Confusion between Two Amphotericin B Formulations Leading to a Paediatric Rehospitalisation.

A heavily immunosuppressed, 43-kg, 9-year-old patient was recovering from a bone marrow transplant. Primary prophylaxis against invasive fungal infections was liposomal amphotericin B (AmBisome®, 2.3 mg/kg [100 mg] two times per week). Once home, following a first amphotericin B infusion, he presented with strong diarrhoea and vomiting; this was repeated after the second infusion. The clinical situation worsened rapidly and the patient was rehospitalised. On admission, he presented with acute renal failure. During the 2-week hospitalisation, renal function recovered progressively. A few days after returning home, a new administration of amphotericin B was again followed by diarrhoea and vomiting, together with shivering and fever. The child was again rapidly rehospitalised. Investigation revealed that the community pharmacist, relying on drug software, had selected an inappropriate substitute drug: the patient had been administered amphotericin B deoxycholate (Fungizone®) and not liposomal amphotericin B. Depending on the indication, intravenous AmBisome® is usually administered at a dose between 3 and 5 mg/kg bodyweight; this dose can be increased to up to 10 mg/kg/day. Intravenous Fungizone®, however, should be administered using an initial dose of 0.25 mg/kg bodyweight, up to a recommended 1-mg/kg/day dose. The child had thus received 100 mg of Fungizone®, or ten times the recommended dose.

Small Cell Carcinoma of the Ovary, Hypercalcemic Type: Report of a Bilateral Case in a Teenager Associated with SMARCA4 Germline Mutation.

Small cell carcinoma of the ovary, hypercalcemic type (SCCOHT), is a highly aggressive neoplasm that typically occurs in young females. Paraneoplastic hypercalcemia is associated in two thirds of the cases. Recent studies demonstrated that this rare tumor harbors the same molecular features of malignant rhabdoid tumor secondary to SMARCA4/BRG1 mutations. We illustrate herein a typical bilateral case of SCCOHT with comprehensive molecular characterization in a 14-year-old girl. We also discuss the value of SMARCA4 immunostaining in the diagnostic approach of undifferentiated ovarian and pelvic malignancies.

[Management of febrile neutropenias in adolescents and young adults: Differences of practice between adult and pediatric units]. / Gestion des aplasies fébriles chez les adolescents et jeunes adultes: différences de pratiques entre secteurs adultes et pédiatriques.

INTRODUCTION: Adolescents and young adults (AYA, 15-25years old) with cancer are treated either in adult or pediatric units. Management of febrile neutropenia (FN) is different between these units. Monitoring rules and indications of hospitalization are often stricter in pediatrics. This study evaluates if these differences influence the occurrence of complications. METHODS: The medical records of AYA patients treated in our institution in the Euro-E-W-I-N-G99 protocol between 01/09/2000 and 31/05/2013 were retrospectively analyzed. We studied febrile neutropenias occurring after VIDE courses, during the induction period. RESULTS: Forty-four patients were included (18 from adult units, 26 from pediatrics). Median age at inclusion was 19.6. After 260 courses of VIDE, we observed a median of 2 FN per adult and 3 per pediatric patient (P=0.2). Hospitalization occurred in median 1.5 time per adult and 3 per pediatric patient (P=0.008). Median cumulated length of stay was 4.5days for adults versus 16 days for pediatric patients (P=0.008). There was no significant difference for survival, number of documented infections, transfusions, dose modifications, chemotherapy delay, need for intensive care, infection after post-induction surgery. CONCLUSION: AYA treated in adult services are less frequently hospitalized for FN with no difference in morbi-mortality. Homogeneous recommendations could be made for these patients, whatever the units they are treated in.

[Genetic alterations in neuroblastoma and their usefulness for clinical management]. / Les altérations génétiques dans le neuroblastome et leur apport pour la prise en charge thérapeutique.

Neuroblastoma, the most frequent solid extracranial tumor of childhood, is characterized by a wide variability of its clinical course. The most important clinical prognostic markers are stage and age at diagnosis, but these markers are insufficient to predict outcome reliably and to determine treatment intensity. Recent evidence indicates that neuroblastoma can be considered as a "genetic disease", firstly by the recent observation that certain alleles of specific genes significantly increase the relative risk to develop neuroblastoma, and the discovery of mutations in genes such as ALK or PHOX2B in rare familial cases. On the other hand, a large number of recurrent genetic somatic alterations have been described in neuroblastoma. Recent technological advances, such as array-CGH (comparative genomic hybridisation), now enable the analysis of these markers in a single step and allow the definition of genomic profiles associated with typical clinical features. Numerical chromosome alterations are observed more frequently in tumors of younger children with localised disease and a good prognosis, whereas segmental chromosome alterations are found more frequently in tumors of older children with advanced stages of disease and a poorer outcome. Future therapeutic stratification schemes can make use of the tumor genomic profile by proposing less intense treatment for infants with a neuroblastoma harboring a favorable tumor genomic profile, while intensifying treatment in case of a defavorable tumor genomic profile. Such approaches require standardisation of the molecular techniques and the interpretation of results for application in international trials.