A protocol to develop clinical guidelines for inclusion‐body myositis

Jones, Katherine; Sejersen, Thomas; Amato, Anthony A.; Hilton‐Jones, David; Schmidt, Jens; Wallace, Amanda; Badrising, Umesh A.; Rose, Michael R.

doi:10.1002/mus.25036

Cited by 10 publications

(6 citation statements)

References 20 publications

(34 reference statements)

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“…Recently, the IBM guideline development group developed a protocol to produce best practice clinical guidelines for IBM. 27 In this study, the 6MWD (primary efficacy endpoint) was chosen as the physical performance measure of choice in the IBM population based on data from the proof-ofconcept study. 15 The 6MWD is a standardised test, 28,29 approved by the US FDA as an acceptable measure of physical function in IBM participants to assess therapeutic drug effects.…”

Section: Discussionmentioning

confidence: 99%

Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

Hanna

Badrising

Benveniste

et al. 2019

The Lancet Neurology

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Background: To assess the efficacy, safety, and tolerability of bimagrumab (fully human monoclonal antibody) in participants with inclusion body myositis (IBM). Methods: This multicentre, double-blind, placebo-controlled study (RESILIENT; ClinicalTrials.gov, number NCT01925209) was conducted between September 26, 2013 and January 06, 2016 at academic clinical sites in Europe, the USA, Australia, and Japan. Eligible participants (aged 36-85 years [inclusive]; modified 2010 MRC criteria) were randomly assigned (1:1:1:1) using blocked randomisation schedule (block size=4) to receive intravenous infusions of bimagrumab 10, 3, 1 mg/kg, or placebo every 4 weeks for at least 48 weeks. All study participants, sponsor, investigators, site personnel, and those performing assessments were masked to treatment assignment. 6-minute walking distance (6MWD; primary outcome measure) was assessed at Week 52 in the primary analysis population. A multivariate normal repeated measures model was used to analyse data on 6MWD. Safety was assessed by recording adverse events (AEs), electrocardiography, echocardiography, hematology, urinalysis, and blood chemistry.

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Section: Discussionmentioning

confidence: 99%

Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

Hanna

Badrising

Benveniste

et al. 2019

The Lancet Neurology

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“…In view of the positive case series in this devastating disease that often is associated with severe dysphagia, aspiration pneumonia and an increased mortality [ 48 ], a probatory treatment with IVIG for 6 months with a dose of 1-2 g/kg every 6–8 weeks appears to be justifiable in such patients [ 47, 156, 157 ], yet its use may be restricted by national reimbursement policies. An international group of experts does currently establish the standard of care for IBM including all pharmacological and non-pharmacological treatments [ 158 ].…”

Section: Treatment Of Ibmmentioning

confidence: 99%

Current Classification and Management of Inflammatory Myopathies

Schmidt

2018

JND

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225

199

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Inflammatory disorders of the skeletal muscle include polymyositis (PM), dermatomyositis (DM), (immune mediated) necrotizing myopathy (NM), overlap syndrome with myositis (overlap myositis, OM) including anti-synthetase syndrome (ASS), and inclusion body myositis (IBM). Whereas DM occurs in children and adults, all other forms of myositis mostly develop in middle aged individuals. Apart from a slowly progressive, chronic disease course in IBM, patients with myositis typically present with a subacute onset of weakness of arms and legs, often associated with pain and clearly elevated creatine kinase in the serum. PM, DM and most patients with NM and OM usually respond to immunosuppressive therapy, whereas IBM is largely refractory to treatment. The diagnosis of myositis requires careful and combinatorial assessment of (1) clinical symptoms including pattern of weakness and paraclinical tests such as MRI of the muscle and electromyography (EMG), (2) broad analysis of auto-antibodies associated with myositis, and (3) detailed histopathological work-up of a skeletal muscle biopsy. This review provides a comprehensive overview of the current classification, diagnostic pathway, treatment regimen and pathomechanistic understanding of myositis.

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“…The sensitivity of Bclinicopathologically defined IBM^was reported as 15% and clinically defined IBM as 57% [29]. Clinical guidelines for diagnosis and management of IBM are yet to be published [30].…”

Section: Diagnosismentioning

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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A protocol to develop clinical guidelines for inclusion‐body myositis

Cited by 10 publications

References 20 publications

Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

Current Classification and Management of Inflammatory Myopathies

Inclusion Body Myositis: Update on Pathogenesis and Treatment

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