Autoinflammatory diseases: a Latin American multicenter study according to age and sex / Doenças autoinflamatórias: um estudo multicêntrico latino-americano de acordo com o sexo e a idade

ABSTRACT Objective: To evaluate autoinflammatory diseases (AID) according to age at diagnosis and sex, and response to therapy in a large population. Methods: This is a cross-sectional observational study of a Latin American registry using a designed web system for data storage, collected between 2015 and 2018. Any altered findings during follow-up were recorded. The forms were translated into Portuguese and Spanish, including demographic, clinical, laboratory, genetic and treatment characteristics. Results: We included 152 patients, 51.3% male and 75% Caucasian. The median age at disease onset was 2.1 years (0-15.6 years) and median age at diagnosis 6.9 years (0-21.9 years); 111 (73%) were children (0-9 years old), and 41 (27%) were adolescents and young adults (AYA) (10-21 years old). Periodic fever, aphthous stomatitis, pharyngitis, and adenitis syndrome (PFAPA) occurred in 46/152 (30%), chronic non-bacterial osteomyelitis (CNO) in 32/152 (21%), and familial Mediterranean fever (FMF) in 24/152 (15.7%). PFAPA was significantly higher in young children than in AYA (38.7% vs. 7.3%, p<0.001), while CNO were lower (13.5% vs. 41.5%, p<0.001). The frequency of females was significantly higher in CNO (28.4% vs. 14.1%, p=0.031) and lower in FMF (8.1% vs. 23.1%, p=0.011). The most used drugs were glucocorticoids, non-steroidal anti-inflammatory drugs (NSAID), and colchicine. Glucocorticoids and colchicine treatment were used in all AID with good to moderate response. However, cryopyrin-associated periodic syndromes (CAPS) seemed unresponsive to glucocorticoids. NSAIDs and methotrexate were the main medications used to treat CNO. Conclusions: Differences among AID patients were observed in the LA population regarding sex and age at disease diagnosis.

RESUMO Objetivo: Avaliar as doenças autoinflamatórias (DAI) de acordo com sexo e idade no momento do diagnóstico e a resposta terapêutica em uma grande população. Métodos: Este é um estudo observacional transversal de um registro latino-americano que usou um sistema de dados coletados entre 2015 e 2018. Quaisquer achados alterados ao longo do acompanhamento foram registrados. Os formulários foram traduzidos para os idiomas português e espanhol, incluindo características demográficas, clínicas, laboratoriais, genéticas e de tratamento. Resultados: Incluímos 152 pacientes, sendo 51,3% do sexo masculino e 75% da raça branca. A média de idade de início da doença foi de 2,1 anos (0-15,6 anos) e a média de idade de diagnóstico 6,9 anos (0-21,9 anos); 111 (73%) eram crianças (0-9 anos) e 41 (27%) adolescentes/adultos jovens (10-21 anos). A síndrome de febre periódica, estomatite aftosa, faringite e adenite (PFAPA) ocorreu em 46/152 (30%), osteomielite não bacteriana crônica (CNO) em 32/152 (21%) e febre familiar do Mediterrâneo (FMF) em 24/152 (15,7%). A PFAPA foi significativamente maior em crianças pequenas (38,7 vs. 7,3%, p<0,001), e a CNO, em adolescentes/adultos jovens (13,5 vs. 41,5%, p<0,001). A frequência do sexo feminino foi significativamente maior na CNO (28,4 vs. 14,1%, p=0,031) e menor na FMF (8,1 vs. 23,1%, p=0,011). Os medicamentos mais utilizados foram glicocorticoides, anti-inflamatórios não esteroidais (AINE) e colchicina. O tratamento com glicocorticoides e colchicina foi usado em todas as DAI com resposta boa a moderada. No entanto, as síndromes periódicas associadas à criopirina (CAPS) pareciam não responder aos glicocorticoides. AINE e metotrexato foram os principais medicamentos utilizados no tratamento da CNO. Conclusões: Diferenças de pacientes com DAI foram observadas na população latino-americana em pacientes agrupados por sexo e idade ao diagnóstico da doença.

Childhood-onset systemic lupus erythematosus (cSLE) and malignancy: a nationwide multicentre series review

Abstract Background Increased malignancy frequency is well documented in adult-systemic lupus erythematosus (SLE), but with limited reports in childhood-onset SLE (cSLE) series. We explored the frequency of malignancy associated with cSLE, describing clinical and demographic characteristics, disease activity and cumulative damage, by the time of malignancy diagnosis. Method A retrospective case-notes review, in a nationwide cohort from 27 Pediatric Rheumatology centres, with descriptive biopsy-proven malignancy, disease activity/damage accrual, and immunosuppressive treatment were compiled in each participating centre, using a standard protocol. Results Of the 1757 cSLE cases in the updated cohort, 12 (0.7%) developed malignancy with median time 10 years after cSLE diagnosis. There were 91% females, median age at cSLE diagnosis 12 years, median age at malignancy diagnosis 23 years. Of all diagnosed malignancies, 11 were single-site, and a single case with concomitant multiple sites; four had haematological (0.22%) and 8 solid malignancy (0.45%). Median (min-max) SLEDAI-2 K scores were 9 (0-38), median (min-max) SLICC/ACR-DI (SDI) score were 1 (1-5) Histopathology defined 1 Hodgkin's lymphoma, 2 non-Hodgkin's lymphoma, 1 acute lymphoblastic leukaemia; 4 gastrointestinal carcinoma, 1 squamous cell carcinoma of the tongue and 1 anal carcinoma; 1 had sigmoid adenocarcinoma and 1 stomach carcinoid; 3 had genital malignancy, being 1 vulvae, 1 cervix and 1 vulvae and cervix carcinomas; 1 had central nervous system oligodendroglioma; and 1 testicle germ cell teratoma. Conclusion Estimated malignancy frequency of 0.7% was reported during cSLE follow up in a multicentric series. Median disease activity and cumulative damage scores, by the time of malignancy diagnoses, were high; considering that reported in adult series.

Factors related to the readiness of Brazilian chronic pediatric patients to transition to care in adult clinics

Abstract Objective Advances in medicine have increased the life expectancy of pediatric patients with chronic illnesses, and challenges with the guided transition of adolescents and young adults from pediatric clinics to adult clinics have grown. The aim of this study was to better understand readiness and factors related to this transition process in Brazil. Method In this cross-sectional study of 308 patients aged from 16 to 21 years under follow-up in pediatric specialties, the degree of readiness for transition was assessed using the Transition Readiness Assessment Questionnaire (TRAQ) and its domains. Associations with demographic data, clinical data, socio-economic level, medication adherence, family functionality, and parental satisfaction with health care were evaluated. Results The median TRAQ score was 3.7 (3.2 - 4.2). Better readiness was associated with female patients, socio-economic class A-B, current active employment, higher level of education, not failing any school year, attending medical appointments alone, functional family, and a good knowledge of disease and medications. A low correlation was observed between TRAQ and age. TRAQ presented good internal consistency (alpha-Cronbach 0.86). In the multiple linear regression, TRAQ score showed a significant association with female gender, advanced age, socio-economic class A-B, better knowledge of disease and medications, and independence to attend appointments alone. Conclusion TRAQ instrument can guide healthcare professionals to identify specific areas of approach, in order to support adolescents with chronic disease to set goals for their own personal development and improve their readiness to enter into the adult healthcare system. In this study, some factors were related to better TRAQ scores.

Adherence to treatment of children and adolescents recently diagnosed with auto-immune Rheumatic Diseases during the COVID-19 pandemic

Abstract Objectives Adherence problems have a great impact on auto-immune Rheumatic Diseases (AIRD). The COVID-19 pandemic may have worsened treatment adherence. The aims of this study were to measure treatment adherence to identify an earlier risk of poor adherence and measure families' satisfaction with the health service during the pandemic. Methods Prospective observational study with 50 parents/children and adolescents with recent AIRD diagnosis. Initially, they answered questions (demographic data, disease) and completed the Pediatric Rheumatology Adherence Questionnaire (PRAQ), after 6 months they completed the Morisky-Green Test (MGT), Brief Medication Questionnaire (BMQ), Compliance Questionnaire for Rheumatology (CQR-19) and Pediatric Quality of Life Inventory Questionnaire 3.0 (PedsQlTM-SSS). The patient's medical records from the previous 12 weeks were reviewed for global and medication adherence data. Results The mean global adherence score was 94.3 ± 10.0, for medication adherence 97.3 ± 9.3, and for PRAQ questionnaire 5.2 ± 1.5. The authors observed agreement between MGT, BMQ, CQR-19, PedsQLTM-SSS scores and medication adherence rate, but not with global adherence rate. There were no associations between demographic characteristics, disease diagnosis, and adherence. No associations between PRAQ scores and values and global/medication adherence rates were observed. No variables were shown to be predictors of good adherence. The mean PedsQLTM-SSS rate was 92.1 ± 6.8. Conclusion The high values of MGT, BMQ, CQR-19 questionnaire scores were in agreement with the medication adherence rate. Despite the pandemic, the global and medication adherence rates were good. It was not possible to demonstrate the PRAQ's predictive power. The authors weren't able to establish an association between families' satisfaction and treatment adherence rates.

Could ultrasound and muscle elastography be associated with clinical assessment, laboratory and nailfold capillaroscopy in juvenile dermatomyositis patients?

Abstract Background Juvenile Dermatomyositis (JDM) is the most common idiopathic inflammatory myopathy in children. Imaging exams are useful for muscle assessment, with ultrasonography (US) being a promising tool in detecting disease activity and tissue damage. There are few studies about muscle elastography. Objectives Our aim was to associate clinical, laboratory, and nailfold capillaroscopy (NC) assessments with US in JDM patients; and to compare the findings of US and Strain Elastography (SE) from patients and healthy controls. Methods An analytic cross-sectional study was performed with JDM patients and healthy controls. Patients underwent clinical exam to access muscle strength and completed questionnaires about global assessment of the disease and functional capacity. Patients were submitted to NC and measurement of muscle enzymes. All subjects underwent US assessment, using gray scale, Power Doppler (PD), and SE. Results Twenty-two JDM patients and fourteen controls, aged between 5 and 21 years, matched for age and sex were assessed. In qualitative and semi-quantitative gray scale, we observed a higher frequency of alterations in patients (p < 0.001), while in PD, there was a higher frequency of positivity in patients' deltoids and anterior tibialis (p < 0.001). Active disease was associated with an important change in the semi-quantitative gray scale in deltoids (p = 0.007), biceps brachii (p = 0.001) and quadriceps femoris (p = 0.005). The SE demonstrated a high negative predictive value of 87.2. Conclusion US was able, through gray scale, to differentiate JDM patients from controls, while PD achieved such differentiation only for deltoids and anterior tibialis. The semi-quantitative gray scale showed disease activity in proximal muscles. SE was not able to differentiate patients from controls.

Does the use of panoramic radiography add information in the temporomandibular joint evaluation in Juvenile Idiopathic Arthritis patients? A case control study

Abstract Objective To determine the frequency of radiographic changes in the temporomandibular joint, in a representative population of patients with Juvenile Idiopathic Arthritis (JIA) and to compare with findings in healthy controls matched by sex and age. Patients and Methods One hundred and thirty-seven panoramic radiographies (PR) from JIA patients of a pediatric rheumatology outpatient clinic were prospectively evaluated and compared to 137 PR from healthy individuals. Results 102 (74.5%) JIA patients and 47 (34.3%) controls showed at least one radiological alteration (p < 0.001). The following radiographic alterations were more frequently observed in JIA patients than in controls: erosion (p < 0.001), altered condylar morphology (p < 0.001), disproportion between condylar process and the coronoid process (p < 0.001) and accentuated curve in the antegonial notch (p = 0.002). Twenty patients (14.6%) presented the four radiographic alterations simultaneously compared to only two controls (1.5%) (p < 0.001). Conclusion Due to the difference in the frequency of findings in the PR of patients and controls, we concluded that PR has value as a screening tool. In the presence of major changes in the mandible head in the PR of patients with a confirmed diagnosis of JIA, MRI should be considered to detect an active inflammatory process in this joint.

Does [18F]F-FDG-PET/MRI add metabolic information to magnetic resonance image in childhood-onset Takayasu's arteritis patients? A multicenter case series

Abstract Background: The observation that 2-deoxy-2[18F]fluoro-D-glucose-positron emission tomography/magnetic resonance imaging ([18F]F-FDG-PET/MRI) revealed high-grade arterial wall FDG uptake, without arterial wall thickening with contrast-enhancement, in a considerable number of c-TA patients in our previous study, encouraged us to compare patients with both PET and MR angiography (MRA) positives, with those with PET positive but MRA negative. Our aim was to evaluate the relevance of these two imaging modalities together. Methods: A three-center cross-sectional study with 17 patients who fulfilled the EULAR/PRINTO/PReS criteria for c-TA and who underwent [18F]F-FDG-PET/MRI was previously performed. Herein we compared patients/vessels with positive PET (arterial wall 18F-FDG uptake higher than liver) and positive MRA (arterial wall thickening with contrast-enhancement)—group 1, with those with positive PET but negative MRA—group 2. Results: Median disease duration of 17 c-TA patients was 10.4 years. Nine patients were classified as group 1 and six as group 2. Median of metabolic inflammatory volume (MIV) of all arterial segments was significantly higher in group 1 (2346 vs. 1177 cm3; p = 0.036). Fifty-four (19%) from 284 available arterial segments presented positive findings in vessel wall in one or both images. Positive findings were concordant between PET and MRA in only 13% arterial segments (group 1); most changes (28-59.6%) that were discordant between both images, were positive in PET and negative in MRA (group 2). Conclusions: Our study demonstrated that [18F]F-FDG-PET/MRI added information about inflammation in vessel wall of c-TA patients. Prospective multicenter studies are needed in order to get solid data to guide immunosuppressive tapering and withdrawal.

Official Position of the Brazilian Association of Bone Assessment and Metabolism (ABRASSO) on the evaluation of body composition by densitometry—part II (clinical aspects): interpretation, reporting, and special situations

Abstract Objective: To present an updated and evidence-based guideline for the use of dual-energy x-ray absorptiometry (DXA) to assess body composition in clinical practice. Materials and methods: This Official Position was developed by the Scientific Committee of the Brazilian Association of Bone Assessment and Metabolism ( Associação Brasileira de Avaliação Óssea e Osteometabolismo , ABRASSO) and experts in the field who were invited to contribute to the preparation of this document. The authors searched current databases for relevant publications in the area of body composition assessment. In this second part of the Official Position, the authors discuss the interpretation and reporting of body composition parameters assessed by DXA and the use of DXA for body composition evaluation in special situations, including evaluation of children, persons with HIV, and animals. Conclusion: This document offers recommendations for the use of DXA in body composition evaluation, including indications, interpretation, and applications, to serve as a guiding tool in clinical practice and research for health care professionals in Brazil.

Paediatric inflammatory multisystem syndrome temporally associated with SARS-CoV-2 (PIMS-TS): a Brazilian cohort

Abstract Background: Paediatric inflammatory multisystem syndrome (PIMS) associated with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has been described since mid-April 2020 with the first reports coming from Europe. Our objective was to describe the characteristics of patients among the Brazilian population. Methods: A multicenter retrospective study was conducted with the participation of five pediatric rheumatology centers in Brazil during the period from March to November 2020. Children and adolescents with PIMS temporally associated with SARS-CoV-2 (TS) who met the definition criteria for the disease according to the Royal College of Paediatrics and Child Health were included. Demographic, clinical, laboratory, therapeutic characteristics and molecular and serological diagnosis of SARS-CoV-2 infection were described. Results: Fifty-seven children and adolescents with PIMS-TS were evaluated, 54% female, with a median age of 8 (3-11) years. Most (86%) were previously healthy, with asthma being the main comorbidity, present in 10% of the patients. Fever was the main manifestation, present in all patients, followed by mucocutaneous and gastrointestinal features, present in 89% and 81% of the patients, respectively. Myocarditis occurred in 21% of the patients and in 68% of them required intensive care. The Kawasaki disease phenotype occurred in most patients (77%). All patients had elevated inflammatory markers, with elevated CRP being the most found (98%). Anemia and lymphopenia were present in 79% and 72%, respectively. Laboratory evidence of SARS-CoV-2 was found in 77% of the patients, with 39% positive RT-PCR and 84% positive serology for SARS-CoV-2. An immunomodulatory treatment was performed in 91% of the patients, with 67% receiving intravenous immunoglobulin (IVIG) associated with glucocorticoid, 21% receiving IVIG, and 3.5% receiving glucocorticoid. The median length of hospitalization was 10 days. Conclusions: This study showed a high morbidity of PIMS-TS in Brazilian children, with a prolonged length of hospitalization and a high rate of admission to pediatric intensive care unit. Multicenter prospective studies are needed to assess the morbidity of the disease in the medium and long term.

Factor analysis of the performance at the medical residency exam, progress test and clerkship rotations performance coefficient / Análise fatorial do desempenho no exame de residência médica, teste de progresso e coeficiente de desempenho nos estágios do internato

Abstract: Introduction: The relationships between the students' performance on medical residency exams and progress tests and medical clerkship rotations are not well established. Objective: The objective of this study was to measure the correlations between grades on progress tests and clerkship rotations assessments and the medical residency exam and determine which performance had the strongest correlation with the final medical residency exam. Methods: This was a retrospective and longitudinal study with correlation analyses of grades on progress tests from the 1st to 6th year of medical school, the clerkship rotations performance coefficient (5th and 6th years of school) and the final medical residency exam in a cohort of students enrolled in a federal public medical school using factor analysis. Students who performed the progress tests from the 1st to 6th year were included. Results: Of 123 students enrolled in the first year of medical school in 2009, 114 (92.7%) performed the progress tests during the six years and were included. The average grades on the progress tests from 1 to 10 were 2.67 (1st year), 3.01 (2nd year), 4.19 (3rd year), 4.01 (4th year), 5.19 (5th year), and 6.38 (6th year). The average grades in the clerkship rotations were 8.32 (5th year) and 8.26 (6th year). The average score on the theoretical medical residency exam was 7.53 and the final result of the medical residency exam was 8.05. Factor analysis detected three domains with greater correlation strength that accounted for 76.3% of the model variance. Component 1 was identified as the coefficient of academic performance (CAP) 5th, CAP 6th and final medical residency exam grades, whereas component 2 was constituted by the grades of the 5th and 6th years progress tests and the third component comprised the progress tests of the 2nd, 3rd and 4th years. Conclusions: Grades on the progress tests, the clerkship rotations assessments and the final medical residency exam were correlated. Moreover, the performance during the medical clerkship rotations showed the strongest correlations with medical residency exam grades.

Resumo: Introdução: As relações entre o desempenho dos alunos nos exames de residência médica e testes de progresso e os estágios no internato médico não estão bem estabelecidas. Objetivo: Este estudo teve como objetivos medir as correlações entre as notas nos testes de progresso e as notas no internato e o resultado final do exame de residência médica, e determinar qual desempenho teve a maior correlação com o exame final da residência médica. Método: Trata-se de um estudo retrospectivo e longitudinal com análises de correlação de notas em provas de progresso do primeiro ao sexto ano do curso de Medicina, coeficiente de desempenho de estágios do internato (quinto e sexto anos) e notas do exame final de residência médica em uma coorte de alunos matriculados em uma Faculdade de Medicina de uma instituição pública federal, usando análise fatorial. Foram incluídos os alunos que realizaram os testes de progresso do primeiro ao sexto ano. Resultado: Dos 123 alunos matriculados no primeiro ano do curso de Medicina em 2009, 114 (92,7%) realizaram os testes de progresso durante os seis anos letivos e foram incluídos. As notas médias nos testes de progresso de 1 a 10 foram 2,67 (primeiro ano), 3,01 (segundo ano), 4,19 (terceiro ano), 4,01 (quarto ano), 5,19 (quinto ano) e 6,38 (sexto ano). As notas médias nos estágios foram 8,32 (quinto ano) e 8,26 (sexto ano). A nota média no exame teórico da residência médica foi 7,53; e a média no exame final da residência, 8,5. A análise fatorial detectou três domínios com maior força de correlação que responderam por 76,3% da variância do modelo. O componente 1 foi identificado como coeficiente de rendimento acadêmico (CAP) 5º, CAP 6º e o resultado final do exame de residência médica, o componente 2 foi formado pelas notas das provas de progresso do quinto e sextos anos, e o terceiro componente compreendeu as notas do progresso do segundo, terceiro e quarto anos. Conclusão: As notas das provas de progresso, as avaliações do internato e o exame final de residência médica apresentaram correlações significantes. Além disso, o desempenho durante o internato apresentou maior correlação com as notas do exame final de residência médica.

Spotlight on latent tuberculosis infection screening for juvenile idiopathic arthritis in two countries, comparing high and low risk patients

Abstract Background: Rheumatic diseases are associated with an increase in overall risks of tuberculosis (TB). The aim of this study was to evaluate the frequency of TB and the frequency of latent TB infection (LTBI), in clinical practice, for juvenile idiopathic arthritis (JIA) patients from high and low risk of TB incidence endemic countries. Methods: This is an international, multicenter, cross-sectional, observational study of data collection from Brazil and Registry of Portugal at REUMA.PT. The inclusion criteria were patients with Juvenile Idiopathic Arthritis (JIA) with age ≤ 18 years who underwent screening for Mycobacterium tuberculosis infection [tuberculin skin test (TST) and/or interferon gamma release assay (IGRA)]. Chest X-rays and history of exposure to TB were also assessed. Results: 292 JIA patients were included; mean age 14.3 years, mean disease duration 7.5 years, 194 patients (66.4%) performed only TST, 14 (4.8%) only IGRA and 84 (28.8%) both. The frequency of LTBI (10.6%) and TB was similar between the two countries. The reasons for TB screening were different; in Brazil it was performed more often at JIA onset while in Portugal it was performed when starting Disease Modified Anti-Rheumatic Drugs (DMARD) treatment (p < 0.001). Isoniazid therapy was prescribed in 40 (13.7%) patients (31 with LTBI and 9 with epidemiologic risks and/or due to contact with sick people). Only three patients (1%) developed active TB. Conclusion: We found nearly 10% of patients with LTBI, a small percentage of patients with treatment due to epide-miologic risks and only 1% with active TB. Distinct reasons and screening methods for LTBI were observed between the two countries.

Sleep and restless legs syndrome in female adolescents with idiopathic musculoskeletal pain / Sono e síndrome das pernas inquietas em adolescentes do sexo feminino com dor musculoesquelética idiopática

Abstract Objectives: To assess the presence of restless legs syndrome, periodic leg movement, and sleep disorders in female adolescents with idiopathic musculoskeletal pain through a sleep scale and polysomnography, and to compare these data in adolescents without pain history. Method: Twenty-six adolescents diagnosed with idiopathic musculoskeletal pain followed in a pain outpatient clinic and 25 healthy controls matched by age and education were recruited. The restless legs syndrome criteria were evaluated according to the International Restless Legs Syndrome Study Group, the Sleep Disturbance Scale for Children was completed, nocturnal polysomnography was performed, and anxiety symptoms were recorded. Results: The mean age of idiopathic musculoskeletal pain adolescents was 13.9 ± 1.6 years; in controls, it was 14.4 ± 1.4 years. One adolescent in the control group (4 %) and nine patients with idiopathic musculoskeletal pain (34.6 %) fulfilled the restless legs syndrome criteria (p = 0.011). The authors did not observe significant differences in Sleep Disturbance Scale for Children scores between the groups in all components: disorders of initiating and maintaining sleep (p = 0.290), sleep breathing disorders (p = 0.576), disorders of arousal (p = 0.162), sleep-wake transition disorders (p = 0.258), disorder of excessive daytime somnolence (p = 0.594), and sleep hyperhidrosis (p = 0.797). The neurophysiological, respiratory, and periodic leg movement parameters were similar in both groups. Having anxiety was not associated with restless legs syndrome (p = 0.11). Three patients with idiopathic musculoskeletal pain (11.5 %) presented restless legs syndrome and periodic leg movement simultaneously, which was absent in the control group. Conclusion: Female adolescents with idiopathic musculoskeletal pain present criteria for RLS more frequently than healthy adolescents. However, this study did not observe relevant changes in objective and subject sleep variables.

Resumo Objetivos: Avaliar a presença de síndrome das pernas inquietas, movimento periódico das pernas e distúrbios do sono em adolescentes do sexo feminino com dor musculoesquelética idiopática por meio da escala do sono e da polissonografia e comparar esses dados em adolescentes sem histórico de dor. Método: Foram recrutados 26 adolescentes diagnosticados com dor musculoesquelética idiopática acompanhados em um ambulatório de dor e 25 controles saudáveis pareados por idade e escolaridade. Avaliamos os critérios da síndrome das pernas inquietas de acordo com o Grupo Internacional de Estudos de Síndrome das Pernas Inquietas, a Escala de Distúrbios do Sono em Crianças, a polissonografia noturna e os sintomas de ansiedade. Resultados: A idade média dos adolescentes com dor musculoesquelética idiopática foi 13,9 ± 1,6 anos e dos controles foi 14,4 ± 1,4 anos. Um adolescente no grupo de controle (4%) e nove pacientes com dor musculoesquelética idiopática (34,6%) atenderam aos critérios da síndrome das pernas inquietas (p = 0,011). Não observamos diferenças significativas nos escores da Escala de Distúrbios do Sono em Crianças entre os grupos em todos os componentes: distúrbios do início e da manutenção do sono (p = 0,290), distúrbios respiratórios do sono (p = 0,576), distúrbios do despertar (p = 0,162), distúrbios da transição sono-vigília (p = 0,258), sonolência diurna excessiva (p = 0,594) e hiperidrose do sono (p = 0,797). Os parâmetros neurofisiológicos, respiratórios e o movimento periódico das pernas foram semelhantes nos dois grupos. Ansiedade não foi associada à síndrome das pernas inquietas (p = 0,11). Três pacientes com dor musculoesquelética idiopática (11,5%) apresentaram síndrome das pernas inquietas e movimento periódico das pernas simultaneamente, situação ausente no grupo de controle. Conclusão: As adolescentes do sexo feminino com dor musculoesquelética idiopática apresentaram critérios para síndrome das pernas inquietas com mais frequência do que as adolescentes saudáveis. Contudo, não observamos mudanças relevantes nas variáveis do sono objetivas e subjetivas.

Lipid profile of pediatric patients with chronic rheumatic diseases - a retrospective analysis

SUMMARY AIM To describe the prevalence of dyslipidemia in children and adolescents with autoimmune rheumatic diseases (ARDs), particularly juvenile idiopathic arthritis (JIA), juvenile systemic lupus erythematosus (jSLE), and juvenile dermatomyositis (JDM). METHODS Retrospective cross-sectional study conducted in the pediatric rheumatology outpatient clinic. We evaluated 186 children and adolescents between the ages of 5 and 19 years. The medical records were reviewed for the following data: demographic and clinical features, disease activity, and lipid profile (triglycerides (TG), total cholesterol (TC), low density lipoprotein (LDL-C), high density lipoprotein (HDL-C) and very low density lipoprotein (VLDL-C)). In addition, non-HDL cholesterol was calculated as TC minus HDL-C. The cut-off points proposed by the American Academy of Pediatrics were used to classify the lipid profile. RESULTS Dyslipidemia was observed in 128 patients (68.8%), the most common being decreased HDL-C (74 patients, 39.8%). In the JIA group there was an association between the systemic subtype and altered LDL-C and NHDL-C, which demonstrated a more atherogenic profile in this subtype (p=0.027 and p=0.017, respectively). Among patients with jSLE, the cumulative corticosteroid dose was associated with an increase in LDL-C (p=0.013) and with a decrease in HDL-C (p=0.022). CONCLUSION Dyslipidemia is common in children and adolescents with ARDs, especially JIA, jSLE, and JDM, and the main alteration in the lipid profile of these patients was decreased HDL-C.

RESUMO OBJETIVO Descrever a prevalência de dislipidemias em crianças e adolescentes com doenças reumáticas autoimunes (Drai), em particular artrite idiopática juvenil (AIJ), lúpus eritematoso sistêmico juvenil (Lesj) e dermatomiosite juvenil (DMJ). MÉTODOS Estudo transversal retrospectivo realizado no ambulatório de reumatologia pediátrica. Foram avaliados 186 crianças e adolescentes com idades entre 5 e 19 anos. Foram coletados dos prontuários dados demográficos, clínicos, atividade de doença e perfil lipídico (triglicérides (TG), colesterol total (CT) e frações LDL-c (low density lipoprotein); HDL-c (high density lipoprotein) e VLDL-c (very low density lipoprotein). Foi também calculada a fração não HDL do colesterol (CT-NHDL -c). Para classificação do perfil lipídico, foram adotados os pontos de corte propostos pela American Academy of Pediatrics. RESULTADOS A dislipidemia foi observada em 128 pacientes (68,8%), sendo a mais comum a diminuição do HDL-c em 74 (39,8%). No grupo AIJ houve uma associação entre o subtipo sistêmico com alteração de LDL-c e NHDL-c, mostrando um perfil mais aterogênico neste subtipo (p=0,027 e 0,017, respectivamente). Em relação aos pacientes com Lesj, podemos observar que a dose cumulativa de CTC teve associação com o aumento do LDL-c (p=0,013) e com a diminuição do HDL-c (p=0,022). CONCLUSÃO A dislipidemia é frequente em crianças e adolescentes com Drai, em especial, AIJ, Lesj e DMJ, e a principal alteração no perfil lipídico desses pacientes foi a diminuição do HDL-c.

Virtual learning environment in pediatric rheumatology for pediatric residents / Ambiente virtual de aprendizagem em reumatologia pediátrica para residentes em pediatria

ABSTRACT Objective: To develop, implement and evaluate an online virtual learning environment (VLE) on pediatric rheumatology, aimed at pediatric residents, analyzing its effectiveness and satisfaction rates. Methods: A total of 92 first and second year pediatric residents at two pediatric reference centers were invited to participate in the study. Residents were randomized into a case group (that answered the pre-course test, attended the six virtual pediatric rheumatology modules, and then responded to the post-course test and a satisfaction questionnaire) and a control group (that only answered the pre-course test and, after 4 weeks, the post-course test). Results: Forty-seven residents (51%) completed their participation. In the case group (n=24), the mean percentage of correct answers was 14% higher on the post-course test (p<0.001). The number of correct answers was larger in the case group than in the control one (n=23) in the post-course test (p=0.045). In the assessment of satisfaction with VLE use, residents considered the site easy to navigate (91%), suitable as a learning tool (91%), and attractive in design (79%). They reported poor prior knowledge in pediatric rheumatology (91%) and agreed that there was good learning with the methodology (75%). Conclusions: The virtual learning environment in pediatric rheumatology proved to be an effective teaching tool with high satisfaction rates, providing pediatrician residents with adequate knowledge regarding the initial assessment and management of children with rheumatic diseases.

RESUMO Objetivo: Elaborar, implementar e avaliar um ambiente virtual de aprendizagem online em reumatologia pediátrica, direcionado aos residentes em pediatria, analisando sua efetividade e seus índices de satisfação. Métodos: Foram convidados 92 residentes de pediatria do primeiro e segundo anos de dois centros de referência em pediatria. Os residentes foram divididos, de forma randomizada, em grupo caso (que respondeu ao teste pré-curso, assistiu aos seis módulos virtuais de reumatologia pediátrica e, ao término das aulas virtuais, respondeu ao teste pós-curso e ao questionário de satisfação) e grupo controle (que apenas respondeu ao teste pré-curso e, após quatro semanas, ao teste pós-curso). Resultados: Completaram a participação 47 (51%) residentes. No grupo caso (n=24), o percentual de acertos foi 14% maior no teste pós-curso (p<0,001). Houve um percentual de acertos maior no grupo caso em relação ao grupo controle (n=23) na comparação do resultado do teste pós-curso (p=0,045). Na avaliação da satisfação em relação ao uso do ambiente virtual, os residentes consideraram o site de fácil navegação (91%), adequado como ferramenta de aprendizagem (91%) e com design atrativo (79%). Eles relataram um conhecimento prévio ruim em reumatologia pediátrica (91%) e concordaram que houve um bom aprendizado por meio da metodologia (75%). Conclusões: O ambiente virtual de aprendizado em reumatologia pediátrica mostrou-se uma ferramenta de ensino eficaz e com altos índices de satisfação na sua utilização, fornecendo ao residente em pediatria um conhecimento adequado para avaliação e conduta inicial de pacientes com doenças reumáticas da infância.

Adverse drug reactions associated with treatment in patients with chronic rheumatic diseases in childhood: a retrospective real life review of a single center cohort

Abstract Background: Adverse drug reactions (ADRs) are the sixth leading causes of death worldwide; monitoring them is fundamental, especially in patients with disorders like chronic rheumatic diseases (CRDs). The study aimed to describe the ADRs investigating their severity and associated factors and resulting interventions in pediatric patients with CRDs. Methods: A retrospective, descriptive and analytical study was conducted on a cohort of children and adolescents with juvenile idiopathic arthritis (JIA), juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM). The study evaluated medical records of the patients to determine the causality and the management of ADRs. In order to investigate the risk factors that would increase the risk of ADRs, a logistic regression model was carried out on a group of patients treated with the main used drug. Results: We observed 949 ADRs in 547 patients studied. Methotrexate (MTX) was the most frequently used medication and also the cause of the most ADRs, which occurred in 63.3% of patients, followed by glucocorticoids (GCs). Comparing synthetic disease-modifying anti-rheumatic drugs (sDMARDs) vs biologic disease-modifying antirheumatic drugs (bDMARDs), the ADRs attributed to the former were by far higher than the latter. In general, the severity of ADRs was moderate and manageable. Drug withdrawal occurred in almost a quarter of the cases. In terms of risk factors, most patients who experienced ADRs due to MTX, were 16 years old or younger and received MTX in doses equal or higher than 0.6 mg/kg/week. Patients with JIA and JDM had a lower risk of ADRs than patients with JSLE. In the multiple regression model, the use of GCs for over 6 months led to an increase of 0.5% in the number of ADRs. Conclusions: Although the ADRs highly likely affect a wide range of children and adolescents with CRDs they were considered moderate and manageable cases mostly. However, triggers of ADRs need further investigations.(AU)

Comparison of urinary parameters, biomarkers, and outcome of childhood systemic lupus erythematosus early onsetlupus nephritis

Abstract Background: Urinary parameters, anti-dsDNA antibodies and complement tests were explored in patients with childhood-Systemic Lupus Erythematosus (cSLE) early-onset lupus nephritis (ELN) from a large multicenter cohort study. Methods: Clinical and laboratory features of cSLE cases with kidney involvement at presentation, were reviewed. Disease activity parameters including SLEDAI-2 K scores and major organ involvement at onset and follow up, with accrued damage scored by SLICC-DI, during last follow up, were compared with those without kidney involvement. Autoantibodies, renal function and complement tests were determined by standard methods. Subjects were grouped by presence or absence of ELN. Results: Out of the 846 subjects enrolled, mean age 11.6 (SD 3.6) years; 427 (50.5%) had ELN. There was no significant difference in the ELN proportion, according to onset age, but ELN frequency was significantly higher in non-Caucasians (p = 0.03). Hematuria, pyuria, urine casts, 24-h proteinuria and arterial hypertension at baseline, all had significant association with ELN outcome (p < 0.001). With a similar follow up time, there were significantly higher SLICC-DI damage scores during last follow up visit (p = 0.004) and also higher death rates (p < 0.0001) in those with ELN. Low C3 (chi-square test, p = 0.01), but not C3 levels associated significantly with ELN. High anti-dsDNA antibody levels were associated with ELN (p < 0.0001), but anti-Sm, anti-RNP, anti-Ro, anti-La antibodies were not associated. Low C4, C4 levels, low CH50 and CH50 values had no significant association. High erythrocyte sedimentation rate (ESR) was associated with the absence of ELN (p = 0.02). Conclusion: The frequency of ELN was 50%, resulting in higher morbidity and mortality compared to those without ELN. The urinary parameters, positive anti-dsDNA and low C3 are reliable for discriminating ELN.(AU)

Early detection of poor adherence to treatment of pediatric rheumatic diseases: pediatric rheumatology adherence questionnaire - a pilot study / Detecção precoce da má adesão ao tratamento em doenças reumáticas pediátricas: questionário de adesão em reumatologia pediátrica - estudo piloto

ABSTRACT Objective: To develop a questionnaire that allows the early detection of patients at risk for poor adherence to medical and non-medical treatment in children and adolescents with chronic rheumatic diseases. Methods: The Pediatric Rheumatology Adherence Questionnaire (PRAQ) was applied in recently diagnosed patients within a period of one to four months after confirmation of the rheumatic disease. After six months, the patients' adherence to the medical and non-medical treatment was assessed. An internal consistency analysis was conducted to eliminate redundant questions in the PRAQ. Results: A total of 33 patients were included in the pilot study. Six months after the PRAQ had been applied, poor global adherence was observed in seven (21.2%) patients and poor adherence to medical treatment in eight (24.2%) patients. No correlation was observed between the PRAQ scores and the percentages of adherence, as well as the stratification for each index, except for a tendency to a correlation between socioeconomic index and poor adherence to medical treatment (p=0.08). A new PRAQ questionnaire with 25 of the 46 original questions was generated as a result of the reliability analysis. Conclusions: The usefulness of this questionnaire in clinical practice should be still evaluated. Due to the importance of a tool for the early detection of rheumatic patients at risk of poor adherence to treatment, the new PRAQ questionnaire should be reviewed and applied in a larger study to better define its validity and reliability.

RESUMO Objetivo: Desenvolver um questionário que permitisse a detecção precoce de pacientes em risco de má adesão ao tratamento medicamentoso e não medicamentoso para crianças e adolescentes com doenças reumáticas crônicas. Métodos: O Questionário de Adesão em Reumatologia Pediátrica (QARP) foi aplicado em pacientes recentemente diagnosticados, de um a quatro meses após a confirmação da doença reumática. Seis meses depois, foi avaliada a adesão do paciente ao tratamento medicamentoso e não medicamentoso. Foi realizada uma análise de consistência interna para eliminar questões redundantes no QARP. Resultados: Ao todo, 33 pacientes foram incluídos no estudo-piloto. Seis meses após a aplicação do QARP, observou-se baixa adesão global em sete pacientes (21,2%) e baixa adesão ao tratamento medicamentoso em oito (24,2%) pacientes. Não foi encontrada correlação entre os escores QARP e as porcentagens de adesão, bem como a estratificação para cada indicador, com exceção de uma tendência a uma correlação entre o indicador socioeconômico e a baixa adesão ao tratamento medicamentoso (p=0,08). Um novo questionário QARP com 25 das 46 perguntas originais foi gerado como resultado da análise de confiabilidade. Conclusões: A utilidade deste questionário na prática clínica ainda deve ser avaliada. Devido à importância de uma ferramenta para a detecção precoce de pacientes reumáticos em risco de má adesão ao tratamento, o novo questionário QARP deve ser revisado e aplicado em um estudo maior para que sua validade e confiabilidade sejam mais bem definidas.

Translation and validation of the Transition Readiness Assessment Questionnaire (TRAQ) / Tradução e validação do Questionário de Avaliação do Preparo para a Transição (TRAQ)

Abstract Objective: To translate and validate the Brazilian Portuguese version of the Transition Readiness Assessment Questionnaire in a population of adolescents and young adults with chronic rheumatologic disorders. This questionnaire evaluates the patient's readiness for making the transition from the pediatric health service to adult care. Methods: The four-phase methodology for the translation and validation of generic questionnaires was followed, including translation, back-translation, pilot testing and clinical validation of the final tool. The confirmatory factor analysis was used for clinical validation and the Cronbach's alpha coefficient was used to assess the overall internal consistency of the final tool. Results: A total of 150 patients with a mean age of 17.0 years (SD = 2.2 years, range 14-21 years) were enrolled for the final tool validation. Of those, 71 patients had juvenile systemic lupus erythematosus (47.3%), 64 had juvenile idiopathic arthritis (42.7%), and 15 had juvenile dermatomyositis (10%). During the confirmatory factor analysis, the dimension "Talking with providers" consisting of two questions, was considered as not fitting the translated questionnaire due to a very high ceiling effect and was therefore excluded. All other translated items favorably contributed to the overall consistency of the final tool; removing that dimension did not result in a substantial increase in Cronbach's alpha, which was 0.776. Conclusions: The Brazilian Portuguese version of the Transition Readiness Assessment Questionnaire was validated in a population of transitional patients with chronic rheumatologic disorders, after one dimension from the original questionnaire was excluded. It is a non-specific disease questionnaire; thus, it can be used to evaluate the transition readiness of Brazilian patients with other chronic diseases.

Resumo Objetivo: Traduzir para o português brasileiro e validar o Questionário de Avaliação do Preparo para a Transição em uma população de adolescentes e adultos jovens com doenças reumáticas crônicas. Este questionário avalia o preparo do paciente para realizar a transição do serviço de saúde pediátrico para a assistência ao adulto. Métodos: Seguimos a metodologia de quatro etapas para a tradução e validação de questionários genéricos que inclui tradução, retrotradução, teste piloto e validação clínica do instrumento final. Utilizamos Análise Fatorial Confirmatória e Coeficiente Alfa de Cronbach para testar a validade do instrumento e sua consistência interna. Resultados: Responderam ao questionário traduzido e adaptado 150 pacientes. A média de idade foi de 17,0 anos (DP = 2,2 anos, variação 14-21 anos). Tinham o diagnóstico de lúpus eritematoso sistêmico juvenil 71 pacientes (47,3%), 64 (42,7%) artrite idiopática juvenil e 15 (10%) dermatomiosite juvenil. Durante a análise fatorial confirmatória, a dimensão "Falando com a Equipe Médica" contendo duas questões teve que ser removida devido à presença de expressivo efeito teto. Todas as outras questões restantes contribuíram favoravelmente para aumentar a consistência interna do questionário, obteve-se um Coeficiente Alfa de Cronbach de 0,776. Conclusões: O Questionário de Avaliação do Preparo para a Transição na sua versão em português brasileiro pode ser validado em uma população de pacientes com doenças reumáticas crônicas em transição, com a exclusão de uma dimensão do questionário original. Por ser um questionário não específico para doenças reumáticas, poderá ser utilizado para avaliar o preparo para a transição de outros pacientes brasileiros com doenças crônicas.

Percepção e enfrentamento da dor em crianças e adolescentes com fibromialgia juvenil e artrite idiopática juvenil poliarticular / Pain perception and pain coping mechanisms in children and adolescents with juvenile fibromyalgia and polyarticular juvenile idiopathic arthritis

RESUMO Objetivo: Mensurar e comparar a dor musculoesquelética em pacientes com fibromialgia juvenil (FMJ) e em pacientes com artrite idiopática juvenil poliarticular (AIJ); e avaliar e comparar a percepção e o enfrentamento da dor. Métodos: Foram avaliados, em estudo transversal, 150 crianças e adolescentes (e seus respectivos pais), divididos em três grupos: FMJ, AIJ e controles saudáveis. A mensuração e o enfrentamento da dor foram realizados por meio de instrumentos específicos. Para a avaliação da percepção da dor, desenvolveram-se três vinhetas com simulação de situações que pudessem gerar dor: aplicação de injeção, queda de bicicleta e isolamento social. Os pais e os pacientes responderam individualmente quanto à percepção da dor em cada situação. Resultados: As maiores notas de dor, os menores escores de enfrentamento da dor, as maiores notas para a percepção da dor nas vinhetas e os piores índices de qualidade de vida relacionada à saúde foram observados nos pacientes com FMJ, quando comparados aos pacientes com AIJ e aos controles. O mesmo padrão foi observado com os respectivos pais. Conclusões: Pacientes com AIJ e FMJ se comportam diferentemente em relação à percepção da dor e ao desenvolvimento de técnicas para o enfrentamento da dor. A dor deve ser avaliada sob diferentes perspectivas para um planejamento mais individualizado e efetivo do tratamento desses pacientes.

ABSTRACT Objective: To measure and compare musculoskeletal pain in patients with juvenile fibromyalgia (JFM) and polyarticular juvenile idiopathic arthritis (JIA), and to evaluate and compare pain perception and pain coping mechanisms in these patients. Methods: In this cross sectional study, we evaluated 150 children and adolescents, and their respective parents, from 3 different groups: JFM, polyarticular JIA, and healthy controls. Pain intensity and pain coping mechanisms were measured using specific questionnaires. Pain perception was evaluated according to three illustrations simulating situations that might cause pain: a shot, a bicycle fall, and social isolation. The patients' parents also filled out the questionnaires and provided a pain score that matched their child's perception of pain for each illustration. Results: The highest pain scores, the lowest pain coping strategy scores, the highest pain perception scores for all three illustrations, and the worse health related to quality of life indicators were observed in the JFM group, when compared to the JIA and control groups. The same pattern was observed with their parents. Conclusions: Patients with JIA and JFM behave differently in relation to pain perception and the development pain coping mechanisms. Pain should be evaluated from different perspectives for an individualized and efficient treatment of patients.