Skeletal involvement in Gaucher disease: An observational multicenter study of prognostic factors in the Argentine Gaucher disease patients.

Patients with Gaucher type 1 (GD1) throughout Argentina were enrolled in the Argentine bone project to evaluate bone disease and its determinants. We focused on presence and predictors of bone lesions (BL) and their relationship to therapeutic goals (TG) with timing and dose of enzyme replacement therapy (ERT). A total of 124 patients on ERT were enrolled in a multi-center study. All six TG were achieved by 82% of patients: 70.1% for bone pain and 91.1% for bone crisis. However, despite the fact that bone TGs were achieved, residual bone disease was present in 108 patients on ERT (87%) at time 0. 16% of patients showed new irreversible BL (bone infarcts and avascular osteonecrosis) despite ERT, suggesting that they appeared during ERT or were not detected at the moment of diagnosis. We observed 5 prognostic factors that predicted a higher probability of being free of bone disease: optimal ERT compliance; early diagnosis; timely initiation of therapy; ERT initiation dose ≥45 UI/kg/EOW; and the absence of history of splenectomy. Skeletal involvement was classified into 4 major phenotypic groups according to BL: group 1 (12.9%) without BL; group 2 (28.2%) with reversible BL; group 3 (41.9%) with reversible BL and irreversible chronic BL; and group 4 (16.9%) with acute irreversible BL. Our study identifies prognostic factors for achieving best therapeutic outcomes, introduces new risk stratification for patients and suggests the need for a redefinition of bone TG. Am. J. Hematol. 91:E448-E453, 2016. © 2016 Wiley Periodicals, Inc.

Safety and efficacy of anticoagulant therapy in pediatric catheter-related venous thrombosis (EINSTEIN-Jr CVC-VTE).

Anticoagulant treatment of pediatric central venous catheter-related venous thromboembolism (CVC-VTE) has not been specifically evaluated. In EINSTEIN-Jr, 500 children with any VTE received rivaroxaban or standard anticoagulants. A predefined analysis of the CVC-VTE cohort was performed. Children with CVC-VTE (age, birth to 17 years) were administered rivaroxaban or standard anticoagulants during the 1-month (children <2 years) or 3-month (all other children) study period. Predefined outcomes were recurrent VTE, change in thrombotic burden on repeat imaging, and bleeding. Predictors for continuation of anticoagulant therapy beyond the study period were evaluated. One hundred twenty-six children with symptomatic (n = 76, 60%) or asymptomatic (n = 50, 40%) CVC-VTE received either rivaroxaban (n = 90) or standard anticoagulants (n = 36). There was no recurrent VTE (0%; 95% confidence interval [CI], 0.0%-2.8%). Three children had the principal safety outcome: none had major bleeding and 3 children had clinically relevant nonmajor bleeding (2.4%; 95% CI, 0.7%-6.5%), all in the rivaroxaban arm. Complete or partial vein recanalization occurred in 57 (55%) and 38 (37%) of 103 evaluable children, respectively. Results were similar for symptomatic and asymptomatic CVC-VTE. Continuation of anticoagulant therapy beyond the study period occurred in 61 (48%) of children and was associated with residual VTE but only in children <2 years (odds ratio [OR], 20.9; P = .003) and continued CVC use (OR, 6.7; P = .002). Anticoagulant therapy appeared safe and efficacious and was associated with reduced clot burden in most children with symptomatic or asymptomatic CVC-VTE. Residual VTE and continued CVC use were associated with extended anticoagulation. This trial was registered at www.clinicaltrials.gov as #NCT02234843.

Análisis de la mortalidad en enfermedades Hemato-oncológicas malignas en pediatría en hospitales públicos de Argentina / Analysis of Mortality due to Hemato-Oncological Pediatrics Malignancies in Public Hospitals in Argentina

INTRODUCCION: En Argentina, la mortalidad por enfermedades malignas en edad pediátrica ocupa un lugar relevante y sus causas todavía no han sido estudiadas en el país. OBJETIVO: Analizar las tasas, causas y etapas de los fallecimientos relacionados con neoplasias en centros públicos seleccionados, desde enero de 2000 a diciembre de 2010. METODOS: Se analizaron las historias clínicas de los pacientes fallecidos por cáncer en centros registrados en el Registro Oncopediátrico Hospitalario Argentino (ROHA) y en los registros individuales de los servicios de Hemato-Oncología. Se clasificaron las causas de mortalidad, la etapa en la cual se produjo el óbito y su relación con el tratamiento o con la patología de base. Se pesquisaron las causas de comorbilidad y las demoras en el diagnóstico y tratamiento. RESULTADOS: En 13 centros se analizó exitosamente un promedio >70...(AU)

INTRODUCTION: In Argentina, the mortality of pediatric malignant diseases occupies an important place causes have not yet been studied in the country. OBJECTIVE:To analyze mortality rates, causes and moment of death related to neoplasias in selected public centers from January 2000 until December 2010. METHODS: The analysis was conducted in clinical records of patients who died due to cancer. The cases were registered in the Argentine Hospital Oncopediatric Registry (ROHA)and by different registries belonging to hemato-oncological departments. Mortality causes were classified according to the phase of therapy when the event occurred and the relation shipof death with the treatment or underlying disease. Causes of comorbility and delays in diagnosis/treatment were also analyzed. RESULTS: In 13 centers, more than 70...(AU)

Análisis de la mortalidad en enfermedades Hemato-oncológicas malignas en pediatría en hospitales públicos de Argentina / Analysis of Mortality due to Hemato-Oncological Pediatrics Malignancies in Public Hospitals in Argentina

INTRODUCCIÓN: En Argentina, la mortalidad por enfermedades malignas en edad pediátrica ocupa un lugar relevante y sus causas todavía no han sido estudiadas en el país. OBJETIVO: Analizar las tasas, causas y etapas de los fallecimientos relacionados con neoplasias en centros públicos seleccionados, desde enero de 2000 a diciembre de 2010. MÉTODOS: Se analizaron las historias clínicas de los pacientes fallecidos por cáncer en centros registrados en el Registro Oncopediátrico Hospitalario Argentino (ROHA) y en los registros individuales de los servicios de Hemato-Oncología. Se clasificaron las causas de mortalidad, la etapa en la cual se produjo el óbito y su relación con el tratamiento o con la patología de base. Se pesquisaron las causas de comorbilidad y las demoras en el diagnóstico y tratamiento. RESULTADOS: En 13 centros se analizó exitosamente un promedio >70...

INTRODUCTION: In Argentina, the mortality of pediatric malignant diseases occupies an important place causes have not yet been studied in the country. OBJECTIVE:To analyze mortality rates, causes and moment of death related to neoplasias in selected public centers from January 2000 until December 2010. METHODS: The analysis was conducted in clinical records of patients who died due to cancer. The cases were registered in the Argentine Hospital Oncopediatric Registry (ROHA)and by different registries belonging to hemato-oncological departments. Mortality causes were classified according to the phase of therapy when the event occurred and the relation shipof death with the treatment or underlying disease. Causes of comorbility and delays in diagnosis/treatment were also analyzed. RESULTS: In 13 centers, more than 70...

Análisis de la mortalidad en enfermedades hemato-oncológicas malignas en pediatría: estudio multicéntrico en 14 hospitales públicos argentinos / Study of mortality due to hemato-oncological malignancies during childhood: multicenter study in 14 public hospitals in Argentina

INTRODUCCION: La mortalidad producida de forma temprana y durante diferentes fases del tratamiento de los niños con enfermedades hemato-oncológicas malignan es una de las causas de fracaso de los tratamientos actuales. Las tasas de mortalidad y sus causas no han sido estudiadas en Argentina.OBJETIVO: Analizar las tasas, causas y etapas de los fallecimientos relacionados con neoplasias en 14 centros públicos desde enero de 2000 a diciembre de 2010.METODOS: Se analizaron las historias clínicas de los pacientes fallecidos por cáncer en 14 centros identificados en el Registro Onco-Hematológico Argentino y en los registros individuales de los servicios de Hemato-Oncología. Se clasificaron las causas de muerte, la etapa en la cual se produjo el óbito y su relación con el tratamiento o con la patología de base. Se pesquisaron las causas de co-morbilidad y las demoras en el diagnóstico y tratamiento, así como las particularidades de cada centro.RESULTADOS: En 13 centros se analizó exitosamente un promedio >70% de los óbitos, y en el restante se obtuvieron datos del 35%. La tasa de mortalidad fue >40% en 3 centros, ≥30% en 7 y 30% de mortalidad en los primeros 5 años analizados, la tasa disminuyó a 20% de los pacientes fallecidos. La principal causa de muerte en las etapas tempranas del diagnóstico y tratamiento correspondió a infecciones severas.CONCLUSIONES: Es indispensable desarrollar políticas de salud para revertir la elevada mortalidad en niños con cáncer en Argentina. Para ello, se deben aplicar programas que incluyan reformas estructurales, formación de recursos humanos y equipamiento, además de un trabajo colaborativo continuo y eficiente.

INTRODUCTION:The early mortality occurred at different stages of the treatment agains hemato-oncological malignancies during childhood is one of the causes of failure of modern therapies. In Argentina, the mortality rates and their causes have not been studied yet.OBJECTIVE: To analyze mortality rates, causes and moment of death in children with malignant diseases in 14 public centers from January 2000 until December 2010.METHODS: The analysis was conducted in clinical records of patients who died due to malignant diseases in 14 centers. The cases were identified by the national register for hematology and oncology diseases (ROHA) and by different registers belonging to hemato-oncological departments. Causes were classified according to the phase of therapy when the event occurred and the relationship of death with the treatment or underlying disease. Causes of co-morbility, delays in diagnosis/during treatment and particular features of the centers were also analyzed.RESULTS: In 13 centers, more than 70% of the deaths were successfully analyzed, while there was information of 35% for the remaining one. The mortality rate was >40% in 3 centers, ≥30% in 7 and 30% of mortality during the first 5 years of the analyzed period, the rate decreased to <25% as a hemato-oncologist joined the medical staff. The access to information was difficult in all the centers. In 5 of them there were delays during the diagnostic evaluation and treatment in about 20% of the dead patients. The main cause of death during early phases of diagnosis and treatment was related to severe infectious complications.CONCLUSIONS: It is fundamental to develop appropriate health policies in order to decrease mortality rates among children with cancer in Argentina. They should include programs for structural reform, human resource development and equipment supply, as well as continuous cooperative work.

Análisis de la mortalidad en enfermedades hemato-oncológicas malignas en pediatría: estudio multicéntrico en 14 hospitales públicos argentinos / Study of mortality due to hemato-oncological malignancies during childhood: multicenter study in 14 public hospitals in Argentina

INTRODUCCION: La mortalidad producida de forma temprana y durante diferentes fases del tratamiento de los niños con enfermedades hemato-oncológicas malignan es una de las causas de fracaso de los tratamientos actuales. Las tasas de mortalidad y sus causas no han sido estudiadas en Argentina.OBJETIVO: Analizar las tasas, causas y etapas de los fallecimientos relacionados con neoplasias en 14 centros públicos desde enero de 2000 a diciembre de 2010.METODOS: Se analizaron las historias clínicas de los pacientes fallecidos por cáncer en 14 centros identificados en el Registro Onco-Hematológico Argentino y en los registros individuales de los servicios de Hemato-Oncología. Se clasificaron las causas de muerte, la etapa en la cual se produjo el óbito y su relación con el tratamiento o con la patología de base. Se pesquisaron las causas de co-morbilidad y las demoras en el diagnóstico y tratamiento, así como las particularidades de cada centro.RESULTADOS: En 13 centros se analizó exitosamente un promedio >70% de los óbitos, y en el restante se obtuvieron datos del 35%. La tasa de mortalidad fue >40% en 3 centros, ≥30% en 7 y <25% en 4. En un centro con >30% de mortalidad en los primeros 5 años analizados, la tasa disminuyó a <25% con la incorporación de una hemato-oncóloga. El acceso a los datos fue dificultoso en todos los centros. En 5 de ellos, hubo demoras en el diagnóstico y/o tratamiento en >20% de los pacientes fallecidos. La principal causa de muerte en las etapas tempranas del diagnóstico y tratamiento correspondió a infecciones severas.CONCLUSIONES: Es indispensable desarrollar políticas de salud para revertir la elevada mortalidad en niños con cáncer en Argentina. Para ello, se deben aplicar programas que incluyan reformas estructurales, formación de recursos humanos y equipamiento, además de un trabajo colaborativo continuo y eficiente.

INTRODUCTION:The early mortality occurred at different stages of the treatment agains hemato-oncological malignancies during childhood is one of the causes of failure of modern therapies. In Argentina, the mortality rates and their causes have not been studied yet.OBJECTIVE: To analyze mortality rates, causes and moment of death in children with malignant diseases in 14 public centers from January 2000 until December 2010.METHODS: The analysis was conducted in clinical records of patients who died due to malignant diseases in 14 centers. The cases were identified by the national register for hematology and oncology diseases (ROHA) and by different registers belonging to hemato-oncological departments. Causes were classified according to the phase of therapy when the event occurred and the relationship of death with the treatment or underlying disease. Causes of co-morbility, delays in diagnosis/during treatment and particular features of the centers were also analyzed.RESULTS: In 13 centers, more than 70% of the deaths were successfully analyzed, while there was information of 35% for the remaining one. The mortality rate was >40% in 3 centers, ≥30% in 7 and <25% in 4. At a center which had >30% of mortality during the first 5 years of the analyzed period, the rate decreased to <25% as a hemato-oncologist joined the medical staff. The access to information was difficult in all the centers. In 5 of them there were delays during the diagnostic evaluation and treatment in about 20% of the dead patients. The main cause of death during early phases of diagnosis and treatment was related to severe infectious complications.CONCLUSIONS: It is fundamental to develop appropriate health policies in order to decrease mortality rates among children with cancer in Argentina. They should include programs for structural reform, human resource development and equipment supply, as well as continuous cooperative work.