Update on the pharmacological treatment of adult myositis

Oddis, Chester V.

doi:10.1111/joim.12511

Cited by 50 publications

(48 citation statements)

References 73 publications

(105 reference statements)

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“…Moreover, IVIg seems to achieve disease control in patients with severe muscle weakness (i.e. dropped head syndrome) or dysphagia [2,[8][9][10][11].…”

Section: Discussionmentioning

confidence: 99%

A Case of Refractory Dysphagia in the Context of Polymyositis Treated with Subcutaneous Immunoglobulin and Review of the Literature

Vacchi¹

2017

Clin Med Rev Case Rep

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Polymyositis (PM) is a systemic inflammatory disease affecting muscles and several other organs, including gastrointestinal system, in which the main symptom is dysphagia.Here, we describe a case of a 57-years-old woman presenting a difficult-to-manage dysphagia related to polymyositis, treated with benefit with Subcutaneous Immunoglobulins (SCIg). Beyond symptomatic therapies, immunosuppressive therapies (glucocorticoids, methotrexate, azathioprine, mycophenolatemofetil, and cyclosporine) are the main treatment of PM related dysphagia. Nowadays, few data are available to evaluate effectiveness and safety of intravenous immunoglobulins on esophageal involvement in refractory PM and only anecdotal cases are reported about the use of SCIg in the treatment of dysphagia in inflammatory myopathies (including inclusion body myositis). Up to date, this is the second observation describing an improvement of dysphagia in a subject affected by PM treated with SCIg; further studies are needed to evaluate the effectiveness of SCIg in the management of these patients.

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“…Moreover, IVIg seems to achieve disease control in patients with severe muscle weakness (i.e. dropped head syndrome) or dysphagia [2,[8][9][10][11].…”

Section: Discussionmentioning

confidence: 99%

A Case of Refractory Dysphagia in the Context of Polymyositis Treated with Subcutaneous Immunoglobulin and Review of the Literature

Vacchi¹

2017

Clin Med Rev Case Rep

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“…Inclusion body myositis is an exception from the other IIMs as it has not been shown to respond to immunosuppressant agents, including glucocorticoids. In fact, inclusion body myositis has only one indicated pharmaceutical option at this time, intravenous immunoglobulin G, discussed below [24]. The remaining pharmaceuticals discussed are options for treatment of IIM subcategories, excluding inclusion body myositis.…”

Section: Therapiesmentioning

confidence: 99%

“…AZA is often preferred in patients with ILD, starting at a dose of 50 mg/day with increasing to a goal of 1.5 mg/kg/day. However, contradictory results have been reported in studies comparing MTX versus AZA in regard to survival benefit [3,24]. …”

Section: Therapiesmentioning

confidence: 99%

“…IVIG is one of the only treatments indicated for use in inclusion body myositis, specifically in patients suffering from oropharyngeal dysfunction [24,28]. Multiple mechanisms of action have been reported for IVIG including disruption of the F c region causing interference with cytokine and complement activity [28].…”

Section: Therapiesmentioning

confidence: 99%

“…Multiple mechanisms of action have been reported for IVIG including disruption of the F c region causing interference with cytokine and complement activity [28]. IVIG therapy is generally performed by infusion at a dose of 2 gm/kg/month [24]. Dalakas et al [7] conducted small population studies demonstrating efficacy of IVIG in a double-blind, controlled trial of IVIG therapy in 15 patients with refractory dermatomyositis.…”

Section: Therapiesmentioning

confidence: 99%

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Idiopathic Inflammatory Myopathies: A Review of the Classification and Impact of Pathogenesis

Mandel

Malemud

Askari

2017

IJMS

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Idiopathic inflammatory myopathies (IIMs) are a group of autoimmune muscle diseases with significant morbidity and mortality. This review details and updates the pathogenesis and emerging importance of myositis-specific antibodies in the development of IIMs. An increase in the understanding of how these myositis-specific antibodies play a role in IIMs has led to the further categorization of IIMs from the traditional polymyositis versus dermatomyositis, to additional subcategories of IIMs such as necrotizing autoimmune myositis (NAM). The diagnosis of IIMs, including manual muscle testing, laboratory studies, and non-invasive imaging have become important in classifying IIM subtypes and for identifying disease severity. Treatment has evolved from an era where glucocorticoid therapy was the only option to a time now that includes traditional steroid-sparing agents along with immunoglobulin therapy and biologics, such as rituximab.

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CD19-Targeting CAR T Cells for Myositis and Interstitial Lung Disease Associated With Antisynthetase Syndrome

Pecher

Hensen

Klein

et al. 2023

JAMA

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ImportanceAutoimmune disorders can affect various organs and if refractory, can be life threatening. Recently, CD19-targeting–chimeric antigen receptor (CAR) T cells were efficacious as an immune suppressive agent in 6 patients with refractory systemic lupus erythematosus and in 1 patient with antisynthetase syndrome.ObjectiveTo test the safety and efficacy of CD19-targeting CAR T cells in a patient with severe antisynthetase syndrome, a complex autoimmune disorder with evidence for B- and T-cell involvement.Design, Setting, and ParticipantsThis case report describes a patient with antisynthetase syndrome with progressive myositis and interstitial lung disease refractory to available therapies (including rituximab and azathioprine), who was treated with CD19-targeting CAR T cells in June 2022 at University Hospital Tübingen in Tübingen, Germany, with the last follow-up in February 2023. Mycophenolate mofetil was added to the treatment to cotarget CD8+ T cells, hypothesized to contribute to disease activity.ExposurePrior to treatment with CD19-targeting CAR T cells, the patient received conditioning therapy with fludarabine (25 mg/m2 [5 days before until 3 days before]) and cyclophosphamide (1000 mg/m2 [3 days before]) followed by infusion of CAR T cells (1.23×106/kg [manufactured by transduction of autologous T cells with a CD19 lentiviral vector and amplification in the CliniMACS Prodigy system]) and mycophenolate mofetil (2 g/d) 35 days after CD19-targeting CAR T-cell infusion.Main Outcomes and MeasuresThe patient’s response to therapy was followed by magnetic resonance imaging of the thigh muscle, Physician Global Assessment, functional muscle and pulmonary tests, and peripheral blood quantification of anti-Jo-1 antibody levels, lymphocyte subsets, immunoglobulins, and serological muscle enzymes.ResultsRapid clinical improvement was observed after CD19-targeting CAR T-cell infusion. Eight months after treatment, the patient’s scores on the Physician Global Assessment and muscle and pulmonary function tests improved, and there were no detectable signs of myositis on magnetic resonance imaging. Serological muscle enzymes (alanine aminotransferase, aspartate aminotransferase, creatinine kinase, and lactate dehydrogenase), CD8+ T-cell subsets, and inflammatory cytokine secretion in the peripheral blood mononuclear cells (interferon gamma, interleukin 1 [IL-1], IL-6, and IL-13) were all normalized. Further, there was a reduction in anti-Jo-1 antibody levels and a partial recovery of IgA (to 67% of normal value), IgG (to 87%), and IgM (to 58%).Conclusions and RelevanceCD19-targeting CAR T cells directed against B cells and plasmablasts deeply reset B-cell immunity. Together with mycophenolate mofetil, CD19-targeting CAR T cells may break pathologic B-cell, as well as T-cell responses, inducing remission in refractory antisynthetase syndrome.

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Update on the pharmacological treatment of adult myositis

Abstract: Abstract. Oddis CV

Cited by 50 publications

References 73 publications

A Case of Refractory Dysphagia in the Context of Polymyositis Treated with Subcutaneous Immunoglobulin and Review of the Literature

A Case of Refractory Dysphagia in the Context of Polymyositis Treated with Subcutaneous Immunoglobulin and Review of the Literature

Idiopathic Inflammatory Myopathies: A Review of the Classification and Impact of Pathogenesis

CD19-Targeting CAR T Cells for Myositis and Interstitial Lung Disease Associated With Antisynthetase Syndrome

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