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This case report describes a 23-year-old male patient who presented with right chylothorax as the initial manifestation of a severe flare of systemic lupus erythematosus (SLE) and secondary Evans syndrome. Chylothorax and chylous ascites are rare features of SLE that can occur due to the accumulation of triglyceride-rich fluid in serous cavities. However, they have never been reported as the initial manifestation of a lupus flare. Evans syndrome is a rare disease characterized by autoimmune hemolytic anemia and immune thrombocytopenia, which can be secondary to SLE. The concomitant occurrence of both chylothorax and Evans syndrome in the setting of systemic lupus erythematosus has never been described, and the exact causative mechanisms of both entities are yet to be fully understood. In this report, we discuss our approach to this challenging case to broaden the understanding of the clinical manifestations of systemic lupus erythematosus. Our findings emphasize the importance of considering rare features of systemic lupus erythematosus and secondary diseases when evaluating patients with the disease.
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INTRODUCTION: The Evans syndrome (ES) is a rare, often chronic, relapsing and treatment-refractory hematological disorder. We described the clinical features, diagnostic workup, treatment and outcome in patients with ES. METHOD: We performed a retrospective chart review of patients aged < 18 years with ES admitted to a tertiary center in Brazil from 2001 to 2021. The analysis of the data was primarily descriptive, using median, interquartile range and categorical variables presented in absolute frequencies. MAIN RESULTS: Twenty patients (12 female, 8 male) were evaluated in this study. The median age at the initial cytopenia was 4.98 years (1.30-12.57). The ES was secondary in nine cases (45%), of which six patients (30%) showed autoimmune disease (AID) or primary immunodeficiencies (PID) and one presented a spontaneous recovery. Steroids and intravenous immunoglobulin were first-line therapy in 19 cases. Twelve patients (63%) required second-line treatments (rituximab, cyclosporine, splenectomy, sirolimus, cyclophosphamide, mycophenolate mofetil, azathioprine and eltrombopag). The median follow-up period was 2.41 years (1.4 -7.52). One patient (5%) died of underlying neuroblastoma, one case (5%) was lost to follow-up and four patients (20%) received a medical discharge. The median age for the 14 remaining cases was 12.6 years. Twelve patients (85.7%) were in complete response (CR) with no therapies. Two patients (14.3%) were in CR with chronic therapy. CONCLUSION: As ES may be a symptom of AID and PID, a thorough rheumatological, immunologic and genetic workup and a careful follow-up are essential. The second-line treatment remains a dilemma. Further prospective studies are needed to address the optimal therapeutic combinations, morbidity and mortality in this disorder.
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Abstract Introduction The Evans syndrome (ES) is a rare, often chronic, relapsing and treatment-refractory hematological disorder. We described the clinical features, diagnostic workup, treatment and outcome in patients with ES. Method We performed a retrospective chart review of patients aged < 18 years with ES admitted to a tertiary center in Brazil from 2001 to 2021. The analysis of the data was primarily descriptive, using median, interquartile range and categorical variables presented in absolute frequencies. Main results Twenty patients (12 female, 8 male) were evaluated in this study. The median age at the initial cytopenia was 4.98 years (1.30-12.57). The ES was secondary in nine cases (45%), of which six patients (30%) showed autoimmune disease (AID) or primary immunodeficiencies (PID) and one presented a spontaneous recovery. Steroids and intravenous immunoglobulin were first-line therapy in 19 cases. Twelve patients (63%) required second-line treatments (rituximab, cyclosporine, splenectomy, sirolimus, cyclophosphamide, mycophenolate mofetil, azathioprine and eltrombopag). The median follow-up period was 2.41 years (1.4 -7.52). One patient (5%) died of underlying neuroblastoma, one case (5%) was lost to follow-up and four patients (20%) received a medical discharge. The median age for the 14 remaining cases was 12.6 years. Twelve patients (85.7%) were in complete response (CR) with no therapies. Two patients (14.3%) were in CR with chronic therapy. Conclusion As ES may be a symptom of AID and PID, a thorough rheumatological, immunologic and genetic workup and a careful follow-up are essential. The second-line treatment remains a dilemma. Further prospective studies are needed to address the optimal therapeutic combinations, morbidity and mortality in this disorder.
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Humanos , Masculino , Feminino , Lactente , Pré-Escolar , Criança , Adolescente , Púrpura Trombocitopênica Idiopática , Anemia Hemolítica Autoimune , Pediatria , Lúpus Eritematoso SistêmicoRESUMO
ABSTRACT Background: Secondary immune thrombocytopenia (ITP) is a heterogeneous and unpredictable disease associated with various underlying conditions. Objective: The objective of the study was to investigate clinical evolution and chronicity predictors in secondary ITP. Methods: Patients treated at an academic medical center during 2008-2019 were stratified by age as children <16 years and adults >16 years. Responses to steroids, intravenous immunoglobulin G (IVIG), rituximab, and eltrombopag were classified as response (R) and complete response (CR). Risk factors for chronic ITP were determined by multiple regression with uni- and multi-variate analysis. Results: Eighty-three patients were included, 37 children and 46 adults. The most frequent associated conditions were infections 53%, systemic lupus erythematosus (SLE) 24%, thyroid disease 9.6%, and Evans syndrome 3.6%. Response to first-line treatment in the whole cohort was 94%; CR 45.7%; and R 50.6%. Initial response to steroids alone was 91.3% (n = 21/23), rituximab plus high-dose dexamethasone (HDD) 93.3% (n = 14/15); children receiving IVIG alone 100% (n=12/12); and eltrombopag in adults 100% (n = 3/3). Relapse was documented in 19.4% of children and 34% of adults, at a median time of 15 and 2 months, respectively; 30.4% of adults (15.2% from the miscellaneous group, 10.9% SLE-associated, and 4.3% infection-associated) and 18.9% of children followed a chronic course; age ≥10 years and platelets ≥20 × 109/L were risk factors for chronic ITP in children. Conclusion: Evolution was heterogeneous: a better and more sustained response was documented in the infections group compared to SLE or the miscellaneous group. (REV INVEST CLIN. 2021;73(1):31-8)
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Humanos , Lactente , Pré-Escolar , Criança , Adolescente , Adulto , Pessoa de Meia-Idade , Idoso , Adulto Jovem , Púrpura Trombocitopênica Idiopática/diagnóstico , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Encaminhamento e Consulta , Doença Crônica , Estudos Retrospectivos , Resultado do Tratamento , HematologiaRESUMO
Primary immune thrombocytopenia (ITP) is an intriguing autoimmune disease characterized by autoantibodies against platelets and megakaryocytes. Clinical outcomes, response to treatment, and chronicity predictors were investigated. Patients with newly diagnosed primary ITP treated at a hematology referral center from 2008 to 2018 with complete medical and recent medication history were stratified by age as children < 16 years and adults > 16 years. Responses to treatment including steroids, splenectomy, rituximab, and eltrombopag were classified as response (R) and complete (CR). Factors for developing chronic ITP were determined by multiple regression with uni- and multivariate analysis. p < 0.05 was considered significant. A total of 175 patients were included, 52 children and 123 adults; women predominated with 57.7%. Response to first-line treatment in the whole cohort was 86.18%, CR 43.42% and R 42.76%. The initial response to steroids alone was 83.9% (n = 52/62), rituximab plus high-dose dexamethasone (HDD) 87.2% (n = 34/39), eltrombopag plus HDD 90.9% (n = 10/11), and children receiving IVIG alone 100% (n = 8/8); 9 children were under clinical observation and achieved spontaneous response; loss of response was documented in 15.21% children and 28.3% adults with a median time of 15.95 and 4.07 months respectively; 37.39% of adults and 30.76% of children progressed to a chronic course. Platelets ≥ 20 × 109/L and age ≥ 6 years were risk factors for chronic ITP in the univariate analysis in the adult and children groups, respectively. Clinical course and treatment outcomes for ITP are considerably heterogeneous. Higher platelet counts at diagnosis in adults and age ≥ 6 years in children were associated with an increased risk of chronicity.
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Benzoatos/administração & dosagem , Dexametasona/administração & dosagem , Hidrazinas/administração & dosagem , Imunoglobulinas Intravenosas/administração & dosagem , Púrpura Trombocitopênica Idiopática/terapia , Pirazóis/administração & dosagem , Rituximab , Esplenectomia , Adolescente , Adulto , Fatores Etários , Idoso , Idoso de 80 Anos ou mais , Criança , Pré-Escolar , Doença Crônica , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Pessoa de Meia-Idade , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/sangue , Estudos RetrospectivosRESUMO
La púrpura trombocitopénica inmunológica es una enfermedad autoinmune, benigna, de aparición frecuente, caracterizada por la presencia de anticuerpos dirigidos contra las glicoproteínas de la membrana plaquetaria que producen una disminución del recuento plaquetario y manifestaciones hemorrágicas cutáneo-mucosas. El diagnóstico de esta entidad se realiza por exclusión de otras causas de trombocitopenia. El síndrome de Guillain-Barré es también una enfermedad de naturaleza autoinmune donde la pérdida de la tolerancia inmunológica trae como consecuencia la aparición de anticuerpos dirigidos contra los gangliósidos de los nervios periféricos. Se presenta una paciente femenina de 40 años con diagnóstico de una púrpura trombocitopénica inmunológica crónica que comenzó con una parálisis motora ascendente, sin toma respiratoria, parálisis facial y dolor intenso en las regiones dorsal y lumbar. Fue diagnosticada como un síndrome de Guillain-Barré e inmediatamente se comenzó tratamiento con vitaminoterapia y esteroides a altas dosis. Después de varios meses de seguimiento y rehabilitación presentó una evolución satisfactoria con remisión de todos los síntomas neurológicos
The immunologic thrombocytopenic purpura is an autoimmune, benign, of frequent appearance disease characterized by the presence of antibodies directed to glycoproteins of platelet membrane producing a decrease of platelet count and cutaneous-mucosal hemorrhagic manifestations. The Guillain-BarrÚ syndrome is also a disease autoimmune by origin where the loss of immunological tolerance causes the appearance of antibodies directed to gangliosides of peripheral nerves. This is the case of female patient aged 40 diagnosed with a chronic immunologic thrombocytopenic purpura beginning with an ascendant motor paralysis, without respiratory compromise, facial paralysis and intense pain in dorsal and lumbar regions and also a diagnosis of Guillain-BarrÚ syndrome with immediate treatment based on vitamin-therapy and high dose of steroids. After some months of follow-up and rehabilitation there was a satisfactory evolution with remission of all neurological symptoms
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Humanos , Adulto , Feminino , Esteroides/uso terapêutico , Púrpura Trombocitopênica Idiopática/complicações , Síndrome de Guillain-Barré/complicações , Síndrome de Guillain-Barré/tratamento farmacológico , Vitaminas/uso terapêuticoRESUMO
Quadros trombocitopênicos são descritos nos cães como resultado na queda da produção efetiva de plaquetas ou aumento na sua distribuição, utilização e sequestro. A Trombocitopenia Imunomediada caracteriza-se pela distribuição dos trombócitos pelo sistema imune mediada por anticorpos e complemento dentro do baço, fígado e medula óssea. As circunstâncias que levam à formação de anticorpos anti-plaquetas são indeterminadas. Neste relato foi observado um cão macho, dez anos de idade, da raça poodle, atendido em um consultório veterinário apresentando quadro de trombocitopenia severa. Após administração de terapia imunossupressiva, com uso de prednisona e danazol, houve remissão do quadro trombocitopênico. Após 90 dias de tratamento o animal apresentava-se clinicamente sadio, com eritrograma e plaquetograma inalterados. A ocorrência deste caso de trombocitopenia imunomediada deve servir com alerta para a ocorrência desta doença e como base para o diagnóstico diferencial de algumas hemoparasitoses importantes e rotineiras que levam à quadros trombocitopênicos semelhantes
Thrombocytopenia may result from the failure to effectively produce platelets or from accelerated platelet destruction, utilization and sequestration. Autoimmune Thrombocytopenia is characterized by the destruction of the thrombocytes by the immune system mediated by antibodies and complement inside of the spleen, liver and bone marrow. The circumstances leading to formation of antiplatelet antibody remain unknown. In this case, it was observed a male dog, ten years old, poodle, was assisted at a veterinary clinic presenting severe thrombocytopenia. After immunosuppressive therapy, with prednisone and danazol, the thrombocytopenia was suppressed. After 90 days of treatment, the animal came clinically healthy, with red blood and platelet unaffected. The report of an autoimmune thrombocytopenia in a dog is an alert for the existence of this disease and should be useful for the differential diagnosis of some important and routinist hemoparasitosis causing similar thrombocytopenia
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Animais , Cães , Cães , Terapia de Imunossupressão/veterinária , Trombocitopenia/diagnóstico , Trombocitopenia/veterináriaRESUMO
Quadros trombocitopênicos são descritos nos cães como resultado na queda da produção efetiva de plaquetas ou aumento na sua distribuição, utilização e sequestro. A Trombocitopenia Imunomediada caracteriza-se pela distribuição dos trombócitos pelo sistema imune mediada por anticorpos e complemento dentro do baço, fígado e medula óssea. As circunstâncias que levam à formação de anticorpos anti-plaquetas são indeterminadas. Neste relato foi observado um cão macho, dez anos de idade, da raça poodle, atendido em um consultório veterinário apresentando quadro de trombocitopenia severa. Após administração de terapia imunossupressiva, com uso de prednisona e danazol, houve remissão do quadro trombocitopênico. Após 90 dias de tratamento o animal apresentava-se clinicamente sadio, com eritrograma e plaquetograma inalterados. A ocorrência deste caso de trombocitopenia imunomediada deve servir com alerta para a ocorrência desta doença e como base para o diagnóstico diferencial de algumas hemoparasitoses importantes e rotineiras que levam à quadros trombocitopênicos semelhantes(AU)
Thrombocytopenia may result from the failure to effectively produce platelets or from accelerated platelet destruction, utilization and sequestration. Autoimmune Thrombocytopenia is characterized by the destruction of the thrombocytes by the immune system mediated by antibodies and complement inside of the spleen, liver and bone marrow. The circumstances leading to formation of antiplatelet antibody remain unknown. In this case, it was observed a male dog, ten years old, poodle, was assisted at a veterinary clinic presenting severe thrombocytopenia. After immunosuppressive therapy, with prednisone and danazol, the thrombocytopenia was suppressed. After 90 days of treatment, the animal came clinically healthy, with red blood and platelet unaffected. The report of an autoimmune thrombocytopenia in a dog is an alert for the existence of this disease and should be useful for the differential diagnosis of some important and routinist hemoparasitosis causing similar thrombocytopenia(AU)