Burden of illness and healthcare resource use in United States patients with sporadic inclusion body myositis

Capkun, Gorana; Callan, Aoife; Tian, Haijun; Wei, Zhongyuan; Zhao, Changgeng; Agashivala, Neetu; Barghout, Victoria

doi:10.1002/mus.25686

Cited by 11 publications

(15 citation statements)

References 36 publications

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“…Dysphagia in patients with IIM not only affects quality of life [ 71 ], but is also associated with severe complications such as weight loss [ 30 , 138 ] or aspiration pneumonia [ 24 , 29 , 30 , 32 , 68 , 116 , 143 , 144 , 145 , 146 ]. Pneumonia after aspiration is particularly dangerous as this condition can be fatal [ 24 , 29 , 30 , 32 , 116 , 143 , 144 , 145 , 147 ].…”

Section: Resultsmentioning

confidence: 99%

The Impact of Dysphagia in Myositis: A Systematic Review and Meta-Analysis

Labeit

Pawlitzki

Ruck

et al. 2020

JCM

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(1) Background: Dysphagia is a clinical hallmark and part of the current American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) diagnostic criteria for idiopathic inflammatory myopathy (IIM). However, the data on dysphagia in IIM are heterogenous and partly conflicting. The aim of this study was to conduct a systematic review on epidemiology, pathophysiology, outcome and therapy and a meta-analysis on the prevalence of dysphagia in IIM. (2) Methods: Medline was systematically searched for all relevant articles. A random effect model was chosen to estimate the pooled prevalence of dysphagia in the overall cohort of patients with IIM and in different subgroups. (3) Results: 234 studies were included in the review and 116 (10,382 subjects) in the meta-analysis. Dysphagia can occur as initial or sole symptom. The overall pooled prevalence estimate in IIM was 36% and with 56% particularly high in inclusion body myositis. The prevalence estimate was significantly higher in patients with cancer-associated myositis and with NXP2 autoantibodies. Dysphagia is caused by inflammatory involvement of the swallowing muscles, which can lead to reduced pharyngeal contractility, cricopharyngeal dysfunction, reduced laryngeal elevation and hypomotility of the esophagus. Swallowing disorders not only impair the quality of life but can lead to serious complications such as aspiration pneumonia, thus increasing mortality. Beneficial treatment approaches reported include immunomodulatory therapy, the treatment of associated malignant diseases or interventional procedures targeting the cricopharyngeal muscle such as myotomy, dilatation or botulinum toxin injections. (4) Conclusion: Dysphagia should be included as a therapeutic target, especially in the outlined high-risk groups.

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Section: Resultsmentioning

confidence: 99%

The Impact of Dysphagia in Myositis: A Systematic Review and Meta-Analysis

Labeit

Pawlitzki

Ruck

et al. 2020

JCM

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“…Despite the inflammatory infiltrate on muscle biopsy, IBM remains refractory to immunotherapy. Although IBM does not usually affect longevity, patients can be markedly disabled at advanced stages, which markedly affects their quality of life and is associated with high economic burden [2]. This resulted in a continuous strive to better understand the disease pathogenesis, and identify novel therapeutic targets.…”

Section: Introductionmentioning

confidence: 99%

Inclusion Body Myositis: Update on Pathogenesis and Treatment

2018

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Inclusion body myositis is the most common acquired myopathy after the age of 50. It is characterized by progressive asymmetric weakness predominantly affecting the quadriceps and/or finger flexors. Loss of ambulation and dysphagia are major complications of the disease. Inclusion body myositis can be associated with cytosolic 5'-nucleotidase 1A antibodies. Muscle biopsy usually shows inflammatory cells surrounding and invading non-necrotic muscle fibers, rimmed vacuoles, congophilic inclusions, and protein aggregates. Disease pathogenesis remains poorly understood and consists of an interplay between inflammatory and degenerative pathways. Antigen-driven, clonally restricted, cytotoxic T cells represent a main feature of the inflammatory component, whereas abnormal protein homeostasis with protein misfolding, aggregation, and dysfunctional protein disposal is the hallmark of the degenerative component. Inclusion body myositis remains refractory to treatment. Better understanding of the disease pathogenesis led to the identification of novel therapeutic targets, addressing both the inflammatory and degenerative pathways.

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“…Currently, there is no effective therapy for IBM 1 . Even with current immunotherapies, most patients become wheelchair dependent, affecting their quality of life and causing a high economic burden with a likely underestimated annual overall healthcare cost to IBM patients more than twice that of age-matched non-IBM counterparts 7–10 .…”

Section: Introductionmentioning

confidence: 99%

Association between TDP-43 and mitochondria in inclusion body myositis

Huntley

Gao

Termsarasab

et al. 2019

Laboratory Investigation

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Inclusion body myositis (IBM) is the most common cause of primary myopathy in individuals ages 50 years and over, and is pathologically characterized by protein aggregates of p62 and mislocalized cytoplasmic TDP-43 as well as mitochondrial abnormalities in affected muscle fibers. Our recent studies have shown the accumulation of TDP-43 in mitochondria in neurons from patients with amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD), and revealed mitochondria as critical mediators of TDP-43 neurotoxicity. In this study, we investigated the association between mitochondria and TDP-43 in biopsied skeletal muscle samples from IBM patients. We found that IBM pathological markers TDP-43, phosphorylated TDP-43, and p62 all coexisted with intensively stained key subunits of mitochondrial oxidative phosphorylation complexes I-V in the same skeletal muscle fibers of patients with IBM. Further immunoblot analysis showed increased levels of TDP-43, truncated TDP-43, phosphorylated TDP-43, and p62, but decreased levels of key subunits of mitochondrial oxidative phosphorylation complexes I and III in IBM patients compared to aged matched control subjects. This is the first demonstration of the close association of TDP-43 accumulation with mitochondria in degenerating muscle fibers in IBM and this association may contribute to the development of mitochondrial dysfunction and pathological protein aggregates.

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Burden of illness and healthcare resource use in United States patients with sporadic inclusion body myositis

Abstract: sIBM imposes a substantial burden on U.S. patients in terms of additional healthcare usage and associated costs. Muscle Nerve 56: 861-867, 2017.

Cited by 11 publications

References 36 publications

The Impact of Dysphagia in Myositis: A Systematic Review and Meta-Analysis

The Impact of Dysphagia in Myositis: A Systematic Review and Meta-Analysis

Inclusion Body Myositis: Update on Pathogenesis and Treatment

Association between TDP-43 and mitochondria in inclusion body myositis

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