Proteus syndrome is a life-threatening segmental overgrowth syndrome caused by a mosaic gain-of-function AKT1 variant. There are no effective treatments for Proteus syndrome. Miransertib is an AKT1 inhibitor that, prior to this study, has been evaluated only in adult oncology trials.
View Article and Find Full Text PDFJ Radioanal Nucl Chem
August 2018
The objective was to study changes in water-saturated biodiesel irradiated by electron beam and to analyse them considering the influence of absorbed dose. Based on obtained results it can be concluded that irradiation did not affect ester groups in FAME molecules, but strongly influenced on double bonds. Total ester content decreased linearly with the increase in absorbed dose, causing FAME not to meet the requirement of PN-EN 14214 concerning the ester content (96.
View Article and Find Full Text PDFIntravenous (IV) busulfan test dose pharmacokinetics (PK) has been shown to accurately predict once-daily dose requirements and improve outcomes in adult transplant patients, but there are limited data to support this approach in children. Test doses of busulfan ∼0.8 mg/kg were infused over 2 to 3 hours, followed by serial sampling to 4-6 hours postinfusion in pediatric hematopoietic stem cell transplant recipients (n = 5).
View Article and Find Full Text PDFThe safety, tolerability, and pharmacokinetics of the liposomal formulation of amphotericin B (L-AMB) were evaluated in 40 immunocompromised children and adolescents. The protocol was an open-label, sequential-dose-escalation, multidose pharmacokinetic study with 10 to 13 patients in each of the four dosage cohorts. Each cohort received daily dosages of 2.
View Article and Find Full Text PDFAntimicrob Agents Chemother
December 2016
Liposomal amphotericin B (LAmB) is widely used in the treatment of invasive fungal disease (IFD) in adults and children. There are relatively limited pharmacokinetic (PK) data to inform optimal dosing in children that achieves systemic drug exposures comparable to those of adults. Our objective was to describe the pharmacokinetics of LAmB in children aged 1 to 17 years with suspected or documented IFD.
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