RESUMO
OBJECTIVE: To describe the clinical characteristics, treatment, and outcomes of a multinational cohort of patients with macrophage activation syndrome (MAS) and thrombotic microangiopathy (TMA). STUDY DESIGN: International pediatric rheumatologists were asked to collect retrospectively the data of patients with the co-occurrence of MAS and TMA. Clinical and laboratory features of patients with systemic juvenile idiopathic arthritis (sJIA)-associated MAS and TMA were compared with those of an historical cohort of patients with sJIA and MAS. RESULTS: Twenty-three patients with MAS and TMA were enrolled: 17 had sJIA, 2 systemic lupus erythematosus, 1 juvenile dermatomyositis, 1 mixed connective tissue disease, and 2 undifferentiated connective tissue disease. Compared with the historical cohort of MAS, patients with sJIA with coexistent MAS and TMA had higher frequencies of renal failure and neurologic involvement, hemorrhage, jaundice, and respiratory symptoms, as well as more severe anemia and thrombocytopenia, higher levels of alanine aminotransferase, lactate dehydrogenase, bilirubin and D-dimer, and lower levels of albumin and fibrinogen. They also required admission to the intensive care unit more frequently. Among patients tested, complement abnormalities and reduced ADAMTS13 activity were observed in 64.3% and 44.4% of cases, respectively. All patients received glucocorticoids. Treatment for TMA included plasma-exchange, eculizumab, and rituximab. CONCLUSIONS: The possible coexistence of MAS and TMA in rheumatic diseases may be underrecognized. This association should be considered in patients with MAS who develop disproportionate anemia, thrombocytopenia, and lactate dehydrogenase increase, or have multiorgan failure.
Assuntos
Artrite Juvenil/fisiopatologia , Síndrome de Ativação Macrofágica/fisiopatologia , Microangiopatias Trombóticas/fisiopatologia , Adolescente , Anticorpos Monoclonais Humanizados/uso terapêutico , Antirreumáticos/uso terapêutico , Artrite Juvenil/complicações , Artrite Juvenil/tratamento farmacológico , Biomarcadores/sangue , Criança , Pré-Escolar , Glucocorticoides/uso terapêutico , Humanos , Síndrome de Ativação Macrofágica/complicações , Síndrome de Ativação Macrofágica/tratamento farmacológico , Troca Plasmática , Estudos Retrospectivos , Microangiopatias Trombóticas/complicações , Microangiopatias Trombóticas/tratamento farmacológicoRESUMO
OBJECTIVE: Long-term outcome of steroid-sensitive idiopathic nephrotic syndrome (SSNS) in children is usually considered benign, although data on follow-up into adulthood are scarce. The aim of this study was to investigate adults who had childhood SSNS regarding their relapse rate, growth, and renal and extrarenal morbidity. STUDY DESIGN: Adult patients (n=42, 26 males) were evaluated at a median age of 28.0 (18.1 to 46.9) years and a median follow-up of 22.0 (2.9 to 39.0) years since diagnosis. RESULTS: Fourteen of 42 (33%) patients relapsed in adulthood. The number of relapses during childhood and adolescence and a complicated course-administration of steroid-sparing medication such as cyclophosphamide, chlorambucil, and cyclosporin A-were identified as risk factors. Final adult height (median SD score -0.4, range -3.3 to +1.3) and body mass index (BMI) were normal. Renal function was normal in all patients, and overall morbidity was low. Only eight patients (three males) had children. Cytotoxic therapy was identified as a major factor contributing to childlessness. CONCLUSION: Relapses in adulthood were common in pediatric patients with SSNS. Growth and renal function were normal, and overall morbidity was low. Yet, transition to an adult nephrologist is recommended for all children with relapsing SSNS.