About

Cerebral Palsy impacts 17 million people worldwide, 0.2% of the global population.

Cerebral palsy (CP) is a disorder of movement and posture secondary to an insult to the developing brain. While the early brain injury is static, the functional problems are progressive and life-long. Gross motor function plateaus in childhood and declines in adolescence with loss of mobility and the progression of deformity. The disability increases with age and ageing occurs earlier.

Children with CP have major co-morbidities that are likely to impact outcomes and costs of care. Based on CP registers, a recent review by CI Novak identified that in children diagnosed with CP at 5 years:

• 3 in 4 were in pain;
• 1 in 2 had an intellectual disability;
• 1 in 3 could not walk;
• 1 in 3 had hip displacement;
• 1 in 4 could not talk;
• 1 in 4 had epilepsy;
• 1 in 4 had a behaviour disorder;
• 1 in 4 had bladder control problems;
• 1 in 5 had a sleep disorder;
• 1 in 5 dribbled;
• 1 in 10 were blind;
• 1 in 15 were tube fed; and
• 1 in 25 were deaf.

In addition, co-morbidities impact on growth (children with CP are shorter), nutrition (underweight), feeding, communication and executive function.

The European CP registers have identified pain in adolescence as the major comorbidity that reduces participation in education and social functioning. There is a paucity of interventions to optimise function early, to increase participation in education and improve quality of life.

In terms of economic burden, CP is the fifth most expensive health condition in Australia. The ensuing disability and costs of care incur significant health and long term social costs (0.14% of GDP, AU$1.47 billion p.a., with an average yearly cost of AU$43,431 per individual). The evidence for increasing cost effectiveness in treatment is sparse. Our population cohorts have examined costs of medical/allied health care and consequences but few studies have evaluated the cost effectiveness of interventions. As CP is an early brain injury, there is greater opportunity to mitigate the brain injury.

Our focus will be on earlier rehabilitation to maximise adaption of the brain with motor learning focused rehabilitation and neuroprotectants. We have the opportunity to utilise both the ACPR and NZ CP registers to improve early detection of CP, to conduct earlier clinical trials, to test curative agents, and to test the efficacy of early brain rehabilitation on outcomes at 5 years when children are ascertained in the CP registers.

People with CP have identified prevention, cure and reduction of severity as their highest research priorities in an international survey conducted by CI Novak and AI McIntyre. A collaborative effort using multi-centre trials and “big data” aggregation is the best solution for achieving CP consumer research priorities, hence an Australasian CP Clinical Trials Network is urgently needed.

How will health outcomes for children with CP be further improved in 5 years?

• The rate of CP will continue to decline by 30% as reported by our Australian CP Register. 
• The severity of CP will be reduced as the proportion of independently ambulant children with CP will continue to increase from 65% to 80%, with substantial cost and societal implications.
• >75% of infants with CP including Aboriginal and Torres strait Islander and Culturally and Linguistically Diverse infants (CALD) will be accurately diagnosed in the first 3 months post-term, 1.5 years earlier than current practice due to implementation of our International CPG.
• >75% infants at high risk of CP will have been recruited to clinical trials of neuroprotectants and/or rehabilitation within the first year of life.
• Effective interventions implemented nationally will lead to enhanced functional outcomes, with increased participation in education and improved quality of life for families.
• Effective interventions that enhance motor, manual, cognitive and communication outcomes in infancy rather than childhood will optimise neuroplasticity leading to better school readiness.
• New treatments found to be effective in RCTs will be measured for cost effectiveness by our health economist (AI Comans) ensuring rapid implementation of both effective and efficient interventions via the National Disability Insurance Scheme (NDIS).

Figure: National and International Translation in the Australian CP Clinical Trials Network

This CRE will be led by a Management Committee consisting of the ten Chief Investigators led by CIA Boyd as chair supported by a full time CRE Manager. The Management committee will maintain close communication through second monthly teleconferences (six per year).

Each of the five Research Themes will be chaired by 1-2 CIs:

1. Epidemiology (CI McIntyre, AI Badawi), Pre-clinical and neuroprotection (CIs Hunt, Miller, Colditz),
2. Early Detection and Neuroimaging (CI Fripp, AI Morgan)
3. Clinical Trials (CI Boyd, Novak),
4. Knowledge Translation and Implementation (CI Novak), and
5. Engagement and Health Policy (CI Sakzewski).

Each theme panel will include at least 2-3 Chief Investigators, 2-3 Associate Investigators, a lead postdoctoral fellow, an Early Career Researcher/Mid-career fellow, and a representative from the Consumer Council. The Clinical Trials theme also includes key AIs in topic areas (AI Ware Biostatistician, AI Whittingham Psychologist, AI Comans Health Economist). Each theme will monitor research productivity according to the KPIs. Regular meetings of the 5 Themes bi-monthly by ZOOM and at annual meeting (face to face).

Figure: Governance Structure indicateinng frequency of meetings per year in brackets

Each theme panel will select suitable pilot clinical trials to propose to proceed to national multi-site studies, critiquing proposals for proof of concept studies with key expertise across the network to enhance competiveness of grant proposals. The themes will award post-graduate top-up stipends to the top ranked PhD scholars (10), and training fellowships to 18 post-doctoral (11 new) and clinical fellows. They will also oversee the mentorship program, identify areas of training need, and plan national/international collaborations. The KT and Implementation Committee will prioritise effective interventions for development of international CPG. Five international experts form the International External Review Panel of the CRE (Professors Cioni, Fehlings, Anderson, Miller, Dan) who will perform an annual review of KPIs (quantity/quality of outputs, spread/depth of impact) and translation to consumers. The evaluation committee will be chaired by one international scientist rotating each year, meeting annually.