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CCI-007, a novel small molecule with cytotoxic activity against infant leukemia with MLL rearrangementsIdentified CCI-007 as a novel small molecule that displays rapid toxicity towards a subset of MLL-r, CALM-AF10 and SET-NUP214 leukemia cell lines
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Successful Treatment of Congenital Erythroleukemia With Low-Dose Cytosine ArabinosideWe report a term male with congenital acute erythroleukemia who achieved sustained remission with low-dose cytosine arabinoside alone
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The role of CCN family genes in haematological malignanciesHaematological malignancies, although a broad range of specific disease types, continue to show considerable overlap in classification, and patients are...
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Glucocorticoid resistance in T-lineage acute lymphoblastic leukaemia is associated with a proliferative metabolismWe examined the baseline profile of a panel of T-ALL cell lines to determine factors that contribute to GC resistance without prior drug selection.
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Efficacy of DYRK1A inhibitors in novel models of Down syndrome acute lymphoblastic leukemiaDespite significant advances, outcomes for children with Down syndrome (DS, trisomy 21) who develop acute lymphoblastic leukemia remain poor. Reports of large DS-ALL cohorts have shown that children with DS have inferior event-free survival and overall survival compared to children without DS.
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Biologic and Clinical Analysis of Childhood Gamma Delta T-ALL Identifies LMO2/STAG2 Rearrangements as Extremely High RiskAcute lymphoblastic leukemia expressing the gamma delta T-cell receptor (γδ T-ALL) is a poorly understood disease. We studied 200 children with γδ T-ALL from 13 clinical study groups to understand the clinical and genetic features of this disease. We found age and genetic drivers were significantly associated with outcome.
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Minimising Adverse Drug Reactions and Verifying Economic Legitimacy-Pharmacogenomics Implementation in Children (MARVEL- PIC): protocol for a national randomised controlled trialDNA-informed prescribing (termed pharmacogenomics, PGx) is the epitome of personalised medicine. Despite international guidelines existing, its implementation in paediatric oncology remains sparse.
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Invasive fungal infections in children with acute lymphoblastic leukaemia: Results from four Australian centres, 2003-2013Invasive fungal infections are more common in children with high-risk acute lymphoblastic leukaemia and in relapsed disease
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Taste evaluation of a novel midazolam tablet for pediatric patients: In vitro drug dissolution, in vivo animal taste aversion and clinical taste perception profilesThis study utilized in vitro, in vivo and clinical data to evaluate the palatability of a novel midazolam chocolate tablet.
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Systematic chemical and molecular profiling of MLL-rearranged infant acute lymphoblastic leukemia reveals efficacy of romidepsinIdentified romidepsin as a promising therapeutic for mixed lineage leukemia (MLL)-rearranged infant acute lymphoblastic leukemia