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A novel, palatable paediatric oral formulation of midazolam: Pharmacokinetics, tolerability, efficacy and safetyWe conclude that the novel chocolate-based formulation of midazolam provides improved tolerability while remaining efficacious
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Optimized peripheral blood progenitor cell mobilization for autologous hematopoietic cell transplantation in children with high-risk and refractory malignanciesOur approach to hematopoietic progenitor cells mobilization resulted in highly effective HPC harvest in children and adolescents with high-risk cancers
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Primary central nervous system lymphoma: Initial features, outcome, and late effects in 75 children and adolescentsChildren with Primary Central Nervous System Lymphoma and no immunodeficiency have a good outcome
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Rare childhood cancers—an increasing entity requiring the need for global consensus and collaborationRare childhood cancers have not benefited to the same extent from the gains that have been made for their frequently occurring counterparts.
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Childhood craniopharyngioma: 20-year institutional experience in Western AustraliaAlthough neurocognitive, psychological and behavioural problems were noted for some patients during medical review, only 20% of patients were formally assessed.
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Deregulated expression of connective tissue growth factor is linked to poor outcome in human cancerConnective tissue growth factor (CTGF/CCN2) has long been associated with human cancers. The role it plays in these neoplasms is diverse and tumour specific.
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Updates in infant acute lymphoblastic leukemia and the potential for targeted therapyOutcomes for infants diagnosed under 1 year of age with KMT2A-rearranged acute lymphoblastic leukemia (ALL) have remained stagnant over the past 20 years. Successive treatment protocols have previously focused on intensification of conventional chemotherapy, but increased treatment-related toxicity and chemoresistance have led to a plateau in survival.
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Systematic In Vitro Evaluation of a Library of Approved and Pharmacologically Active Compounds for the Identification of Novel Candidate Drugs for KMT2A-Rearranged LeukemiaPatients whose leukemias harbor a rearrangement of the Mixed Lineage Leukemia (MLL/KMT2A) gene have a poor prognosis, especially when the disease strikes in infants. The poor clinical outcome linked to this aggressive disease and the detrimental treatment side-effects, particularly in children, warrant the urgent development of more effective and cancer-selective therapeutics.
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Successful treatment of a child with acute monoblastic leukaemia who relapsed with T-cell acute lymphoblastic leukaemia: A rare lineage switchRishi S. Kotecha MB ChB (Hons) MRCPCH FRACP PhD Co-Head, Leukaemia Translational Research rishi.kotecha@health.wa.gov.au Co-Head, Leukaemia
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Constitutive Activation of RAS/MAPK Pathway Cooperates with Trisomy 21 and Is Therapeutically Exploitable in Down Syndrome B-cell LeukemiaChildren with Down syndrome (constitutive trisomy 21) that develop acute lymphoblastic leukemia (DS-ALL) have a 3-fold increased likelihood of treatment-related mortality coupled with a higher cumulative incidence of relapse, compared with other children with B-cell acute lymphoblastic leukemia (B-ALL).