Favorable outcomes with reduced steroid use in juvenile dermatomyositis.

BACKGROUND: High-intensity glucocorticoid regimens are commonly used to induce and maintain remission in Juvenile Dermatomyositis but are associated with several adverse side-effects. Evidence-based treatment guidelines from North American and European pediatric rheumatology research societies both advocate induction with intravenous pulse steroids followed by high dose oral steroids (2 mg/kg/day), which are then tapered. This study reports the time to disease control with reduced glucocorticoid dosing. METHODS: We retrospectively reviewed the records at a single tertiary-care children's hospital of patients diagnosed with Juvenile Dermatomyositis between 2000 and 2014 who had a minimum of 2 years of follow-up. The primary outcome measure was time to control of muscle and skin disease. Additional outcome measures included glucocorticoid dosing, effect of treatment on height, frequency of calcinosis, and complications from treatment. RESULTS: Of the 69 patients followed during the study period, 31 fulfilled inclusion criteria. Median length of follow-up was 4.58 years, (IQR 3-7.5). Myositis control was achieved in a median of 7.1 months (IQR 0.9-63.4). Cutaneous disease control was achieved in a median of 16.7 months (IQR 4.3-89.5). The median starting dose of glucocorticoids was 0.85 mg/kg/day, (IQR 0.5-1.74). The median duration of steroid treatment was 9.1 months, (IQR 4.7-17.4), while the median duration of any pharmacotherapy was 29.2 months (IQR 10.4 to 121.3). Sustained disease control off medications was achieved in 21/31 (68%) patients by the end of review. Persistent calcinosis was identified in only one patient (3%). CONCLUSION: Current accepted treatment paradigms for Juvenile Dermatomyositis include oral glucocorticoids beginning at 2 mg/kg/day and reduced over a prolonged time period. However, our results suggest that treatment using reduced doses and duration with early use of steroid-sparing agents is comparably effective in achieving favorable outcomes in Juvenile Dermatomyositis.

Assessment of traditional and non-traditional risk factors for premature atherosclerosis in children with juvenile dermatomyositis and pediatric controls.

BACKGROUND: Children with juvenile dermatomyositis (JDM), the most common inflammatory myopathy of childhood, may be at increased risk of premature atherosclerosis given a host of traditional and non-traditional risk factors. The primary aim of this study was to determine the underlying frequency of premature atherosclerosis in children with JDM compared to pediatric controls using flow-mediated dilation as a measure of endothelial function. METHODS: Children and adolescents with and without JDM were evaluated for traditional atherosclerotic risk factors and assessment of endothelial function, using Endothelial Pulse Amplitude Testing (Endo-PAT). RESULTS: In this study, 75% of pediatric controls were of Black or Hispanic descent (compared to 55% in the JDM group) and 70% were found to live in a household with a medium income less than $50,000/year (compared to 45% in the JDM group). Among traditional atherogenic risk factors, lipoprotein A appeared to be different between controls and JDM patients (66 nmol/L and 16.5 nmol/L, respectively). Using a reactive hyperemia index (RHI) < 1.67 as evidence of endothelial dysfunction, 75% of controls were defined as having endothelial dysfunction compared to 50% in JDM group. When controlled for lipoprotein A as an atherogenic confounder, JDM patients were found to have a 41% increase in RHI, thus indicating less endothelial dysfunction compared to controls. CONCLUSIONS: In this study, we have shown that atherogenic risk factors are present in the pediatric population and may be associated with endothelial dysfunction, even at very young ages. Despite increasing concerns that children with rheumatologic disorders may be at increased risk of developing premature atherosclerosis, traditional and sociodemographic features may play a greater role in the ultimate development of cardiovascular disease.

Predictive factors for long-term outcome in polymyositis/dermatomyositis-associated interstitial lung diseases.

BACKGROUND: Interstitial lung disease (ILD) is strongly associated with polymyositis (PM), dermatomyositis (DM), and clinically amyopathic dermatomyositis (CADM). It is also related to mortality. Previous studies have highlighted that the acute form of PM/DM/CADM-associated ILD (PM/DM/CADM-ILD) has a poor short-term prognosis. However, little is known about the long-term clinical features of patients with PM/DM/CADM-ILD. The aim of the present study is to clarify the clinical characteristics and the predictive factors for long-term outcomes in patients with PM/DM/CADM-ILD. METHODS: Thirty-four patients with PM/DM/CADM-ILD who were followed up for more than 12 months were analyzed retrospectively. The patients were classified as "stable" or "deterioration" according to respiratory symptoms, serial changes in forced vital capacity (FVC) or arterial oxygen pressure, and radiologic findings during the follow-up period. RESULTS: Twenty-six patients (76%) were in the stable group and eight patients (24%) were in the deterioration group. Home oxygen therapy was performed in six cases in the deterioration group because of chronic respiratory failure due to progression of ILD. The deterioration group, in comparison to the stable group, had a significantly lower %FVC and a higher positive rate for the anti-PL-7 antibody. Multivariate logistic regression analysis revealed that a positive anti-PL-7 antibody test and a lower %FVC were independently associated with deterioration during long-term follow-up. CONCLUSIONS: Patients with PM/DM/CADM-ILD are at risk for chronic respiratory failure due to the deterioration of ILD during long-term follow-up. The presence of anti-PL-7 antibody and a lower %FVC at initial diagnosis may predict long-term deterioration in patients with PM/DM/CADM-ILD.

Protecting Bone Health in Pediatric Rheumatic Diseases: Pharmacological Considerations.

Bone health in children with rheumatic conditions may be compromised due to several factors related to the inflammatory disease state, delayed puberty, altered life style, including decreased physical activities, sun avoidance, suboptimal calcium and vitamin D intake, and medical treatments, mainly glucocorticoids and possibly some disease-modifying anti-rheumatic drugs. Low bone density or even fragility fractures could be asymptomatic; therefore, children with diseases of high inflammatory load, such as systemic onset juvenile idiopathic arthritis, juvenile dermatomyositis, systemic lupus erythematosus, and those requiring chronic glucocorticoids may benefit from routine screening of bone health. Most commonly used assessment tools are laboratory testing including serum 25-OH-vitamin D measurement and bone mineral density measurement by a variety of methods, dual-energy X-ray absorptiometry as the most widely used. Early disease control, use of steroid-sparing medications such as disease-modifying anti-rheumatic drugs and biologics, supplemental vitamin D and calcium, and promotion of weight-bearing physical activities can help optimize bone health. Additional treatment options for osteoporosis such as bisphosphonates are still controversial in children with chronic rheumatic diseases, especially those with decreased bone density without fragility fractures. This article reviews common risk factors leading to compromised bone health in children with chronic rheumatic diseases and discusses the general approach to prevention and treatment of bone fragility.

[New therapeutic approaches for polymyositis and dermatomyositis]. / A polymyositis és dermatomyositis modern kezelési lehetoségei.

Inflammatory myopathies are chronic, immune-mediated diseases characterized with progressive proximal muscle weakness. They encompass a variety of syndromes with protean manifestations. The aims of therapy are to increase muscle strength, prevent the development of contractures, and to manage the systemic manifestations of the disease. This is a complex treatment which requires routine and wide knowledge. The most important task is to recognize the disease and guide the patient to immunologic center. Although the first line of therapy continues to include corticosteroids, there are a multitude of agents available for treating patients with myositis. There are several different immunosuppressive agents which may be applied alone or in combination with each other, as well as an increasing number of novel and exciting biologic agents targeting molecules participating in the pathogenesis of inflammatory myopathy. Physiotherapy and rehabilitation in the remission period may significantly improve the functional outcome of patients with these disorders.

Dermatomyositis.

Dermatomyositis is one of the idiopathic inflammatory myopathies with characteristic cutaneous manifestations including the heliotrope rash, Gottron's papules, cuticular changes including periungual telangiectasia, a photodistributed erythema or poikiloderma, and a scaly alopecia. Dermatomyositis has been linked to cancer, particularly ovarian cancer. Cancer-associated disease is more commonly found in older patients, and when present, is associated with a poor prognosis. A childhood form of the disease exists and is frequently complicated by the development of calcinosis. Dermatomyositis is a systemic disorder and whereas the skin and muscles are the most commonly affected organs, patients may have arthralgias, arthritis, oesophageal disease, or cardiopulmonary dysfunction. Recently described serological abnormalities, known as myositis-specific antibodies, add credence to the notion that this disorder is distinct from all other collagen-vascular diseases, and may lead to important discoveries about the pathogenesis of the inflammatory myopathies, which are not currently of practical use in the clinic or office. Management of the patient with myositis usually includes systemic corticosteroids with or without an immunosuppressive agent. Cutaneous disease is more difficult to manage, but antimalarials, methotrexate, and intravenous immunoglobulin are effective in small, often open-label, studies.

Dermatomiositis juvenil: perfil clínico en niños mexicanos / Juvenile dermatomyositis. Clinical profile in Mexican children

Objetivo. Conocer el perfil clínico de pacientes pediátricos con dermatomiositis juvenil (DMJ), en función del tiempo de evolución de la enfermedad al momento del diagnóstico. Diseño. Retrospectivo, longitudinal, descriptivo y observacional. Contexto. Servicio de Inmunología, Instituto Nacional de Pediatría, Ciudad de México. Hospital pediátrico de tercer nivel. Pacientes. Todos los expedientes de pacientes entre 0 y 17 años de edad que fueron diagnosticado con DMJ, que cumplieron con dos o más criterios diagnósticos de Bohan y Peter. Se dividió a la población en dos grupos según tiempo de evolución al momento del diagnóstico: en el grupo 1 (G1) los pacientes con 6 meses o menos y en el grupo 2 (G2), aquellos que tenían más de 6 meses. Material y Métodos. Se investigó sexo, edad, antecedentes de importancia, signos y síntomas, enzimas musculares, estudios inmunológicos, serología para hepatitis A, B, y C y Toxoplasma gondii, electromiografía (EMG), biopsia muscular, serie esofagogástrica (SEG), radiografía de tórax, pruebas de función respiratoria (PFR), electrocardiograma (ECG), electroencefalograma (EEG), valoración oftálmica y renal. Resultados. Se encontraron 102 pacientes, 57 en el G1 y 45 en el G2, la edad promedio fue de 9 años 2 meses con desviación estándar (DE) de 3 años 7 meses. Predominó el sexo femenino en relación 2.3:1. La evoluación del padecimiento tuvo un promedio de 9 meses 10 días con DE de 10 meses 15 días. Los síntomas y signos más frecuentes en ambos grupos fueron el exantema en heliotropo y la debilidad muscular. En el G2 fueron más fecuentes la artritis (p=0.04), hipertricosis (p=0.05) y calcinosis (p=0.03). La ALT (alanin transferasa) fue mayor (p=0.05) y la PCR (proteína C reactiva) positiva con mayor frecuencia (p=0.03) en el G1. Los anticuerpos aCL (anticardiolipina) fueron positivos en cuatro pacientes del G1. Los anticuerpos antitoxoplasma fueron positivos en cuatro pacientes del G1 y uno del G2. No se observaron diferencias significativas entre los grupos al comparar los resultados de EMG, biopsia muscular, SEG, Rx de tórax, PFR, ECG, EEG y valoraciones oftálmica y renal. Discusión. El hecho de que no se detectaran diferencias significativas entre los grupos en la mayor parte de los parámetros estudiados, sugiere que el tiempo de evolución no afecta la expresión clínica de la enfermedad. La artritis, calcinosis e hipertricosis se relacionan con cronicidad. La PCR es un marcador de inflamación del G1 y puede estar en relación al grado de inflamación muscular. Es posible que los anticuerpos aCL y antitoxoplasma fueran positivos con mayor frecuencia en el G1, en relación con el grado de vasculitis; sin embargo, se necesitará evaluar prospectivamente un mayor número de casos para hacer conclusiones válidas

Dermatomyositis: correlative MR imaging and P-31 MR spectroscopy for quantitative characterization of inflammatory disease.

Magnetic resonance (MR) imaging and phosphorus-31 MR spectroscopy were used to examine four patients with dermatomyositis and five control subjects. T2-weighted images of the thigh muscles of patients showed increased signal intensity, with focal and inhomogeneous involvement predominantly in the vastus lateralis and secondarily in the vastus intermedius and vastus medialis. T1 and T2 values of the vastus lateralis in patients were significantly higher than those of the control subjects. T1 values of the rectus femoris and biceps femoris with more generalized inflammation were moderately elevated but still significantly higher than those of the control subjects. P-31 MR spectra of the quadriceps muscles were obtained during rest, during exercise at two graded levels, and in recovery. Concentrations of adenosine triphosphate and phosphocreatine (PCr) in the diseased muscles were 30% below normal values, and the inorganic phosphate/PCr ratios were increased in the patients' muscles at rest and throughout exercise. The T1 and T2 values as well as the P-31 metabolite data correlated with symptoms and clinical assessment.