Pediatric blood & cancer
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Pediatric blood & cancer · Feb 2016
Design of the DOVE (Determining Effects of Platelet Inhibition on Vaso-Occlusive Events) trial: A global Phase 3 double-blind, randomized, placebo-controlled, multicenter study of the efficacy and safety of prasugrel in pediatric patients with sickle cell anemia utilizing a dose titration strategy.
Sickle cell disease (SCD) is an inherited blood disorder characterized by painful vaso-occlusive crises (VOC) with limited treatment options, particularly for children. Emerging knowledge of the pathophysiology of SCD suggests antiplatelet therapies may hold promise for treatment of VOC. Multiple small studies have evaluated antiplatelet agents on the frequency of VOC with varying results, but there has not been an adequately powered study to definitively determine the effect of antiplatelet agents on VOC. Prasugrel, a third-generation thienopyridine that irreversibly inhibits platelet activation and aggregation, is approved in adults with acute coronary syndrome managed with percutaneous coronary intervention. ⋯ DOVE presents a unique opportunity to determine whether antiplatelet therapy reduces frequency of patient-reported VOC and daily vaso-occlusive pain in a global study of children with SCA. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.
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Pediatric blood & cancer · Jan 2016
Multicenter StudyHydroxyurea Therapy for Children With Sickle Cell Anemia in Sub-Saharan Africa: Rationale and Design of the REACH Trial.
Sickle cell anemia (SCA) is an inherited hematological disorder that causes a large but neglected global health burden, particularly in Africa. Hydroxyurea represents the only available disease-modifying therapy for SCA, and has proven safety and efficacy in high-resource countries. In sub-Saharan Africa, there is minimal use of hydroxyurea, due to lack of data, absence of evidence-based guidelines, and inexperience among healthcare providers. ⋯ REACH will provide data that address critical gaps in knowledge for the treatment of SCA in sub-Saharan Africa. By developing local expertise with the use of hydroxyurea and helping to establish treatment guidelines, the REACH trial results will have the potential to transform care for children with SCA in Africa.
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Pediatric blood & cancer · Jan 2016
A Prospective Cohort Quality Improvement Study to Reduce the Time to Antibiotics for New Fever in Neutropenic Pediatric Oncology Inpatients.
Fever and neutropenia (F&N) is a pediatric oncology emergency due to the risk of disseminated infection. Quality improvement (QI) efforts to improve time to antibiotics for F&N in the emergency department have been documented, but the issue has not been studied in the established inpatient setting. ⋯ Daily discussion of a fever contingency plan appears effective in decreasing the time to antibiotics for neutropenic pediatric oncology inpatients with new fever, likely by circumventing the need for multi-level discussion of the antibiotic plan when fever is identified.
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Pediatric blood & cancer · Jan 2016
Metastatic Low-Grade Gliomas in Children: 20 Years' Experience at St. Jude Children's Research Hospital.
Patients with low-grade gliomas (LGG), which are the most common childhood brain tumors, have excellent long-term survival. Dissemination of LGG is rare. Robust data on the incidence, presentation, patterns of dissemination, disease behavior, outcome, and best-management approaches do not exist. We describe 20 years of follow-up of children with metastatic LGG. ⋯ This study describes the longest follow-up of children with metastatic LGG. LGG is underestimated and entails major morbidity and mortality. Prospective studies are needed to learn the true incidence, study the biology, and determine the best approaches to diagnosis, treatment, and follow-up.
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Pediatric blood & cancer · Jan 2016
Trajectory of Material Hardship and Income Poverty in Families of Children Undergoing Chemotherapy: A Prospective Cohort Study.
Poverty is correlated with negative health outcomes in pediatric primary care, and is emerging as a negative prognostic indicator in pediatric oncology. However, measures of poverty amenable to targeted intervention, such as household material hardship (HMH)--including food, energy, and housing insecurity--have not been described in pediatric oncology. We describe the trajectory of family reported HMH and income poverty at a pediatric oncology referral center in New England with high psychosocial supports. ⋯ Low-income and HMH are prevalent in a significant proportion of newly diagnosed pediatric oncology families at a large referral center. Despite psychosocial supports, the proportion of families experiencing unmet basic needs increases during chemotherapy to nearly one in three families. HMH provides a quantifiable and remediable measure of poverty in pediatric oncology. Interventions to ameliorate this concrete component of poverty could benefit a significant proportion of pediatric oncology families.