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Research
Priorities for improved management of acute rheumatic fever and rheumatic heart diseaseThe results suggest that timely injection and patient education are priorities for managing ARF and RHD, particularly focusing on child-to-adult transition care.
Research
Knowledge translation lessons from an audit of Aboriginal Australians with acute coronary syndrome presenting to a regional hospitalThis audit is used as a case study of translating knowledge processes in order to identify the factors that support equity-oriented knowledge translation.
Research
Exploiting the reactive oxygen species imbalance in high-risk paediatric acute lymphoblastic leukaemia through auranofinThe prognosis for high-risk childhood acute leukaemias remains dismal and established treatment protocols often cause long-term side effects in survivors. This study aims to identify more effective and safer therapeutics for these patients.
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Research
Delayed airway epithelial repair is correlated with airway obstruction in young adults born very pretermNasal epithelial cells from young adults with a history of very preterm birth show delayed closure following scratch-wounding. Repair correlated with lung function, suggesting epithelial barrier integrity may play a role in preterm-associated lung disease.
Research
The potential of antisense oligonucleotide therapies for inherited childhood lung diseasesAntisense oligonucleotides are an emerging therapeutic option to treat diseases with known genetic origin. In the age of personalised medicines, antisense oligonucleotides can sometimes be designed to target and bypass or overcome a patient's genetic mutation, in particular those lesions that compromise normal pre-mRNA processing. Antisense oligonucleotides can alter gene expression through a variety of mechanisms as determined by the chemistry and antisense oligomer design.
Research
Blinatumomab Added to Chemotherapy in Infant Lymphoblastic LeukemiaKMT2A-rearranged acute lymphoblastic leukemia (ALL) in infants is an aggressive disease with 3-year event-free survival below 40%. Most relapses occur during treatment, with two thirds occurring within 1 year and 90% within 2 years after diagnosis. Outcomes have not improved in recent decades despite intensification of chemotherapy.
Research
Survival Outcomes of Children with Relapsed or Refractory Myeloid Leukemia Associated with Down syndromeChildren with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population.
Research
Acute Lymphoblastic Leukemia in Infants: A Distinctive, High-Risk Subtype of Childhood Acute Lymphoblastic LeukemiaAcute lymphoblastic leukemia (ALL) in infants younger than 1 year of age is an aggressive, high-risk subtype of childhood ALL. Infant ALL with KMT2A-r is characteristically poorly responsive to chemotherapy and hematopoietic stem cell transplantation. New strategies, such as molecularly targeted therapies and immunotherapies, are in development and show promise in preclinical models and early phase studies.
News & Events
Drug find could represent big win for our little patientsDr Rishi Kotecha knows too well the devastation of a leukaemia diagnosis in a child, treating children as a consultant at Princess Margaret Hospital.