CLASSIFICATION AND TREATMENT OF THE JUVENILE IDIOPATHIC INFLAMMATORY MYOPATHIES
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CLASSIFICATION OF THE JUVENILE IDIOPATHIC INFLAMMATORY MYOPATHIES
Clinical and histopathologic features have been useful in defining different groups of myositis patients that share similar disease courses, responses to therapy, and prognosis. A clinicopathologic classification of myositis subsets has long been recognized in adults with IIMs. * Recent studies suggest that children with myositis share similar clinical characteristics, histopathologic features and disease courses as adults within these distinct subgroups,
TREATMENT APPROACHES FOR JUVENILE DERMATOMYOSITIS AND JUVENILE POLYMYOSITIS
The published experiences with treatment of juvenile IIMs, in large part focused on JDM, are few, and our ability to draw conclusions is limited. The rarity of these diseases has resulted in a lack of randomized, controlled studies; thus, conclusions regarding treatment consist of an accumulation of possibly biased, open-label observations. Treatment reports are usually drawn from the experience of a single referral center and are often retrospective. With the small number of patients, studies
AGENTS FOR SEVERE OR LIFE-THREATENING DISEASE
Successful experience with daily oral cyclophosphamide has been reported in four JDM patients whose disease was refractory to steroids and methotrexate; all improved with a daily regimen of 50 to 75 mg/m 2, with remission induced in three. Hemorrhagic cystitis and herpes zoster infections required discontinuation in two patients.209 Reports of pulse intravenous cyclophosphamide and chlorambucil have been limited to adult IIM patients. Results with pulse cyclophosphamide in treating adult
EXTRASKELETAL MUSCLE DISEASE: CUTANEOUS DISEASE, CALCINOSIS, AND OSTEOPOROSIS
A thorough review of the therapy of extramuscular disease, including cardiopulmonary disease, gastrointestinal involvement, and arthritis, has recently been published 3; most experience in these areas has been derived from adults. For this reason, only the treatment of cutaneous disease, new approaches in treating calcinosis, and treatment of osteoporosis will be addressed.
Cutaneous disease is often neglected in the treatment of JDM. Although the histopathology of skin lesions may resemble that
PERSONAL VIEWPOINT ON TREATMENT OF JUVENILE DERMATOMYOSITIS
It is clear from published data that there are many different approaches to treat JDM. Although certain general therapeutic approaches for JDM have been suggested (Table 7), therapy in each patient must be individualized, taking into account disease severity, prognostic risk factors, and risks for and history of adverse events from medications.
Currently available data suggest that morbidity from juvenile and adult IIM remains high, 47, 73, 314 resulting from delay of treatment of disease, 30,
CONCLUSION
Although JDM is the most common of the juvenile IIMs, a number of other distinct clinicopathologic entities, serologic subsets, and environmentally triggered myositis syndromes are being recognized in children. These subsets seem to parallel their counterparts in adults in defining relatively homogeneous groups of patients sharing similar clinical features, responses to therapy, and prognoses. Additional serologic and environmentally associated myositis subsets are likely to be recognized,
ACKNOWLEDGMENTS
We thank Elizabeth Adams, MD, and Karyl Barron, MD, for critical reading of the manuscript, Ira Targoff, MD, for his immunologic expertise, Paul Plotz, MD, and John Klippel, MD, of NIAMS for support of our research studies on the natural history and disease activity of juvenile myositis, and the patients and physicians who have participated in the Childhood Myositis Heterogeneity Study for helping to better define the classification of the juvenile idiopathic inflammatory myopathies.
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Skin disease is more recalcitrant than muscle disease: A long-term prospective study of 184 children with juvenile dermatomyositis
2021, Journal of the American Academy of DermatologyMedications received by patients with juvenile dermatomyositis
2018, Seminars in Arthritis and RheumatismCitation Excerpt :Since high doses of daily oral prednisone of longer duration reportedly have led to better functional outcomes and less frequent calcinosis [5], patients with JDM have been treated primarily with high doses of oral prednisone. Based on open-label and retrospective studies, methotrexate (MTX), intravenous pulse methylprednisolone (IVMP), intravenous immunoglobulin (IVIG), cyclosporine and cyclophosphamide were later introduced to decrease the exposure to daily oral prednisone, as well as the duration of active disease [6–10]. In recent years, additional therapeutic options for treatment-refractory patients that were often supported by open-label trials and retrospective case series have included mycophenolate mofetil (MMF), tacrolimus, anti-TNF therapy, and rituximab, among others [3,11–13].
Critical review of the role of intravenous immunoglobulins in idiopathic inflammatory myopathies
2017, Seminars in Arthritis and RheumatismCitation Excerpt :This study found that despite greater initial disease activity, subjects in the IVIg group maintained similar or even lower disease activity than controls, from 30 days to 4 years after diagnosis, particularly in the steroid-resistant subjects, suggesting a beneficial effect of early IVIg in controlling short- and long-term disease activity in severe or refractory JDM. Several other uncontrolled studies have reported improvement in steroid-resistant, steroid-dependent or with severe myopathic involvement in 63–86% of juvenile IIM subjects treated with monthly IVIg of varying duration (Table 5) [47–49]. However, relapse was reported in 14–67% of initially responsive subjects when IVIg was discontinued [47,48,50].
Pediatric Systemic Lupus Erythematosus, Juvenile Dermatomyositis, Scleroderma, and Vasculitis
2016, Kelley and Firestein's Textbook of Rheumatology: Volumes 1-2, Tenth EditionJuvenile dermatomyositis: A 20-year retrospective analysis of treatment and clinical outcomes
2015, Pediatrics and Neonatology
Address reprint requests to Lisa G. Rider, MD, Laboratory of Molecular and Developmental Immunology, Division of Monoclonal Antibodies, Center for Biologics Evaluation and Research, Food and Drug Administration, Building 29B, Room 2G11, HFM-561, 8800 Rockville Pike, Bethesda, MD 20892
This work was supported by the intramural programs of CBER, FDA and NIAMS, NIH. The opinions expressed herein are the authors' and do not necessarily represent the positions of the US Food and Drug Administration or the Center for Biologics Evaluation and Research.
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From the Laboratory of Molecular and Developmental Immunology, Division of Monoclonal Antibodies, Center for Biologics Evaluation and Research, Food and Drug Administration, Bethesda, Maryland