We describe the clinical features of 28 patients with juvenile dermatomyositis (JDM) and 1 patient with adult-onset dermatomyositis (DM), all of whom developed lipodystrophy (LD) that could be categorized into 1 of 3 phenotypes, generalized, partial, or focal, based on the pattern of fat loss distribution. LD onset was often delayed, beginning a median of 4.6 years after diagnosis of DM. Calcinosis, muscle atrophy, joint contractures, and facial rash were DM disease features found to be associated with LD. Panniculitis was associated with focal lipoatrophy while the anti-p155 autoantibody, a newly described myositis-associated autoantibody, was more associated with generalized LD. Specific LD features such as acanthosis nigricans, hirsutism, fat redistribution, and steatosis/nonalcoholic steatohepatitis were frequent in patients with LD, in a gradient of frequency and severity among the 3 sub-phenotypes. Metabolic studies frequently revealed insulin resistance and hypertriglyceridemia in patients with generalized and partial LD. Regional fat loss from the thighs, with relative sparing of fat loss from the medial thighs, was more frequent in generalized than in partial LD and absent from DM patients without LD. Cytokine polymorphisms, the C3 nephritic factor, insulin receptor antibodies, and lamin mutations did not appear to play a pathogenic role in the development of LD in our patients. LD is an under-recognized sequela of JDM, and certain DM patients with a severe, prolonged clinical course and a high frequency of calcinosis appear to be at greater risk for the development of this complication. High-risk JDM patients should be screened for Address reprint requests to: Lisa G. Rider, MD, Environmental Autoimmunity Group, National Institute of Environmental Health Sciences, National Institutes of Health, CRC 4-2352, MSC 1301, 10, Center Drive, Bethesda, MD 20892-1301. Fax: 301-451-5588; email: E-mail: riderl@mail.nih.gov. * These authors contributed equally to this work. † Contributors to this study are listed in the appendix. NIH Public AccessAuthor Manuscript Medicine (Baltimore) NIH-PA Author ManuscriptNIH-PA Author Manuscript NIH-PA Author Manuscript metabolic abnormalities, which are common in generalized and partial LD and result in much of the LD-associated morbidity. Further study is warranted to investigate the pathogenesis of acquired LD in patients with DM.

Objective Treat to target (T2T) is a strategy of adjusting treatment until a target is reached. An international task force recommended T2T for juvenile idiopathic arthritis (JIA) treatment. Implementing T2T in a standard and reliable way in clinical practice requires agreement on critical elements of: (1) target setting, (2) T2T strategy, (3) identifying barriers to implementation, and (4) eligible patients. A consensus conference was held amongst Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) stakeholders to inform a statement of understanding regarding the PR-COIN approach to T2T. Methods PR-COIN stakeholders including health care providers (16) and parents (4), were invited to form a voting panel. Using the nominal group technique, two rounds of voting were held to address the above four areas to select the top 10 responses in rank order. Results Incorporation of patient goals ranked most important when setting a treatment target. Use of shared decision making (SDM), tracking measurable outcomes, and adjusting treatment to achieve goals were voted as top elements of T2T strategy. Workflow considerations, and provider buy-in were identified as key barriers to T2T implementation. Patients with JIA, with poor prognostic factors and at risk for high disease burden were leading candidates for a T2T approach. Conclusion This consensus conference identified the importance of incorporating patient goals as part of target setting, and influence of patient stakeholder involvement in drafting treatment recommendations. The network approach to T2T will be modified to address the above findings including solicitation of patient goals, optimizing SDM, and better workflow integration.

Objectives Anti-endothelial cell antibodies (AECA) are detected in multiple forms of vasculitis or vasculopathy, including juvenile dermatomyositis (JDM). High levels of tropomyosin alpha-4 chain (TPM4) gene expression in cutaneous lesions and TPM4 protein expression in some ECs have been proven. Furthermore, the presence of autoantibodies to tropomyosin proteins have been discovered in dermatomyositis. We therefore investigated whether anti-TPM4 autoantibodies are an AECA in JDM and are correlated with clinical features of JDM. Methods The expression of TPM4 protein in cultured normal human dermal microvascular EC was investigated by Western blotting. Plasma samples from 63 children with JDM, 50 children with polyarticular juvenile idiopathic arthritis (pJIA), and 40 healthy controls (HC) were tested for the presence of anti-TPM4 autoantibodies using an ELISA. Clinical features were compared between JDM patients with and without anti-TPM4 autoantibodies. Results Autoantibodies to TPM4 were detected in the plasma of 30% of JDM, 2% of pJIA (P < 0.0001), and 0% of HC children (P < 0.0001). In JDM, anti-TPM4 autoantibodies were associated with the presence of cutaneous ulcers (53%, P = 0.02), shawl sign rash (47%, P = 0.03), mucous membrane lesions (84%, P = 0.04) and subcutaneous edema (42%,　P<0.05). Anti-TPM4　autoantibodies significantly correlated with use of intravenous steroids and intravenous immunoglobulin therapy in JDM (both P = 0.01). The total number of medications received was higher in patients with anti-TPM4 autoantibodies (P = 0.02). Conclusion Anti-TPM4 autoantibodies are detected frequently in children with JDM and are novel myositis-associated autoantibodies. Their presence correlates with vasculopathic and other cutaneous manifestations of JDM that may be indicative of more refractory disease.