics in the treatment of myositis has been exciting, it has also highlighted the scarcity of validated outcome measures. The purpose of our review is to highlight the updated classification criteria of myositis, newly reported clinical phenotypes associated with myositis autoantibodies, the measurement of outcomes, and emerging treatments in the field.
Topics: Autoantibodies; Autoimmune Diseases; Dermatomyositis; Humans; Muscle, Skeletal; Myositis; Myositis, Inclusion Body
PubMed: 32046904
DOI: 10.1016/j.berh.2019.101484 -
Current Neurology and Neuroscience... May 2024Immune-mediated necrotizing myopathy (IMNM), characterized by acute or subacute onset, severe weakness, and elevated creatine kinase levels, poses diagnostic and... (Review)
Review
PURPOSE OF REVIEW
Immune-mediated necrotizing myopathy (IMNM), characterized by acute or subacute onset, severe weakness, and elevated creatine kinase levels, poses diagnostic and therapeutic challenges. This article provides a succinct overview of IMNM, including clinical features, diagnostic strategies, and treatment approaches.
RECENT FINDINGS
Recent insights highlight the different clinical presentations and therapeutic options of IMNM stratified by autoantibody positivity and type. Additionally, recent findings call into question the reported link between statin use and IMNM. This review synthesizes current knowledge on IMNM, emphasizing its distinct clinical features and challenging management. The evolving understanding of IMNM underscores the need for a comprehensive diagnostic approach that utilizes a growing range of modalities. Early and aggressive immunomodulatory therapy remains pivotal. Ongoing research aims to refine diagnostic tools and therapeutic interventions for this challenging muscle disorder, underscoring the importance of advancing our understanding to enhance patient outcomes.
Topics: Humans; Muscle, Skeletal; Necrosis; Myositis; Autoimmune Diseases; Muscular Diseases; Autoantibodies
PubMed: 38589696
DOI: 10.1007/s11910-024-01337-y -
Best Practice & Research. Clinical... Jun 2022Idiopathic inflammatory myopathies are a heterogeneous set of systemic inflammatory disorders primarily affecting muscle. Signs and symptoms vary greatly between and... (Review)
Review
Idiopathic inflammatory myopathies are a heterogeneous set of systemic inflammatory disorders primarily affecting muscle. Signs and symptoms vary greatly between and within subtypes, requiring supportive laboratory and pathologic evidence to confirm the diagnosis. Several studies are typical assessments for patients with suspected inflammatory myopathy, including muscle enzymes, autoimmune markers, imaging, and muscle biopsy. Misdiagnoses of myositis are not only related to the overlap of clinical phenotype with non-inflammatory myopathies, but also due to the limitations of diagnostic tests employed. Since many of the investigative tests are non-specific, they share features with other disorders, including muscular dystrophies, endocrine, toxic, and metabolic myopathies, and other neuromuscular or rheumatologic conditions. Recognizing the limitations of tests and understanding the shared features between inflammatory and non-inflammatory myopathies can help prevent misdiagnosing myositis with one of its several mimics.
Topics: Autoantibodies; Biomarkers; Biopsy; Dermatomyositis; Humans; Myositis; Myositis, Inclusion Body
PubMed: 35752578
DOI: 10.1016/j.berh.2022.101764 -
Brain and Nerve = Shinkei Kenkyu No... May 2024Sporadic inclusion body myositis (sIBM) is an intractable and progressive skeletal muscle disease of unknown etiology. Muscle biopsy typically reveals endomysial... (Review)
Review
Sporadic inclusion body myositis (sIBM) is an intractable and progressive skeletal muscle disease of unknown etiology. Muscle biopsy typically reveals endomysial inflammation, invasion of mononuclear cells into non-necrotic fibers, and rimmed vacuoles, suggesting that inflammation and degeneration co-exist in the pathomechanism. According to a nationwide survey conducted by a research team of the Ministry of Health, Labor, and Welfare, the number of patients is increasing in Japan as well. The clinical progression shows a slow and chronic deterioration. sIBM is usually diagnosed five years after onset. Muscle weakness and atrophy in the quadriceps, wrist flexors, and finger flexors are typical neurological findings of sIBM. Dysphagia and asymmetric weakness are often found. Serum creatine kinase is usually below 2,000 IU/L. sIBM is generally refractory to current therapy, such as steroids or immunosuppressants. Understanding the pathomechanism of sIBM is crucial for developing effective therapeutic strategies.
Topics: Myositis, Inclusion Body; Humans; Disease Progression
PubMed: 38741510
DOI: 10.11477/mf.1416202657 -
Neuromuscular Disorders : NMD Jan 2020
Topics: Autoantibodies; Biomarkers; Congresses as Topic; Dermatomyositis; Humans; Myositis; Netherlands
PubMed: 31791867
DOI: 10.1016/j.nmd.2019.10.005 -
Annals of the Rheumatic Diseases Jun 2024With improved understanding of disease pathogenesis and availability of outcome measures, there has been a remarkable increase in the number of therapeutic clinical...
With improved understanding of disease pathogenesis and availability of outcome measures, there has been a remarkable increase in the number of therapeutic clinical trials in idiopathic inflammatory myopathies (myositis) over the last three years reaching as many as five trials per site. These trials share similar design and inclusion/exclusion criteria resulting in a competitive clinical trial landscape in myositis. While these are exciting times for the myositis field, we have a number of concerns about the design and conduct of the myositis trials. These include competitive landscape, lengthy placebo arms, underrepresentation of minority groups among participants, use of patient reported outcome measures with limited/no data on validity in myositis, antiquated disease classification criteria, and unclear performance of the ACR/EULAR Myositis Response Criteria in skin-predominant patients despite inclusion of these patients in trials. In this viewpoint, we further discuss these concerns and offer potential solutions such as including patient perspectives in the trial design and adoption of innovative frameworks.
Topics: Humans; Clinical Trials as Topic; Myositis; Patient Reported Outcome Measures; Research Design
PubMed: 38216318
DOI: 10.1136/ard-2023-224652 -
Rheumatology (Oxford, England) May 2022
Topics: Adolescent; Adult; Arthritis, Juvenile; Child; Humans; Myositis; Rheumatology
PubMed: 35355064
DOI: 10.1093/rheumatology/keac115 -
Current Rheumatology Reports Aug 2020The use of lipid-lowering therapies in patients with idiopathic inflammatory myopathies (IIM) is complicated and there are no guidelines for diagnosing, monitoring, or... (Review)
Review
PURPOSE OF REVIEW
The use of lipid-lowering therapies in patients with idiopathic inflammatory myopathies (IIM) is complicated and there are no guidelines for diagnosing, monitoring, or treating atherosclerotic cardiovascular disease (ASCVD) in this group of patients.
RECENT FINDINGS
The use of lipid-lowering therapies, especially statins, is recommended in patients with increased risk for ASCVD, which includes patients with inflammatory diseases, based on recent American College of Cardiology/American Heart Association (ACC/AHA) guidelines for ASCVD management. There is accumulating evidence that patients with IIM are at increased risk for ASCVD, similar to other inflammatory diseases. Lipid-lowering therapies have side effects that may be pronounced or confounding in myositis patients, potentially limiting their use. Statins are specifically contraindicated in patients with anti 3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) antibodies. Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors have been shown to be safe and potentially beneficial in patients with IIM. Here, we propose a framework for (1) ASCVD risk assessment and treatment based on ACC/AHA ASCVD primary prevention guidelines; (2) myositis disease monitoring while undergoing lipid-lowering therapy; and (3) management of statin intolerance, including, indications for the use of PCSK9 inhibitors.
Topics: Cardiovascular Diseases; Humans; Hydroxymethylglutaryl-CoA Reductase Inhibitors; Lipids; Myositis; PCSK9 Inhibitors
PubMed: 32845379
DOI: 10.1007/s11926-020-00942-3 -
Neuromuscular Disorders : NMD Oct 2021Myositis in childhood can occur under different conditions and with various aetiologies, juvenile dermatomyositis (jDM) being by far the most frequent entity. The exact... (Review)
Review
Myositis in childhood can occur under different conditions and with various aetiologies, juvenile dermatomyositis (jDM) being by far the most frequent entity. The exact diagnostic workup and precise assessment of muscular as well as extramuscular involvement of organs in these systemic autoimmune diseases are relevant for specific and adjunct treatment of complications. Many new insights have become available with respect to the pathophysiological concepts as well as modern diagnostic measures and therapeutic approaches. Autoantibody detection in the serum of children with myositis is one of the major novelties that has become widely used and that is indeed helpful for diagnostic and prognostic measures. The pathophysiological relevance of type I interferons in jDM has been studied intensively in the past years. jDM is now seen as an acquired interferonopathy and first therapeutic consequences have been drawn from this pathogenic finding with the use of Janus-kinase inhibitors for severe and not otherwise treatable children.
Topics: Autoantibodies; Child; Dermatomyositis; Humans; Myositis; Prognosis
PubMed: 34736626
DOI: 10.1016/j.nmd.2021.08.007