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        Safety and immunogenicity of a primary series and booster dose of the meningococcal serogroup B-factor H binding protein vaccine (MenB-FHbp) in healthy children aged 1–9 years: two phase 2 randomised, controlled, observer-blinded studies

        The meningococcal serogroup B-factor H binding protein vaccine (MenB-FHbp) is licensed for use in children aged 10 years or older for protection against invasive serogroup B meningococcal disease. Because young children are at increased risk of invasive meningococcal disease, MenB-FHbp clinical data in this population are needed. ...

        Research

        Clinical Implications of Minimal Residual Disease Detection in Infants With KMT2A-Rearranged Acute Lymphoblastic Leukemia Treated on the Interfant-06 Protocol

        Infant acute lymphoblastic leukemia (ALL) is characterized by a high incidence of KMT2A gene rearrangements and poor outcome. We evaluated the value of minimal residual disease (MRD) in infants with KMT2A-rearranged ALL treated within the Interfant-06 protocol, which compared lymphoid-style consolidation (protocol IB) versus myeloid-style consolidation (araC, daunorubicin, etoposide/mitoxantrone, araC, etoposide). ...

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        Clinical evidence for synergy between immunotherapy and radiotherapy (SITAR)

        Previous preclinical and clinical trials have shown promising antitumour activity and toxicity profile when employing the ‘Synergy between Immunotherapy and Radiotherapy’ (SITAR) strategy. Approximately, one in seven radiation therapy studies currently recruiting is investigating SITAR. ...

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        SJ-ELiOT: St Jude - Phase 1 Evaluation of LY2606368, Molecularly-Targeted CHK1/2i Therapy, in Combination with Cyclophosphamide or Gemcitabine for Children and Adolescents with Refractory or Recurrent Medulloblastoma Brain Tumours

        Nick Raelene Gottardo Endersby NG R MBChB FRACP PhD BSc (Hons) PhD Co-Head, Brain Tumour Research Brainchild Fellow; Co-Head, Brain Tumour Research nicholas.gottardo@telethonkids.org.au raelene.endersby@telethonkids.org.au Co-Head, Brain Tumour ...

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        Pertussis immunisation in infancy and atopic outcomes: A protocol for a population-based cohort study using linked administrative data

        The burden of IgE-mediated food allergy in Australian born children is reported to be among the highest globally. This illness shares risk factors and frequently coexists with asthma, one of the most common noncommunicable diseases of childhood. ...

        Research

        Using causal directed acyclic graphs (DAGs) to select patient-important outcomes in transplantation trials—interventions to treat polyomavirus infection as an example

        Tom Snelling T BMBS DTMH GDipClinEpid PhD FRACP Head, Infectious Disease Implementation Research 08 6319 1817 tom.snelling@telethonkids.org.au Head, Infectious Disease Implementation Research Professor Tom Snelling is Head of the Infectious Disease ...

        Research

        A novel transcriptional signature identifies T-cell infiltration in high-risk paediatric cancer

        Molecular profiling of the tumour immune microenvironment (TIME) has enabled the rational choice of immunotherapies in some adult cancers. In contrast, the TIME of paediatric cancers is relatively unexplored. We speculated that a more refined appreciation of the TIME in childhood cancers, rather than a reliance on commonly used biomarkers such as tumour mutation burden (TMB), neoantigen load and PD-L1 expression, is an essential prerequisite for improved immunotherapies in childhood solid cancers. ...

        Research

        Challenges in the Management of Childhood Intracranial Germ Cell Tumors in Middle-Income Countries: A 20-Year Retrospective Review From a Single Tertiary Center in Malaysia

        A higher incidence of pediatric intracranial germ cell tumors (iGCTs) in Asian countries compared with Western countries has been reported. In Malaysia, the literature regarding pediatric iGCTs have been nonexistent. The aim of this study was to review the management, survival, and long-term outcomes of pediatric iGCTs at a single tertiary center in Malaysia. ...

        Research

        'Walking their walk': Reducing conflict between families of ill children and the medical profession

        I review recent high-profile court cases to underscore the changing landscape of conflict and the central role that the media (and social media in particular) can play ...

        Research

        Interactions between acute lymphoblastic leukemia and bone marrow stromal cells influence response to therapy

        The cure rate for pediatric patients with B precursor acute lymphoblastic leukemia (pre-B ALL) is steadily improving, however relapses do occur despite... ...

        Research

        Meningiomas in children and adolescents: a meta-analysis of individual patient data

        The epidemiological, prognostic, and therapeutic features of child and adolescent meningioma are poorly defined... ...

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        An introduction to clinical trial design

        This manuscript will give a brief overview of clinical trial design including the strengths and limitations of various approaches ...

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