EVALUATION OF THE EFFECTIVENESS OF EXPANDED NEWBORN SCREENING BY TANDEM MASS SPECTROMETRY
Funder
National Health and Medical Research Council
Funding Amount
$375,250.00
Summary
Newborn babies in Australia are routinely tested for certain treatable disorders. Testing began in the 1960's with systematic testing for phenylketonuria, a rare amino acid enzyme defect. It causes severe mental retardation which can only be prevented if treatment is begun in the first few weeks of life. By 1997, only three other disorders, congenital hypothyroidism, cystic fibrosis, and galactosaemia, had been added to the testing protocol as tests became available. Using the new technology of ....Newborn babies in Australia are routinely tested for certain treatable disorders. Testing began in the 1960's with systematic testing for phenylketonuria, a rare amino acid enzyme defect. It causes severe mental retardation which can only be prevented if treatment is begun in the first few weeks of life. By 1997, only three other disorders, congenital hypothyroidism, cystic fibrosis, and galactosaemia, had been added to the testing protocol as tests became available. Using the new technology of tandem mass spectrometry (MSMS) it is now possible to screen for up to 30 extremely rare, treatable metabolic disorders simultaneously and cheaply, but it is not clear how effective this is. A formal trial of MSMS screening, randomly assigning babies to be tested or not tested, does not seem feasible because of the rarity of the individual disorders (most with a birth prevalence much less than 1: 50,000). Huge numbers would be needed in the trial for statistical significance. We began MSMS screening in NSW April 1998 and in South Australia in February 1999. Victoria is proposing to start screening now, but there are as yet no plans for this screening in the other states. We would like to assess the effectiveness of MSMS newborn screening using the best possible evidence drawn from all data available in the whole of Australia. We plan to undertake an economic evaluation, comparing costs and benefits such as development, hospitalisations, medical complications and other outcome measures, in screened and unscreened babies and also assess harms from screening. Because only 6 specialised laboratories in Australia, in Brisbane (2), Sydney, Melbourne, Adelaide, and Perth can diagnose these disorders, we are confident that we know of all diagnosed cases of the disorders in question. We hope to be able to show whether or not there is a benefit to affected babies by implementing newborn screening tests for these rare diseases.Read moreRead less
Reducing Vaccine Preventable Diseases In Children: Using National Active Hospital-based Surveillance To Evaluate And Improve Immunisation Program Performance
Funder
National Health and Medical Research Council
Funding Amount
$1,049,916.00
Summary
This proposal aims to evaluate and improve upon immunisation policy and programs for the prevention of severe childhood influenza and pertussis. Using our successful Paediatric Active Enhanced Disease Surveillance (PAEDS) network in 6 major children's hospitals across Australia, we will identify gaps in the evidence base needed to better control influenza and pertussis and in particular to ensure programs are delivered in such a way so as to address health inequalities.
The Role Of Emerging Gastrointestinal Viruses In The First Two Years Of Life: A Birth Cohort Study
Funder
National Health and Medical Research Council
Funding Amount
$392,534.00
Summary
Gastro illnesses are common and potentially serious early in life. We are aiming to find out more about these illnesses by following ~140 children from birth to their second birthday, with parents collecting a dirty nappy swab every week. Our laboratory will test these samples for a wide range of known and new gastro viruses, and we will also be looking for, as yet, undiscovered viruses. This information will allow us to document the burden of these illnesses in young children and their families ....Gastro illnesses are common and potentially serious early in life. We are aiming to find out more about these illnesses by following ~140 children from birth to their second birthday, with parents collecting a dirty nappy swab every week. Our laboratory will test these samples for a wide range of known and new gastro viruses, and we will also be looking for, as yet, undiscovered viruses. This information will allow us to document the burden of these illnesses in young children and their families.Read moreRead less
Implementing, Evaluating And Translating Pragmatic Strategies To Prevent Prenatal Alcohol Exposure (PAE), And Treat Fetal Alcohol Spectrum Disorders (FASD)
Funder
National Health and Medical Research Council
Funding Amount
$340,038.00
Summary
Drinking alcohol when pregnant places the unborn child at risk of lifelong brain damage, that we call Fetal Alcohol Spectrum Disorders (FASD). We can prevent FASD by raising awareness of the harms of drinking in pregnancy, and supporting women not to drink. For those with FASD, treatment programs can help reduce learning and behavioural problems. Our research team work with communities and service providers to implement FASD Prevention and Treatment strategies, and raise awareness of FASD.
Long Term Outcomes Following Early Infection And Inflammation In Cystic Fibrosis Lung Disease
Funder
National Health and Medical Research Council
Funding Amount
$75,006.00
Summary
This study involves the long-term follow up of birth cohort of 98 infants with cystic fibrosis, with the objectives of assessing the impact of lower airway infection and inflammation in infancy and early childhood on the development of CF lung disease over 20-25 years, and of describing the development of the lower airway microbiota in early CF lung disease and its relationship to clinical outcomes.
A Population-based Longitudinal Assessment Of Early Life Vitamin D And Risk Of Food Allergy
Funder
National Health and Medical Research Council
Funding Amount
$466,086.00
Summary
There has been a dramatic and unexplained increase in food allergy over recent decades. The increase in food allergy may relate to a concordant increase in early vitamin D insufficiency, however this hypothesis has never been directly tested. This project will use existing/funded samples from two NHMRC funded studies to conduct a detailed investigation of (i) the determinant and predictors of early life vitamin D insufficiency; and (ii) the association of vitamin D insufficiency and food allergy ....There has been a dramatic and unexplained increase in food allergy over recent decades. The increase in food allergy may relate to a concordant increase in early vitamin D insufficiency, however this hypothesis has never been directly tested. This project will use existing/funded samples from two NHMRC funded studies to conduct a detailed investigation of (i) the determinant and predictors of early life vitamin D insufficiency; and (ii) the association of vitamin D insufficiency and food allergy.Read moreRead less
Evaluating Neonatal Intensive Care For Tiny Babies In The 2000s - Is It Still Effective, Efficient And Available?
Funder
National Health and Medical Research Council
Funding Amount
$246,333.00
Summary
Most extremely tiny babies, those of birthweight less than 1000 g, need intensive care to survive. However some survivors have substantial problems with their long-term health. Since intensive care is costly we must be sure that it is money well spent. From the late 1970s until the late 1990s in the state of Victoria neonatal intensive care has been increasingly effective, with large increases in the long-term survival rate, from 25% in 1979-80, to 73% in 1997. Its efficiency has been relatively ....Most extremely tiny babies, those of birthweight less than 1000 g, need intensive care to survive. However some survivors have substantial problems with their long-term health. Since intensive care is costly we must be sure that it is money well spent. From the late 1970s until the late 1990s in the state of Victoria neonatal intensive care has been increasingly effective, with large increases in the long-term survival rate, from 25% in 1979-80, to 73% in 1997. Its efficiency has been relatively high and stable over time, comparing favorably with many other health care programmes, both intensive and non-intensive. It has been increasingly available, with fewer than 10% of ELBW infants born outside major hospitals with intensive care nurseries, and more tiny babies offered intensive care. We now need to know if these benefits have been maintained for tiny babies born in the 2000s. Hence we need to assess the long-term health of tiny babies born in Victoria in 2005.Read moreRead less
RCT Of The Intragastric Balloon And Lifestyle Intervention Versus Lifestyle Intervention Alone In Obese Adolescents
Funder
National Health and Medical Research Council
Funding Amount
$598,976.00
Summary
For the first time in adolescents, Perth researchers will trial placement of a fluid-filled silicone balloon in the stomach. It will be used in conjunction with a lifestyle intervention, and so is expected to increase weight loss. The balloons are safe, effective at adult weight loss and an alternative to invasive surgery. Adolscent obesity rates are rising and treatment options are limited. The non-surgical balloon may be the alternative for adolescents who would previously had to have surgery.