EACD 2020: How the use of functional assessments in clinical trials provides meaningful information for physicians treating children with cerebral palsy

By Andreas Lysandropoulos MD, PhD, Vice President Head Global Medical Affairs Neuroscience at Ipsen

An estimated 17 million people worldwide are affected by cerebral palsy (CP).1 One of the most common forms of the condition is spastic CP, which affects approximately 80% of patients.1-2

While there is no cure for CP, the associated spasticity can be managed well with a combination of physical therapies and medications. Even minor adjustments to treatment management can deliver significant improvements in quality of life for these patients. This is why I’m so pleased that the Ipsen Neuroscience team is sharing new research at the annual European Academy of Childhood of Childhood Disability (EACD) scientific meetings, taking place between 25-28 November.

The ability to successfully evaluate the impact that treatments can have for people with CP is critical in continuing to advance the science and improve treatment and quality of life for these patients. At present, most clinical trials in this area rely on the Modified Ashworth Scale (MAS) which works by measuring the resistance of a muscle to passive stretching. However, new data shared at EACD utilizes Assisting Hand Assessment (AHA) to measure how effectively a child uses their affected hand in conjunction with their well-functioning hand, a cutting-edge outcome measure in this area.

In CP patients with one-sided impairments (hemiparesis), AHA provides a new perspective on hand function evaluation by measuring how well the affected hand is used in conjunction with the well-functioning hand. For the young people affected by CP, children who want to play and explore the world around them, the ability to use their hands is key.3 AHA uses a play session with specific toy kits for children and adolescents, encouraging the spontaneous use of both hands, to enable researchers to evaluate hand function in a natural environment; not assessing what children can do, but what they actually do when faced with a more spontaneous, real-life problem requiring them to use their hands. Measuring the efficacy of therapies in this way is directly linked to how children with CP live – evaluating the real impact on their day-to-day lives, and enabling physicians to guide treatment planning and evaluate treatment effects.

Evaluating cutting-edge outcome measures such as AHA is another example of how Ipsen puts patients at the center of everything we do, right down to designing our clinical trials – delivering on our commitment to patient centricity even in challenging pediatric conditions. This research, and studies like it, will help to create and support solutions that meet the real needs of patients and help to improve their lives.

References

  1. Cerebral Palsy Foundation. Key Facts. Available at: yourcpf.org/statistics. Accessed November 2020.
  2. Cook Children’s. Cerebral Palsy. Available at: https://cookchildrens.org/neurology/conditions/Pages/cerebral-palsy.aspx. Accessed November 2020.
  3. Krumlinde-Sundholm L, Holmefur M, Kottorp A, Eliasson AC. The Assisting Hand Assessment: current evidence of validity, reliability, and responsiveness to change. Dev Med Child Neurol. 2007 Apr;49(4):259-64. doi: 10.1111/j.1469-8749.2007.00259.x. PMID: 17376135.
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