RESUMO
Objetivo: Estimar el impacto económico del emicizumab en pacientes con hemofilia A (HA) e inhibidor en un hospital de tercer nivel, comparándolo con las alternativas terapéuticas. Métodos: Se estimó el coste anual del tratamiento de la HA e inhibidor con complejo protrombínico activado (aPCC), factor VII recombinante (rFVIIa) y emicizumab, y varias estrategias terapéuticas: profiláctica, a demanda e inmunotolerancia (ITI). Las dosis utilizadas, localización, frecuencia y gravedad de los sangrados se obtuvieron de la literatura. Resultados: El coste medio anual de la estrategia a demanda con aPCC/rFVIIa y de la estrategia profiláctica fueron 309.523 y 354.866 , en un paciente pediátrico y 808.928 y 926.574 en un adulto, respectivamente. El coste de la ITI fue 619.644 y 1.029.399 en el paciente pediátrico y adulto, respectivamente. Respecto a la estrategia profiláctica, el coste del tratamiento con emicizumab fue un 27,7% menor en el paciente pediátrico (240.255 ) y un 50,8% menor en el adulto (427.266 ).Conclusiones: Emicizumab, además de aportar mejoras clínicas y de calidad de vida a los pacientes con HA, ofrece ventajas económicas frente a los agentes bypass. (AU)
Purpose: Estimate the economic impact of the use of emicizumab in patients with hemophilia A (HA) and inhibitor in a third level hospital, comparing it with the different therapeutic alternatives.Methods: HA and inhibitor annual cost treatment with activated prothrombin complex (aPCC), recombinant factor VII (rFVIIa) and emicizumab was estimated. The patients were stratified in pediatrics (<14 years) and adults (>14 years). Several strategies were considered: prophylactic, on demand and immune tolerance induced (ITI). The dose used and the bleeding location, frequency and severity were obtained from the literature.Results: The average annual cost of on demand strategy with aPCC and rFVIIa and prophylactic strategy in a pediatric patient was 309,523 and 354,866 respectively, and 808,928 and 926,574 in an adult patient, respectively. ITI cost was 619,644 and 1,029,399 in pediatric and adult patient respectively. Regard prophylactic strategy, the treatment cost with emicizumab was 27.7% lower in pediatric patients (240,255 ) and 50.8% lower in adult patients (427,266 ). (AU)
Assuntos
Humanos , Custos de Medicamentos , Custos e Análise de Custo , Hemofilia A/tratamento farmacológico , Protrombina , Fator VIIRESUMO
BACKGROUND: The aim of this study is to analyze the global prevalence of carriers of heterozygous hemoglobinopathies among pregnant women settled in Lanzarote. PATIENTS AND METHODS: A epidemiologic cross-sectional observational investigation was undertaken to study the prevalence of hemoglobinopathies in 2,436 pregnant women in Lanzarote. The techniques of primary screening were hemoglobin electrophoresis on cellulose acetate at alkaline pH for the detection of hemoglobin variants, and the quantification of HbA2 and HbF for the diagnosis of b thalassemia trait. The study to confirm the diagnosis of structural hemoglobinopathies was based on hemoglobin electrophoresis on citrate agar at acid pH, isolectric focusing and high-performance liquid chromatography (HPLC). The molecular characterization of b thalassemia trait (HbA2 >3.5%) was carried out by techniques using a real time PCR procedure with specific fluorescently labelled hybridization probes and allele-specific amplification (PCR-ARMS). RESULTS: The global prevalence of hemoglobinopathies was 11.90 corresponding to 9.44 for structural hemoglobinopathies and 2.46 for heterozygous b thalassemias. A variant hemoglobin was detected on 23 women and the distribution was as follows: thirteen carriers of hemoglobin S, seven HbC trait, two HbD trait and one "unstable" hemoglobin. 82.6% of the variant hemoglobins found were from migrant population from Africa and America. CONCLUSIONS: The high prevalence of carriers of structural hemoglobinopathies in Lanzarote justifies the initiation of programs for screening for hemoglobinopathies to prevent the emergence of severe states causing disease.
Assuntos
Hemoglobinopatias/epidemiologia , Complicações Hematológicas na Gravidez/epidemiologia , Estudos Transversais , Árvores de Decisões , Feminino , Hemoglobinopatias/sangue , Humanos , Gravidez , Complicações Hematológicas na Gravidez/sangue , Prevalência , Espanha/epidemiologiaRESUMO
The anti-CD20 monoclonal antibody rituximab has shown benefit in some patients diagnosed of severe cold agglutinin disease. Here we report our experience with rituximab in a patient with chronic haemolysis due to refractory cold agglutinin disease. An increase in the haemoglobin level was observed seven months later from rituximab administration and with a follow-up of 17 months, the patient maintains the haematological response. We suggest that rituximab can play an important role in the treatment of adult patients with refractory cold agglutinin disease with anaemia requiring transfusion. However only a few case reports of rituximab treatment in this haemolytic disease is available and there is need of large prospective studies that allow elucidate the better schedule of administration, the duration of the clinical effect, factors predictive of clinical outcome, the possible benefit of the association with other drugs and the possibility to achieve delayed and maintained responses.
Assuntos
Anemia Hemolítica Autoimune/tratamento farmacológico , Anticorpos Monoclonais/uso terapêutico , Fatores Imunológicos/uso terapêutico , Anticorpos Monoclonais Murinos , Antígenos CD20/imunologia , Humanos , Masculino , Pessoa de Meia-Idade , Rituximab , Fatores de TempoRESUMO
Fundamento: El objetivo de este trabajo es el estudio de la prevalencia de las hemoglobinopatías heterocigotas en las mujeres gestantes residentes en Lanzarote. Pacientes y métodos: Se ha puesto en marcha un estudio epidemiológico observacional transversal para determinar la prevalencia de hemoglobinopatías en 2.436 mujeres gestantes en Lanzarote. El método diagnóstico de despistaje para las hemoglobinas variantes fue la electroforesis de hemoglobinas en acetato de celulosa a pH alcalino y para la b talasemia la cuantificación de hemoglobinas A2 y fetal. El estudio de confirmación de una hemoglobinopatía estructural se basó en la electroforesis de hemoglobinas en agar citrato a pH ácido, el isoelectroenfoque y la cromatografía líquida de alta resolución (HPLC). El estudio molecular de la b talasemia (HbA2 > 3,5%) se realizó con técnicas de PCR en tiempo real y sondas marcadas con fluorógenos y la PCR con amplificación de alelos específicos (PCR-ARMS). Resultados: La prevalencia global de portadores de hemoglobinopatías fue 11,90, de los que 9,44 eran hemoglobinopatías estructurales y 2,46 b talasemias heterocigotas. Se detectó una hemoglobina variante en 23 mujeres y la distribución fue: trece casos con hemoglobinas S, siete portadoras de HbC, dos de HbD y una hemoglobina inestable. El 82,6% de las hemoglobinas variantes correspondían a población inmigrante con origen en Africa y América. Conclusiones: La alta prevalencia de portadores de hemoglobinopatías estructurales en Lanzarote justifica la puesta en marcha de programas prospectivos de detección de portadores para prevenir la aparición de las formas severas de la enfermedad
Background: The aim of this study is to analyze the global prevalence of carriers of heterozygous hemoglobinopathies among pregnant women settled in Lanzarote. Patients and methods: A epidemiologic cross-sectional observational investigation was undertaken to study the prevalence of hemoglobinopathies in 2,436 pregnant women in Lanzarote. The techniques of primary screening were hemoglobin electrophoresis on cellulose acetate at alkaline pH for the detection of hemoglobin variants, and the quantification of HbA2 and HbF for the diagnosis of b thalassemia trait. The study to confirm the diagnosis of structural hemoglobinopathies was based on hemoglobin electrophoresis on citrate agar at acid pH, isolectric focusing and high-performance liquid chromatography (HPLC). The molecular characterization of b thalassemia trait (HbA2 >3.5%) was carried out by techniques using a real time PCR procedure with specific fluorescently labelled hybridization probes and allele-specific amplification (PCR-ARMS). Results: The global prevalence of hemoglobinopathies was 11.90 corresponding to 9.44 for structural hemoglobinopathies and 2.46 for heterozygous b thalassemias. A variant hemoglobin was detected on 23 women and the distribution was as follows: thirteen carriers of hemoglobin S, seven HbC trait, two HbD trait and one unstable hemoglobin. 82.6% of the variant hemoglobins found were from migrant population from Africa and America. Conclusions: The high prevalence of carriers of structural hemoglobinopathies in Lanzarote justifies the initation of programs for screening for hemoglobinopathies to prevent the emergence of severe states causing disease
Assuntos
Feminino , Adulto , Humanos , Hemoglobinopatias/diagnóstico , Hemoglobinopatias/epidemiologia , Hemoglobinopatias/prevenção & controle , Eletroforese/métodos , Cromatografia Líquida de Alta Pressão/métodos , Protocolos Clínicos , Cromatografia/métodos , Complicações na Gravidez/diagnóstico , Complicações Hematológicas na Gravidez/diagnóstico , Complicações Hematológicas na Gravidez/epidemiologia , Espanha/epidemiologia , Eletroforese/classificação , Demografia , Estudos Transversais , Grupos de RiscoRESUMO
El anticuerpo monoclonal anti-CD20 rituximab ha demostrado ser útil en el tratamiento de pacientes diagnosticados de enfermedad hemolítica severa por crioaglutininas. Presentamos nuestra experiencia con el empleo de rituximab en un paciente con hemólisis crónica por una enfermedad por crioaglutininas refractaria. Siete meses después de la administración del rituximab se detectó un aumento de la tasa de hemoglobina y durante el seguimiento durante 17 meses se mantuvo la respuesta hematológica. El rituximab puede jugar un papel en el tratamiento de individuos adultos con enfermedad por crioaglutininas refractarias a otros tratamientos con anemia dependientes de transfusión. Sin embargo, existen escasas series de casos que describan el empleo de rituximab en esta enfermedad hemolítica y son necesarios estudios prospectivos amplios que permitan aclarar el mejor esquema de administración, los posibles factores predictores del resultado, el beneficio de su asociación con otros fármacos y la posibilidad de obtener respuestas diferidas y mantenidas
The anti-CD20 monoclonal antibody rituximab has shown benefit in some patients diagnosed of severe cold agglutinin disease. Here we report our experience with rituximab in a patient with chronic haemolysis due to refractory cold agglutinin disease. An increase in the haemoglobin level was observed seven months later from rituximab administration and with a follow-up of 17 months, the patient maintains the haematological response. We suggest that rituximab can play an important role in the treatment of adult patients with refractory cold agglutinin disease with anaemia requiring transfusion. However only a few case reports of rituximab treatment in this haemolytic disease is available and there is need of large prospective studies that allow elucidate the better schedule of administration, the duration of the clinical effect, factors predictive of clinical outcome, the possible benefit of the association with other drugs and the possibility to achieve delayed and maintained responses
Assuntos
Masculino , Pessoa de Meia-Idade , Humanos , Anemia Hemolítica Autoimune/complicações , Anemia Hemolítica Autoimune/diagnóstico , Anemia Hemolítica Autoimune/tratamento farmacológico , Anticorpos Monoclonais , Hemólise , Imunossupressores/farmacocinética , Imunossupressores/uso terapêutico , Antígenos CD20 , Anemia Hemolítica/complicações , Anemia Hemolítica/diagnóstico , Imunossupressores/administração & dosagem , Imunossupressores/análiseRESUMO
No disponible
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Pessoa de Meia-Idade , Masculino , Humanos , Artrite Gotosa , Protocolos de Quimioterapia Combinada Antineoplásica , Linfoma não HodgkinRESUMO
No disponible
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Adulto , Masculino , Humanos , Tuberculose , Infecções por HIV , Carcinoma de Células Escamosas , Neoplasias PulmonaresRESUMO
No disponible
