Correction to: Predictive and prognostic clinical and pathological factors of nivolumab efficacy in non-small-cell lung cancer patients.

Clinical and Translational Oncology.

Predictive and prognostic clinical and pathological factors of nivolumab efficacy in non-small-cell lung cancer patients.

BACKGROUND: Immunotherapy increases overall response rate (ORR) and overall survival (OS) in patients with non-small-cell lung cancer (NSCLC). Prognostic and predictive factors are a high need. PATIENTS AND METHODS: Retrospective review of NSCLC patients treated with nivolumab was performed. Analyzed variables included age, sex, stage, performance status (PS), location of metastases, presence of tumour-related symptoms and comorbidities, number of metastasis locations, previous chemotherapy, anti-angiogenic and radiotherapy treatments, and analytical data from the standard blood count and biochemistry. RESULTS: A total of 175 patients were included. Median age was 61.5 years, 73.1% were men, 77.7% were ECOG-PS 0-1, and 86.7% were included with stage IV disease. Histology was non-squamous in 77.1%. Sixty-five received nivolumab in second line (37.1%). Thirty-eight patients had brain metastasis (22%), and 39 (22.3%) liver metastasis and 126 (72%) had more than one metastatic location. The ORR was 15.7% with median Progression free survival (PFS) 2.8 months and median OS 5.81 months. Stage III vs IV and time since the beginning of the previous line of treatment ≥ 6 vs < 6 months were associated with better response. PS 2, time since the previous line of treatment < 6 vs ≥ 6 months, and more than one metastatic location were independently associated with shorter OS in multivariable analysis (7.8 vs 2.7 months, 11.2 vs 4.6 months, and 9.4 vs 5.1 month). Finally, time since the previous treatment < 6 vs ≥ 6 months and more than one metastatic location were independently associated with shorter PFS in multivariable analysis (4.3 vs 2.3 months and 4.7 vs 2.3 months). CONCLUSION: Poor PS, short period of time since the previous treatment, and more than one metastatic location were associated with poorer prognostic.

[Sickle cell disease: registry of the Spanish Society of Pediatric Hematology]. / Enfermedad de células falciformes: registro de la Sociedad Española de Hematología Pediátrica.

INTRODUCTION: Sickle cell disease (SCD), a genetic anemia, is currently an emerging health problem in Spain. Since 2000, The Spanish Society of Pediatric Hematology has maintained a registry of these patients. The data corresponding to 2004 are presented herein. PATIENTS AND METHODS: Information was sent by different national hospitals. Pediatric patients with SCD followed-up during 2003 were registered in the first quarter of 2004. Data on epidemiology, diagnosis, treatment and outcome in each patient were gathered. RESULTS: A total of 138 patients in 24 national hospitals were registered. Of these, 99 were still under follow-up. There was no significant difference in sex. The mean age was 8.2 years. Seventy-eight percent of the patients were homozygous. Forty-four percent were born in Africa but 76% had abnormal genes originating in Africa. Neurophysiologic disorders were detected in 36% of the patients. Symptomatic treatment was given in 65%, hydroxyurea in 27%, hypertransfusional therapy in 3%, and chelation therapy, indicated for ferric overload, was provided in 4%. None of the patients underwent stem cell transplantation. Acute complications requiring hospitalization occurred in 21%, and chronic complications were observed in 27%. The most frequent chronic complications were delayed height and weight gain and liver and biliary tract disorders. Two patients died. CONCLUSIONS: This study confirms a highly significant increase in the prevalence of pediatric patients with SCD in the last 4 years, requiring greater resources to be devoted to the diagnosis and follow-up of this disease.

[Early detection of sickle cell anemia and other hemoglobinopathies in neonates in the Autonomous Community of Madrid. A pilot study]. / Detección precoz neonatal de anemia falciforme y otras hemoglobinopatías en la comunidad autónoma de Madrid. Estudio piloto.

OBJECTIVE: To determine the incidence of sickle cell anemia and other hemoglobinopathies in the neonatal population of the Autonomous Community of Madrid and to determine the need for a screening program. METHODS: The study was performed with the same blood spot specimen dried on filter paper used for congenital hypothyroidism and congenital adrenal hyperplasia screening. All neonates born in the public and private hospitals of the Autonomous Community of Madrid were included and universal-type screening was performed. High-performance liquid chromatography (HPLC) was used to detect variant hemoglobins. The variant automated system was used to separate and identify hemoglobin F, A1c, A, S, C, A2/E and D. To confirm variant hemoglobins, specific HPLC for -thalassemia (ion exchange) and globin chains (reversed phase) with a more expanded gradient were used. RESULTS: A total of 29 253 specimens were screened and 98 cases of variant hemoglobins were detected. The overall incidence was 1/299. There were five cases of sickle cell disease (HbFS and HbFS(tal), with an incidence of 1/5.851, and 71 cases of sickle cell traits (1/412). CONCLUSIONS: These results confirm the need to include screening for sickle cell disease and other hemoglobinopathies in our neonatal program.

[Renal cell carcinoma in children]. / Carcinoma de células renales.

Renal cell carcinoma is infrequent in children; consequently it is important to communicate its diagnosis and follow up. The behavior of this type of tumor is better characterized in adults and in this setting the treatment of choice is surgical resection. However, chemo- and radiotherapy for metastatic tumors has not been well defined. Our objective was to report the experience of a single pediatric institution in the diagnosis and treatment of renal cell carcinoma and to review the literature on this subject. We retrospectively reviewed patients diagnosed with renal cell carcinoma in the last twenty years. Only three patients were found, and we describe their clinical features and therapeutic approach. Although renal cell carcinoma is rare in children, clinical suspicion of this disease in children older than 5 years with renal masses is very important since the diagnostic and therapeutic approach differs from that for Wilms' tumor. The main prognostic factors seem to be staging and complete resection. Multicenter collaboration is required to standardize the treatment of tumors in advanced stages and to define the role of allogeneic stem cell transplantation in metastatic tumors.

[Perineal bag versus urethral catheterization of suprapubic aspiration for the diagnosis of urinary tract infections in infants in emergency units]. / Bolsa perineal versus sondaje uretral o punción suprapúbica para el diagnóstico de infección urinaria en el lactante en las unidades de urgencias.

OBJECTIVE: Our objective was to determine the utility of urine cultures collected by sterile perineal bags as a method of diagnosis of urinary tract infection in infants. PATIENTS AND METHODS: Forty-two patients, aged 0 to 27 months, were diagnosed with urinary tract infections based on the growth of more than 100,000 colonies/ml in two urine cultures collected by sterile bags. Confirmation of the infection was done by urine cultures obtained by suprapubic aspiration or urethral catheterization. A urinalysis was simultaneously performed. RESULTS: Urinary tract infection was confirmed in only 6 out of 42 patients. The positive predictive value of the sterile bag was 14%, increasing to 42% combined with an abnormal urinalysis. CONCLUSIONS: The sterile perineal bag is not an accurate method to collect urine for diagnosis of urinary tract infections in febrile infants or those who need prompt diagnosis and treatment.

[Radiologic features of lung diseases in HIV infection in children]. / Manifestaciones radiológicas de la patología pulmonar en la infección VIH pediátrica.

OBJECTIVES: The purpose of this study was to assess the incidence of respiratory involvement in HIV-infected children, along with the radiological manifestations of the various HIV-related diseases. MATERIAL AND METHODS: A retrospective review of the medical records of 65 HIV-infected children (63 vertically-infected and 2 through blood transfusion) has been carried out. The mean time of follow-up since the diagnosis of HIV infection was 32 +/- 27 months, beginning January 1987. Patients have been classified into 5 categories: Pneumocystis carinii (PC) pneumonia, lymphoid interstitial pneumonitis (LIP), lobar pneumonia, acute respiratory distress and a miscellaneous group. Plain chest radiographies were performed according to clinical criteria and at least every 6 months in asymptomatic patients. Diagnosis into categories was based upon chest X-ray findings, along with several etiological diagnostic criteria depending on the category. RESULTS: Respiratory involvement occurred in 32 children (49%). The most common diseases were PC pneumonia in 7 patients, LIP in 8 children, and lobar pneumonia and acute respiratory distress in 6 and 7 cases, respectively. PC pneumonia in children younger than one year had a good clinical and radiological correlation. LIP diagnosis has been based only on radiological criteria with the typical pattern. We want to highlight the disappearance of the radiological findings in 3 cases over time. Interestingly, several patients had other etiologic diagnosis, such as S. pneumoniae pneumonia, miliary tuberculosis, and two patients had acute respiratory distress, one caused by adenovirus and the other by enterovirus. CONCLUSION: Respiratory involvement is common in HIV-infected children. Clinical and radiological manifestations are variable. Plain chest radiography plays a very important role in the management and follow-up of these patients.

[The results of treatment by the Madrid Pediatric Oncology Group according to the BFM protocols in non-Hodgkin's B-cell lymphoma and acute B-cell lymphoblastic leukemia in pediatric patients]. / Resultados del tratamiento según protocolos BFM del linfoma no Hodgkin B y de la leucemia linfoblástica aguda de células B en pacientes pediátricos por el GOPM.

The objective of this report is to present the results of the BFM group in the treatment of 41 children with non-Hodgkin's B cell lymphoma and acute B cell lymphoblastic leukemia according to the BFM 86 and 90 protocols. Forty-one children, between 2 and 16 years of age, were treated from November 1987 to October 1993. Of these, 25 were treated with the BFM 86 protocol (18 non-Hodgkin's B cell lymphomas and 7 acute B cell lymphoblastic leukemias) and the rest with the BFM 90 protocol (15 non-Hodgkin's B cell lymphomas and 1 acute B cell lymphoblastic leukemia). Complete remission was achieved in 97.5% of the patients. A relapse occurred in 12.5% of the cases. Currently, 80.4% remain in continuous complete remission and 17% have died. The 5 year actuarial survival rate of those treated with the BFM 86 and 90 protocols was 79% and 87%, respectively, and event free survival in the same period was 76% and 87%, respectively. There was no statistically significant difference in the results obtained with the two treatment protocols.

Enfermedad de células falciformes: registro de la Sociedad Española de Hematología Pediátrica / Sickle cell disease: registry of the spanish society of pediatric hematology

Introducción La enfermedad de células falciformes (ECF), anemia de origen genético, es un problema de salud emergente en España. La Sociedad Española de Hematología Pediátrica realiza desde el año 2000 un registro de estos pacientes. Se presentan los datos correspondientes a la recogida de 2004. Pacientes y métodos De la información enviada por diferentes hospitales nacionales se registraron en el primer trimestre de 2004 los pacientes pediátricos con ECF en seguimiento en 2003 y se recogieron variables epidemiológicas, relativas al diagnóstico, tratamiento y evolución de cada paciente. Resultados Se registraron un total de 138 enfermos, 99 aún en seguimiento, de 24 hospitales nacionales, sin diferencia significativa entre sexos y con una media de edad de 8,2 años. Eran homozigotos (SS) el 78 %. El 44 % habían nacido en África, pero tenían genes anómalos procedentes de África el 76 %. Se detectaron anomalías neuropsiquiátricas en el 36 % de los pacientes investigados. Se hizo tratamiento sólo sintomático en el 65 %, tratamiento con hidroxiurea en el 27 %, terapia hipertransfusional en el 3 % y quelación por sobrecarga férrica en el 4 %. Ningún paciente fue sometido a trasplante de progenitores hematopoyéticos ese año. Presentaron complicaciones agudas con necesidad de hospitalización el 21 % y complicaciones crónicas el 27 %. De éstas, las más frecuentes fueron los retrasos en la curva ponderoestatural y trastornos hepatobiliares. Dos pacientes fallecieron. Conclusiones Se confirma en los últimos 4 años un incremento nacional muy significativo de pacientes pediátricos con ECF, que obligaría a un mayor esfuerzo en su diagnóstico y seguimiento

Introduction Sickle cell disease (SCD), a genetic anemia, is currently an emerging health problem in Spain. Since 2000, The Spanish Society of Pediatric Hematology has maintained a registry of these patients. The data corresponding to 2004 are presented herein. Patients and methods Information was sent by different national hospitals. Pediatric patients with SCD followed-up during 2003 were registered in the first quarter of 2004. Data on epidemiology, diagnosis, treatment and outcome in each patient were gathered. Results A total of 138 patients in 24 national hospitals were registered. Of these, 99 were still under follow-up. There was no significant difference in sex. The mean age was 8.2 years. Seventy-eight percent of the patients were homozygous. Forty-four percent were born in Africa but 76 % had abnormal genes originating in Africa. Neurophysiologic disorders were detected in 36 % of the patients. Symptomatic treatment was given in 65 %, hydroxyurea in 27 %, hypertransfusional therapy in 3 %, and chelation therapy, indicated for ferric overload, was provided in 4 %. None of the patients underwent stem cell transplantation. Acute complications requiring hospitalization occurred in 21 %, and chronic complications were observed in 27 %. The most frequent chronic complications were delayed height and weight gain and liver and biliary tract disorders. Two patients died. Conclusions This study confirms a highly significant increase in the prevalence of pediatric patients with SCD in the last 4 years, requiring greater resources to be devoted to the diagnosis and follow-up of this disease

Detección precoz neonatal de anemia falciforme y otras hemoglobinopatías en la comunidad autónoma de Madrid. Estudio piloto / Early detection of sickle cell anemia and other hemoglobinopathies in neonates in the autonomous community of Madrid. A pilot study

Objetivo: Conocer la incidencia de anemia falciforme y otras hemoglobinopatías en la población neonatal de la comunidad de Madrid y determinar la necesidad de realizar su cribado neonatal. Métodos El estudio se realiza sobre el mismo espécimen de sangre seca impregnada en papel, que se utiliza para la detección precoz de hipotiroidismo congénito e hiperplasia suprarrenal congénita. Se han incluido especímenes de sangre de recién nacidos, procedentes de todos los hospitales públicos y privados del ámbito de la comunidad de Madrid (CAM). El estudio se ha realizado de forma universal. La detección de variantes de hemoglobina se lleva a cabo mediante cromatografía líquida de alta resolución (HPLC). El sistema automático VARIANT separa e identifica las hemoglobinas F, A1c, A, S, C, E/A2 y D. La presencia de variantes se confirma con cromatografías específicas para betatalasemia (intercambio iónico) y cadenas de globina (fase reversa), con gradientes más expandidos. Resultados Se han analizado un total de 29.253 especímenes de sangre y se han detectado 98 casos con alguna variante de hemoglobina, con una incidencia global de 1/299, cinco de ellos fueron diagnosticados de anemia falciforme (HbFS y HbFS tal), con una incidencia de 1/5.851 y 71 casos con rasgo falciforme, con una incidencia de 1/412.Conclusiones Los resultados obtenidos reflejan la necesidad de incluir la detección de anemia falciforme dentro del programa de cribado neonatal de la comunidad de Madrid (AU)

Efectos secundarios tardíos en pacientes pedíátricos diagnosticados de cáncer. Alteraciones endocrinas / Late adverse in pediatric patients diagnosed with cancer: endocrine changes

En los últimos 25 años, con la aplicación de los nuevos fármacos quimioterápicos en combinación con radioterapia y/o cirugía, se han conseguido importantes avances en la supervivencia de los pacientes diagnosticados de cáncer, con mayor expresión en la población infantil, en la que se ha llegado a conseguir un 65-70 por ciento, alcanzando el 90 por ciento en algunos tipos de tumores, como en el tumor de Wilms y el linfoma de Hodgkin. Tan altas supervivencias conllevan el riesgo de sufrir efectos adversos tardíos directamente relacionados con las técnicas terapéuticas empleadas, y su interacción con el paciente y los factores tumorales. La mayor vulnerabilidad de los tejidos a los efectos secundarios del tratamiento está incrementada durante los periodos de rápida proliferación. Cualquier sistema del organismo puede ser lugar de elección para la aparición de secuelas postratamiento. Una de las alteraciones más frecuentes es la disminución del crecimiento lineal del niño, presente en el 30 por ciento de los pacientes, seguida de las alteraciones endocrinas y de la aparición de segundos tumores lineal del niño, presente en el 30 por ciento de los pacientes, seguida de las alteraciones endocrinas y de la aparición de segundos tumores (AU)

Carcinoma de células renales / Renal cell carcinoma in children

El carcinoma renal es poco frecuente en niños, por lo que es importante comunicar el diagnóstico y seguimiento de estos casos. Su comportamiento en adultos es más conocido, y se sabe que el tratamiento de elección es la extirpación quirúrgica. Sin embargo, el tratamiento de los tumores metastásicos con quimioterapia y/o radioterapia no está bien definido. Nuestro objetivo es aportar la experiencia de un centro en el diagnóstico y tratamiento del carcinoma renal pediátrico, así como revisar la bibliografía al respecto. Se revisan de forma retrospectiva los pacientes diagnosticados de carcinoma renal en los últimos 20 años. La muestra está formada por 3 niños, y se describen las características clínicas de los pacientes y su tratamiento. Se insiste en que aunque el carcinoma renal es una entidad rara en niños, es importante sospecharlo en pacientes mayores de 5 años con masas renales, ya que la aproximación diagnóstica y terapéutica difiere del tumor de Wilms. La estadificación y la resección completa parecen ser los factores pronósticos fundamentales. Es necesaria la colaboración de varios centros para la estandarización del tratamiento de los tumores en estadios avanzados, valorando el papel del trasplante alogénico de progenitores hematopoyéticos en tumores metastásicos (AU)