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.

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.

[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.

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.

[Musculoskeletal pain: a common initial sign of acute lymphoblastic leukaemia]. / Dolor musculoesquelético: una forma de inicio frecuente de leucemia linfoblástica aguda.

Musculoskeletal pain is a common complaint in paediatrics usually due to benign diseases. Nevertheless neoplasms, particularly acute leukaemia, must be considered in the differential diagnosis. During the last 9 months 4 of the 9 patients diagnosed with acute leukaemia at our hospital presented with a limp, arthralgias, lumbar or bony pain. We describe these cases and review the clinical and analytical parameters that help to differentiate benign pain from that associated with a malignant disease. The early detection of these processes may represent a significant improvement in their prognosis.

[Antipyretic effectiveness of ibuprofen and paracetamol]. / Eficacia de ibuprofeno y paracetamol como antitérmicos.

OBJECTIVE: To compare the antipyretic effectiveness of ibuprofen and paracetamol and to evaluate the possible influence of patients' sex, weight, height and underlying disease on effectiveness. PATIENTS AND METHODS: A total of 166 children with fever, defined as a temperature equal to or above 38 degrees C, were enrolled. Of these, 80 were given paracetamol at a dose of 15 mg per kg and 86 were given 7 mg of ibuprofen per kg. Temperature was recorded at 60, 120,180 and 240 minutes after drug administration. Data were statistically analyzed, including analysis of paired data. RESULTS: Ninety percent of the children became afebrile at some time during the study with both paracetamol and ibuprofen. Seventy-four percent of the patients remained afebrile 4 hours after drug administration. The mean temperatures obtained with ibuprofen versus paracetamol were 37.66 +/- 0.73 vs 37.8 +/- 0.65, p = 0.22 one hour after drug administration; 37.09 +/- 0.83 vs 37.29 +/- 0.71, p = 0.14 two hours after drug administration; 37.12 +/- 1.05 vs 37.28 +/- 0.87, p = 0.64 three hours after drug administration; and 37.40 +/- 1.12 vs 37.46 +/- 1.00, p = 0.72 four hours after drug administration. The maximum rate of temperature decrease was achieved during the first 60 minutes after drug administration (-1.32 +- 0.83 with ibuprofen vs -1.09 +/- 0.77 with paracetamol, p = 0.10). In children aged between 5 and 12 years, ibuprofen achieved significantly lower temperatures than paracetamol (38.00 +/- 0.65 vs 37.45 +/- 0.43, p = 0.02 at 1 hour; 36.71 +/- 0.66 vs 37.60 +/- 0.93, p = 0.01 at 2 hours; 36.80 +/- 0.79 vs 37.67 +/- 1.12, p = 0.03 at 3 hours). Analysis by weight, height or underlying disease revealed no significant differences. CONCLUSIONS: Both ibuprofen and paracetamol proved to be successful in reducing temperature. The effectiveness of ibuprofen and paracetamol was similar, except in children aged more than 5 years old, in whom ibuprofen was more effective. Weight, sex and underlying disease had no influence on effectiveness.