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IVIG has been widely used for dermatomyositis but with limited evidence-based support. In a 16-week randomized trial, the percentage of patients with improvement was greater with IVIG than with placebo.

This study explored the effect of graded nursing and feeding guidance combined with enteral nutrition support in juvenile dermatomyositis children with swallowing dysfunction. Sixty-four juvenile dermatomyositis children with swallowing dysfunction admitted in Children's Hospital of Hebei Province from January 2022 to January 2023 were recruited as the study participants. They were randomly divided into an experimental group and a control group. The control group accepted routine nursing. The experimental group received graded nursing care, feeding guidance, enteral nutrition support, and routine nursing. After intervention, relative to the control group, the experimental group had better grade of water swallow test, higher total effectiveness rate, higher quality of life scores, higher levels of nutritional indexes and immune function indexes, lower incidence of complications and higher nursing satisfaction. We conclude that graded nursing, feeding guidance, and enteral nutrition support can improve the swallowing function, promote the nutritional status and immune function, along with decrease the incidence of complications in juvenile dermatomyositis children with swallowing dysfunction. Cette étude a exploré l'effet des soins infirmiers progressifs et des conseils nutritionnels associés à une nutrition entérale chez les enfants atteints de dermatomyosite juvénile présentant des troubles de la déglutition. Soixante-quatre enfants atteints de dermatomyosite juvénile présentant des troubles de la déglutition, admis à l'hôpital pour enfants de la province du Hebei entre janvier 2022 et janvier 2023, ont été recrutés comme participants à l'étude. Ils ont été répartis aléatoirement en un groupe expérimental et un groupe témoin. Le groupe témoin a accepté les soins infirmiers de routine. Le groupe expérimental a bénéficié de soins infirmiers progressifs, de conseils nutritionnels, d'une nutrition entérale et de soins infirmiers de routine. Après l'intervention, par rapport au groupe témoin, le groupe expérimental a obtenu une meilleure note au test de déglutition à l'eau, un taux d'efficacité globale plus élevé, des scores de qualité de vie plus élevés, des indices nutritionnels et immunitaires plus élevés, une incidence plus faible de complications et une plus grande satisfaction vis-à-vis des soins infirmiers. Nous concluons que les soins infirmiers progressifs, les conseils nutritionnels et la nutrition entérale peuvent améliorer la déglutition, favoriser l'état nutritionnel et la fonction immunitaire, et réduire l'incidence de complications chez les enfants atteints de dermatomyosite juvénile présentant des troubles de la déglutition.

Objective To assess the safety and clinical efficacy of allogeneic mesenchymal stem cell transplantation (MSCT) in a small-scale pilot study with 10 patients with drug-resistant polymyositis (PM) or dermatomyositis (DM). Methods A single-arm trial involving 10 patients with DM/PM who were either refractory to standard treatment, or had severe systemic involvement. All patients consented and underwent allogeneic MSCT. Clinical and laboratory manifestations were compared before and after MSCT. Results Improvements were seen in serum creatine kinase (CK), CK-MB, patient global assessment by visual analogue scale and muscle strength by manual muscle test in all patients, as well as improvement in interstitial lung disease in selected patients. Improvement in chronic non-healing skin ulcers was noted in one patient. Clinical responses were also seen in patients undergoing a second MSCT for recurrence of disease. Conclusion MSCT appears safe and effective in drug-resistant patients with DM/PM. Larger-scale studies including a control group receiving standard treatment are needed to assess the long-term efficacy of allogeneic MSCT in refractory patients with DM/PM.

Dermatomyositis is a chronic autoimmune disease with distinctive cutaneous eruptions and muscle weakness, and the pathophysiology is characterised by type I interferon (IFN) dysregulation. This study aims to assess the efficacy, safety, and target engagement of dazukibart, a potent, selective, humanised IgG1 neutralising monoclonal antibody directed against IFNβ, in adults with moderate-to-severe dermatomyositis. This multicentre, double-blind, randomised, placebo-controlled, phase 2 trial was conducted at 25 university-based hospitals and outpatient sites in Germany, Hungary, Poland, Spain, and the USA. Adults aged 18–80 years with skin-predominant dermatomyositis were enrolled during stages 1, 2, and 2a, and had to have a Cutaneous Dermatomyositis Disease Area and Severity Index-Activity (CDASI-A) score of 14 or more and at least one unsuccessful systemic treatment with standard of care; whereas those with muscle-predominant dermatomyositis were enrolled in stage 3 and had to have active moderate muscle involvement. Patients were randomly assigned using an interactive response technology system to dazukibart 600 mg or placebo in stage 1; dazukibart 600 mg, dazukibart 150 mg, or placebo in stage 2; dazukibart 600 mg then placebo, dazukibart 150 mg then placebo, placebo then dazukibart 600 mg, or placebo then dazukibart 150 mg in stage 2a; and dazukibart 600 mg then placebo or placebo then dazukibart 600 mg in stage 3. For stage 2a and stage 3, treatments were switched at week 12. Patients, investigators, outcome assessors, and funders were masked to the treatment assignment. Dazukibart and placebo were administered intravenously on day 1 every 4 weeks, up to and including week 8 (stages 1 and 2, and stages 2a and 3 for patients starting dazukibart), or on week 12 every 4 weeks, up to and including week 20 (stages 2a and 3 for patients who started placebo and switched to dazukibart). The primary outcome for the skin-predominant cohorts was the change from baseline in CDASI-A score at week 12 assessed in the full analysis set (FAS; stage 1) and the pooled skin FAS (stages 1, 2, and 2a), and safety in the muscle-predominant cohort. This study is registered with ClinicalTrials.gov, NCT03181893. Between Jan 23, 2018, and Feb 23, 2022, 125 adults were assessed and 50 were excluded. 75 patients were randomly assigned and treated (15 to dazukibart 150 mg, 37 to dazukibart 600 mg, and 23 to placebo). Most patients were female (53 [93%] of 57 in the skin-predominant cohort vs 13 [72%] of 18 in the muscle-predominant cohort and four [7%] vs five [28%] were male). In the FAS in stage 1 at week 12, the mean change from baseline in CDASI-A for dazukibart 600 mg was –18·8 (90% CI –21·8 to –15·8; placebo-adjusted difference –14·8 [–20·3 to –9·4]; p<0·0001). In the pooled skin FAS at week 12, the mean change from baseline in CDASI-A for the dazukibart 600 mg group was –19·2 (–21·5 to –16·8; placebo-adjusted difference –16·3 [–20·4 to –12·1]; p<0·0001), whereas the dazukibart 150 mg group was –16·6 (–19·8 to –13·4; placebo-adjusted difference –13·7 [–18·3 to –9·0]; p<0·0001). Treatment-emergent adverse events occurred in 12 (80%) of 15 patients in the dazukibart 150 mg group versus 30 (81%) of 37 in the dazukibart 600 mg group versus 18 (78%) of 23 in the placebo group, with the most common being infections and infestations (two [13%] vs 12 [32%] vs seven [30%]). Four (11%) patients in the dazukibart 150 mg group and one (4%) in the placebo group reported serious adverse events. One patient in stage 3 received dazukibart 600 mg then placebo and died during follow-up due to haemophagocytic lymphohistiocytosis and macrophage activation syndrome. Dazukibart resulted in a pronounced reduction in disease activity and was generally well tolerated, supporting IFNβ inhibition as a highly promising therapeutic strategy in adults with dermatomyositis. Pfizer.

Idiopathic inflammatory myopathies (IIMs) are a diverse group of diseases characterized by proximal muscle weakness and inflammation in skeletal muscle. Phenotypically, the subtypes include dermatomyositis, polymyositis, inclusion body myositis, and amyopathic dermatomyositis. The most common IIM in children is juvenile dermatomyositis (JDM). In contrast to adult dermatomyositis (DM), children are likely to have frequent relapses, vasculopathy, and long-term metabolic and other complications like lipodystrophy, insulin resistance, and calcinosis. Significant advances in our understanding of pathogenesis, disease course, and treatment of JDM has changed the therapeutic landscape and improved outcomes in children. Myositis-specific autoantibodies and myositis-associated autoantibodies have unique clinical associations, disease course and help predict response to therapy. A multidisciplinary approach including exercise programs and psychosocial support is essential. The first line of treatment is a combination of corticosteroids and methotrexate (MTX). Other targeted immunosuppressive therapy is used in refractory cases. Early recognition and timely referral to a specialist center remain pivotal to improving the mortality and morbidity associated with this disease.

Lenabasum, a non-psychoactive cannabinoid type-2 receptor (CB2R) agonist, has shown promise in reducing cutaneous disease in Dermatomyositis (DM) patients. Lenabasum activates two distinct receptors: CB2R and the nuclear peroxisome proliferator-activated receptor-γ (PPARγ). Our goal was to investigate the dominant mechanism of action leading to pathogenic IFNβ reduction by lenabasum (through CB2R or PPARγ) across leukocytes. We utilized whole blood leukocytes from 14 DM patients and grouped patients as in vitro responders or non-responders. We stimulated leukocytes in vitro in the presence of CB2R and PPARγ inhibitors and lenabasum. Intracellular and extracellular marker expression was analyzed by flow cytometry. CD4 + T ( p  <  0.05 ), monocyte-derived dendritic cells ( p  =  0.06 ), and intermediate monocytes (iMs) ( p  <  0.05 ) activate a CB2R-mediated lenabasum pathway in responders. Responder B cells ( p  <  0.01 ), CD8 + T cells ( p  <  0.01 ), and non-classical monocytes ( p  =  0.06 ) activate a co-dependent CB2R/PPARγ-mediated lenabasum pathway. Lenabasum can independently activate CB2R or PPARγ in myeloid dendritic cells ( p  <  0.05 ). Responder plasmacytoid dendritic cells ( p  <  0.05 ) and classical monocytes ( p  <  0.01 ) activate a PPARγ-mediated lenabasum pathway. CB2R was increased in certain responder CB2R-mediated cell populations compared to non-responders. Lenabasum elevated cyclooxygenase-2 or 15-lipoxygenase-1 levels in all responder CB2R-mediated cell populations except iMs. Baseline cell-to-cell CB2R/PPARγ testing could be useful to select ideal lenabasum candidates. Trial Registration: Registered at ClinicalTrials.gov (Identifier: NCT03813160) on 2019–01-23. Sponsored by Corbus Pharmaceuticals Inc.

ObjectivesTo study the effects of abatacept on disease activity and on muscle biopsy features of adult patients with dermatomyositis (DM) or polymyositis (PM).MethodsTwenty patients with DM (n=9) or PM (n=11) with refractory disease were enrolled in a randomised treatment delayed-start trial to receive either immediate active treatment with intravenous abatacept or a 3 month delayed-start. The primary endpoint was number of responders, defined by the International Myositis Assessment and Clinical Studies Group definition of improvement (DOI), after 6 months of treatment. Secondary endpoints included number of responders in the early treatment arm compared with the delayed treatment arm at 3 months. Repeated muscle biopsies were investigated for cellular markers and cytokines.Results8/19 patients included in the analyses achieved the DOI at 6 months. At 3 months of study, five (50%) patients were responders after active treatment but only one (11%) patient in the delayed treatment arm. Eight adverse events (AEs) were regarded as related to the drug, four mild and four moderate, and three serious AEs, none related to the drug. There was a significant increase in regulatory T cells (Tregs), whereas other markers were unchanged in repeated muscle biopsies.ConclusionsIn this pilot study, treatment of patients with DM and PM with abatacept resulted in lower disease activity in nearly half of the patients. In patients with repeat muscle biopsies, an increased frequency of Foxp3+ Tregs suggests a positive effect of treatment in muscle tissue.

ObjectiveStudies of dermatomyositis (DM) are frequently limited to single-centre cohorts. We used two large nationally representative US cohorts to conduct a descriptive epidemiological study of the characteristics, treatments and outcomes of patients with incident DM.MethodsThis retrospective study identified two DM inception cohorts using (1) commercial claims and (2) electronic health record (EHR) data from the Excellence Network in Rheumatology to Innovate Care and High-impact research (ENRICH), a community rheumatology practice-based research network. Patient characteristics, treatments and healthcare utilisation were assessed using the 18 months before and 12 months after diagnosis in claims and the 12 months before and after diagnosis in EHR data.ResultsWe identified 2475 patients (claims) and 1196 patients (EHR) with incident DM. Among 998 patients in the EHR cohort with available laboratory data, 472 had available myositis panel results, with 165 (35.0%) having a positive myositis-specific antibody. Glucocorticoid use was common, 68.7% and 73.8% in the two cohorts, respectively, with initial doses most often >20 mg/day; among glucocorticoid users, mean cumulative dose was 1407 mg in the claims cohort. Hydroxychloroquine, methotrexate and mycophenolate were the most commonly used immunomodulatory therapies. During follow-up in the claims data cohort, incidence per 1000 person-years was 92.2, 15.3, 6.4, 2.9 and 2.1 for all-cause hospitalisation, malignancy, interstitial lung disease, gastrostomy tube placement and myocarditis, respectively.ConclusionAdministrative claims and EHR data can be leveraged to assess treatment patterns and longitudinal outcomes/disease manifestations in incident dermatomyositis cohorts. This study highlights a high burden of glucocorticoid exposure, significant heterogeneity in treatment and high healthcare utilisation in this population.

To the Editor: The placebo-controlled, phase 3 trial of intravenous immune globulin (IVIG) in patients with dermatomyositis that was conducted by Aggarwal et al. (Oct. 6 issue) 1 represents an important advance. It is unfortunate that the trial excluded patients with skin-predominant disease, including those with muscle disease that had resolved. This group comprised more than 20% of patients in a population-based study. 2 In the trial, it is clear that IVIG treatment was effective for skin disease, given the clinically significant improvement observed in the score on the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), a validated tool that captures . . .