Impacto económico de emicizumab en pacientes con hemofilia a con inhibidor en un hospital de tercer nivel / Economic impact of emicizumab in patients with haemophilia a with inhibitor in a third level hospital

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)

Evaluación de la utilidad del coeficiente de difusión aparente en resonancia magnética para la diferenciación del grado tumoral de los tumores cerebrales pediátricos / Evaluating the apparent diffusion coefficient in MRI studies as a means of determining paediatric brain tumour stages

Introducción: El coeficiente de difusión aparente (ADC) de la resonancia magnética parece relacionarse con el grado de celularidad de los tumores de sistema nervioso central. Su utilidad para diferenciar el grado tumoral y tipo histológico de los tumores es controvertido. Material y métodos: Estudio retrospectivo de los pacientes pediátricos con diagnóstico de tumor de sistema nervioso central desde enero-2008 a diciembre-2013. Se revisan edad, sexo, localización del tumor y anatomía patológica. Las medidas de ADC y ratio ADC (cociente ADC tumoral/ADC tejido sano) se llevaron a cabo por 2 neurorradiólogos expertos, ciegos al diagnóstico histológico. Se calcula el valor ADC y el ratio ADC y se comparan sus valores con los diagnósticos anatomopatológicos. Resultados: Se incluyen 55 pacientes. La mediana de edad fue 6 años. Los diagnósticos anatomopatológicos fueron: astrocitoma pilocítico (40%), ependimoma anaplásico (16,4%), ganglioglioma (10,9%), glioblastoma (7,3%), meduloblastoma (5,5%), y otros (20%). El 64% fueron de bajo grado (BG) y el 36% de alto grado (AG). La media de ADC fue 1,3 en los de BG y 0,9 en los de AG (p = 0,004). La media de ratio ADC fue de 1,5 y 1,2 (p = 0,025) respectivamente. No hubo diferencias significativas en el ADC/ratio ADC entre los distintos tipos histológicos. Conclusiones: El ADC y ratio ADC son una herramienta útil en la diferenciación por imagen del grado tumoral en los tumores cerebrales pediátricos, sin sustituir a la anatomía patológica

Background: The apparent diffusion coefficient (ADC) in MRI seems to be related to cellularity in brain tumours. Its utility as a tool for distinguishing between histological types and tumour stages remains controversial. Procedures: We retrospectively evaluated children diagnosed with CNS tumours between January 2008 and December 2013. Data collected were age, sex, histological diagnosis, and location of the tumour. We evaluated the ADC and ADC ratio and correlated those values with histological diagnoses. Results: The study included 55 patients with a median age of 6 years. Histological diagnoses were pilocytic astrocytoma (40%), anaplastic ependymoma (16.4%), ganglioglioma (10.9%), glioblastoma (7.3%), medulloblastoma (5.5%), and other (20%). Tumours could also be classified as low-grade (64%) or high-grade (36%). Mean ADC was 1.3 for low-grade tumours and 0.9 for high-grade tumours (p = .004). Mean ADC ratios were 1.5 and 1.2 for low and high-grade tumours respectively (p = .025). There were no significant differences in ADC/ADC ratio between different histological types. Conclusion: ADC and ADC ratio may be useful in imaging-study based differential diagnosis of low and high-grade tumours, but they are not a substitute for an anatomical pathology study

Evaluating the apparent diffusion coefficient in MRI studies as a means of determining paediatric brain tumour stages. / Evaluación de la utilidad del coeficiente de difusión aparente en resonancia magnética para la diferenciación del grado tumoral de los tumores cerebrales pediátricos.

BACKGROUND: The apparent diffusion coefficient (ADC) in MRI seems to be related to cellularity in brain tumours. Its utility as a tool for distinguishing between histological types and tumour stages remains controversial. PROCEDURES: We retrospectively evaluated children diagnosed with CNS tumours between January 2008 and December 2013. Data collected were age, sex, histological diagnosis, and location of the tumour. We evaluated the ADC and ADC ratio and correlated those values with histological diagnoses. RESULTS: The study included 55 patients with a median age of 6 years. Histological diagnoses were pilocytic astrocytoma (40%), anaplastic ependymoma (16.4%), ganglioglioma (10.9%), glioblastoma (7.3%), medulloblastoma (5.5%), and other (20%). Tumours could also be classified as low-grade (64%) or high-grade (36%). Mean ADC was 1.3 for low-grade tumours and 0.9 for high-grade tumours (p=.004). Mean ADC ratios were 1.5 and 1.2 for low and high-grade tumours respectively (p=.025). There were no significant differences in ADC/ADC ratio between different histological types. CONCLUSION: ADC and ADC ratio may be useful in imaging-study based differential diagnosis of low and high-grade tumours, but they are not a substitute for an anatomical pathology study.

Adopción internacional de Etiopía en un período de 5 años / International adoption from Ethiopia in a 5-year period

INTRODUCCIÓN: La adopción internacional (AI) en nuestro país es un fenómeno reciente, siendo Etiopía uno de los países de origen más frecuentes. El objetivo principal del estudio es determinar la prevalencia de enfermedades infecciosas en los niños adoptados procedentes de Etiopía y valorar su estado nutricional. MÉTODO: Estudio de cohortes prospectivo, observacional. Se evalúa una cohorte de 251 niños adoptados en España procedentes de Etiopía (período del 1 de enero del 2006 y al 31 de diciembre del 2010). RESULTADOS: La edad media fue de 7 meses (90% de ellos menores de 5 años). Se detectó alguna anomalía en la exploración física en el 56,6%. La mitad de la muestra presentó un peso por debajo del tercer percentil, encontrándose algún grado de malnutrición en el 65% de los niños. Desde el punto de vista infeccioso, el hallazgo más frecuente fue la afectación cutánea. El 15,1% de los pacientes presentaba diarrea en el momento de la exploración. En las pruebas analíticas destacan: leucocitosis > 15.000/μL en el 8,8% e hipertransaminasemia en el 29% de los pacientes. La serología de VIH fue positiva en el 4,8% de los casos, mientras que la PCR-VIH fue positiva en el 0,8%. CONCLUSIONES: Predomina la adopción en los 5 primeros años de la vida. El bajo peso fue el hallazgo más frecuente en esta muestra. Las enfermedades infecciosas deberían ser sistemáticamente descartadas, incluso con la utilización de PCR-VIH dado que la exposición al virus no es infrecuente

INTRODUCTION: An increase in the number of internationally adopted children has been observed in the last few years. The country of origin that has experienced a greater increase is Ethiopia. The health of internationally adopted children from Ethiopia has not been extensively assessed to date. The main objective of the study is to determine the prevalence of infectious diseases in children adopted from Ethiopia, and to assess their nutritional status. METHOD: A prospective, observational cohort study was conducted using the medical records of 251 children adopted from Ethiopia to Spain in the period from Jan 1, 2006 and December 31, 2010. RESULTS: The mean age of the children was 7 months (range 1-120). Abnormalities were detected on physical examination in 56.6%. In 90% of cases the child was less than 5 years-old. Half of the sample had a weight below the third percentile, with some degree of malnutrition in 65% of the children. HIV exposure was not uncommon (4.8%). CONCLUSIONS: Low weight and acute gastroenteritis were the main findings in this cohort. Infectious diseases should be systematically assessed

Anemia hemolítica tardía secundaria al tratamiento con artesunato por vía intravenosa / Delayed haemolytic anemia due to intravenous artesunate

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Infección por virus respiratorio sincitial en los pacientes inmunodeprimidos en un hospital pediátrico: experiencia de 5 años / Respiratory syncitial virus in immunocompromised patients in a pediatric hospital: 5 years experience

INTRODUCCIÓN: La infección por virus respiratorio sincitial (VRS) causa importante morbimortalidad en pacientes inmunodeprimidos. MÉTODOS: Estudio descriptivo en un hospital pediátrico de los casos de infección por VRS en pacientes inmunodeprimidos de las unidades de Hemato-Oncología e Inmunodeficiencias en el periodo 2008-2012. RESULTADOS: Se diagnosticaron 19 casos de infección por VRS. Nueve pacientes requirieron ingreso, 2 en Unidad de Cuidados Intensivos Pediátrica. Cinco pacientes precisaron tratamiento con ribavirina y/o palivizumab. No se produjeron fallecimientos. CONCLUSIÓN: La infección por VRS es potencialmente grave en los pacientes pediátricos inmunodeprimidos

INTRODUCTION: Respiratory syncytial virus (RSV) infection is associated with an increase in morbidity and mortality in immunocompromised hosts. METHODS: A description is presented of all cases of RSV infection in immunocompromised pediatric patients in Hematology and Oncology and Immunodeficiency Units between 2008 and 2012. RESULTS: Nineteen patients were diagnosed with RSV infection. Nine patients required in-patient care and 2 required Pediatric Intensive Care Unit. Five patients were treated with specific therapy (ribavirin ± palivizumab). No deaths occurred in the study period. CONCLUSION: RSV infection may be severe in immunocompromised pediatric patients

[International adoption from Ethiopia in a 5-year period]. / Adopción internacional de Etiopía en un período de 5 años.

INTRODUCTION: An increase in the number of internationally adopted children has been observed in the last few years. The country of origin that has experienced a greater increase is Ethiopia. The health of internationally adopted children from Ethiopia has not been extensively assessed to date. The main objective of the study is to determine the prevalence of infectious diseases in children adopted from Ethiopia, and to assess their nutritional status. METHOD: A prospective, observational cohort study was conducted using the medical records of 251 children adopted from Ethiopia to Spain in the period from Jan 1, 2006 and December 31, 2010. RESULTS: The mean age of the children was 7 months (range 1-120). Abnormalities were detected on physical examination in 56.6%. In 90% of cases the child was less than 5 years-old. Half of the sample had a weight below the third percentile, with some degree of malnutrition in 65% of the children. HIV exposure was not uncommon (4.8%). CONCLUSIONS: Low weight and acute gastroenteritis were the main findings in this cohort. Infectious diseases should be systematically assessed.

[Respiratory syncitial virus in immunocompromised patients in a pediatric hospital: 5 years experience]. / Infección por virus respiratorio sincitial en los pacientes inmunodeprimidos en un hospital pediátrico: experiencia de 5 años.

INTRODUCTION: Respiratory syncytial virus (RSV) infection is associated with an increase in morbidity and mortality in immunocompromised hosts. METHODS: A description is presented of all cases of RSV infection in immunocompromised pediatric patients in Hematology and Oncology and Immunodeficiency Units between 2008 and 2012. RESULTS: Nineteen patients were diagnosed with RSV infection. Nine patients required in-patient care and 2 required Pediatric Intensive Care Unit. Five patients were treated with specific therapy (ribavirin ± palivizumab). No deaths occurred in the study period. CONCLUSION: RSV infection may be severe in immunocompromised pediatric patients.

Hemophagocytic lymphohistiocytosis secondary to Epstein Barr virus and Leishmania co-infection in a toddler.

This is the report of an EBV+Leishmanial co-infection. The patient developed hemophagocytic syndrome (HLH) and was treated with the standard HLH-2004 protocol. However, PCR in bone marrow discovered this secondary cause for HLH. In endemic countries, visceral leishmaniasis should be considered in the differential diagnosis even in EBV-related HLH, as chemotherapy toxicity may be avoided.

Bradicardia sinusal secundaria al uso de corticoides en bolo / Sinus bradycardia secondary to the use of pulse corticosteroids

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Citopenias autoinmunes: tratamiento según el diagnóstico / Autoimmune cytopenias: Diagnosis Before Treatment

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