RESUMO
OBJECTIVE: To analyze, in a cohort of pediatric patients with recurrent pericarditis undergoing anti-interleukin (IL)-1 treatment: the agent and dosing used as first-line treatment, the long-term efficacy of IL-1 blockers, the percentage of patients achieving a drug-free remission, and the presence of variables associated with drug-free remission. STUDY DESIGN: Data were collected from patients' charts. The annualized relapse rate (ARR) was used for evaluation of treatment efficacy, and bivariate logistic regression analysis was used for variables associated with drug-free remission. RESULTS: Fifty-eight patients, treated between 2008 and 2018, were included in the study (mean follow-up. 2.6 years). Of the 56 patients treated with first-line drugs, 14 not responsive patients were underdosed. Fifty-seven patients were treated with anakinra: the ARR before and during daily treatment was 3.05 and 0.28, respectively (P < .0001); an increase to 0.83 was observed after the reduction/withdrawal of treatment (P < .0001). The switch from anakinra to canakinumab (5 patients) was associated to an increase of the ARR (0.49 vs 1.46), but without statistical significance (P = .215). At last follow-up, only 9 of the 58 patients had withdrawn all treatments. With the limits of a retrospective study and the heterogeneity between the patients enrolled in the study, a shorter duration of treatment with anakinra was the only variable associated with drug-free remission. CONCLUSIONS: This study shows that most pediatric patients with recurrent pericarditis needing IL-1 blockade received an inadequate treatment with first-line agents. The effectiveness of anakinra is supported by this study, but few patients achieved drug-free remission. The different rate of response to anakinra and canakinumab may suggest a possible role of IL-1α in the pathogenesis of recurrent pericarditis.
Assuntos
Proteína Antagonista do Receptor de Interleucina 1 , Pericardite , Humanos , Criança , Proteína Antagonista do Receptor de Interleucina 1/uso terapêutico , Estudos Retrospectivos , Interleucina-1/uso terapêutico , Padrão de Cuidado , Resultado do Tratamento , Pericardite/tratamento farmacológico , RecidivaRESUMO
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 evaluate the long-term response and safety of interleukin-1 receptor antagonist (anakinra) in recurrent pericarditis. STUDY DESIGN: Fifteen patients (12 children, 3 adults) were enrolled in a multicenter retrospective study. All the patients were corticosteroid-dependent and 14 had received colchicine. Anakinra was given at 1-2 mg/kg/d. The primary outcome of the study was a reduction of at least 70% of disease flares after anakinra treatment compared with the pretreatment period. Secondary outcomes were: (1) number of complete or partial responders to anakinra and time for complete response; (2) number of patients who discontinued other ongoing treatments (non-steroidal anti-inflammatory drugs, corticosteroid, colchicine) and time needed for discontinuation; (3) number of relapses during continuous anakinra treatment; and (4) number of relapses during anakinra tapering or discontinuation. RESULTS: All patients treated had a complete response within a few days and were able to rapidly withdraw concomitant treatments, including corticosteroids. During daily treatment, no patient had a relapse of the disease; 14 patients started tapering and 6 of them experienced a relapse, with a prompt response after anakinra reintroduction. Overall, after a median follow-up of 39 months (range 6-57), a 95% reduction of flares was observed compared with pretreatment period. CONCLUSION: The long-term use of anakinra in monotherapy is associated with persistent control of recurrent pericarditis.
Assuntos
Corticosteroides/uso terapêutico , Colchicina/uso terapêutico , Resistência a Medicamentos , Proteína Antagonista do Receptor de Interleucina 1/administração & dosagem , Pericardite/tratamento farmacológico , Adolescente , Corticosteroides/efeitos adversos , Criança , Estudos de Coortes , Colchicina/efeitos adversos , Relação Dose-Resposta a Droga , Esquema de Medicação , Feminino , Seguimentos , Humanos , Masculino , Pessoa de Meia-Idade , Pericardite/diagnóstico , Recidiva , Estudos Retrospectivos , Índice de Gravidade de Doença , Fatores de Tempo , Resultado do Tratamento , Adulto JovemRESUMO
We report a family with pyogenic sterile arthritis, pyoderna and acne syndrome (PAPA). The proband presented several episodes of sterile pyogenic arthritis and became unresponsive to glucocorticoids. After treatment with the tumor necrosis factor inhibitor etanercept, the disease underwent rapid and sustained clinical remission. Production of tumor necrosis factor-alpha by mononuclear cells of the proband and of the affected relatives was abnormally elevated.