Fri Jun 28, 2019 22:00
Abstract Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. The hypomethylating agent, azacitidine, has been used as a bridging therapy to transplant. However, no patients have been treated with azacitidine without an HSCT post azacitidine. We report on an infant with JMML with somatic KRAS G12A mutation and monosomy 7 who achieved sustained remission following azacitidine monotherapy. He...
Fri Jun 28, 2019 12:05
Fri Jun 28, 2019 12:04
Abstract We report two patients with leukaemia driven by the rare CNTRL‐FGFR1 fusion oncogene. This fusion arises from a t(8;9)(p12;q33) translocation, and is a rare driver of biphenotypic leukaemia in children. We used RNA sequencing to report novel features of expressed CNTRL‐FGFR1, including CNTRL‐FGFR1 fusion alternative splicing. From this knowledge, we designed and tested a Droplet Digital PCR assay that detects CNTRL‐FGFR1 expression to approximately one cell in 100 000 using fusion breakpoint‐specific...
Fri Jun 28, 2019 12:03
Abstract Background Pediatric oncohematologic patients are a high‐risk population for clinical deterioration that might require pediatric intensive care unit (PICU) admission. Several studies have described outcomes and mortality predictors for patients post hematopoietic stem cell transplantation (HSCT), but fewer data exist regarding the category of non‐HSCT patients. Procedure All oncohematologic non‐HSCT patients ≤18 years requiring PICU admission from 1998 to 2015 in our tertiary‐care...
Fri Jun 28, 2019 12:03
Abstract Routinely measurable biomarkers as predictors for adverse outcomes in febrile neutropenia could improve management through risk stratification. This systematic review assesses the predictive role of biomarkers in identifying events such as bacteraemia, clinically documented infections, microbiologically documented infection, severe sepsis requiring intensive care or high dependency care and death. This review collates 8319 episodes from 4843 patients. C‐reactive protein (CRP), interleukin...
Fri Jun 28, 2019 12:03
Abstract Background/Objectives Stagnant blood flow present in slow‐flow vascular malformations can lead to localized intravascular coagulopathy (LIC), measured by elevated D‐dimer levels, low fibrinogen, and/or thrombocytopenia. LIC can lead to localized thrombosis and/or bleeding, resulting in pain, swelling, and functional limitations. Patients with complex vascular malformations treated with sirolimus show clinical improvement in these symptoms. We hypothesized that the clinical benefits of...
Fri Jun 28, 2019 12:03
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