2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects.

Objective: The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods: Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. Results: Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. Conclusion: Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.

2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis: An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative.

To develop response criteria for juvenile dermatomyositis (DM). We analysed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute per cent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91-98% for minimal improvement, 92-94% and 94-99% for moderate improvement, and 91-98% and 85-86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (p=0.009-0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (p<0.006). The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute per cent change in core set measures, with thresholds for minimal, moderate, and major improvement.

2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis: An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative.

OBJECTIVE: To develop response criteria for juvenile dermatomyositis (DM). METHODS: We analyzed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. RESULTS: Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute percent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91-98% for minimal improvement, 92-94% and 94-99% for moderate improvement, and 91-98% and 85-86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (P = 0.009-0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (P < 0.006). CONCLUSION: The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute percent change in core set measures, with thresholds for minimal, moderate, and major improvement.

Nephrotic syndrome due to immunoglobulin M mesangial glomerulonephritis preceding juvenile idiopathic arthritis.

The association between nephrotic syndrome and juvenile idiopathic arthritis have rarely been described in pediatric patients. We report a child with steroid-responsive nephrotic syndrome, with frequent relapses, who presented with a new relapse of nephrotic syndrome associated with arthritis and uveitis at 21 months in remission after treatment with chlorambucil. Juvenile idiopathic arthritis was diagnosed and kidney biopsy examination showed mesangial glomerulonephritis with immunoglobulin M deposits. To our knowledge, only 2 cases of nephrotic syndrome preceding juvenile idiopathic arthritis have been reported, one without histopathology assessment and the other with minimal change disease. Although mesangial glomerulonephritis with nephrotic syndrome and juvenile idiopathic arthritis could have been coincidental, the immune pathogenic mechanism accepted for both diseases suggests they could be related.

Discrimination between incomplete and atypical Kawasaki syndrome versus other febrile diseases in childhood: results from an international registry-based study.

OBJECTIVES: Kawasaki syndrome (KS) is an acute systemic vasculitis of unknown origin predominantly affecting young children. Early diagnosis is crucial to prevent cardiac complications. However, the differential diagnosis of patients with the incomplete or atypical form of the disease poses a heavy challenge for the paediatrician. Our aim was to evaluate the prevalence of incomplete and atypical cases among children with KS and to identify clinical and laboratory variables that may help differentiate incomplete and atypical KS from other febrile diseases at this age. METHODS: We established an international registry to recruit patients with KS, including those with incomplete and atypical forms. The control group included age-matched febrile children admitted to the hospital with a variety of diseases mimicking KS. The aim was to define clinical or laboratory clues to help in the discrimination of incomplete and atypical KS patients from others. RESULTS: Two hundred and twenty-eight patients with incomplete KS (78%) and atypical KS (22%) were compared to 71 children with other febrile diseases. Patients with incomplete and atypical KS presented a statistically significant higher frequency of mucosal changes, conjunctivitis, extremity abnormalities and perineal desquamation compared to the group of other febrile diseases. In addition, C-reactive protein and platelet counts were significantly higher in incomplete and atypical KS compared to the other group. CONCLUSIONS: This is the largest series of incomplete and atypical KS patients of non East-Asian ancestry: we suggest that in patients with the aforementioned clinical features and laboratory evidence of systemic inflammation in terms of increased C-reactive protein and platelet counts an echocardiogram should be performed and diagnosis of KS considered.

Epidemiología de la fiebre reumática y valvulopatía reumática en la Argentina y Latinoamérica / Epidemiology of rheumatic fever and rheumatic valvular disease in Argentina and Latin America

Se consideran la forma de presentación, evolución y pronóstico de 1.807 pacientes con fiebre reumática (FR) o valvulopatía reumática (VR) examinados en un período de 44 años (1938-1982) en Hospitales de Niños de la ciudad de Buenos Aires. En FR aguda ha disminuido la frecuencia y severidad de la carditis (70% al 46%). Asimismo, la evolución y pronóstico es más favorable: mayor porcentaje de pacientes sin valvulopatía residual (17% al 33%), menor número de fallecidos (14% al 4%) y de valvulopatías severas (39% al 26%). El pronóstico depende de la presencia y severidad de la carditis inicial. En el estudio colaborativo OPS-SIC sobre 691 pacientes con FR aguda, efectuado en siete países de Latinoamérica en los años 1975-1980, se demuestra que en la carditis leve (sin agrandamiento cardíaco) quedan con valvulopatía el 35% de los pacientes, en la moderada (con agrandamiento cardíaco) el 65% y en la severa (con insuficiencia cardíaca) el 95% de los pacientes. La VR residual al año de la carditis fue similar en los diferentes países de Latinomérica, con predominio de la insuficiencia mitral. En cambio, son diferentes a otros países en desarrollo de Africa o Asia como la India, especialmente en relación a la estenosis mitral juvenil (34% en Nueva Delhi y 5% en Buenos Aires) y la insuficiencia tricuspídea (21% en la India y 2,5% en Buenos Aires) (AU)

Estreptococos beta hemolíticos aislados en Buenos Aires y su relevancia en la epidemiología de las infecciones estreptocóccicas, fiebre reumática y glomerulonefritis aguda / Beta hemolytic streptococci isolated in Buenos Aires and its relevance for the epidemiology of streptococcal infections, rheumatic fever, and acute glomerulonephritis

Se aislaron 118 cepas de estreptococos beta hemolíticos (EBH) de hisopados de fauces de niños de Buenos Aires, que presentaban angina y/o faringitis, con el objeto de realizar su agrupamiento y tipificación. Las mismas fueron enviadas, debidamente acondicionadas, a la Universidad de Minnesota, EE.UU. Ciento dos cepas (86%) fueron estreptococos beta hemolíticos del grupo A (EBHA) y 16 (14%) fueron del grupo C y G. Los cuatro tipos de cepas EBHA más frecuentemente hallados fueron: T 12 Factor de opacidad 22 M 6 T 6 M 1 T 1 T 11 Factor de opacidad 78 Se comenta la relación entre EBHA y sitios específicos de infección y su asociación con secuelas no supurativas, así como la existencia de cepas "reumatogénicas" y "nefritogénicas" (AU)

Epidemiología de la fiebre reumática y valvulopatía reumática en la Argentina y Latinoamérica / Epidemiology of rheumatic fever and rheumatic valvular disease in Argentina and Latin America

Se consideran la forma de presentación, evolución y pronóstico de 1.807 pacientes con fiebre reumática (FR) o valvulopatía reumática (VR) examinados en un período de 44 años (1938-1982) en Hospitales de Niños de la ciudad de Buenos Aires. En FR aguda ha disminuido la frecuencia y severidad de la carditis (70% al 46%). Asimismo, la evolución y pronóstico es más favorable: mayor porcentaje de pacientes sin valvulopatía residual (17% al 33%), menor número de fallecidos (14% al 4%) y de valvulopatías severas (39% al 26%). El pronóstico depende de la presencia y severidad de la carditis inicial. En el estudio colaborativo OPS-SIC sobre 691 pacientes con FR aguda, efectuado en siete países de Latinoamérica en los años 1975-1980, se demuestra que en la carditis leve (sin agrandamiento cardíaco) quedan con valvulopatía el 35% de los pacientes, en la moderada (con agrandamiento cardíaco) el 65% y en la severa (con insuficiencia cardíaca) el 95% de los pacientes. La VR residual al año de la carditis fue similar en los diferentes países de Latinomérica, con predominio de la insuficiencia mitral. En cambio, son diferentes a otros países en desarrollo de Africa o Asia como la India, especialmente en relación a la estenosis mitral juvenil (34% en Nueva Delhi y 5% en Buenos Aires) y la insuficiencia tricuspídea (21% en la India y 2,5% en Buenos Aires)

Estreptococos beta hemolíticos aislados en Buenos Aires y su relevancia en la epidemiología de las infecciones estreptocóccicas, fiebre reumática y glomerulonefritis aguda / Beta hemolytic streptococci isolated in Buenos Aires and its relevance for the epidemiology of streptococcal infections, rheumatic fever, and acute glomerulonephritis

Se aislaron 118 cepas de estreptococos beta hemolíticos (EBH) de hisopados de fauces de niños de Buenos Aires, que presentaban angina y/o faringitis, con el objeto de realizar su agrupamiento y tipificación. Las mismas fueron enviadas, debidamente acondicionadas, a la Universidad de Minnesota, EE.UU. Ciento dos cepas (86%) fueron estreptococos beta hemolíticos del grupo A (EBHA) y 16 (14%) fueron del grupo C y G. Los cuatro tipos de cepas EBHA más frecuentemente hallados fueron: T 12 Factor de opacidad 22 M 6 T 6 M 1 T 1 T 11 Factor de opacidad 78 Se comenta la relación entre EBHA y sitios específicos de infección y su asociación con secuelas no supurativas, así como la existencia de cepas "reumatogénicas" y "nefritogénicas"

Taponamiento cardíaco en artritis reumatoidea juvenil / Cardiac tamponade in juvenile rheumatoid arthritis

El taponamiento cardíaco en artritis reumatoidea juvenil (ARJ) ha sido comunicado excepcionalmente; se ha observado acompañando a brotes sistémicos de la misma, a cuya sintomatología se agregó tos, disnea, respiración superficial y precordialgias como síntomas prodrómicos. En este trabajo se comunican dos pacientes con ARJ de comienzo sistémico que en un segundo brote de su enfermedad desarrollan pericarditis con taponamiento cardíaco. La pericardiocentesis resultó útil en el primer paciente pero el segundo requirió pericardiotomía; no se observaron diferencias evolutivas con el uso de esteroides intrapericárdicos y en el seguimiento a largo plazo ambos desarrollaron pericarditis crónica con derrame. Las características histológicas del pericardio en el segundo paciente fueron compatibles con pericarditis granulomatosa crónica (AU)

Taponamiento cardíaco en artritis reumatoidea juvenil / Cardiac tamponade in juvenile rheumatoid arthritis

El taponamiento cardíaco en artritis reumatoidea juvenil (ARJ) ha sido comunicado excepcionalmente; se ha observado acompañando a brotes sistémicos de la misma, a cuya sintomatología se agregó tos, disnea, respiración superficial y precordialgias como síntomas prodrómicos. En este trabajo se comunican dos pacientes con ARJ de comienzo sistémico que en un segundo brote de su enfermedad desarrollan pericarditis con taponamiento cardíaco. La pericardiocentesis resultó útil en el primer paciente pero el segundo requirió pericardiotomía; no se observaron diferencias evolutivas con el uso de esteroides intrapericárdicos y en el seguimiento a largo plazo ambos desarrollaron pericarditis crónica con derrame. Las características histológicas del pericardio en el segundo paciente fueron compatibles con pericarditis granulomatosa crónica