Development and preliminary validation of a paediatric-targeted MRI scoring system for the assessment of disease activity and damage in juvenile idiopathic arthritis.

OBJECTIVES: To develop and validate a paediatric-targeted MRI scoring system for the assessment of disease activity and damage in juvenile idiopathic arthritis (JIA). To compare the paediatric MRI score with the adult-designed. Outcome Measures in Rheumatology Clinical Trials-Rheumatoid Arthritis MRI Score (RAMRIS), whose suitability for assessing growing joints was tested. METHODS: In 66 patients with JIA the clinically more affected wrist was studied. Thirty-nine patients had a 1-year MRI follow-up. Two readers independently assigned the paediatric score and the RAMRIS to all studies. Validation procedures included analysis of reliability, construct validity and responsiveness to change. A reduced version of the bone erosion score was also developed and tested. RESULTS: The paediatric score showed an excellent reproducibility (interclass correlation coefficient >0.9). The interobserver agreement of RAMRIS was moderate for bone erosions and excellent for bone marrow oedema (BMO). The paediatric score and RAMRIS provided similar results for construct validity. The responsiveness to change of the paediatric score was moderate for synovitis and bone erosion, and poor for BMO and did not improve when RAMRIS was applied. The reduced version of the bone erosion was valuable for the assessment of joint damage, and provided time-saving advantages. CONCLUSION: The results demonstrate that the paediatric MRI score is a reliable and valid method for assessing disease activity and damage in JIA. Unexpectedly, the RAMRIS provides acceptable suitability for use in the paediatric age group. Further work, especially in a longitudinal setting, is required before defining the most suitable MRI scale for assessing growing joints.

Predictors of poor response to methotrexate in polyarticular-course juvenile idiopathic arthritis: analysis of the PRINTO methotrexate trial.

OBJECTIVES: To determine whether baseline demographic, clinical, articular and laboratory variables predict methotrexate (MTX) poor response in polyarticular-course juvenile idiopathic arthritis. METHODS: Patients newly treated for 6 months with MTX enrolled in the Paediatric Rheumatology International Trials Organization (PRINTO) MTX trial. Bivariate and logistic regression analyses were used to identify baseline predictors of poor response according to the American College of Rheumatology pediatric (ACR-ped) 30 and 70 criteria. RESULTS: In all, 405/563 (71.9%) of patients were women; median age at onset and disease duration were 4.3 and 1.4 years, respectively, with anti-nuclear antibody (ANA) detected in 259/537 (48.2%) patients. With multivariate logistic regression analysis, the most important determinants of ACR-ped 70 non-responders were: disease duration > 1.3 years (OR 1.93), ANA negativity (OR 1.77), Childhood Health Assessment Questionnaire (CHAQ) disability index > 1.125 (OR 1.65) and the presence of right and left wrist activity (OR 1.55). Predictors of ACR-ped 30 non-responders were: ANA negativity (OR 1.92), CHAQ disability index > 1.14 (OR 2.18) and a parent's evaluation of child's overall well-being < or = 4.69 (OR 2.2). CONCLUSION: The subgroup of patients with longer disease duration, ANA negativity, higher disability and presence of wrist activity were significantly associated with a poorer response to a 6-month MTX course.

EULAR/PRINTO/PRES criteria for Henoch-Schönlein purpura, childhood polyarteritis nodosa, childhood Wegener granulomatosis and childhood Takayasu arteritis: Ankara 2008. Part I: Overall methodology and clinical characterisation.

OBJECTIVES: To report methodology and overall clinical, laboratory and radiographic characteristics for Henoch-Schönlein purpura (HSP), childhood polyarteritis nodosa (c-PAN), c-Wegener granulomatosis (c-WG) and c-Takayasu arteritis (c-TA) classification criteria. METHODS: The preliminary Vienna 2005 consensus conference, which proposed preliminary criteria for paediatric vasculitides, was followed by a EULAR/PRINTO/PRES - supported validation project divided into three main steps. Step 1: retrospective/prospective web-data collection for HSP, c-PAN, c-WG and c-TA, with age at diagnosis

Agreement between multi-dimensional and renal-specific response criteria in patients with juvenile systemic lupus erythematosus and renal disease.

OBJECTIVES: To evaluate change over time and level of agreement of renal-specific and multi-dimensional measures in juvenile systemic lupus erythematosus (SLE) with renal disease. METHODS: An analysis was made of 205/557 children with baseline 24-hour proteinuria >or=0.5 g. Data were collected at baseline, 6-, 12- and 24-month intervals. Using the Systemic Lupus International Collaborating Clinics (SLICC) renal index (change in proteinuria and urine sediment) as gold standard, responsiveness and discriminative ability analyses were used to identify key renal and multi-dimensional disease activity and damage measures for the evaluation of response to therapy. We also evaluated the kappa agreement between SLICC renal index and PRINTO/ACR juvenile SLE criteria (change in proteinuria, physician and parents evaluations, disease activity, health related quality of life [HRQOL]). RESULTS: Children with renal disease compared to children without renal disease, had a lower female rate and higher disease activity/response rate (p-values <0.01) but similar damage levels. Large responsiveness (standardised response mean >or=0.8) and statistical significant discriminative ability with the SLICC renal index 4 levels of response (improved, partially improved, stable and worsened) were observed for renal specific measures (proteinuria, urine sediment, renal sub-scores, p<0.0001) and for multi-dimensional variables (disease activity level and physician evaluation p<0.001). Agreement between the SLICC renal index and PRINTO/ACR criteria was moderate (0.57; 95% confidence intervals: 0.44-0.71). CONCLUSIONS: We propose to incorporate multi-dimensional measures (physician and parents' evaluations, disease activity and HRQOL), in addition to renal specific measures, in future clinical trials in juvenile SLE with renal involvement.

[Clinical- epidemiological characteristics and predictors of coronary complications in children of Argentina with Kawasaki disease]. / Características clínico-epidemiológicas y predictores de complicaciones coronarias en niños de Argentina con enfermedad de Kawasaki.

BACKGROUND: Kawasaki disease (EK) is an acute systemic vasculitis with a risk of developing coronary aneurysms. AIM: To describe the clinical and epidemiological characteristics of children with EK in Argentina and to analyse the risk factors for the development of coronary's complications (CC). METHODS: Multicenter, retrospective, cross-sectional, observational and analytical study. It included patients younger than 18 years of age diagnosed with EK in hospitals in Argentina, between January the 1st, 2010 and December the 31th, 2013. RESULTS: N = 193 subjects. Age: medium: 29 months. Total incidence 5 cases / 10,000 hospital discharges. CC was observed in 15.5% of patients. Increased risk factors for CC: Elevated number of days with fever at the time of treatment placement (p = 0.0033); Increased of: heart frequency (p = 0.0021), erythrosedimentation (ESR) (p = 0.005), C-reactive protein (CRP) (p < 0.0001), leukocytes (p = 0.0006), neutrophils (p = 0.0021); Decreased of hematocrit (p = 0.0007) and hemoglobin (p < 0.0001).Association with CC: non-coronary cardiological alterations (OR = 10,818); PCR greater than 68 mg /L (OR = 11,596); leukocytes greater than 20,000 / mm3 (OR = 4.316); and ESR greater than 64 mm / 1 hour (OR = 4.267). CONCLUSION: The most frequent form of presentation was complete EK, the risk of CC was higher in males, younger than 5 years old, the risk factors (clinical and laboratory) were similar to those described in the literature.

[Treatment with sublingual desmopressin in two infants with hydranencephaly and central diabetes insipidus]. / Tratamiento con desmopresina sublingual en dos lactantes con hidranencefalia y diabetes insípida central.

Central diabetes insipidus is a rare disease in children caused by a deficiency of vasopressin. Its main clinical manifestations are polyuria and polydipsia. Brain malformations are one of the main causes. Desmopressin is the synthetic drug of choice for the treatment. One of the routes of administration is sublingual and its use in infants is very limited. We describe two infants with central diabetes insipidus and hydranencephaly who were successfully treated with sublingual desmopressin.

La diabetes insípida central es una patología infrecuente en pediatría ocasionada por un déficit de vasopresina. Sus manifestaciones clínicas principales son poliuria y polidipsia. Las malformaciones cerebrales son una de las principales causas. La desmopresina es la droga sintética de elección para el tratamiento. Una de las vías de administración es la sublingual y su uso en lactantes es muy limitado. Se describe a dos lactantes con hidranencefalia y diabetes insípida central que fueron tratados satisfactoriamente con desmopresina sublingual.

Características clínico-epidemiológicas y predictores de complicaciones coronarias en niños de Argentina con enfermedad de Kawasaki / Clinical- epidemiological characteristics and predictors of coronary complications in children of Argentina with Kawasaki disease

Resumen Introducción: La enfermedad de Kawasaki (EK) es una vasculitis sistémica aguda con riesgo de desarrollar aneurismas coronarios. Objetivos: Describir características clínico-epidemiológicas en niños con diagnóstico de EK en Argentina. Analizar factores de riesgo para el desarrollo de complicaciones coronarias (CC). Población y Métodos: Estudio multicéntrico, retrospectivo, transversal, observacional y analítico. Incluyó pacientes bajo 18 años de edad, con diagnóstico de EK en hospitales de Argentina, entre el 1 de enero de 2010 y el 31 de diciembre de 2013. Resultados: N = 193 sujetos. Edad: mediana: 29 meses. Tasa promedio total país 5 casos/10.000 egresos hospitalarios. Presentaron CC 15,5%. Mayor riesgo de CC: Mayor cantidad de días de fiebre al momento de colocación del tratamiento (p = 0,0033); Aumento de: frecuencia cardíaca (p = 0,0021), eritrosedimentación (VSG) (p = 0,005), proteína C reactiva (PCR) (p < 0,0001), leucocitosis (p = 0,0006), neutrofilia (p = 0,0021); Disminución de hematocrito (p = 0,0007) y hemoglobina (p < 0,0001). Asociación con CC: alteraciones cardiológicas no coronarias (ORv10.818); PCR mayor de 68 mg/L (OR = 11.596); leucocitos mayores a 20.000/mm3 (OR= 4.316); y VSG mayor de 64 mm/1° hora (OR = 4.267). Conclusión: La forma de presentación más frecuente fue EK completa, el riesgo de CC fue mayor en varones, menores de 5 años de edad, los factores de riesgo (clínicos y de laboratorio) fueron semejantes a los descritos en la bibliografía.

Background: Kawasaki disease (EK) is an acute systemic vasculitis with a risk of developing coronary aneurysms. Aim: To describe the clinical and epidemiological characteristics of children with EK in Argentina and to analyse the risk factors for the development of coronary's complications (CC). Methods: Multicenter, retrospective, cross-sectional, observational and analytical study. It included patients younger than 18 years of age diagnosed with EK in hospitals in Argentina, between January the 1st, 2010 and December the 31th, 2013. Results: N = 193 subjects. Age: medium: 29 months. Total incidence 5 cases / 10,000 hospital discharges. CC was observed in 15.5% of patients. Increased risk factors for CC: Elevated number of days with fever at the time of treatment placement (p = 0.0033); Increased of: heart frequency (p = 0.0021), erythrosedimentation (ESR) (p = 0.005), C-reactive protein (CRP) (p < 0.0001), leukocytes (p = 0.0006), neutrophils (p = 0.0021); Decreased of hematocrit (p = 0.0007) and hemoglobin (p < 0.0001).Association with CC: non-coronary cardiological alterations (OR = 10,818); PCR greater than 68 mg /L (OR = 11,596); leukocytes greater than 20,000 / mm3 (OR = 4.316); and ESR greater than 64 mm / 1 hour (OR = 4.267). Conclusion: The most frequent form of presentation was complete EK, the risk of CC was higher in males, younger than 5 years old, the risk factors (clinical and laboratory) were similar to those described in the literature.

Tratamiento con desmopresina sublingual en dos lactantes con hidranencefalia y diabetes insípida central / Treatment with sublingual desmopressin in two infants with hydranencephaly and central diabetes insipidus

La diabetes insípida central es una patología infrecuente en pediatría ocasionada por un déficit de vasopresina. Sus manifestaciones clínicas principales son poliuria y polidipsia. Las malformaciones cerebrales son una de las principales causas. La desmopresina es la droga sintética de elección para el tratamiento. Una de las vías de administración es la sublingual y su uso en lactantes es muy limitado. Se describe a dos lactantes con hidranencefalia y diabetes insípida central que fueron tratados satisfactoriamente con desmopresina sublingual.

Central diabetes insipidus is a rare disease in children caused by a deficiency of vasopressin. Its main clinical manifestations are polyuria and polydipsia. Brain malformations are one of the main causes. Desmopressin is the synthetic drug of choice for the treatment. One of the routes of administration is sublingual and its use in infants is very limited. We describe two infants with central diabetes insipidus and hydranencephaly who were successfully treated with sublingual desmopressin.