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Novel CT domain-encoding splice forms of CTGF/CCN2 are expressed in B-lineage acute lymphoblastic leukaemiaConnective tissue growth factor (CTGF/CCN2) has been shown previously to be aberrantly expressed in a high proportion of paediatric precursor B cell acute...
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Heterogeneity in mechanisms of emergent resistance in pediatric T-cell acute lymphoblastic leukemiaBiological changes associated with T-ALL relapse and resistance are stochastic and highly individual
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A Novel Missense Mutation Affecting the N-terminal Domain of SAP Protein in X-linked Lymphoproliferative DiseaseWe have revealed a novel SH2D1A gene mutation in a patient with XLP resulting in fulminant refractory EBV-driven HLH, which is a recognized severe complication
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Risk factors for symptomatic venous thromboembolism during therapy for childhood acute lymphoblastic leukemiaWe found two known risk factors in a large cohort of children treated for ALL and identified other factors associated with venous thromboembolism
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Challenges posed by COVID-19 to children with cancerDevelopment of standardised guidance by national and regional authorities for reducing the risk of SARS-CoV-2 transmission to children with cancer
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Immunogenicity of the inactivated influenza vaccine in children who have undergone allogeneic haematopoietic stem cell transplantThis study provides evidence to support annual inactivated influenza vaccine administration to children following allogeneic haematopoietic stem cell transplant
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Invasive fungal disease and antifungal prophylaxis in children with acute leukaemia: a multicentre retrospective Australian cohort studyInvasive fungal disease (IFD) is a common and important complication in children with acute myeloid leukaemia (AML). We describe the epidemiology of IFD in a large multicentre cohort of children with AML.
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KMT2A-rearranged acute lymphoblastic leukaemiaKMT2A-rearranged acute lymphoblastic leukaemia (ALL) represents a high risk subtype of childhood ALL. Historical treatment strategies have comprised of intensification with conventional chemotherapy. However, outcomes have remained consistently poor compared to the advances that have been seen for other ALL subtypes, particularly for infants diagnosed before their first birthday
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Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AMLHematopoietic cell transplantation (HCT) is an effective treatment for pediatric patients with high-risk, refractory, or relapsed acute myeloid leukemia (AML). However, a large proportion of transplanted patients eventually die due to relapse. To improve overall survival, we propose a combined strategy based on cord blood (CB)-HCT with the application of AML-specific T cell receptor (TCR)-engineered T cell therapy derived from the same CB graft.
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Precision-guided treatment in high-risk pediatric cancersRecent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment.