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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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New therapeutic opportunities from dissecting the pre-B leukemia bone marrow microenvironmentWe provide evidence that targeting leukemia-induced bone loss is a therapeutic strategy for pre-B ALL
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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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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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Receptor mutation is not a common mechanism of naturally occurring glucocorticoid resistance in leukaemia cell linesGlucocorticoids (GCs) are among the most important drugs for the treatment of acute lymphoblastic leukaemia (ALL).
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Preclinical Assessment of Dactinomycin in KMT2A-Rearranged Infant Acute Lymphoblastic LeukemiaInfants with KMT2A-rearranged B-cell acute lymphoblastic leukemia (ALL) have high rates of relapse and poor survival compared with children. Few new therapies have been identified over the past twenty years. The aim of this study was to identify existing anti-cancer agents that have the potential to be repurposed for the treatment of infant ALL.
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Polyamine depletion limits progression of acute leukaemiaCancer cells are addicted to polyamines, polycations essential for cellular function. While dual targeting of cellular polyamine biosynthesis and polyamine uptake is under clinical investigation in solid cancers, preclinical and clinical studies into its potential in haematological malignancies are lacking. Here we investigated the preclinical efficacy of polyamine depletion in acute leukaemia.
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The critical role of the bone marrow stromal microenvironment for the development of drug screening platforms in leukemiaExtensive research over the past 50 years has resulted in significant improvements in survival for patients diagnosed with leukemia. Despite this, a subgroup of patients harboring high-risk genetic alterations still suffer from poor outcomes. There is a desperate need for new treatments to improve survival, yet consistent failure exists in the translation of in vitro drug development to clinical application.
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Pharmacokinetics of PEGasparaginase in Infants with Acute Lymphoblastic LeukemiaPEGasparaginase is known to be a critical drug for treating pediatric acute lymphoblastic leukemia (ALL), however, there is insufficient evidence to determine the optimal dose for infants who are less than one year of age at diagnosis. This international study was conducted to identify the pharmacokinetics of PEGasparaginase in infants with newly diagnosed ALL and gather insight into the clearance and dosing of this population.