Haploidentical Hematopoietic Stem Cell Transplantation in Pediatric Patients with Acquired Hypocellular Bone Marrow Failure.

Children with acquired hypocellular bone marrow failure of unknown cause (AHBMF) are usually diagnosed either with severe aplastic anemia (SAA) or refractory cytopenia of childhood (RCC). Patients with AHBMF who lack a matched donor and who failed or relapsed after immunosuppressive therapy (IST) need alternative therapies. Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) offers a curative treatment for these patients. We report a multicenter Spanish experience with haplo-HSCT in pediatric patients with AHBMF. Eleven pediatric patients (SAA, n = 9; RCC, n = 2) underwent haplo-HSCT with different lymphodepletion strategies. Most patients (10 of 11) had previously failed to respond or relapsed after IST. The conditioning regimen was reduced intensity in SAA and myeloablative in RCC. Patients with SAA received low-dose radiotherapy as part of their conditioning regimen. All patients engrafted. Viral reactivation was common (8 of 11). Acute GVHD grade ≥II was seen in 5 patients. Chronic GVHD was diagnosed in 4 of the long-term survivors. Transplantation-associated microangiopathy was a frequent complication in SAA patients and was related to worse outcome. Two patients died of transplantation-related complications. Overall survival was 81%, with a median follow-up of 36 months. Haplo-HSCT can be a successful salvage curative treatment for pediatric patients with AHBMF, but with significant toxicities that must be addressed. Transplantation-associated microangiopathy was the most critical complication.

[Cancer in the first 18 months of life]. / Cáncer en los primeros 18 meses de vida.

INTRODUCTION: Oncological-haematological disease continues to be the first cause of non-traumatic mortality in childhood, as well as a significant cause of morbidity. The patient less than 18-months-old has special clinical, diagnostic, and therapeutic features that all paediatricians are interested in determining, with the aim of achieving greater survival and a lower morbidity throughout the lives of their patients. MATERIAL AND METHODS: A retrospective, descriptive study was carried out using the clinical, diagnostic, and therapeutic variables in patients less than 18-months-old diagnosed with an oncological-haematological that received chemotherapy in a Paediatric Oncology Unit between January 2007 and August 2019. RESULTS: A total of 72 patients were diagnosed with 76 cancers that required chemotherapy. The most common cancer was leukaemia (21 patients), followed by neuroblastoma (15 patients), and tumours of the central nervous system (12 patients). The presentation of "life-threatening symptoms" was seen in 20.8% of cases, particularly in tumours of neural origin (13/15). Although 18% of patients showed no symptoms on diagnosis, just over half (51%) of the diagnoses took place in the "advanced stages". Particularly in the case of solid tumours in which 23.6% were diagnosed with metastases. A significant percentage of genetic alterations implicated in the aetiopathogenesis of the different cancers were found. CONCLUSIONS: Cancer in the first stages of life is a diagnostic and therapeutic challenge due to its phenotypical diversity, its genetic load, and its therapeutic difficulties. Knowledge of its particular features is essential for its early and effective approach.

[Descriptive study of malaria cases in the paediatric population in a reference hospital in Valencia, Spain, between 1993 and 2015]. / Estudio descriptivo de los casos de malaria en la población pediátrica en un hospital de referencia de Valencia, España, entre 1993 y 2015.

INTRODUCTION: Malaria is considered to be the fourth leading cause of infant mortality after pneumonia, complications related to premature birth, and perinatal asphyxia. MATERIAL AND METHODS: A retrospective and descriptive study of cases of malaria confirmed and treated by the Paediatric Infectious Diseases Unit (age lower than 15 years) at the La Fe Hospital, Valencia, over the period 1993 to 2015. RESULTS: A total of 54 cases of paediatric malaria were diagnosed in the period 1993-2015, with 51.8% of these occurring in males, and 46.2% of patients were aged below 5 years. The majority of children came from Equatorial Guinea (68.5%). Only 5.6% had received antimalarial prophylaxis. Plasmodium falciparum was found to be the causal species in 81.4% of cases. Seven patients (13%) presented with complicated malaria. The most widely used treatment was quinine, either alone or in combination with other drugs. Atovaquone/proguanil was used from 2010 onwards and was indicated in 20.3% of the patients. The combination of artesunate/piperaquine/dihydroartemisinin began to be used in 2013. No deaths or relevant side effects were reported, and the clinical response was favourable in all children (100%). CONCLUSIONS: Malaria is still a prevalent disease in this population, a consequence of immigration, and tourism to endemic countries. Malaria should be considered as a likely diagnosis in a febrile child who comes from, or has travelled to, an endemic region in the past year.