Histiocitosis de células de Langerhans con afectación gastrointestinal / Gastrointestinal involvement in Langerhans cell histiocytosis

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Linfoma no Hodgkin: excelentes resultados a expensas de elevada toxicidad del tratamiento / Non-Hodgkin lymphoma: Excellent results at the expense of the high toxicity of the treatment

Introducción : Los linfomas son la tercera neoplasia maligna en los niños y, dentro de ellos, los linfomas no Hodgkin (LNH) representan tan solo el 7% del cáncer en menores de 15 años. La quimioterapia constituye actualmente el tratamiento de elección. El objetivo de este estudio es analizar la toxicidad secundaria al tratamiento en pacientes pediátricos diagnosticados de LNH. Material y métodos: Estudio retrospectivo de pacientes diagnosticados de LNH de células B maduras tratados según el protocolo LMB 2001, desde enero del 2007 hasta febrero del 2014. Se recogieron los datos referentes al diagnóstico, el tratamiento y las toxicidades que desarrollaron durante el mismo. Resultados: Se diagnosticaron 20 LNH de células B maduras: 16 linfomas Burkitt, 2 linfomas difusos de células grandes y 2 leucemias maduras. Un 65% de los pacientes se clasificó al diagnóstico en un estadio de alto grado (III-IV). Los procesos infecciosos graves, la mielosupresión severa, las alteraciones hepáticas y la mucositis fueron las toxicidades más frecuentes. La supervivencia global fue del 95% (19/20). Un paciente falleció por causas no relacionadas con su enfermedad. Conclusión: La mayoría de los pacientes diagnosticados de LNH de células B maduras experimentan toxicidades grado III y IV durante el tratamiento, a pesar de lo cual la supervivencia es excelente (AU)

Introduction: Lymphomas are the third malignancy in children, and within them non-Hodgkin lymphoma (NHL) accounts for just 7% of cancers in children under 15 years old. Chemotherapy is currently the treatment of choice. The objective of this study is to analyze the toxicity caused by the treatment in pediatric patients diagnosed with NHL. Material and methods: A retrospective study was conducted on patients diagnosed with mature B-cell NHL, treated according to the LMB protocol 2001, from January 2007 to February 2014. Data concerning the diagnosis, treatment and toxicities that developed in the patients during the same period were collected. Results: A total of 20 mature B-cell NHL cases were diagnosed: 16 Burkitt lymphomas, 2 diffuse large cell lymphomas and 2 mature leukemias. Almost two-thirds (65%) of patients were classified in a high grade stage (III-IV) at diagnosis. Serious infectious processes, severe myelosuppression, liver abnormalities, and mucositis were the most frequent toxicities. Overall survival was 95% (19/20). One patient died of causes unrelated to the illness. Conclusion: Despite the excellent survival rate, most patients diagnosed with NHL mature B cells experience grade III and IV toxicities during treatment (AU)

El cáncer en el mundo adolescente / Cancer in the adolescent world

Existen diferencias tanto clínicas como psicológicas dentro de la oncología según los grupos de edad. La fase madurativa en la que se encuentran en el diagnóstico, la histología del tumor, la respuesta a los tratamientos y las complicaciones a largo plazo, no van a ser las mismas si se es adulto, niño o adolescente. El adolescente con cáncer debe enfrentarse con un doble estresor: tener un diagnóstico que "amenaza" la vida asociado a un tratamiento invasivo, además del desafío que supone la transición en su desarrollo de la infancia a la adultez. La repercusión de la enfermedad onco-hematológica en la adolescencia es claramente devastadora. Una intervención multidisciplinar debe ofrecer una mayor calidad de vida tanto al paciente como a su familia. Los aspectos somáticos son la base del tratamiento. No obstante, para conseguir una buena adherencia y adaptación, es necesario contar con el soporte de un equipo integrado por médicos, enfermeras, auxiliares, maestros, trabajadores sociales, psicólogos, voluntarios... que abarque de forma global tanto los aspectos físicos y biológicos como el resto de aspectos (emocionales, sociales y de cuidados). El objetivo es facilitar el proceso y adaptación a la enfermedad, tratamiento y vuelta a la normalidad, e incluso facilitar una buena despedida de éstos, cuando sea necesaria (AU)

There are both clinical and psychological differences within oncology according to the age groups. The development phase of the patient at the time of the diagnosis, tumor histology, response to treatments and long-term complications are not going to be the same if the patient is an adult, child or adolescent. The adolescent with cancer should be approached with a double stressor: having a diagnosis that is life "threatening" associated to an invasive treatment in addition to the challenge entailed by the transition in development from childhood to adulthood. The repercussion of the onco-hematology disease in the adolescent is clearly devastating. A multidisciplinary intervention should offer greater quality of life both to the patient and family. The somatic aspects are the basis of the treatment. However, in order to achieve good adherence and adaptation, support is needed from a team made up of physicians, nurses, auxiliaries, teachers, social workers, psychologists, volunteers, etc., that globally covers both physical and biological aspects as well as the remaining aspects (emotional, social and care). The aim is to facilitate the process and adaptation to the disease, treatment and return to normality and even facilitate a warm farewell, when necessary (AU)

Proyecto de gestión clínica "Unidad de cáncer de adolescentes y adultos jóvenes" / Clinical management project "Adolescent and Young Adult Cancer Unit"

Se describe la incidencia del cáncer en los adolescentes y adultos jóvenes y las dificultades que presenta su asistencia integral en la actualidad. Se propone la creación de unidades de gestión clínica específicas para ese segmento poblacional (AU)

The incidence of cancer in adolescents and young adults and the difficulties currently entailed in the comprehensive care are described. The creation of specific clinical management units for this population segment is proposed (AU)

[Non-Hodgkin lymphoma: Excellent results at the expense of the high toxicity of the treatment]. / Linfoma no Hodgkin: excelentes resultados a expensas de elevada toxicidad del tratamiento.

INTRODUCTION: Lymphomas are the third malignancy in children, and within them non-Hodgkin lymphoma (NHL) accounts for just 7% of cancers in children under 15 years old. Chemotherapy is currently the treatment of choice. The objective of this study is to analyze the toxicity caused by the treatment in pediatric patients diagnosed with NHL. MATERIAL AND METHODS: A retrospective study was conducted on patients diagnosed with mature B-cell NHL, treated according to the LMB protocol 2001, from January 2007 to February 2014. Data concerning the diagnosis, treatment and toxicities that developed in the patients during the same period were collected. RESULTS: A total of 20 mature B-cell NHL cases were diagnosed: 16 Burkitt lymphomas, 2 diffuse large cell lymphomas and 2 mature leukemias. Almost two-thirds (65%) of patients were classified in a high grade stage (iii-iv) at diagnosis. Serious infectious processes, severe myelosuppression, liver abnormalities, and mucositis were the most frequent toxicities. Overall survival was 95% (19/20). One patient died of causes unrelated to the illness. CONCLUSION: Despite the excellent survival rate, most patients diagnosed with NHL mature B cells experience grade iii and iv toxicities during treatment.

All children with malignant rhabdoid tumors should be treated initially with intensive chemotherapy.

Malignant rhabdoid tumors (MRT) of soft tissues are aggressive tumors, which can be detected in almost any part of the body. MRT are rare, and very few cases have been reported in the literature. Prognosis of these tumors is extremely poor despite intensive therapy. Some risk factors such as young age or disseminated disease are associated with an aggressive and almost always lethal clinical course. Some clinicians even recommend initial palliative care due to this outcome. We report a case of metastatic MRT in a 6-month-old child with excellent initial response to chemotherapy.

Citopenias autoinmunes: tratamiento según el diagnóstico. Respuesta de los autores / Autoimmune cytopenias: Diagnosis Before Treatment. Reply by the authors

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Actualización del tratamiento con L-asparraginasa en Pediatría / Update on L-asparaginase treatment in paediatrics

La L-asparraginasa (L-ASP) es una de las piedras angulares del tratamiento de la leucemia linfoblástica aguda y del linfoma no Hodgkin. Es una enzima de origen bacteriano con capacidad de transformar la L-Asparragina en ácido aspártico; la depleción extracelular de este aminoácido inhibe la síntesis proteica en los linfoblastos induciendo su apoptosis. Numerosos estudios han demostrado que los tratamientos con L-ASP mejoran la supervivencia de estos pacientes, pero existen diferencias en las características de las 3 formulaciones disponibles en la actualidad. Este artículo revisa la dosificación, actividad y efectos secundarios de las 2 L-ASP derivadas de Escherichia coli (la nativa y la pegilada) y de la única derivada de Erwinia chrysanthemi (Erwinia ASP). A pesar de su indiscutible indicación en los últimos 50 años, siguen existiendo numerosos puntos de controversia, y su uso todavía sigue marcado por los efectos secundarios derivados de la inhibición de la síntesis proteica. La vida media corta de las formas nativas y la vía de administración intramuscular, la más utilizada hasta el momento, afecta la calidad de vida de estos pacientes por la frecuencia con la que han de acudir al centro hospitalario y las múltiples punciones que conlleva. Por ello, los estudios más recientes pretenden valorar otras alternativas como la formulación de vida media más larga (L-ASP pegilada) y la vía intravenosa, con resultados alentadores. Aun así, son necesarios más estudios para establecer cuál es la formulación y la vía de administración indicada en primera línea, la dosificación óptima y el manejo de los efectos adversos (AU)

L-asparaginase (L-ASP) is one of the cornerstones of the treatment of acute lymphoblastic leukemia and non-Hodgkin lymphoma. It is an enzyme of bacterial origin capable of transforming L-asparagine to aspartic acid. The extracellular depletion of L-asparagine inhibits protein synthesis in lymphoblasts, inducing their apoptosis. Numerous studies have demonstrated that treatment with L-ASP improves survival of patients, but there are clear differences in the characteristics of the three currently available formulations. This article reviews the dosage, activity and side effects of the two L-ASP derived from Escherichia coli (native and pegylated), and the one derived from Erwinia chrysanthemi (Erwinia ASP). Despite its indisputable indication over the past50 years, there are still many points of contention, and its use is still marked by the side effects of the inhibition of protein synthesis. The short half-life of native forms, and the most frequently used parenteral administration by intramuscular injections, affects the quality of life of the patients. Therefore, recent studies claim to evaluate alternatives, such as the formulation of longer half-life pegylated L-ASP, and the use of intravenous formulations. There are encouraging results to date with both preparations. Still, further studies are needed to establish which should be the formulation and frontline indicated route of administration, optimal dosing, and management of adverse effects (AU)

Irinotecán y temozolomida en tumores sólidos pediátricos en recaída o refractarios / Irinotecan plus temozolomide in refractory or relapsed pediatric solid tumors

Introducción: El pronóstico de los tumores sólidos pediátricos refractarios o en recaída es infausto, y actualmente no existen tratamientos estandarizados para este tipo de situaciones. La combinación irinotecán y temozolomida ha demostrado su utilidad en adultos como segunda línea de tratamiento en distintos tumores sólidos. En pacientes pediátricos ya ha demostrado su eficacia en sarcoma de Ewing, neuroblastoma o rabdomiosarcoma en recaída o refractario. Pacientes y métodos: Se ha realizado un estudio retrospectivo de 32 pacientes pediátricos con tumores sólidos en recaída o refractarios que fueron tratados con irinotecán y temozolomida desde septiembre de 2005 hasta junio de 2012 en el Servicio de Oncología del Hospital Infantil Niño Jesús. Se analizaron las características clínicas, el tratamiento realizado, la toxicidad presentada y la respuesta obtenida. Resultados: Treinta y dos pacientes recibieron un total de 180 ciclos. De 30 pacientes valorables, 10 (33%) presentaron respuesta positiva (2 remisión completa y 8 remisión parcial) y 8 (27%) mantuvieron enfermedad estable. El 94% de los pacientes que obtuvo respuesta lo hicieron en los 4 primeros ciclos. De los 180 ciclos analizados, solo 50 (28%) presentaron toxicidad y, de ellos, solo 15 (8%) fueron de grado III-IV . La toxicidad más frecuente fue la diarrea que apareció en 18 pacientes. Todos los pacientes recibieron el tratamiento de forma ambulatoria, excepto 3 que precisaron ingreso por síntomas relacionados con su enfermedad de base. Conclusión: La combinación irinotecán y temozolomida es bien tolerada y activa frente a tumores sólidos pediátricos refractarios al tratamiento o en recaída (AU)

Introduction: The prognosis of refractory or relapsed pediatric solid tumors is very poor, and there is no standard treatment for this condition. The combination of irinotecan and temozolomide has proved useful in adults as a second-line treatment of different solid tumors. In pediatric patients, this combination has been effective in Ewing's sarcoma, neuroblastoma, and relapsed or refractory rhabdomyosarcoma. Patients and methods: A retrospective study was conducted on 32 pediatric patients with refractory or relapsed solid tumors, who were treated with irinotecan and temozolomide in the Oncology Department at Children's Hospital Niño Jesus from September 2005 to June 2012. The clinical characteristics, treatment performed, toxicity and outcome, were analyzed. Results: Thirty-two patients received a total of 180 cycles. Of the 30 evaluable patients, 10 (33%) had a positive response (2 complete remission and 8 partial remission), and in 8 (27%) the disease remained stable. Almost all (94%) of the patients achieved a response in the first four cycles. Of the 180 cycles analyzed, only 50 (28%) had toxicity, and of these only 15 (8%) were grade III-IV . The most common toxicity was diarrhea appearing in 18 patients. All patients received ambulatory treatment, except three of them who required hospitalization due to symptoms of their underlying disease. Conclusion: The combination of irinotecan and temozolomide is well tolerated and active against pediatric refractory or relapsed solid tumors (AU)

Programa de Investigación del Servicio de Oncohematología y Transplantes del H. Infantil Universitario Niño Jesús / Research Program and Transplantation Service Oncohematology H. Infantil Universitario Niño Jesús

Se describen las líneas de investigación clínica y experimental del Servicio. Se detalla la actividad asistencial con especial énfasis en el trasplante de progenitores hematopoyéticos (AU)

The clinical and experimental research lines of the Department are described. The care activity with special emphasis on hematopoietic stem cell transplantation is detailed (AU)

[Update on L-asparaginase treatment in paediatrics]. / Actualización del tratamiento con L-asparraginasa en Pediatría.

L-asparaginase (L-ASP) is one of the cornerstones of the treatment of acute lymphoblastic leukemia and non-Hodgkin lymphoma. It is an enzyme of bacterial origin capable of transforming L-asparagine to aspartic acid. The extracellular depletion of L-asparagine inhibits protein synthesis in lymphoblasts, inducing their apoptosis. Numerous studies have demonstrated that treatment with L-ASP improves survival of patients, but there are clear differences in the characteristics of the three currently available formulations. This article reviews the dosage, activity and side effects of the two L-ASP derived from Escherichia coli (native and pegylated), and the one derived from Erwinia chrysanthemi (Erwinia ASP). Despite its indisputable indication over the past50 years, there are still many points of contention, and its use is still marked by the side effects of the inhibition of protein synthesis. The short half-life of native forms, and the most frequently used parenteral administration by intramuscular injections, affects the quality of life of the patients. Therefore, recent studies claim to evaluate alternatives, such as the formulation of longer half-life pegylated L-ASP, and the use of intravenous formulations. There are encouraging results to date with both preparations. Still, further studies are needed to establish which should be the formulation and frontline indicated route of administration, optimal dosing, and management of adverse effects.

Rituximab en el tratamiento de citopenias autoinmunitarias refractarias a tratamientos convencionales / Role of rituximab in the management of refractory autoimmune cytopenia

Objetivo: Evaluar el papel del rituximab en el tratamiento de citopenias autoinmunitarias refractarias a tratamientos convencionales. Material y métodos: Estudio descriptivo longitudinal constituido por una serie de casos clínicos (n = 7) durante el período comprendido entre 2003 y 2010. Resultados: Se recogen 7 pacientes: 4, trombocitopenia inmune primaria; 2, anemia hemolítica autoinmunitaria, y 1, neutropenia autoinmunitaria. Un paciente había recibido trasplante de progenitores hematopoyéticos. La dosis fue de 375mg/m2/semana. Cuatro pacientes recibieron 4 dosis y 3 pacientes recibieron 2, 6 y 8 dosis cada uno. Cinco de los pacientes (71%) cumplieron criterios de respuesta global (completa en 4 pacientes y parcial en 1). A las 8,5 semanas, respondieron la mitad de los pacientes (rango: 3,5-19,5). La mediana de la duración de la respuesta fue de 35,5 semanas (rango: 12,5-53,5). El 100% de los respondedores pudo disminuir su tratamiento previo. Se registraron 2 recaídas. No se registran efectos adversos graves. Conclusiones: El 71% de los pacientes de esta serie responden al tratamiento, disminuyendo el 100% de los respondedores su tratamiento previo. Rituximab es un tratamiento bien tolerado, sin efectos secundarios graves en el período de seguimiento estudiado (AU)

Objectives: This study examined the efficacy of rituximab in children with refractory autoimmune cytopenia. Material and methods: Longitudinal descriptive study comprising a series of clinical cases (n=7) during the period 2003 to 2010. Results: A series 7 patients were included (4 had primary immune thrombocytopenia, 2 autoimmune hemolytic anemia, and 1 autoimmune neutropenia). One patient had received stem cell transplantation. Rituximab was administered intravenously to all patients at a dose of 375mg/mg2 weekly. Four patients received 4 doses. Three patients received 2, 6, and 8 doses, respectively. Overall, 5 patients responded (4 complete responses plus 1 partial response). The median time to achieve complete response was 8.5 weeks (range: 3.5-19.5 weeks). Two patients achieved complete response in the first 3.5 weeks, and the remaining 3 patients between 8.5 and 19.5 weeks. The median time of response was 35.5 weeks (range: 12.5-53.5 weeks). Two patients relapsed. No serious adverse events were recorded. Conclusions: Overall, seventy one percent of patients in this study respond to treatment, 100% of responders decrease their previous treatment. Rituximab was a well tolerated and no related serious side effects were recorded during the study period (AU)

[Irinotecan plus temozolomide in refractory or relapsed pediatric solid tumors]. / Irinotecán y temozolomida en tumores sólidos pediátricos en recaída o refractarios.

INTRODUCTION: The prognosis of refractory or relapsed pediatric solid tumors is very poor, and there is no standard treatment for this condition. The combination of irinotecan and temozolomide has proved useful in adults as a second-line treatment of different solid tumors. In pediatric patients, this combination has been effective in Ewing's sarcoma, neuroblastoma, and relapsed or refractory rhabdomyosarcoma. PATIENTS AND METHODS: A retrospective study was conducted on 32 pediatric patients with refractory or relapsed solid tumors, who were treated with irinotecan and temozolomide in the Oncology Department at Children's Hospital Niño Jesus from September 2005 to June 2012. The clinical characteristics, treatment performed, toxicity and outcome, were analyzed. RESULTS: Thirty-two patients received a total of 180 cycles. Of the 30 evaluable patients, 10 (33%) had a positive response (2 complete remission and 8 partial remission), and in 8 (27%) the disease remained stable. Almost all (94%) of the patients achieved a response in the first four cycles. Of the 180 cycles analyzed, only 50 (28%) had toxicity, and of these only 15 (8%) were grade iii-iv. The most common toxicity was diarrhea appearing in 18 patients. All patients received ambulatory treatment, except three of them who required hospitalization due to symptoms of their underlying disease. CONCLUSION: The combination of irinotecan and temozolomide is well tolerated and active against pediatric refractory or relapsed solid tumors.

[Role of rituximab in the management of refractory autoimmune cytopenia]. / Rituximab en el tratamiento de citopenias autoinmunitarias refractarias a tratamientos convencionales.

OBJECTIVES: This study examined the efficacy of rituximab in children with refractory autoimmune cytopenia. MATERIAL AND METHODS: Longitudinal descriptive study comprising a series of clinical cases (n=7) during the period 2003 to 2010. RESULTS: A series 7 patients were included (4 had primary immune thrombocytopenia, 2 autoimmune hemolytic anemia, and 1 autoimmune neutropenia). One patient had received stem cell transplantation. Rituximab was administered intravenously to all patients at a dose of 375 mg/mg(2) weekly. Four patients received 4 doses. Three patients received 2, 6, and 8 doses, respectively. Overall, 5 patients responded (4 complete responses plus 1 partial response). The median time to achieve complete response was 8.5 weeks (range: 3.5-19.5 weeks). Two patients achieved complete response in the first 3.5 weeks, and the remaining 3 patients between 8.5 and 19.5 weeks. The median time of response was 35.5 weeks (range: 12.5-53.5 weeks). Two patients relapsed. No serious adverse events were recorded. CONCLUSIONS: Overall, seventy one percent of patients in this study respond to treatment, 100% of responders decrease their previous treatment. Rituximab was a well tolerated and no related serious side effects were recorded during the study period.

Leucemia linfoblástica aguda diagnosticada y tratada inicialmente como artritis crónica juvenil / Acute lymphoblastic leukaemia initially diagnosed and treated as chronic juvenile arthritis

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¿Qué ha cambiado de los pacientes oncológicos en UCIP? / What has changed in the oncology patients in the PICU?

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[Arsenic trioxide treatment in a patient with recurrent acute promyelocytic leukemia (APL)]. / Tratamiento con trióxido de arsénico en paciente con recaída de leucemia promielocítica aguda.

Acute promyelocytic leukemia (APL) is a special subtype of acute myeloid leukemia (AML) with M3 morphology and specific chromosomal translocation t(15;17) with resultant leukemogenic PML-RARalpha fusion gene. Its main characteristics are therapy response using all-trans-retinoic acid (ATRA) and its high rate of recovery; higher than other subtypes of acute myeloid leukemia. Arsenic trioxide induces a high complete remission rate in patients having an APL relapse. Experience in the use of this drug is still limited in the field of Paediatrics.