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The article offers information on a study related to the epidemiology of Recombinant urate oxidase (rasburicase) use in paediatric patients with acute lymphoblastic leukaemia and non-Hodgkin lymphoma. It notes the use of Pediatric Health Information System database (PHIS) for the study which include demographics, diagnosis codes and daily resource utilization data.

Despite the importance of adverse event (AE) reporting, AEs are under-reported on clinical trials. We hypothesized that electronic medical record (EMR) data can ascertain laboratory-based AEs more accurately than those ascertained manually. EMR data on 12 AEs for patients enrolled on two Children's Oncology Group (COG) trials at one institution were extracted, processed and graded. When compared to gold standard chart data, COG AE report sensitivity and positive predictive values (PPV) were 0-21·1% and 20-100%, respectively. EMR sensitivity and PPV were >98·2% for all AEs. These results demonstrate that EMR-based AE ascertainment and grading substantially improves laboratory AE reporting accuracy. [ABSTRACT FROM AUTHOR]

Although inferior outcomes of children with Down syndrome ( DS) and acute lymphoid leukaemia ( ALL) are established, national supportive care patterns for these patients are unknown. A validated retrospective cohort of paediatric patients diagnosed with ALL from 1999 to 2011 was assembled from the US Pediatric Health Information System ( PHIS) database to examine organ toxicity, sepsis, and resource utilization in children with and without DS. Among 10699 ALL patients, 298 had DS- ALL (2·8%). In a multivariate model, DS was associated with increased risk of cardiovascular (odds ratio [ OR] 2·0, 95% confidence interval [ CI] 1·6-2·7), respiratory ( OR 2·1, 95% CI: 1·6-2·9), neurologic ( OR 3·4, 95% CI 1·9-6·2), and hepatic ( OR 1·4, 95% CI 1·0-1·9) dysfunction and sepsis ( OR 1·8, 95% CI: 1·4-2·4). Children with DS- ALL used significantly more respiratory support, insulin, and anti-infectives, including broad-spectrum Gram-positive agents, quinolones, and azoles. They used significantly fewer analgesics and antiemetics compared to non- DS- ALL children. Ultimately, this study confirms the increased risk of infectious and end-organ toxicity in children with DS- ALL and quantifies important differences in resource utilization between children with DS and non- DS ALL. These findings highlight the importance of investigating the impact of these care variations and developing specific supportive care guidelines for this population. [ABSTRACT FROM AUTHOR]

The bone marrow failure syndromes ( BMFS) are a heterogeneous group of rare blood disorders characterized by inadequate haematopoiesis, clonal evolution, and increased risk of leukaemia. Single nucleotide polymorphism arrays ( SNP-A) have been proposed as a tool for surveillance of clonal evolution in BMFS. To better understand the natural history of BMFS and to assess the clinical utility of SNP-A in these disorders, we analysed 124 SNP-A from a comprehensively characterized cohort of 91 patients at our BMFS centre. SNP-A were correlated with medical histories, haematopathology, cytogenetic and molecular data. To assess clonal evolution, longitudinal analysis of SNP-A was performed in 25 patients. We found that acquired copy number-neutral loss of heterozygosity ( CN- LOH) was significantly more frequent in acquired aplastic anaemia ( aAA) than in other BMFS (odds ratio 12·2, P < 0·01). Homozygosity by descent was most common in congenital BMFS, frequently unmasking autosomal recessive mutations. Copy number variants ( CNVs) were frequently polymorphic, and we identified CNVs enriched in neutropenia and aAA. Our results suggest that acquired CN- LOH is a general phenomenon in aAA that is probably mechanistically and prognostically distinct from typical CN- LOH of myeloid malignancies. Our analysis of clinical utility of SNP-A shows the highest yield of detecting new clonal haematopoiesis at diagnosis and at relapse. [ABSTRACT FROM AUTHOR]