Repeated Prenatal Corticosteroids: Effects On Childhood Development, Behaviour, Growth And Health
Funder
National Health and Medical Research Council
Funding Amount
$718,055.00
Summary
Infants born preterm are at high risk of needing help with their breathing to survive. Corticosteroids given to the mother prior to preterm birth can substantially reduce these risks, although the beneficial effects of these drugs only seem to last seven days. Because of this there has been a tendancy to repeat the dose of prenatal steroids after seven days in women who remain at continued risk of very preterm birth. There has been no formal assessment of whether or not repeating the dose of pre ....Infants born preterm are at high risk of needing help with their breathing to survive. Corticosteroids given to the mother prior to preterm birth can substantially reduce these risks, although the beneficial effects of these drugs only seem to last seven days. Because of this there has been a tendancy to repeat the dose of prenatal steroids after seven days in women who remain at continued risk of very preterm birth. There has been no formal assessment of whether or not repeating the dose of prenatal corticosteroids is beneficial or harmful. In this clinical trial we will test what effect, if any, repeat doses of corticosteroids given to women who remain at risk of pretermbirth, have on children at the age of two years Women are eligible for the trial if at of less than 32 weeks of pregnancy, they have received corticosteroids seven or more days ago, and they are considered to be at continued risk of preterm birth. Women are randomised to one of the two treatment groups. Half the women will receive a weekly intramuscular injection of corticosteroids up to the time of birth or 32 weeks gestation, whichever is earlier, whilst the risk of very preterm birth remains. The other half of the women will receive a saline placebo injection. Chance will decide which treatment the women receives. In this study all children who survive to 2 years corrected age will be assessed to see if they have any problems with their health, growth and development. In particular we will assess how well they can walk, talk, understand, see and hear. The trial will be able to assess whether repeat doses of prenatal corticosteroids are helpful or not for infants at risk of being born very preterm by comparing the short term effects on infant health after birth and whilst in hospital with the effects on the child's later health, growth and development. An economic assessment of repeat doses of prenatal corticosteroids will be made in these children.Read moreRead less
Can Pentoxifylline Improve Long-term Outcomes In Preterm Infants With Late-onset Sepsis Or Necrotizing Enterocolitis – A Pragmatic, Randomized, Controlled Trial
Funder
National Health and Medical Research Council
Funding Amount
$2,901,130.00
Summary
Very preterm infants are at high risk of death and disability. Brain injury is often the result of inflammation caused by infection or bowel disease. To date, there is no treatment to reduce the harmful effects of inflammation. Pentoxifylline reduces inflammation and is a promising, safe and inexpensive treatment option for preterm infants. This study will determine whether Pentoxifylline in addition to antibiotics improves survival without disability in preterm infants.
Does Placental Transfusion Prevent Death And Disability In Very Preterm Infants? Childhood Follow Up In The NHMRC Australian Placental Transfusion Study.
Funder
National Health and Medical Research Council
Funding Amount
$889,406.00
Summary
A million babies are born before 30 weeks gestation worldwide each year. Many die or face a lifetime of disability. Enhancing placental transfusion in these infants by deferred clamping of the umbilical cord (DCC) is a simple procedure that may reduce mortality and major disability in childhood. The Australian Placental Transfusion Study (APTS), the largest ever RCT of deferred clamping, will follow up 1200 children born preterm to evaluate if DCC has childhood benefits at 2 years age.
We are an international team committed to clinical trials to improve survival without disability in newborn babies. We plan a randomised trial to confirm if bovine lactoferrin, an inexpensive dairy protein, reduces death or major morbidity and increases total breast milk intake in 1,500 very low birthweight babies in neonatal intensive care units
What Is The Profile, Burden And Consequences Of Cerebral Palsy (CP) Due To Congenital Cytomegalovirus (CMV) Infection?
Funder
National Health and Medical Research Council
Funding Amount
$87,130.00
Summary
Cytomegalovirus (CMV) is a virus that can be transmitted from mother to the unborn child. It is a potentially preventable cause of cerebral palsy (CP). The incidence of CP due to CMV remains unclear in Australia as screening for CMV is not routinely performed in the newborn. Our preliminary data suggests that severe CP is strongly associated with CMV. Here we will use link recorded data and test CMV in newborn screening cards to determine the burden and profile of CP due to congenital CMV in Aus ....Cytomegalovirus (CMV) is a virus that can be transmitted from mother to the unborn child. It is a potentially preventable cause of cerebral palsy (CP). The incidence of CP due to CMV remains unclear in Australia as screening for CMV is not routinely performed in the newborn. Our preliminary data suggests that severe CP is strongly associated with CMV. Here we will use link recorded data and test CMV in newborn screening cards to determine the burden and profile of CP due to congenital CMV in Australia.Read moreRead less
The Burden Of Late Preterm Birth On Brain Development And 2 Year Outcomes – A Prospective, Longitudinal Cohort Study
Funder
National Health and Medical Research Council
Funding Amount
$838,690.00
Summary
80% of preterm babies are born from 32-36 weeks’ gestation, and are late preterm (LPT). LPT children have more learning problems, but why this occurs is unknown. This study aims to understand the effect of LPT birth on brain development. We will do brain scans at term and assess development at 2 years of age of 200 LPT and 200 full-term children. We expect LPT babies will have subtle alterations in brain development compared with term controls which will be associated with delayed development.
Prophylactic Antibiotics To Prevent Recurrent Lower Respiratory Tract Infections In Children With Neurological Impairment (PARROT) Study
Funder
National Health and Medical Research Council
Funding Amount
$1,210,224.00
Summary
We plan a randomised controlled trial to determine if 12 months of a type of antibiotics (compared to placebo) reduces hospitalisations in children with neurological impairment. Currently this group of children are recurrently hospitalised and some doctors use long term antibiotics but there is no high level evidence for this practice. The study will be undertaken in the UK and Australia and involve 474 children. The study will lead to better clinical care and inform guidelines.
Mucopolysaccharidoses (MPS) are a related group of 11 debilitating genetic disorders affecting children. They result from a reduction or total deficiency of an enzyme required for the removal of carbohydrate structures called glycosaminoglycans (gags). Gag degradation occurs inside the cell in specific organelles termed lysosomes and in the absence of the appropriate enzyme, undegraded gag accumulates in the cell. This leads to a range of clinical symptoms and multiple tissue failure. Symptoms c ....Mucopolysaccharidoses (MPS) are a related group of 11 debilitating genetic disorders affecting children. They result from a reduction or total deficiency of an enzyme required for the removal of carbohydrate structures called glycosaminoglycans (gags). Gag degradation occurs inside the cell in specific organelles termed lysosomes and in the absence of the appropriate enzyme, undegraded gag accumulates in the cell. This leads to a range of clinical symptoms and multiple tissue failure. Symptoms common to more than one MPS type include mental deterioration, blindness, abdominal organ enlargement and bone growth problems leading to short stature and bone loss. My laboratory has had a long-term interest in developing treatment for MPS and our research led to the clinical implementation of enzyme replacement therapy (ERT) for MPS VI in 2005. While providing the first effective, multi-tissue treatment for MPS, our research showed that several tissues were not responsive to ERT. These are the brain, cartilage and cornea, thus children on ERT regimens will still suffer from mental retardation, arthritis and blindness. With the goal of treating these particular tissues we have developed a new approach to MPS therapy called substrate deprivation therapy (SDT). Instead of adding back the missing enzyme, SDT acts by decreasing gag production which in turn reduces the level of accumulated gag in cells. SDT results in the correction of MPS cells in culture and reduces several key clinical symptoms in the mouse model of MPS IIIA. In this proposal we will extend our research to evaluate the effect of SDT on brain and bone-joint pathology. Evaluation of efficacy will take place in the MPS VII mouse which exhibits both brain and bone disease and in a new model of MPS IVA developed specifically for this study which exhibits a joint pathology unique amongst the MPS disorders.Read moreRead less
Skeletal disease is a major problem for children with mucopolysaccharidoses (MPS). Patients suffer from early onset osteoporosis and osteoarthritis, severely affecting their quality of life. We will evaluate a lentiviral gene therapy vector developed in-house for its capacity to transduce bone, cartilage, synovial and ligament cells in a mouse model of MPS VI. Our goal is to generate high level, sustained expression of the deficient MPS enzyme and alter the course of skeletal disease in MPS.
Does Gastrostomy Improve The Lives Of Children With Severe Disability And Their Families?
Funder
National Health and Medical Research Council
Funding Amount
$645,101.00
Summary
Around 750 Australian children are born each year with severe intellectual disability. Problems may include feeding difficulties and frequent hospitalisations. Feeding via a gastrostomy tube into the stomach can be used. We will conduct a data linkage study in NSW and WA, and collect additional data in WA to investigate patterns of gastrostomy use and its safety, effectiveness and costs. Our findings will help the management of poor feeding in intellectual disability.