Estimation Of Transient Increases In Bleeding Risk Associated With Physical Activity In Children With Haemophilia
Funder
National Health and Medical Research Council
Funding Amount
$102,143.00
Summary
Haemophilia A and B are genetic conditions which affect 1 in 7,000 males in Australia. These disorders cause frequent bleeding due to problems with the clotting factor in blood. Over the past decade there has been a move to administer clotting factor to children with haemophilia in order to prevent bleeds and the consequent damage to joints that occurs when bleeds occur in a joint. Participation in vigorous physical activity and sport is thought to increase the risk of bleeding. Because of this, ....Haemophilia A and B are genetic conditions which affect 1 in 7,000 males in Australia. These disorders cause frequent bleeding due to problems with the clotting factor in blood. Over the past decade there has been a move to administer clotting factor to children with haemophilia in order to prevent bleeds and the consequent damage to joints that occurs when bleeds occur in a joint. Participation in vigorous physical activity and sport is thought to increase the risk of bleeding. Because of this, children are often given clotting factor prior to playing sport. However clotting factor is extremely expensive. For example, a boy wanting to play tennis three times a week would require three injections of cIotting factor per week at a cost of approximately $250,000 a year. To date there is no good evidence about which physical activities are likely to increase the risk of bleeding. If this information was available clinicians would be able to optimise timing of administration of clotting factor so that it is administered prior to activities associated with high risk of bleeds. Another reason to quantify risk of bleeds associated with activity is to inform decisions about participation in physical activity. Every boy with haemophilia wants to know if he can play sport or ride a skateboard or jump on a trampoline. Informed decisions about participation require accurate estimates of risk. This study will use an innovative design to provide, for the first time, accurate estimates of the risk of bleeding associated with physical activity. This information will form the basis for clinical practice guidelines regarding participation in physical activity.Read moreRead less
Towards An Evidence-Based Model Of Paediatric Palliative Care
Funder
National Health and Medical Research Council
Funding Amount
$73,722.00
Summary
The aim of this study is to improve the evidence base in paediatric palliative care by determining: How families come to realise the need for palliative care; How health professionals (paediatricians, general practitioners, palliative care workers) come to realise the need for palliative care; What aspects of palliative care are of greatest value to dying children and their families; How families experience the service system as it is currently configured including community-based palliative car ....The aim of this study is to improve the evidence base in paediatric palliative care by determining: How families come to realise the need for palliative care; How health professionals (paediatricians, general practitioners, palliative care workers) come to realise the need for palliative care; What aspects of palliative care are of greatest value to dying children and their families; How families experience the service system as it is currently configured including community-based palliative care providers; The degree to which the views of health professionals accord with those of parents, and the extent to which this influences perceived quality of service delivery; and If, and to what extent the needs of children with malignant conditions differ from those with non-malignant conditions.Read moreRead less
Printhotics: A Digital Solution To Optimise Personalised Children’s Orthotic Devices
Funder
National Health and Medical Research Council
Funding Amount
$645,205.00
Summary
Ankle-foot orthoses (AFOs) are custom plastic braces worn around the foot and ankle that are crucial for the daily function of children with nerve, muscle and brain disorders. However, AFO usage is limited by significant wait times, poor adherence and a disconnect with patient preferences. My project, Printhotics, will generate novel AFO designs and production methods based on a concurrent discovery pipeline of co-design, 3D scanning, machine learning, and 3D printing.
ARDAC Second Phase Study : Antecedents Of Renal Disease In Aboriginal Children And Young Adults
Funder
National Health and Medical Research Council
Funding Amount
$86,570.00
Summary
Kidney disease is over-represented within Indigenous communities across Australia. Through screening 2000 participants, aged between 10 and 20 years of age, for risk factors of kidney disease this study will establish the number of young Indigenous people within NSW who show risk factors for kidney disease and add significant information to the natural history of chronic kidney disease. It is a follow on from the successful first phase study.
I am investigating several areas relating to Paediatric surgery in particular the quality and coverage of paediatric surgical trials. The context for this study is to evaluate the current state of paediatric surgical research for methodological rigour, aiming to establish an evidence base and agenda for future trials to improve outcomes in paediatric surgery. The project will involve epidemiological methodology studies and systematic reviews, data linkage and analysis of existing study datasets.
Downregulation Of N-myc Oncogene Expression As A Therapeutic Strategy For Childhood Neuroblastoma.
Funder
National Health and Medical Research Council
Funding Amount
$145,990.00
Summary
Neuroblastoma is a common cancer of young children which, despite the use of powerful anticancer drugs that cure other childhood cancers, has only a 40% survival rate. Many laboratories have shown that the most aggressive neuroblastoma tumours, which are most resistant to the action of anticancer drugs, have an abnormal number of copies of a cancer-associated gene, called N-myc. Patients whose tumours have multiple N-myc copies have dismal survival prospects, and new treatments for such patients ....Neuroblastoma is a common cancer of young children which, despite the use of powerful anticancer drugs that cure other childhood cancers, has only a 40% survival rate. Many laboratories have shown that the most aggressive neuroblastoma tumours, which are most resistant to the action of anticancer drugs, have an abnormal number of copies of a cancer-associated gene, called N-myc. Patients whose tumours have multiple N-myc copies have dismal survival prospects, and new treatments for such patients are urgently needed. Several studies, using models of neuroblastoma cells growing in the laboratory, have shown that it is possible to create small fragments of genetic material which can specifically switch off the N-myc gene. When this happens, the neuroblastoma cells behave in a less aggressive and malignant way. We have recently shown that these genetic fragments are capable of reducing the growth of tumours in mice which have been genetically manipulated to develop neuroblastoma. We now want to develop new types of genetic fragments (DNAzymes) that will be even more effective at switching off N-myc and inhibiting neuroblastoma development, because these fragments may be extremely valuable for treating neuroblastoma in patients.Read moreRead less
Airway Epithelial Barrier Function, Asthma And Aero-allergen Sensitization.
Funder
National Health and Medical Research Council
Funding Amount
$527,886.00
Summary
There is a strong association between allergy and asthma. This association been almost universally assumed to be causative. However, recent evidence suggests an alternative explanation ie., that the abnormal epithelium in asthma allows or facilitates sensitization to airborne allergens. This project will test this alternative hypothesis using human lung tissue and an animal model.
Understanding The Factors Governing Susceptibility And Outcome In Childhood Infection
Funder
National Health and Medical Research Council
Funding Amount
$276,122.00
Summary
This research seeks to understand why a minority of children are prone to severe and often life-threatening infections and inflammation. It focusses on infections both in preterm infants and in later childhood, which may also be relevant to understanding atherosclerosis. I am also interested in improving the health of recently arrived refugees, by conducting research that allows the development of evidence-based health interventions and developing national policy on refugee health.
Tuberculosis - Transmission, Drug Resistance And Strain Emergence
Funder
National Health and Medical Research Council
Funding Amount
$290,652.00
Summary
Tuberculosis (TB) kills nearly 2 million people each year. The emergence of drug resistant TB in the Asia-Pacific region pose a particular threat to Australia, due to frequent population mixing and ongoing TB transmission that may facilitate its spread within vulnerable communities. The proposed study will develop advanced tools to monitor and limit TB transmission within Australia. It will also provide novel insight into the evolution of the global TB epidemic and key factors that sustain it.