Outcome assessment in the idiopathic inflammatory myopathies

Author(s):  
Lisa G. Rider ◽  
Frederick W. Miller

Due to their rarity, heterogeneity, and multispecialty nature, the myositis syndromes have limited data-driven consensus on appropriate outcome measures. Recently, two international, multispecialty consortia developed new tools and consensus on core set measures of myositis disease activity and damage, as well as response criteria that are now recommended for use as clinical trial endpoints but will also be useful in clinical practice. Magnetic resonance imaging, muscle ultrasound, selected laboratory tests, and immunological biomarkers—including cytokines, chemokines, lymphocyte flow cytometry, and endothelial activation markers—can all be helpful adjuncts to serum muscle enzyme levels in assessing disease activity and damage, but these have not yet been fully validated. Definitions of clinically inactive disease, complete clinical response, and remission have also been proposed but require further validation. These advances should enhance the development of therapies by standardizing our ability to demonstrate their efficacy in treating the idiopathic inflammatory myopathies.

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 504.1-504
Author(s):  
V. Kachkovska ◽  
A. Kovchun ◽  
E. Prystupa ◽  
L. Prystupa

Background:Nailfold capillaroscopy (NFC) is a useful, noninvasive, widely available diagnostic tool in rheumatology practice. We commonly use it to describe patterns of abnormalities in systemic sclerosis however we have enough data which proves NFC usefulness for monitoring idiopathic inflammatory myopathies (IIM) considering it as diagnostic tool, monitoring of disease activity and treatment efficiency. Despite evident clinical relevance of NFC in IIM patients we still do not have clear capillaroscopic images for IIM definition.Objectives:That’s why, the goal of our research was aimed to analyze capillaroscopic peculiarities among IIM patients and find possible associations with clinical and activity data.Methods:69 patients with IIM were examined and 47 IIM patients with capillaroscopic alterations were included in the study, 26 patients with dermatomyositis (DM) and 21 patients with polymyositis (PM) according to the Targoff Criteria (1997). NFC we performed usingDino-Lite CapillaryScope with 200 magnification. We assessed nailfold capillary density (NCD), presence of microhemorrhages, giant, dilated and ramified capillaries, scleroderma patterns (defined as an early, active or late pattern) and neovascular pattern (defined as an active and late scleroderma patterns). To asses disease activity we use Manual Muscle Testing 8 (MMT8), Health Assessment Questionnaire (HAQ), Myositis Disease Activity Assessment Tool (MDAAT), Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), physician’s VAS, patient’s VAS, serum muscle enzymes levels. We divided patients into 4 groups: 1stgroup – 17 DM patients with active disease (8 of them with newly onset disease), 2ndgroup included 9 DM patients with inactive disease, 3rdgroup – 11 PM patients with active disease (5 of them with newly onset disease) and 4thgroup included 10 PM patients with inactive disease.Results:The most common finding was low NCD, 70% of all patients had NCD lower than 6 per 1 mm. NCD for the 1stgroup was 4,4±1,12, 2ndgroup – 6,0±1,0, 3rdgroup – 5,8±1,1, 4thgroup – 9,2±2,1 (F=26,27, p<0,001). Hemorrhages were significantly more common among DM patients and active disease and were observed among 47,1% (p=0,036,χ2=8,574) with no significant difference with regard to the disease onset. Analyzing scleroderma patterns, among 1stgroup of patients – 17,6% had early, 47,1% – active, 35,3% – late pattern, in the 2ndgroup – 66,7% had early pattern, in the 3rdgroup – 27,3% patients with early and 18,2% with active pattern and in the 4thgroup – 50% of patients presented with early pattern (p=0,001,χ2=31,87). Neovascular pattern was found significantly more often among patients with active DM (p=0,001) with no regard to the disease onset. No statistically significant difference in giant and ramified capillaries distribution was found.Conclusion:According to our results, we can admit that the most common capillaroscopic finding was decreased NCD, which were significantly lower among patients with active DM, the same as microhemorrhages and neovascular scleroderma pattern. This data suggests that NCD, microhemorrhages and neovascular scleroderma pattern could be consider as biomarkers of DM activity but not PM, therefore more detailed research with larger numbers of patients are required.Disclosure of Interests:None declared


2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1582.1-1582
Author(s):  
B. H. Egeli ◽  
S. Ergun ◽  
Y. K. Gursoy ◽  
A. Cetin ◽  
S. Ugurlu

Background:Idiopathic inflammatory myopathies (IIM) are essentially treated aiming improvement of muscle function and extra muscular disease manifestations. The backbone of the treatment is corticosteroids enhancing the survival and patient quality of life. The lack of consensus on target-specific immunosuppressive treatment highlights the need for further studies evaluating alternative treatment methods. Rituximab is potentially a glucocorticoid-sparing agent which was reviewed in multiple studies with small sample sizes due to the rarity of the disease.Objectives:Higher statistical power can enhance the trustworthiness of alternative treatment methods yielding the main objective of this study.Methods:This retrospective study was conducted at a tertiary rheumatology center. Patients were diagnosed with an idiopathic inflammatory myopathy (dermatomyositis [DM], polymyositis [PM]) and were treated with rituximab in order to be included in this study. Clinical signs and symptoms of the presentation were noted during the first patient encounter as well as the follow-up. Parameters of disease activity including acute phase reactants, muscle enzyme levels, and disease-specific autoantibodies were analyzed.Results:The study includes 28 patients (20 DM, 8 PM). The age of diagnosis was 43.44 ± 15.77 years, follow-up duration was 60.7 ± 70.7 months. The presenting signs and symptoms of the patients are shown in Figure 1. The parameters of disease activity before and after treatment are summarized in Table 1. The mean corticosteroid dose decreased from 31.429 ±23.934 mg to 10.278 ±12.001 (p=0.001). Other treatment methods were methotrexate (n=18), Intravenous Immunoglobulin (IVIG) (n=7), and cyclophosphamide (n=2). There were not any deaths during the follow-up. Two patients were lost to follow-up.Table 1.The Parameters of Disease Activity Before and After TreatmentBefore TreatmentAfter TreatmentP ValueCPK, mean ± std (U/L)1426 ± 2049.92263.44 ± 265.630.004LDH, mean ± std (U/L)557.5 ± 365379.78 ± 192.10.03AST, mean ± std (U/L)62.52 ± 5930.16 ± 27.590.01ALT, mean ± std (U/L)56.48 ± 49.2127.64 ± 24.520.008ESR, mean ± std (mm/hour)26.38 ± 28.9820.39 ± 18.760.36CRP, mean ± std (mg/L)19.23 ± 46.1512.53 ± 26.670.5RF, mean ± std (U/mL)0 (0)N/AN/AANA, n (%)3 (10.71)N/AN/AFigure 1.The Presenting Signs and Symptoms of the PatientsConclusion:Rituximab is shown to be effective in treating myositis along with corticosteroids as well as a corticosteroid-sparing agent in retrospective studies and open-label clinical trials; however, lack of statistical power should be underlined. Long term decrease in steroid use and decrease in disease activity markers hints the effective use of rituximab as a glucocorticoid sparing agent as well as its safety with minimal side effects.Disclosure of Interests:None declared


Rheumatology ◽  
2020 ◽  
Vol 60 (1) ◽  
pp. 322-332
Author(s):  
Eri Watanabe ◽  
Kazunori Kato ◽  
Takahisa Gono ◽  
Emiko Chiba ◽  
Chihiro Terai ◽  
...  

Abstract Objectives Galectin-3 is involved in various biological activities, including immune activations and fibrosis. Idiopathic inflammatory myopathies (IIMs) are autoimmune diseases of unknown aetiology, often complicated by interstitial lung disease (ILD). The aim of this study was to evaluate the expression of galectin-3 in sera and tissues of patients with IIM and assess the associations of galectin-3 with patient characteristics and disease activity. Results Serum galectin-3 levels were significantly higher in IIM patients than in healthy controls. The serum galectin-3 levels positively correlated with serum levels of inflammatory markers and proinflammatory cytokines/chemokines and the Myositis Intention-to-Treat Activity Index. Stratification analysis revealed that patients with IIM-associated ILD (IIM-ILD) had significantly higher levels of serum galectin-3 than those without IIM-ILD. In addition, patients with acute/subacute interstitial pneumonia had significantly higher levels of serum galectin-3 than those with chronic interstitial pneumonia. Furthermore, serum galectin-3 levels in IIM-ILD patients correlated with the radiological assessments of parenchymal lung involvement and treatment response. Immunohistochemical analysis revealed that galectin-3 was expressed in inflammatory cells of myositis and dermatitis sections, whereas in ILD sections, galectin-3 was expressed in interstitial fibrosis and inflammatory cells. Conclusion Galectin-3 may be involved in the pathogenesis of inflammatory and fibrotic conditions in IIM and can serve as a potential biomarker of disease activity, especially in patients with IIM-ILD.


2017 ◽  
Vol 21 (1) ◽  
pp. 233-239 ◽  
Author(s):  
Joana Sousa Neves ◽  
Daniela Santos Faria ◽  
Marcos Cerqueira ◽  
Maria Carmo Afonso ◽  
Filipa Teixeira

Author(s):  
Josephina A. Vossen

Chapter 42 discusses polymyositis and dermatomyositis, which are idiopathic inflammatory myopathies characterized by muscle inflammation, proximal muscle weakness, and elevated muscle enzymes. Idiopathic inflammatory myopathies represent a heterogeneous group of muscle diseases. Diagnosis is based on clinical examination, muscle enzyme laboratory values, electromyography (EMG), and muscle biopsy. MRI is the most important imaging modality used in diagnosis and management. Radiographs and CT may detect calcinosis. Treatment is primarily by immune suppression.


2014 ◽  
Vol 73 (Suppl 2) ◽  
pp. 702.1-702
Author(s):  
A. Notarnicola ◽  
F. Iannone ◽  
M. Giannini ◽  
S. Paolotti ◽  
A. Scardapane ◽  
...  

Rheumatology ◽  
2020 ◽  
Author(s):  
Judith Wienke ◽  
Jorre S Mertens ◽  
Samuel Garcia ◽  
Johan Lim ◽  
Camiel A Wijngaarde ◽  
...  

Abstract Objectives Vasculopathy is an important hallmark of systemic chronic inflammatory connective tissue diseases (CICTD) and is associated with increased cardiovascular risk. We investigated disease-specific biomarker profiles associated with endothelial dysfunction, angiogenic homeostasis and (tissue) inflammation, and their relation to disease activity in rare CICTD. Methods A total of 38 serum proteins associated with endothelial (dys)function and inflammation were measured by multiplex-immunoassay in treatment-naive patients with localized scleroderma (LoS, 30), eosinophilic fasciitis (EF, 8) or (juvenile) dermatomyositis (34), 119 (follow-up) samples during treatment, and 65 controls. Data were analysed by unsupervised clustering, Spearman correlations, non-parametric t test and ANOVA. Results The systemic CICTD, EF and dermatomyositis, had distinct biomarker profiles, with ‘signature’ markers galectin-9 (dermatomyositis) and CCL4, CCL18, CXCL9, fetuin, fibronectin, galectin-1 and TSP-1 (EF). In LoS, CCL18, CXCL9 and CXCL10 were subtly increased. Furthermore, dermatomyositis and EF shared upregulation of markers related to interferon (CCL2, CXCL10), endothelial activation (VCAM-1), inhibition of angiogenesis (angiopoietin-2, sVEGFR-1) and inflammation/leucocyte chemo-attraction (CCL19, CXCL13, IL-18, YKL-40), as well as disturbance of the Angiopoietin-Tie receptor system and VEGF-VEGFR system. These profiles were related to disease activity, and largely normalized during treatment. However, a subgroup of CICTD patients showed continued elevation of CXCL10, CXCL13, galectin-9, IL-18, TNFR2, VCAM-1, and/or YKL-40 during clinically inactive disease, possibly indicating subclinical interferon-driven inflammation and/or endothelial dysfunction. Conclusion CICTD-specific biomarker profiles revealed an anti-angiogenic, interferon-driven environment during active disease, with incomplete normalization under treatment. This warrants further investigation into monitoring of vascular biomarkers during clinical follow-up, or targeted interventions to minimize cardiovascular risk in the long term.


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