Pediatric blood & cancer
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Pediatric blood & cancer · Aug 2012
ReviewMicronutrients and sickle cell disease, effects on growth, infection and vaso-occlusive crisis: a systematic review.
Patients with Sickle cell disease (SCD) exhibit signs of poor growth, increased susceptibility to infection and recurrent episodes of painful vaso-occlusive crises. Micronutrient deficiencies may increase susceptibility to these outcomes. We conducted a systematic review to assess the strength of evidence for improved outcomes related to micronutrient interventions. ⋯ Zinc supplementation was associated with improved growth and decreased incidence of infection and is a promising intervention in the management of SCD patients. Omega-3 fatty acid supplementation was associated with limited reduction in vaso occlusive crises. This review identifies key knowledge gaps, which are important research priorities for nutritional interventions.
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Pediatric blood & cancer · Aug 2012
Initial testing of the investigational NEDD8-activating enzyme inhibitor MLN4924 by the pediatric preclinical testing program.
MLN4924 is an investigational first-in-class small molecule inhibitor of NEDD8-activating enzyme (NAE). NAE is an essential component of the NEDD8 conjugation pathway, controlling the activity of a subset of ubiquitin-proteasome system (UPS) E3 ligases, multiprotein complexes that transfer ubiquitin molecules to substrate proteins. ⋯ MLN4924 showed potent activity in vitro and in vivo showed tumor growth inhibitory activity against a subset of the PPTP solid tumor and ALL xenografts.