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: To develop and validate a diagnostic score that assists in discriminating primary hemophagocytic lymphohistiocytosis (pHLH) from macrophage activation syndrome (MAS) related to systemic juvenile idiopathic arthritis. STUDY DESIGN: The clinical, laboratory, and histopathologic features of 362 patients with MAS and 258 patients with pHLH were collected in a multinational collaborative study. Eighty percent of the population was assessed to develop the score and the remaining 20% constituted the validation sample. Variables that entered the best fitted model of logistic regression were assigned a score, based on their statistical weight. The MAS/HLH (MH) score was made up with the individual scores of selected variables. The cutoff in the MH score that discriminated pHLH from MAS best was calculated by means of receiver operating characteristic curve analysis. Score performance was examined in both developmental and validation samples. RESULTS: Six variables composed the MH score: age at onset, neutrophil count, fibrinogen, splenomegaly, platelet count, and hemoglobin. The MH score ranged from 0 to 123, and its median value was 97 (1st-3rd quartile 75-123) and 12 (1st-3rd quartile 11-34) in pHLH and MAS, respectively. The probability of a diagnosis of pHLH ranged from <1% for a score of <11 to >99% for a score of ≥123. A cutoff value of ≥60 revealed the best performance in discriminating pHLH from MAS. CONCLUSION: The MH score is a powerful tool that may aid practitioners to identify patients who are more likely to have pHLH and, thus, could be prioritized for functional and genetic testing.
Assuntos
Linfo-Histiocitose Hemofagocítica/diagnóstico , Síndrome de Ativação Macrofágica/diagnóstico , Adolescente , Criança , Pré-Escolar , Diagnóstico Diferencial , Feminino , Humanos , Lactente , Masculino , Reprodutibilidade dos TestesRESUMO
OBJECTIVE: To investigate pediatric intensive care unit (PICU) admission in children with malignant and nonmalignant diseases who developed life-threatening complications. PATIENTS AND METHODS: Between 1999 and 2010, of the 1278 eligible pediatric patients treated for a malignant or nonmalignant disease, 54 were admitted to the PICU for respiratory distress (40.7%), neurological events (33.3%), severe sepsis (14.8%), and organ failure (11.2%). RESULTS: Rate of PICU admission was 4.2%, with a 2-year cumulative incidence of 4.5%. Risk factors associated with higher cumulative incidence of PICU admission were older age at study entry (P=0.003), nonmalignant underlying disease (P=0.015), and hematopoietic stem cell transplantation (P<0.001). Patients with leukemia/lymphoma were more likely to be admitted to the PICU compared with patients with solid tumors (P<0.001). Patients admitted because of organ failure had the highest frequency of death within 90 days. Factors significantly associated with survival at 90 days from PICU admission included: no mechanical ventilation (P<0.001), nonmalignant underlying disease (P=0.030), and year of PICU admission after 2005 (P=0.038). CONCLUSIONS: Nonmalignant disease and use of alternative hematopoietic stem cell transplantation were associated with higher risk of PICU admission. Close cooperation between hematologists and intensivists and definition of criteria for PICU admission and discharge contributed to increase in survival of these patients.