ObjectiveTo describe a German cohort of patients with juvenile dermatomyositis (JDM) and to evaluate clinical manifestations, disease course and prognosis in JDM patients with a certain myositis-specific autoantibody.Methods Cross-sectional data on patients with JDM documented in the National Paediatric Rheumatologic Database in Germany between 2014 and 2016 were analysed. In a subgroup of the cohort, MSAs were determined with a commercial multiplex array, and a retrospective chart review was conducted to specify the clinical phenotype and patient outcome. Results Thetotal cohort consisted of 196 patients with JDM (mean age 12.2±4.0 years, mean disease duration 5.1±3.8 years, 70% female). Apart from typical skin changes and muscle weakness, 41% of patients also had arthritis and/or contractures, 27% had calcinosis and approximately 10% had interstitial lung disease. Immunoblot testing was performed on the sera of 91 (46%) patients, detecting MSAs in 44% of patients. Patient groups with specific MSAs differed in clinical characteristics such as calcinosis, dysphagia, and lung and joint involvement. The extent of muscle weakness evaluated by the Childhood Myositis Assessment Scale was significantly associated with an increased level of creatine kinase. Patients with anti-MDA5 were particularly affected by polyarthritis of the small joints. After 5 years, 51patients of the MSA cohort (56.0%) achieved an inactive disease state, 12/51 (23.5%) were off therapy. ConclusionPatients with JDM in Germany show a broad spectrum of clinical manifestations that can be grouped into homogeneous groups using MSA, which also helps to predict the course and prognosis of the disease.
Purpose – the article shows the current state of the literature regarding business transfers presents the findings of a survey based on it, that can support the transfer process in the future. Research methodology – the article is based on three research methods: (a) desk research, (b) surveys and (c) economic analysis. The three methods are highly interdependent. Findings – the authors conclude that according to literature a lack of appropriate support and services for businesses going through a transfer exist. The survey highlights the challenges and demands that differ quite strongly between old and new member states. Research limitations – the number from the 283 participants who stated that they had already been involved in business transfers is above average and cannot be extrapolated to all companies. Practical implications – the practical implications of the study are highly relevant for decision-makers who want to create a structured approach to a sustainable support for business transfers in their countries. Originality/Value – the article is of high practical relevance. With regards to the evaluation of existing literature, there is a lack of current overviews like this one. The results of the current survey are of particularly importance for the practice of business transfers and their intensive support.
BackgroundNext to weakness of the proximal muscles and typical skin lesions as leading symptoms in juvenile dermatomyositis (JDM) there can be involvement of other organ systems and tissues. Myositis-specific antibodies (MSA) may help to distinguish clinically distinct phenotypesObjectives1) to analyse the clinical presentation of JDM and how diagnosis was verified and 2) to determine the spectrum of MSA and the associated clinical phenotypes in a German cohort of JDM patients.MethodsWe analyzed data of the national pediatric rheumatology database (NPRD), where children and adolescents with chronic rheumatic diseases are documented yearly by means of disease-specific questionnaires. Cross-sectional data of patients with JDM documented between 2014 and 2016 were analyzed. MSA were determined by a commercial multiplex array. To further specify the phenotype and patient’s outcome, an additional retrospective chart review was conducted.ResultsWe identified 186 patients with a diagnosis of JDM (69% female). Mean age at disease onset was 6.8 ± 3.5 years, mean time between first symptoms and first contact to a pediatric rheumatologist was 6.1 ± 8.9 months, mean age at documentation was 11.5 ± 4.4 years. The following diagnostic procedures were pathologic/positive for JDM: Histology 44/147 (30%), electromyography 28/144 (19%), magnetic resonance imaging 103/148 (70%), Creatine kinase (CK) elevation at diagnosis 126/148 patients (85%). At last consultation (mean disease duration 5.0 ± 3.9 years), the mean physician’s global assessment of disease activity (PGA, NRS 0 – 10) was 1.6 ± SD 2.3, the mean manual muscle test (MMT, range 0 – 80, best 80) was 69.7 ± SD 19, the mean disease activity score (DAS, range 0 – 20, best 0) was 4.4 ± D 4.7, the mean CHAQ was 0.5 ± SD 0.8. MSA testing was performed on 88 patients (73% female); 42% tested positive: anti-NXP2 16%, anti-TIF1γ 14%, anti-MDA5 6%, anti-Mi2 3%, anti-synthetase-antibodies 3%. The most common clinical feature was dysphagia in patients with anti-NXP2, calcinosis in patients with anti-TIF1γ, lung involvement in patients with anti-synthetase antibodies, and pronounced muscle weakness in patients with anti-Mi2. Patients with anti-MDA5 were characterized by frequent lung involvement, mucosal ulcers, fever and polyarticular arthritis of small joints. Muscle weakness tested by Childhood Myositis Assessment Score (CMAS) was significantly associated with an increased CK level. At last consultation, 32% and 14% received oral glucocorticoids (GC) < 0.2 mg/kg or ≥ 0.2 mg/kg body weight, respectively, 52% were treated with methotrexate (MTX) and 27% with intravenous immunoglobulins (last 12 months). PGA was ≤ 1 in 65% of patients at last consultation, 22% of those were off therapy.ConclusionDemographic and clinical parameters of patients with JDM in Germany are comparable to JDM-cohorts in other countries. MRI has gained diagnostic importance and is used more than twice as often as biopsy. MSA could be found in almost half of the patients. Clinical phenotypes were associated with ...
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