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
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.