Rapamicina oral: una alternativa en niños con anomalías vasculares complicadas / Oral rapamycin: an alternative in children with complicated vascular abnormalities

Objetivos: El uso del inhibidor mTOR sirolimus ha supuesto unavance en el tratamiento de pacientes con anomalías vasculares complicadas. El objetivo de este estudio es presentar nuestra serie de pacientespediátricos con anomalías vasculares tratados con sirolimus oral y haceruna revisión de la literatura al respecto. Material y métodos: Se realizó un análisis retrospectivo de lospacientes con anomalías vasculares complicadas tratados con sirolimusoral en nuestro centro desde el año 2016. La dosis inicial utilizada fuede 0,8 mg/m2 cada 12 horas y el rango terapéutico de 5-15 ng/ml. Todos los pacientes recibieron profilaxis con trimetoprim-sulfametoxazol. Resultados: Se incluyeron seis niños, tres varones y tres mujeres, con una edad media al inicio del tratamiento de 9,5 años. Trespresentaban una malformación linfática en cabeza y cuello, dos unamalformación venosa en miembro inferior y la última una malformación combinada linfática-venosa a nivel toracoabdominal. Todos habíanrecibido múltiples tratamientos previos sin mejoría. Tras el inicio desirolimus, cinco pacientes mejoraron clínicamente (tiempo medio 3,6meses) y cuatro radiológicamente (tiempo medio 6,6 meses). Se registraron efectos adversos leves y transitorios en tres casos. Actualmente,cinco pacientes continúan con el tratamiento. Conclusiones: El sirolimus oral es un tratamiento eficaz y seguroen pacientes con anomalías vasculares complicadas. Nuestros resultadosapoyan su uso en malformaciones linfáticas y venosas en las que hanfracasado otros tratamientos, presentando buenas respuestas sintomáticasy, en menor medida, radiológicas.(AU)

Objective: Sirolimus mTOR inhibitor represents a major advancein the treatment of patients with complicated vascular abnormalities.The objective of this study was to present our series of pediatric patientswith vascular abnormalities treated with oral sirolimus, and to conducta review of the relevant literature. Materials and methods: A retrospective analysis of patients withcomplicated vascular abnormalities treated with oral sirolimus in ourhealthcare facility from 2016 was carried out. Initial dosage was 0.8 mg/m 2 every 12 hours, and therapeutic range was 5-15 ng/ml. All patientsreceived trimethoprim-sulfamethoxazole prophylaxis. Results: 6 children –3 boys and 3 girls– with a mean age of 9.5years at treatment initiation were included. 3 of them had head and necklymphatic malformation, 2 had lower limb venous malformation, and 1had combined lymphatic-venous malformation at the thoracoabdominal level. They all had received multiple previous treatments withoutimprovement. Following sirolimus initiation, 5 patients had clinicalimprovement (mean time: 3.6 months) and 4 had radiological improvement (mean time: 6.6 months). Mild and transitory adverse effects werenoted in the 3 cases. Today, 5 patients remain under treatment. Conclusions: Oral sirolimus is an effective and safe treatment inpatients with complicated vascular abnormalities. Our results supportsirolimus use in lymphatic and venous malformations in which previoustreatments have failed, with a good symptomatic and, to a lesser extent,radiological response.(AU)

Practical management of pulmonary nodules in the most common pediatric tumors. / Manejo práctico de los nódulos pulmonares en las neoplasias pediátricas más frecuentes.

In pediatric patients with sarcomas, hepatoblastomas, or other types of primary tumors, lung metastases are often found at diagnosis or during follow-up. The wide variety of primary tumors and clinical situations makes management and follow-up of these patients challenging. Chest CT is the best way to detect the dissemination of disease to the lungs. Many pulmonary nodules are nonspecific, and many might not be pathological. Others have characteristics that make them suspicious. Although there are some general features that indicate that a pulmonary nodule is likely to be a metastasis, sometimes the meaning of these features depends on the primary tumor. Furthermore, metastases can develop during the course of the disease, and the protocols for follow-up are different for different primary tumors. We review the different protocols used at our hospital for the primary tumors that most often metastasize to the lungs, including the criteria for lung metastases and the follow-up for each primary tumor.

Oral rapamycin: an alternative in children with complicated vascular abnormalities. / Rapamicina oral: una alternativa en niños con anomalías vasculares complicadas.

OBJECTIVE: Sirolimus mTOR inhibitor represents a major advance in the treatment of patients with complicated vascular abnormalities. The objective of this study was to present our series of pediatric patients with vascular abnormalities treated with oral sirolimus, and to conduct a review of the relevant literature. MATERIAL AND METHODS: A retrospective analysis of patients with complicated vascular abnormalities treated with oral sirolimus in our healthcare facility from 2016 was carried out. Initial dosage was 0.8 mg/m2 every 12 hours, and therapeutic range was 5-15 ng/ml. All patients received trimethoprim-sulfamethoxazole prophylaxis. RESULTS: 6 children -3 boys and 3 girls- with a mean age of 9.5 years at treatment initiation were included. 3 of them had head and neck lymphatic malformation, 2 had lower limb venous malformation, and 1 had combined lymphatic-venous malformation at the thoracoabdominal level. They all had received multiple previous treatments without improvement. Following sirolimus initiation, 5 patients had clinical improvement (mean time: 3.6 months) and 4 had radiological improvement (mean time: 6.6 months). Mild and transitory adverse effects were noted in the 3 cases. Today, 5 patients remain under treatment. CONCLUSIONS: Oral sirolimus is an effective and safe treatment in patients with complicated vascular abnormalities. Our results support sirolimus use in lymphatic and venous malformations in which previous treatments have failed, with a good symptomatic and, to a lesser extent, radiological response.

OBJETIVOS: El uso del inhibidor mTOR sirolimus ha supuesto un avance en el tratamiento de pacientes con anomalías vasculares complicadas. El objetivo de este estudio es presentar nuestra serie de pacientes pediátricos con anomalías vasculares tratados con sirolimus oral y hacer una revisión de la literatura al respecto. MATERIAL Y METODOS: Se realizó un análisis retrospectivo de los pacientes con anomalías vasculares complicadas tratados con sirolimus oral en nuestro centro desde el año 2016. La dosis inicial utilizada fue de 0,8 mg/m2 cada 12 horas y el rango terapéutico de 5-15 ng/ml. Todos los pacientes recibieron profilaxis con trimetoprim-sulfametoxazol. RESULTADOS: Se incluyeron seis niños, tres varones y tres mujeres, con una edad media al inicio del tratamiento de 9,5 años. Tres presentaban una malformación linfática en cabeza y cuello, dos una malformación venosa en miembro inferior y la última una malformación combinada linfática-venosa a nivel toracoabdominal. Todos habían recibido múltiples tratamientos previos sin mejoría. Tras el inicio de sirolimus, cinco pacientes mejoraron clínicamente (tiempo medio 3,6 meses) y cuatro radiológicamente (tiempo medio 6,6 meses). Se registraron efectos adversos leves y transitorios en tres casos. Actualmente, cinco pacientes continúan con el tratamiento. CONCLUSIONES: El sirolimus oral es un tratamiento eficaz y seguro en pacientes con anomalías vasculares complicadas. Nuestros resultados apoyan su uso en malformaciones linfáticas y venosas en las que han fracasado otros tratamientos, presentando buenas respuestas sintomáticas y, en menor medida, radiológicas.

Rapamicina oral: una alternativa en niños con anomalías vasculares complicadas / Oral rapamycin: an alternative in children with complicated vascular abnormalities

OBJETIVOS: El uso del inhibidor mTOR sirolimus ha supuesto un avance en el tratamiento de pacientes con anomalías vasculares complicadas. El objetivo de este estudio es presentar nuestra serie de pacientes pediátricos con anomalías vasculares tratados con sirolimus oral y hacer una revisión de la literatura al respecto. MATERIAL Y MÉTODOS: Se realizó un análisis retrospectivo de los pacientes con anomalías vasculares complicadas tratados con sirolimus oral en nuestro centro desde el año 2016. La dosis inicial utilizada fue de 0,8 mg/m2 cada 12 horas y el rango terapéutico de 5-15 ng/ml. Todos los pacientes recibieron profilaxis con trimetoprim-sulfametoxazol. RESULTADOS: Se incluyeron seis niños, tres varones y tres mujeres, con una edad media al inicio del tratamiento de 9,5 años. Tres presentaban una malformación linfática en cabeza y cuello, dos una malformación venosa en miembro inferior y la última una malformación combinada linfática-venosa a nivel toracoabdominal. Todos habían recibido múltiples tratamientos previos sin mejoría. Tras el inicio de sirolimus, cinco pacientes mejoraron clínicamente (tiempo medio 3,6 meses) y cuatro radiológicamente (tiempo medio 6,6 meses). Se registraron efectos adversos leves y transitorios en tres casos. Actualmente, cinco pacientes continúan con el tratamiento. CONCLUSIONES: El sirolimus oral es un tratamiento eficaz y seguro en pacientes con anomalías vasculares complicadas. Nuestros resultados apoyan su uso en malformaciones linfáticas y venosas en las que han fracasado otros tratamientos, presentando buenas respuestas sintomáticas y, en menor medida, radiológicas

OBJECTIVE: Sirolimus mTOR inhibitor represents a major advance in the treatment of patients with complicated vascular abnormalities. The objective of this study was to present our series of pediatric patients with vascular abnormalities treated with oral sirolimus, and to conduct a review of the relevant literature. MATERIALS AND METHODS: A retrospective analysis of patients with complicated vascular abnormalities treated with oral sirolimus in our healthcare facility from 2016 was carried out. Initial dosage was 0.8 mg/m2 every 12 hours, and therapeutic range was 5-15 ng/ml. All patients received trimethoprim-sulfamethoxazole prophylaxis. RESULTS: 6 children -3 boys and 3 girls- with a mean age of 9.5 years at treatment initiation were included. 3 of them had head and neck lymphatic malformation, 2 had lower limb venous malformation, and 1 had combined lymphatic-venous malformation at the thoracoabdominal level. They all had received multiple previous treatments without improvement. Following sirolimus initiation, 5 patients had clinical improvement (mean time: 3.6 months) and 4 had radiological improve-ment (mean time: 6.6 months). Mild and transitory adverse effects were noted in the 3 cases. Today, 5 patients remain under treatment. CONCLUSIONS: Oral sirolimus is an effective and safe treatment in patients with complicated vascular abnormalities. Our results support sirolimus use in lymphatic and venous malformations in which previous treatments have failed, with a good symptomatic and, to a lesser extent, radiological response

Unidad de Hemato-Oncología Infantil del Hospital Universitario 12 de Octubre / Pediatric Hemato-Oncology Unit of the University Hospital 12 de Octubre

La Unidad de Hemato-Oncología Pediátrica del Hospital 12 de Octubre fue inaugurada hace 35 años. Presentamos una breve descripción histórica de la Unidad, su cartera de servicios y recursos humanos y materiales en la actualidad. Exponemos brevemente la organización de la actividad asistencial, docente e investigadora (AU)

The Pediatric Hemato-Oncology Unit of the University Hospital 12 de Octubre was inaugurated 35 years ago. We are presenting a brief historical description of the unit, its service portfolio and human and material resources at present. We briefly describe the organization of the care, teaching and investigator activity (AU)

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

No disponible

Nutritional and Pharmacological Management during Chemotherapy in a Patient with Propionic Acidaemia and Rhabdomyosarcoma Botryoides.

We present the nutritional and pharmacological management of a 2-year-old girl with a severe form of propionic acidaemia and a genitourinary embryonal rhabdomyosarcoma. This association has not been described before, nor the utilization of chemotherapy in patients with propionic acidaemia.The patient is a girl with neonatal onset of propionic acidaemia, homozygous for the c.2041-2924del3889 mutation in PCCA gene. At 23 months of age she was diagnosed with genitourinary embryonal rhabdomyosarcoma. Conservative surgery, brachytherapy and nine cycles of chemotherapy with iphosphamide, vincristine and actinomycin were recommended by oncologists. Due to the possibility that the child could present decompensations, we elaborated three different courses of treatment: when the patient was stable (treatment 1), intermittent bolus feeding through gastrostomy, containing 70 kcal/kg/day and 1.4 g/kg/day of total protein (0.6 g/kg/day of natural protein and 0.8 g/kg/day of amino acid-based formula) was prescribed; on the chemotherapy-days (treatment 2), diet consisted on continuous feeding, with the same energy and amino acid-based formula but half of natural protein intake; in case of decompensation (treatment 3), we increased by 10% the energy intake, and completely stopped natural protein in the diet but maintaining the amino acid-based formula. On chemotherapy- days carnitine was increased from 100 mg/kg/day to 150 mg/kg/day, and N-carbamylglutamate was added.Through the 7 months with chemotherapy the patient did not suffer decompensations, while she maintained good nutritional status.Enteral continuous feeding by gastrostomy, amino acid-based formula, and preventive use of N-carbamylglutamate during chemotherapy-days are the principal measures we propose in these situations.

[Presence of parents in the emergency room during invasive procedures: do they prefer to be present?]. / Procedimientos invasivos en urgencias: ¿los familiares prefieren estar presentes?

INTRODUCTION: Parents are not usually present during procedures in the paediatric emergency room (ER), although an increasing number of them would like to. Our goal was to find out how parents felt about them being present in ER during procedures. MATERIAL AND METHODS: This is an observational study. Questionnaires were distributed among parents of patients in the ER during January and February 2007. Data included demographic questions as well as the opinion regarding their preference on being present during venipuncture, stitching, lumbar puncture or cardiopulmonary resuscitation. They were also asked about who should take the decision whether to allow the family to be present or not. RESULTS: A total of 98 questionnaires were analyzed. The median age of participants was 32 years-old, of which 84.5% would prefer to be present for venipuncture, 70.4% for stitching, 66.3% for lumbar puncture and 61.2% for cardiopulmonary resuscitation. Venipuncture was performed on 21% of the children, stitching on 4%, and lumbar puncture on 2%. There was no cardiopulmonary resuscitation. Parents considered that the decision about being present should be taken by the professional in 74% (venipuncture), 75% (stitching), 80% (lumbar puncture) and 81% (cardiopulmonary resuscitation). The mean age of the group that preferred to be present was lower (32 vs. 40 years; p = 0.039). CONCLUSIONS: Most parents surveyed preferred to be present during invasive procedures in ER. The more invasive the procedure is the higher reluctance from parents to be present. Most parents thought the decision should be taken by the health care professional, particularly when the procedure is more invasive.

Procedimientos invasivos en urgencias: ¿los familiares prefieren estar presentes? / Presence of parents in the emergency room during invasive procedures: do they prefer to be present?

Introducción: tradicionalmente no se ha permitido a los familiares de los pacientes permanecer con su niño durante los procedimientos invasivos en urgencias pediátricas. Se evalúa la opinión de los padres en cuanto a su preferencia de estar o no presentes. Material y métodos: se trata de un estudio observacional descriptivo. Se realizaron encuestas al azar a familiares de pacientes que fueron atendidos en nuestro servicio de urgencias en enero y febrero de 2007. Se preguntó acerca de su preferencia de estar o no durante las siguientes técnicas invasivas: venopunción, sutura de herida, punción lumbar y reanimación cardiopulmonar. Se preguntó también quién creía que debía tomar la decisión de que estuvieran o no presentes. Resultados: se realizó un total de 98 encuestas. La mediana de edad de los participantes fue 32 años. Preferían estar presentes en las técnicas de venopunción el 84,5% de los familiares; en sutura de la herida, el 70,4%; en punción lumbar, el 66,3%, y en reanimación cardiopulmonar, el 61,2%. Se realizó venopunción al 21% de los niños, sutura de herida al 4%, punción lumbar al 2%; no se realizó ninguna reanimación cardiopulmonar. Los encuestados respondieron que la decisión de estar presente debe ser tomada por el personal sanitario en un 74% en venopunción, el 75% en suturas, el 80% en punción lumbar y el 81% en reanimación cardiopulmonar. La media de edad del grupo que prefiere estar presente difiere significativamente de los que no (32 frente a 40 años). Conclusiones: la mayoría de los familiares encuestados preferirían estar presentes durante los procedimientos invasivos en urgencias pediátricas. A mayor invasividad, menor deseo de los padres de estar presentes. La mayoría de los familiares cree que la decisión de estar o no presente debe ser tomada por el personal sanitario, especialmente cuanto más invasivo es el procedimiento (AU)

Introduction: Parents are not usually present during procedures in the paediatric emergency room (ER), although an increasing number of them would like to. Our goal was to find out how parents felt about them being present in ER during procedures. Material and methods: This is an observational study. Questionnaires were distributed among parents of patients in the ER during January and February 2007. Data included demographic questions as well as the opinion regarding their preference on being present during venipuncture, stitching, lumbar puncture or cardiopulmonary resuscitation. They were also asked about who should take the decision whether to allow the family to be present or not. Results: A total of 98 questionnaires were analyzed. The median age of participants was 32 years-old, of which 84.5% would prefer to be present for venipuncture, 70.4% for stitching, 66.3% for lumbar puncture and 61.2% for cardiopulmonary resuscitation. Venipuncture was performed on 21% of the children, stitching on 4%, and lumbar puncture on 2%. There was no cardiopulmonary resuscitation. Parents considered that the decision about being present should be taken by the professional in 74% (venipuncture), 75% (stitching), 80% (lumbar puncture) and 81% (cardiopulmonary resuscitation). The mean age of the group that preferred to be present was lower (32 vs. 40 years; p=0.039). Conclusions: Most parents surveyed preferred to be present during invasive procedures in ER. The more invasive the procedure is the higher reluctance from parents to be present. Most parents thought the decision should be taken by the health care professional, particularly when the procedure is more invasive (AU)

Presencia familiar durante los procedimientos invasivos en urgencias de pediatría: opinión de los profesionales / The opinion of health care workers concerning the presence of relatives during invasive procedures in the pediatric emergency room

Introducción: Tradicionalmente no se ha permitido a los familiares permanecer junto a los niños durante la realización de procedimientos invasivos en los servicios de urgencias de pediatría, pero cada vez más quieren estar presentes. El objetivo de este estudio es conocer la opinión sobre este aspecto de los profesionales que trabajan en una sección de urgencias de pediatría. Material y métodos: Se trata de un estudio observacional descriptivo. Se distribuyeron encuestas anónimas al personal sanitario que trabaja en la sección de urgencias de pediatría entre los meses de enero y febrero de 2007. Se recogieron datos demográficos y profesionales, y se preguntó respecto a la influencia de la presencia familiar en los niños, los propios familiares y el personal sanitario y sobre quién debe tomar esa decisión. El análisis de los resultados se realizó con el paquete estadístico SPSS versión 13.0.Resultados: Se recogieron un total de 60 encuestas. El 62% de los profesionales considera que la presencia familiar es beneficiosa para el niño, el 26% que lo es para el personal sanitario y el 55%que beneficia a los familiares. El 55% de los encuestados opina que debe ser la persona que realiza el procedimiento quien decida si la familia puede estar o no presente. Si comparamos por grupos, hay variaciones según el escenario clínico por el que se pregunte (colocación de vía periférica, sutura de herida, punción lumbar y reanimación cardiopulmonar) y diferencias estadísticamente significativas según el personal sea médico o no (enfermeros y auxiliares).Conclusiones: Para el personal sanitario la presencia familiar es favorable, aunque menos cuanto más invasivo es el procedimiento realizado. Hay diferencias entre el personal médico y no médico, y es mucho más frecuente en el primer grupo la consideración de quela presencia familiar es positiva para los niños y que ésta debe producirse, así como que los padres deben participar en la decisión, mientras que el grupo de profesionales no médicos cree con más frecuencia que la decisión debe ser tomada por quien realiza el procedimiento invasivo. Estos datos deben contrastarse con las opiniones de los familiares para poder establecer una mejor relación entre el personal sanitario y los familiares de nuestros pacientes(AU)

Introduction: Relatives are not usually present during invasive procedures in the Pediatric Emergency Room (ER), although an increasing number of them would like to be. Our goal was to find out how the health care workers in the ER felt about the presence of relatives during invasive procedures. Material and methods: We carried out an observational study based on anonymous questionnaires distributed among the ER professionals during January and February 2007. The data included demographic and occupational questions, as well as their opinion regarding the influence of the presence of relatives during procedures on the children, the families and the health care workers. The data were analyzed using the SPSS 13.0 statistical software package. Results: Sixty questionnaires were analyzed. Sixty-two percent of the professionals considered that the presence of relatives has beneficial effects on children, 26% considered that it benefited the professionals and 55% that it benefited the relatives. Fifty-five percent of those surveyed considered that the professional should decide whether relatives can be present. When we compared the groups involved, we found differences depending on the clinical scenario (insertion of an intravenous line, wound suturing, lumbar puncture or cardiopulmonary resuscitation). There were also statistical differences when groups of health care workers were compared( physicians versus non-physicians). Conclusions: Health care workers consider beneficial the presence of relatives during procedures in the ER, although the benefit is lesser in more invasive procedures. The consideration of this presence is better among physicians than among non physicians. These data should be compared with the opinion of relatives(AU)