Children represent a minority of total COVID-19 cases, but studies have reported severe disease and death in pediatric patients. Remdesivir (RDV) has recently demonstrated promising results in adults with COVID-19, but few data have been reported to date in children. A nationwide multicenter observational study was conducted on children with confirmed SARS-CoV-2 receiving compassionate treatment with RDV in Spain. Eight patients were included in the study, four infants and four older children [median age 5 years old; IQR 4 months–11.6 years old]. Half of them had complex underlying medical conditions, and the rest were mostly infants (3/4). Six out of eight children needed Pediatric Intensive Care Unit Admission. No RDV-related adverse outcomes were observed in our patients. Seven have reached successful clinical outcome, but one patient with serious clinical status died due to complications. However, she received RDV very late after the first COVID-19 symptom. Conclusions : In our cohort, most of the patients achieved successful clinical outcome, without observing adverse events. Clinical trials of RDV therapy for children with COVID-19 are urgently needed, to assess the safety, tolerability, efficacy, and pharmacokinetics of RDV in children, as this could be an effective treatment in severe cases. What is Known: • Remdesivir has not been approved to treat COVID-19 in children under 12 years old, although the drug is currently being prescribed in critically ill children. • Remdesivir has recently demonstrated promising results in adults with COVID-19, but few data have been reported to date in paediatric population. What is New: • We report a multicentre cohort of children with confirmed SARS-CoV-2 and severe COVID-19 disease receiving remdesivir during the first month of the pandemic in Spain. • No remdesivir-related adverse outcomes were observed in most of the cases. Seven patients reached successful clinical outcome, and one died due to complications (bacterial sepsis). Supplementary Information The online version contains supplementary material available at 10.1007/s00431-020-03876-1.

Severe combined immunodeficiency is an inherited disease with profoundly defective T cells, B cells, and natural killer cells. X-linked severe combined immunodeficiency is the most common form. In this report, we describe a 4-month-old male infant who was admitted to our hospital with progressive breathlessness and abdominal mass. He was diagnosed with hepatoblastoma and presented a pneumocystis jirovecii pneumonia at the beginning of chemotherapy. Definitive diagnosis of X-linked severe combined immunodeficiency was established by DNA analysis of the interleukin 2 receptor gamma chain gene. This case is the first report which describes an X-linked severe combined immunodeficiency patient with hepatoblastoma.

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Background and aimsDespite HLH-04 diagnostic criteria and treatment protocol for hemophagocytic lymphohistiocytosis (HLH) were proposed for both primary (pHLH) and secondary (sHLH) cases, several experts have criticised they are not optimum for the latter.The aims of this investigation are to describe the features of HLH patients that can help to differenciate between primary and secondary cases.MethodsRetrospective descriptive study in of HLH patients from 0 to 14 years, diagnosed according to HLH-04 criteria, in a tertiary Paediatric Hospital during the last 10 years (Jan2007-Jan2017).Clinical, analytical and therapeutic data were retrospectively collected from medical records. pHLH and sHLH features were compared in a bivariant analysis. Statistical significance was considered as p<0.1.ResultsWe found 13 patients, 3pHLH and 10sHLH, whose features are described in Table 1.The median age was 1.5 years (IR: 0.75–3.3), earlier in primary cases (1.0y vs 1.8y), though not significantly. 2 of 3 pHLH had grey hair and neurological impairment and were part of a consanguineous Moroccan family, with cousins diagnosed with Griscelli syndrome, so pHLH was suspected since the begining.CNS injury was more frequent in pHLH (66%) than in sHLH (10%)(p=0.04). There were no significant differences between other organ involvement.At onset, ferritin (p=0.043), neutrophils (p=0.05) and platelets (p=0.28) values were higher in sHLH than in pHLH. Lowest number of neutrophils (p=0.028), highest LDH levels (p=0.036) and minimum platelets count (p=0.07) were higher in sHLH too. There were no differences in other analytical features.Regarding treatment, pHLH cases needed steroids more often (p=0.06), as well as agressive therapies as etoposide (p=0.05) and intrathecal methotrexate (p=0.05).Mortality was significantly higher in primary group (p=0.03).ConclutionSIn our experience pHLH and sHLH have important disparities according to evolution, mortality and treatment.Although 2 of our pHLH had physical appereance and family background that helped with etiological diagnosis, it is not always that easy. In this study, we found pHLH and sHLH have important differences, according to clinical and laboratory findings, that may have implications in subsequent management.Larger multicentric More studies are required to establish specific diagnostic criteria and therapeutic schemes depending on the aetiology.Abstract OC64 Table 1Features of primary and secondary HLH in a tertiary Paediatric Hospital pHLH (n=3) sHLH (n=10)pHLH (n=3)sHLH (n=10) Male, n (%)2 (66.7)3 (30)Age (years), median (IR)1 (0.5–1)1.8 (0.8–4)Aetiology, n (%)Griscelli syndrome: 2 (66.7)Viral infections:6 (60): 3 CMV, 2 EBV, 1MUNC deficiency: 1 (33.3)Enterovirus Rheumatic diseases:2 (20)Length of fever at diagnosis (days), median (IR)7 (2–7)8 (2.7–11.7)Organ involvement Liver2 (66.7)1 (10) CNS2 (66.7)1 (10) Kidney0 (0)1 (10) Hemodinamics0 (0)3 (30) Lung0 (0)1 (10)Hepatomegaly, n (%)3 (100)10 (100)Splenomegaly, n (%)3 (100)9 (90)Haemoglobin (mg/dl), median (IR) aD7.6 (6.9–7.6)9 (8.4–...