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Introduction: In recent years, there have been changes in the management of patients with primary immune thrombocytopenia. In this study, a review is presented of the characteristics and outcomes of children with primary immune thrombocytopenia in a children’s hospital (Hospital Infantil Niño Jesús). Moreover, an analysis is made of the changes in the care of these patients diagnosed before and after 2011, when new guidelines were published by the Spanish Society of Paediatric Haematology Oncology (SEHOP). Material and methods: Data from a cohort of primary immune thrombocytopenia patients followed up in this hospital have been retrospectively reviewed. The statistical package used for the analysis was SPSS Statistics 22.0 (IBM Corp., Chicago, IL, USA). Results: A review is presented on the clinical data from 235 paediatric patients diagnosed with primary immune thrombocytopenia. It was observed that some features at diagnosis, such as age younger than five years and a previous history of infection, influenced the probability of cure. Regarding the changes in the management of patients since 2011, the steroid doses received during the first month and the first year, and the number of days corresponding to the patient’s first admission have both significantly decreased. Splenectomies were also significantly reduced. Conclusions: Since 2011, there have been changes in the medical care of our primary immune thrombocytopenia patients: they receive lower doses of steroids, they stay fewer days in the hospital, and the number of splenectomies has decreased without increasing bleeding or worsening the clinical evolution. Furthermore, it was observed that age younger than 5 years and a history of infection prior to diagnosis were related to higher chances of recovery. Resumen: Introducción: En los últimos años se han experimentado cambios en el manejo de los pacientes con trombocitopenia inmune primaria. En este estudio se revisan las características de los pacientes con trombocitopenia inmune primaria del Hospital Infantil Universitario Niño Jesús y su evolución. Además, analizamos los cambios en el abordaje de los pacientes diagnosticados antes y después del 2011, año en el que se publicó la guía de la Sociedad Española de Pediatría. Material y métodos: Se han revisado retrospectivamente los datos de pacientes con trombocitopenia inmune primaria en seguimiento en nuestro hospital desde el año 2000. El paquete estadístico utilizado para el análisis fue SPSS Statistics 22.0 (IBM Corp, Chicago, IL, EE.UU.). Resultados: Se han revisado 235 pacientes pediátricos con trombocitopenia inmune primaria observando que algunas características al diagnóstico, como la edad menor de 5 años y los antecedentes previos de infección, pueden influir en la probabilidad de recuperación. Con respecto al cambio de manejo de los pacientes, a partir de 2011 las dosis de esteroides recibidas durante el primer mes y el primer año se han reducido de forma significativa, así como el número de días del primer ingreso pasando de 5 a 3 días. Las esplenectomías también se han reducido significativamente. Conclusiones: Desde el año 2011 se han producido cambios en el abordaje de nuestros pacientes: reciben una menor dosis de esteroides, permanecen menos días ingresados y se ha reducido el número de esplenectomías sin aumentar los sangrados y sin disminuir la tasa de respuestas. Además, observamos que la edad menor de 5 años y el antecedente de infección previa al diagnóstico están relacionados con una mayor tasa de recuperación.

Classically, several variables have been related to the disease course of chronic primary immune thrombocytopenia (cITP), though to date, there is no consensus on their clinical relevance. In a recent systematic review, a meta-analysis was made and confirmed the existence of certain cITP-related variables that may be related to prognosis in pediatric patients. We retrospectively analyzed a cohort of patients diagnosed with ITP, identified prognostic variables, and compared our results to the variables described by the authors. A multivariate study revealed that older age at diagnosis and higher platelet count were the only independent variables related to cITP. Children up to age 4 years and those with lower platelet counts (below 20 × 109/L) were at lower risk for cITP.Conclusion: We therefore concluded that only age and platelet count at diagnosis are independent variables that should be considered when evaluating the risk of developing cITP. What is Known: • Around 20% of patients with immune thrombocytopenia progress to chronic disease as determined by a sustained platelet count below 100×109/L for more than 12 months. • A number of variables potentially related to the development of cITP are being studied, such as age, sex, cell count, and previous treatment. What is New: • This is a new group of patients diagnosed with ITP in which the platelet count and age at diagnosis are the only independent variables closely related to cITP. • In this new series, we could not confirm other variables previously related to cITP such as total leukocyte count or the absence of treatment at diagnosis.

PURPOSE: We investigated whether lung ultrasound (US) performed in primary care is useful and feasible for diagnosing community-acquired pneumonia (CAP) compared with chest radiography, as most previous research has been conducted in hospital settings. METHODS: We undertook a prospective observational cohort study of lung US performed in 12 primary care centers. Patients aged 5 years and older with symptoms suggesting CAP were examined with lung US (by 21 family physicians and 7 primary care pediatricians) and chest radiograph on the same day. We compared lung US findings with the radiologist’s chest radiograph report as the reference standard, given that the latter is the most common imaging test performed for suspected CAP in primary care. The physicians had varied previous US experience, but all received a 5-hour lung US training program. RESULTS: The study included 82 patients. Compared with chest radiography, positive lung US findings (consolidation measuring >1 cm or a focal/asymmetrical B-lines pattern) showed a sensitivity of 87.8%, a specificity of 58.5%, a positive likelihood-ratio of 2.12, and a negative likelihood-ratio of 0.21. Findings were similar regardless of the physicians’ previous US training or experience. We propose a practical algorithm whereby patients having consolidation measuring greater than 1 cm or normal findings on lung US could skip chest radiography, whereas patients with a B-lines pattern without consolidation (given its low specificity) would need chest radiography to ensure appropriate management. Lung US was generally performed in 10 minutes or less. CONCLUSION: Point-of-care lung US in primary care could be useful for investigating suspected CAP (avoiding chest radiography in most cases) and is likely feasible in daily practice, as short training programs appear sufficient and little time is needed to perform the scan.

Several genetic diseases, generally considered as congenital diseases, are characterized by bone marrow failure during early childhood. Hematopoietic stem cell transplantation is the only curative treatment for syndromes involving bone marrow failure and thalassemia. In this slate-of-the-art review, we wish to focus on the results of hematopoietic transplantation in treating some of these diseases, with a special emphasis on congenital bone marrow failure and thalassemia. The results of this procedure have improved over the previous years, mainly when performed by experienced teams. New conditioning regimes based on fludarabine and the use of HLA-identical donors have been related with better survivals. In the previous years, donors other than HLA-identical siblings have been increasingly used in patients not responding to conventional measures, but this approach needs to be evaluated in larger studies.

Peripheral blood progenitor cells (PBPC) are increasingly used as a source of stem cells for either autologous or allogeneic hematopoietic transplantation in children. Although technically similar to adult procedures, apheresis in small children worries some operators and physicians that have little experience in managing pediatric patients or donors. However several published series have showed that these procedures, when performed by experienced teams are safe and can obtain enough amount of PBPC for either autologous or allogeneic hematopoietic transplantation. Some technical aspects must be considered in pediatric apheresis due to the size of the patient/donor. Factors that must be evaluated are extracorporeal circuit volume, blood flow rates, type of anticoagulant and vascular access. Other important issue to be considered is the emotional stress for them and their relatives. Leukaphereses in children may be performed with any of the reported continuous blood cell separator without sedation. Adverse events are mainly related either to vascular access or to metabolic or hemodynamic changes. Anyway, taking all these complications together, they are not higher than those adverse events or toxicities reported after general anesthesia and bone marrow harvesting.

Antecedentes: Los niñ;os con síndrome de Down tienen mayor riesgo de leucemia aguda que el resto de la población infantil. Recientemente se ha descrito una evolución favorable de las leucemias mieloides agudas (LMA) en estos niñ;os, mien-tras que las leucemias linfoblásticas agudas (LLA) tienen un pronóstico similar al del resto. Las complicaciones atribuidas a la quimioterapia son la principal causa de morbimortalidad, lo que ocasiona modificaciones de los protocolos de tratamiento Objetivos: Caracterizar las leucemias agudas en niñ;os con síndrome de Down en nuestro centro y la evolución clínica de éstos Métodos y resultados: Entre 1990 y 2002 se han diagnosticado 214 leucemia aguda en el Hospital Niñ;o Jesús (40 LMA y 174 LLA). De ellos, 8 casos (3,8%) eran pacientes con síndrome de Down. Las LMA 2/40 representaron el 5% del total mien-tras que las LLA un 6/174 el 3,4%. La toxicidad hematológica por el tratamiento fue la complicación más habitual, condicionando una elevada frecuencia de procesos infecciosos: neumonías (5/8) y bacteriemias (5/8). Además, las complicaciones obligaron a interrumpir y/o reducir las dosis del tratamiento en todos los pacientesDos niñ;as fallecieron como consecuencia de la toxicidad del tratamiento. Una diagnosticada de LMA desarrolló una sepsis fulminante por Candida, y la otra, diagnosticada de LLA de alto riesgo, falleció por fallo multiorgánico tras recibir altas dosis de metotrexato y arabinósido de citocina (Ara-c) Conclusiones: Los pacientes con síndrome de Down diagnosticados de leucemia aguda presentan una mayor incidencia de complicaciones relacionadas con el tratamiento lo que condiciona de gran modo su pronóstico. Por ello, es importante un tratamiento individualizado de estos niñ;os en unidades cualificadas : Background: Children with Down syndrome (DS) have a higher risk of acute leukemia than the remaining pediatric population. A favorable outcome of acute myeloid leukemia (AML) has recently been described in these patients whereas the prognosis of acute lymphoblastic leukemias (ALL) is similar to that in other children. The main cause of morbidity and mortality in children with Down syndrome are complications related to chemotherapy, leading to numerous modifications in treatment protocols Objectives: To characterize acute leukemias in children with Down syndrome in our center and determine their clinical outcome Methods and results: Between 1990 and 2002, 214 children were diagnosed with acute leukemia at the Niñ;o Jesús Hospital (40 with AML and 174 with ALL). Of these, eight children (3.8%) had Down syndrome. AML (2/40) represented 5% of myeloid leukemias and ALL (6/174) represented 3.4% of lymphoblastic leukemias. The most frequent complication was hematologic toxicity due to chemotherapy, causing a high incidence of infections: pneumonia (5/8) and bacteriemia (5/8). In all patients, these complications led to treatment interruption or dose reductionTwo children died from treatment-related toxicity. Of these, one with AML developed fulminant sepsis due to Candida infection and the other, diagnosed with high risk ALL, died from multiorgan failure after high doses of methotrexate and ARA-C Conclusions: Patients with Down syndrome diagnosed with acute leukemia show a higher incidence of treatment-related complications, which affects their prognosis. Consequently, individualized treatment of these children in qualified units is essential

Myelodysplastic syndromes (MDS) in childhood are rare hematologic diseases. MDS with t(3;5) (NPM/MLF1) is an unusual subtype without a well-defined clinical and prognostic pattern. A poor outcome has been reported, suggesting that hematopoietic transplantation is the only treatment option. Here in we described a 2-year-old child diagnosed with the disease, without a suitable hematopoietic donor, treated early in the disease with chemotherapy. He is alive and well 4 years after the end of treatment. This unusual MDS needs further studies to better understand the disease.

Acute hepatitis with severe hepatic failure is an uncommon manifestation of herpes simplex virus (HSV) infection. It has been described in both immunocompromised and immunocompetent patients and is usually fatal. Due to the better survival after acyclovir treatment in a few reported cases, physicians need to be aware of the characteristic clinical abnormalities so that early diagnosis and treatment can be implemented. The authors describe an adolescent diagnosed with Hodgkin disease who developed fatal hepatic failure secondary to acute HSV. Typical signs and symptoms in patients at risk, when there is no other obvious cause of fulminant hepatitis, should lead to early empirical treatment with acyclovir.

The authors examined data from 166 children who received autologous peripheral blood progenitor cell (PBPC) transplantation to ascertain the incidence of early transplant-related mortality (TRM) and the contributing risk factors. Eleven patients (6.6%) (6 boys, 5 girls) died within 180 days following PBPC infusion. The median age was 4 years (range 2–17). The overall probability of TRM was 6.9 ± 2% at day +180. On univariate analysis, the status of disease at transplantation (complete remission vs. not in complete remission) was identified as the only pretransplant significant predicting factor for TRM (14% of patients who were not in complete remission died within 180 days after PBPC infusion, whereas only 2% of patients in complete remission died) (relative risk [RR] 1.13, 95% confidence interval [CI] 1.01–1.26, P = 0.01). Age, gender, conditioning, and number of CD34+ cells infused were not significantly associated with TRM. In the postinfusion phase, patients who developed multiorgan dysfunction during the neutropenic period, especially when the lung was the first failing organ (RR 16.1, 95% CI 7.16–36.18, P = 0.0001), and those with engraftment syndrome (RR 2.81, 95% CI 1.49–5.24, P = 0.001) had an increased risk for TRM. On multivariate analysis, development of engraftment syndrome was the only significant variable that influenced TRM. In conclusion, the authors found for the first time that engraftment syndrome emerges as the main cause of TRM after autologous PBPC transplantation in children with malignancies.

Secondary acute leukemia (SAL) after treatment of osteosarcoma has been seldom reported in children. Two main mechanisms that can induce SAL have been described. The authors describe two patients who developed SAL after treatment with multiagent therapy. The first patient developed a secondary acute leukemia after treatment with alkylating agents and platinum compounds. The second patient had the clinical and molecular features of a secondary acute leukemia due to topoisomerase II inhibitors. The authors also discuss the data regarding cisplatin-associated SAL. Different molecular and pathogenic agents may be implicated in the development of second malignant neoplasms in long-term survivors of osteosarcomas.