Survival and toxicity outcomes of hematopoietic stem cell transplantation for pediatric patients with Fanconi anemia: a unified multicentric national study from the Spanish Working Group for Bone Marrow Transplantation in Children.

Hematopoietic stem cell transplantation (HSCT) is currently the only curative option for hematological manifestations in patients with Fanconi anemia (FA). We report the outcome of 34 patients with FA inside a collaborative multicenter national study based on recommendations of Spanish Working Group for Bone Marrow Transplantation in Children (GETMON) between 2009 and 2016. Fludarabine-based conditioning regimen was carried out in all patients, with low dose total body irradiation in unrelated transplants. Disease status before HSCT was bone marrow failure (BMF) in 30 patients and myelodysplastic syndrome (MDS) in four. Donors were matched siblings donors (MSD) in 18, matched unrelated donors (MUD) in 15, and one haploidentical donor. All except one patient engrafted. Cumulative incidence of grades II-IV acute graft-versus-host disease (GVHD) was 29% and 11% for chronic GVHD. Median follow-up after HSCT was 6.5 years. Seven patients (21%) died due to transplant-related causes, two (6%) because of MDS relapse, and one (3%) after a squamous cell carcinoma. Overall survival (OS) was 73% at 5 years post-transplant, with no differences between MSD and MUD transplants. OS for patients with BMF was 80% while for MDS was 25%. Our data suggest HSCT can cure hematologic manifestations of most FA patients with BMF.

Nuevos fármacos en oncología pediátrica / New drugs in pediatric oncology

Actualmente el 75-80% de los niños que padecen una neoplasia conseguirán sobrevivir. El resto de pacientes fallecerán a causa del cáncer, la gran mayoría a causa de la progresión de su tumor. A pesar de los grandes avances en los tratamientos antineoplásicos desarrollados en las últimas décadas, aún existen tumores que por su histología, localización o bien por su extensión al diagnóstico, resultan muy difíciles de curar. También tumores inicialmente sensibles a la quimioterapia se hacen resistentes y progresan a pesar de todo tipo de tratamiento. Es indudable que se necesitan más armas terapéuticas para luchar contra estos casos de mal pronóstico. Aunque el desarrollo de nuevos antineoplásicos para tumores infantiles es más lento que en los adultos, varios medicamentos ofrecen resultados preliminares muy esperanzadores: anticuerpos monoclonales, como rituximab y gemtuzumab, ozogamicin en neoplasias hematológicas; inhibidores de la topoisomerasa I, como topotecan e irinotecan en los tumores sólidos; y los antiangiogénicos, en estudio para todo tipo de tumores son algunos ejemplos. Existen muchas drogas en desarrollo, aún en fases muy preliminares. Es de esperar que en no mucho tiempo, gracias a ellas, mejore el pronóstico de los tumores en los que aún nos e consigue una buena supervivencia (AU)

Currently, 75%-80% of children who suffer a neoplasm survive it. The rest of the patients will die because of the cancer, most because of tumor progression. Tumors still exist in spite of the great advances inantineoplastic treatment developed in recent decades. However, these are very difficult to cure due to their histology, location or extension on diagnoses. In addition, tumors that are initially sensitive to chemotherapy become resistant and progress in spite of all the different types of treatment. Undoubtedly, more therapeutic armamentarium is needed to fight against these cases having poor prognosis. Although the development of new antioneoplastics for childhood tumors is slower than in the adults, several drugs offer very encouraging preliminary results. A few examples are monoclonal antibodies such as Rituximab and Gemtuzumab Ozogamicin in blood neoplasms; Topoisomerase I inhibitors such as Topotecan and Irinotecan in solid tumors, and antigenic ones, under study for all types of tumors. There are many drugs still being developed, although in very preliminary phases. It is expected that thanks to them, the prognoses of the tumors for which good survival rate has not been achieved as of yet will improve in a short time (AU)

Caso infantil de polineuropatía del paciente crítico / A paediatric case report of critical illness polyneuropathy

No disponible

Síndromes hemofagocíticos: pensar en ellos... porque existen / Hemophagocitic syndromes: think of them... because they are a reality

No disponible

Secuelas endocrinológicas del tratamiento del cáncer infantil / Endocrine sequellae of childhood cancer survivors

Se presentan las secuelas endocrinológicas y auxiológicas, secundarias a radioterapia y quimioterapia, de 136 niños y adolescentes supervivientes de cáncer. Nuestro objetivo es elaborar un protocolo que facilite su seguimiento.Métodos: Se incluyeron en el estudio 136 niños (78 niños y 57 niñas), todos habían recibido radioterapia y/o quimioterapia, con/sin cirugía. Padecieron el tumor a la edad de 5.4 ñ 3.8 años (0.1-16). El estudio endocrinológico se realizó con 12.0 ñ 4.6 años de edad (2-22). Todos llevaban al menos 2 años en remisión. Recibieron radioterapia 71 niños (45 en el SNC). Dosis de radioterapia: 41 ñ 24.4 Gy (7-98).Resultados: Se observó una pérdida de percentil de talla de 13.5 puntos (centiles). Esta pérdida fue significativamente mayor entre los niños que recibieron radioterapia (18.9 puntos frente a 7.0 sin radioterapia). El peso sufrió un incremento de 5.5 puntos. Se detectaron las siguientes anomalías: talla baja no endocrina en 11 niños, déficit de GH en 19, patología tiroidea en 15, edad ósea/pubertad adelantada en 10, hipogonadismo en 11, y otras en 24 niños. Todas las alteraciones excepto el hipogonadismo fueron más frecuentes en los niños que recibieron radioterapia. El déficit de GH y la pubertad adelantada fueron más frecuentes entre los que recibieron irradiación craneal.Conclusión: Todos los niños supervivientes de cáncer precisan seguimiento a largo plazo (AU)

[Neutropenic enterocolitis in children with cancer]. / La enterocolitis neutropénica en el niño con cáncer.

OBJECTIVES: Neuropenic enterocolitis (NEC) is a destructive lesion of the ileocecal region occurring in cancer patients treated with chemotherapy. Its clinical picture is one of febrile acute abdominal extension with bloody diarrhea and low neutrophil counts. Our aim was to determine the incidence of NEC in children with cancer and to review the indications of surgery in these cases. MATERIAL AND METHODS: The records of children with cancer treated with chemotherapy in the last 6 years at Hospital Infantile La Paz were reviewed. We selected those patients who had abdominal pain and neutropenia and whose physical examination and radiological findings were consistent with NEC. RESULTS: Twelve cases of NEC were diagnosed during this period among 432 malignancies. The symptoms most frequently seen were abdominal pain and distension, nausea and vomiting. The neutrophil count was consistently below 500/ml. All patients were receiving chemotherapy before the onset of the clinical picture. Five children were operated upon. In three of these we found various ileocecal perforations, in one a gastric perforation and in the remaining one a diffuse inflammation of the ileocecal area. Two non-operated patients died from NEC. The remaining children recovered without problems with medical therapy. CONCLUSION: Pediatric surgeons treating neutropenic cancer patients should be familiar with this condition, that must be suspected early in granulocytopenic patients with acute abdominal extension. Aggressive surgical management is indicated in cases with severe peritonitis, bowel perforation or massive lower gastrointestinal bleeding, irrespective of the degree of neutropenia. Medical support should aim at reestablishing normal neutrophil counts.