Biologics in refractory idiopathic inflammatory myositis (IIM): What experience in juvenile vs adult myositis tells us about the use of biologics in pediatric IIM

Patwardhan, Anjali; Spencer, Charles H.

doi:10.1080/14397595.2021.1881027

Cited by 2 publications

(2 citation statements)

References 114 publications

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“…Additionally, myositis can be associated with various rheumatologic diseases and is then named overlap myositis (OM) [ 7 , 8 ]. The currently used classification of myositis, based on pathophysiological and immunological insights, suggests that subtype-specific therapy may be the best treatment option [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

Morphological Characteristics of Idiopathic Inflammatory Myopathies in Juvenile Patients

Schänzer

Rager

Dahlhaus

et al. 2021

Cells

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Background: In juvenile idiopathic inflammatory myopathies (IIMs), morphological characteristic features of distinct subgroups are not well defined. New treatment strategies require a precise diagnosis of the subgroups in IIM, and, therefore, knowledge about the pathomorphology of juvenile IIMs is warranted. Methods: Muscle biopsies from 15 patients (median age 8 (range 3–17) years, 73% female) with IIM and seven controls were analyzed by standard methods, immunohistochemistry, and transmission electron microscopy (TEM). Detailed clinical and laboratory data were accessed retrospectively. Results: Proximal muscle weakness and skin symptoms were the main clinical symptoms. Dermatomyositis (DM) was diagnosed in 9/15, antisynthetase syndrome (ASyS) in 4/15, and overlap myositis (OM) in 2/15. Analysis of skeletal muscle tissues showed inflammatory cells and diffuse upregulation of MHC class I in all subtypes. Morphological key findings were COX-deficient fibers as a striking pathology in DM and perimysial alkaline phosphatase positivity in anti-Jo-1-ASyS. Vascular staining of the type 1 IFN-surrogate marker, MxA, correlated with endothelial tubuloreticular inclusions in both groups. None of these specific morphological findings were present in anti-PL7-ASyS or OM patients. Conclusions: Morphological characteristics discriminate IIM subtypes in juvenile patients, emphasizing differences in aetiopathogenesis and supporting the notion of individual and targeted therapeutic strategies.

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Section: Introductionmentioning

confidence: 99%

Morphological Characteristics of Idiopathic Inflammatory Myopathies in Juvenile Patients

Schänzer

Rager

Dahlhaus

et al. 2021

Cells

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“…Since the results of the 2013 Rituximab in Myositis (RIM) trial, which demonstrated effectiveness of rituximab as a treatment for refractory myositis despite not meeting the primary endpoint of the study, biologic DMARDs have been adopted widely, both in the United States and abroad [ 5 – 9 ]. A growing literature of case reports of patients with refractory JDM has suggested the effectiveness of multiple biologics, including abatacept, tocilizumab, and tumor necrosis factor (TNF) inhibitors in addition to rituximab [ 4 , 10 , 11 ]. However, prospective comparative studies of commonly used biologics in patients with JDM are lacking.…”

Section: Introductionmentioning

confidence: 99%

Treatment escalation patterns to start biologics in refractory moderate juvenile dermatomyositis among members of the Childhood Arthritis and Rheumatology Research Alliance

et al. 2023

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Background Despite new and better treatments for juvenile dermatomyositis (JDM), not all patients with moderate severity disease respond adequately to first-line therapy. Those with refractory disease remain at higher risk for disease and glucocorticoid-related complications. Biologic disease-modifying antirheumatic drugs (DMARDs) have become part of the arsenal of treatments for JDM. However, prospective comparative studies of commonly used biologics are lacking. Methods The Childhood Arthritis and Rheumatology Research Alliance (CARRA) JDM biologics workgroup met in 2019 and produced a survey assessing current treatment escalation practices for JDM, including preferences regarding use of biologic treatments. The cases and questions were developed using a consensus framework, requiring 80% agreement for consensus. The survey was completed online in 2020 by CARRA members interested in JDM. Survey results were analyzed among all respondents and according to years of experience. Chi-square or Fisher’s exact test was used to compare the distribution of responses to each survey question. Results One hundred twenty-one CARRA members responded to the survey (denominators vary for each question). Of the respondents, 88% were pediatric rheumatologists, 85% practiced in the United States, and 43% had over 10 years of experience. For a patient with moderately severe JDM refractory to methotrexate, glucocorticoids, and IVIG, approximately 80% of respondents indicated that they would initiate a biologic after failing 1–2 non-biologic DMARDs. Trials of methotrexate and mycophenolate were considered necessary by 96% and 60% of respondents, respectively, before initiating a biologic. By weighed average, rituximab was the preferred biologic over abatacept, tocilizumab, and infliximab. Over 50% of respondents would start a biologic by 4 months from diagnosis for patients with refractory moderately severe JDM. There were no notable differences in treatment practices between respondents by years of experience. Conclusion Most respondents favored starting a biologic earlier in disease course after trialing up to two conventional DMARDs, specifically including methotrexate. There was a clear preference for rituximab. However, there remains a dearth of prospective data comparing biologics in refractory JDM. These findings underscore the need for biologic consensus treatment plans (CTPs) for refractory JDM, which will ultimately facilitate comparative effectiveness studies and inform treatment practices.

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Biologics in refractory idiopathic inflammatory myositis (IIM): What experience in juvenile vs adult myositis tells us about the use of biologics in pediatric IIM

Cited by 2 publications

References 114 publications

Morphological Characteristics of Idiopathic Inflammatory Myopathies in Juvenile Patients

Morphological Characteristics of Idiopathic Inflammatory Myopathies in Juvenile Patients

Treatment escalation patterns to start biologics in refractory moderate juvenile dermatomyositis among members of the Childhood Arthritis and Rheumatology Research Alliance

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