RESUMO
INTRODUCTION: Although the regular replacement of clotting factor concentrates (prophylaxis) has been well established as the standard of care for severe haemophilia, the high cost of factor concentrates has limited access to prophylaxis in countries with under-developed or developing economies. AIMS: We studied the health gap that could be addressed by providing unlimited access to clotting factor concentrates with implementation of long-term prophylaxis initiated from an early age in life. METHODS: We performed a cross-sectional study of a random, representative sample of boys with moderate and severe haemophilia at three haemophilia treatment centres in Sao Paulo, Brazil, and one centre in Toronto, Canada. RESULTS: Canadian subjects were more often treated with prophylaxis, and began treatment at an earlier age. Fewer Canadian subjects had bleeds within the preceding 6 months (19 vs. 34, P = 0.003). Canadian subjects had lower (better) Pettersson radiographic scores (1.5 vs. 6.0, P = 0.0016), lower (better) Hemophilia Joint Health Scores (5.5 vs. 10.5, P = 0.0038), higher (better) Activity Scale for Kids scores (96.6 vs. 92.0, P = 0.033), more time spent in vigorous activity, and higher (better) social participation scores. CONCLUSIONS: Our findings suggest that increasing access to clotting factor concentrates for young boys with severe haemophilia is a global imperative.
Assuntos
Efeitos Psicossociais da Doença , Países em Desenvolvimento , Recursos em Saúde , Hemofilia A/epidemiologia , Adolescente , Brasil/epidemiologia , Canadá/epidemiologia , Criança , Estudos Transversais , Indicadores Básicos de Saúde , Hemofilia A/diagnóstico , Hemofilia A/terapia , Humanos , Masculino , Qualidade de Vida , Índice de Gravidade de DoençaRESUMO
INTRODUCTION: It is essential to assess the health-related quality of life outcomes of boys with haemophilia in Brazil. The Canadian Haemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) was recently adapted for this population. AIM: To test the construct validity of the Portuguese version of the CHO-KLAT. METHODS: We recruited 50 boys, with moderate [factor VIII (FVIII) level 1-5%] or severe (FVIII level <1%) haemophilia, to participate in a descriptive study to establish a baseline understanding of the current status of boys with haemophilia in Brazil. All boys were required to complete the Brazilian CHO-KLAT and Brazilian Pediatric Quality of Life Inventory (PedsQL) by self-report. We examined the correlation between the CHO-KLAT and PedsQL scores to establish the construct validity of the Brazilian version of the CHO-KLAT. RESULTS: We obtained CHO-KLAT and PedsQL data from 35 boys with severe haemophilia and 15 with moderate haemophilia. They ranged in age from 7.3 to 18.0 years, with a mean of 13.0 years. They reported a mean CHO-KLAT score of 72.3 (range = 44.1-93.9). The mean PedsQL score was 79.9 (range = 45.7-96.7), with physical health (mean of 83.9) being better than psychosocial health (77.8). The Pearson's correlation between CHO-KLAT and PedsQL was 0.47 respectively (P < 0.001). The CHO-KLAT had a moderate and inverse relationship with the degree to which they were bothered by their haemophilia (ρ = -0.53), while the PedsQL had a weaker relationship (ρ = -0.27). CONCLUSION: The results confirm the validity of the Portuguese version of the CHO-KLAT. This measure is now available for clinical trials in boys with haemophilia in Brazil.
Assuntos
Hemofilia A/tratamento farmacológico , Avaliação de Resultados em Cuidados de Saúde/métodos , Adolescente , Brasil , Criança , Humanos , Masculino , Qualidade de VidaRESUMO
OBJECTIVE: To determine whether treatment with tumor necrosis factor alpha (TNFalpha)-blocking agents alters the incidence of new-onset uveitis in patients with juvenile idiopathic arthritis (JIA). STUDY DESIGN: Cohort study based on retrospective chart review. The charts of all 1109 patients with a diagnosis of JIA seen between January 1, 1996, and June 30, 2003, at our clinic were reviewed for diagnosis of uveitis and treatment with TNFalpha inhibitors. Cox regression analysis was performed with anti-TNFalpha treatment as a time-dependent covariate for risk of development of uveitis. RESULTS: We identified 70 patients treated with anti-TNFalpha without a prior diagnosis of uveitis. Two of these 70 patients (2.9%), both treated with etanercept, had development of new-onset uveitis during anti-TNFalpha therapy. One had juvenile psoriatic arthritis diagnosed 4.1 years before onset of uveitis. The other had extended oligoarticular JIA diagnosed 6.4 years before onset of uveitis. We found no statistically significant difference in the risk for development of uveitis between patients with or without anti-TNFalpha treatment. CONCLUSIONS: In our patients with JIA, anti-TNFalpha treatment did not alter the risk for development of new-onset uveitis. However, anti-TNFalpha therapy with etanercept did not prevent the development of uveitis in 2 patients.
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Anticorpos Monoclonais/efeitos adversos , Artrite Juvenil/tratamento farmacológico , Imunoglobulina G/efeitos adversos , Fator de Necrose Tumoral alfa/antagonistas & inibidores , Uveíte/induzido quimicamente , Uveíte/epidemiologia , Adolescente , Criança , Pré-Escolar , Estudos de Coortes , Etanercepte , Feminino , Humanos , Incidência , Lactente , Infliximab , Masculino , Receptores do Fator de Necrose Tumoral , Estudos Retrospectivos , Fatores de RiscoRESUMO
We evaluated the self-esteem and quality of life of 47 children with morphea with the use of the Harter self-perception profile for children and Visual Analog Scale. Most children with morphea have normal self-worth and a high quality of life. Morphea, like some other childhood chronic illnesses, does not impair self-esteem.
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Qualidade de Vida , Esclerodermia Localizada/psicologia , Autoimagem , Adolescente , Criança , Estudos Transversais , Feminino , Humanos , Masculino , Inquéritos e QuestionáriosRESUMO
INTRODUCTION: Localized scleroderma (LS) can cause permanent disability, and there is no universally accepted effective treatment. Methotrexate (MTX) has been shown to be effective in the treatment of systemic sclerosis. OBJECTIVES: To determine the efficacy and tolerability of MTX and corticosteroid therapy in patients with LS. METHODS: MTX, 0.3 to 0.6 mg/kg per week, was given to 10 patients (6 girls, 4 boys; mean age, 6.8 years; mean disease duration before starting treatment, 4 years) with active LS. In addition, pulse intravenous methylprednisolone, 30 mg/kg for 3 days monthly for 3 months, was given to 9 patients at the initiation of therapy. RESULTS: One patient discontinued taking MTX after a month; the remaining 9 patients responded. The median time to response was 3 months (95% CI, 1.15-4.85). One responder discontinued taking MTX after a year because of leukopenia; the LS worsened within 2 months. In another patient LS flared up after 10 months and responded to an increased dose of MTX and intravenous methylprednisolone. At the last follow-up visit, all patients who continued to receive MTX therapy had inactive skin lesions. CONCLUSION: Treatment with MTX and corticosteroids appears to be effective in the treatment of LS and is generally well tolerated. A placebo-controlled study is necessary to confirm the efficacy of MTX therapy in LS.
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Anti-Inflamatórios/uso terapêutico , Fármacos Dermatológicos/uso terapêutico , Glucocorticoides/uso terapêutico , Imunossupressores/uso terapêutico , Metotrexato/uso terapêutico , Metilprednisolona/uso terapêutico , Esclerodermia Localizada/tratamento farmacológico , Administração Oral , Anti-Inflamatórios/administração & dosagem , Criança , Pré-Escolar , Intervalos de Confiança , Fármacos Dermatológicos/administração & dosagem , Fármacos Dermatológicos/efeitos adversos , Quimioterapia Combinada , Feminino , Seguimentos , Glucocorticoides/administração & dosagem , Humanos , Imunossupressores/administração & dosagem , Imunossupressores/efeitos adversos , Injeções Intravenosas , Leucopenia/induzido quimicamente , Masculino , Metotrexato/administração & dosagem , Metotrexato/efeitos adversos , Metilprednisolona/administração & dosagem , Placebos , Recidiva , Indução de RemissãoRESUMO
OBJECTIVES: To determine the prevalence of abnormalities in myocardial perfusion or function in children with systemic lupus erythematosus (SLE), and describe potential factors that may predict their development. STUDY DESIGN: Patients (n = 40; 30 female) were enrolled through the Lupus Clinic at The Hospital for Sick Children between 1990 and 1992. Resting and exercise thallium myocardial perfusion scans, radionuclide angiography with multiple gated acquisition (MUGA), and resting M-mode and two-dimensional echocardiography were performed. RESULTS: All patients were free of symptoms, and none had a history of ischemic heart disease. Their median age was 15.9 years (range 10.5 to 19.8 years) at enrollment. Abnormalities of coronary perfusion were found in 5 (16%) of 31 patients (95% confidence interval: 3%, 29%) and included a large fixed perfusion defect in 1; 5 of 27 MUGA scans showed marginally low left ventricular ejection fractions at rest, whereas all had normal exercise responses. In the group with abnormal thallium scans, three of five patients had antiphospholipid antibodies detected, and two of four had an abnormal plasma lipid profile. This group tended to have a shorter disease duration and had received a lower cumulative dose of corticosteroids; these differences were not statistically significant compared with the group with normal scans. CONCLUSION: Asymptomatic abnormalities of myocardial perfusion occur in children with SLE and are more common than previously suspected. Patients with these abnormalities of myocardial perfusion may be predisposed to the previously recognized early-onset ischemic heart disease seen in adults with SLE.
Assuntos
Coração/diagnóstico por imagem , Lúpus Eritematoso Sistêmico/fisiopatologia , Isquemia Miocárdica/etiologia , Adolescente , Anti-Inflamatórios/uso terapêutico , Anticorpos Antifosfolipídeos/sangue , Criança , Ecocardiografia , Teste de Esforço , Feminino , Glucocorticoides/uso terapêutico , Testes de Função Cardíaca , Humanos , Lipídeos/sangue , Lúpus Eritematoso Sistêmico/tratamento farmacológico , Masculino , Isquemia Miocárdica/diagnóstico , Angiografia Cintilográfica , Esteroides , Tomografia Computadorizada de Emissão de Fóton Único , Função VentricularRESUMO
OBJECTIVE: This study was undertaken to investigate the recent finding of a seasonal difference in the onset of systemic-onset juvenile rheumatoid arthritis (SoJRA). We hypothesized that a seasonal onset pattern might implicate on infectious agent as a cause of SoJRA. METHODS: The date of onset was collected from the records of all patients with SoJRA from 1980 to 1992 at presentation to pediatric rheumatology clinics across Canada. The onset pattern of SoJRA was then compared with incidence data on viral infections obtained for the same period. RESULTS: Across Canada the onset of SoJRA was constant across the seasons. However, in the Prairie region there was a statistically significant seasonal pattern, with peaks in autumn and early spring. We could find no evidence that viral incidence correlated with disease incidence either throughout Canada or in the Prairie region. CONCLUSIONS: If a seasonal infectious agent causes SoJRA, then it is likely only one of several causes and may act only in certain regions. Future studies should be carried out in those areas where SoJRA does have a seasonal onset pattern.
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Artrite Juvenil/epidemiologia , Estações do Ano , Adolescente , Idade de Início , Artrite Juvenil/virologia , Canadá/epidemiologia , Criança , Pré-Escolar , Feminino , Humanos , Incidência , Lactente , Recém-Nascido , Masculino , Viroses/epidemiologiaRESUMO
Two unrelated patients with a family history of rheumatic fever had isolated, acquired chorea. Both index cases, as well as affected family members, had increased expression of the rheumatic B-cell alloantigen D8/17. This test may help differentiate Sydenham chorea from lupus chorea.