A single injection of Dysport (abobotulinumtoxinA) significantly helped children with cerebral palsy to achieve pre-defined functional goals in difficulties such as walking and endurance, according to a study published in the Journal of Child Neurology.
The study, “AbobotulinumtoxinA (Dysport®) Improves Function According to Goal Attainment in Children With Dynamic Equinus Due to Cerebral Palsy,” analyzed data from a randomized and double-blind clinical trial (NCT01249417), conducted on 241 children with cerebral palsy between July 2011 and June 2014.
All of the children, ages 2 to 17, entered the study able to walk, but had a characteristic walking pattern of tiptoeing when taking a step.
The children were divided into three groups. Those in the first two received injections of Dysport in each leg at one of two different doses (10 or 15 units per kilogram, respectively) plus their current standard of care. Those in the third group were injected with an inactive drug (placebo) alongside their current standard of care. (Dysport is an FDA-approved treatment for limb spasticity in children and adults.)
Functional goals were measured using the Goal Attainment Scaling (GAS), a method that relies on a written guide of wanted goals developed between the clinicians and, in this case, the children’s parent or guardian. How well those goals are attained is then judged in follow-up assessments.
Common goals included an improved walking pattern, better balance, fewer trips and falls, and improved endurance. Most parents/guardians selected goals targeting improvements in walking ability as the most relevant.
One month after treatment, the GAS scores of children given either dose of the drug were higher than those in the placebo group, the researchers reported. This result was maintained at week 12.
The best goal attainment scores were noted in an improved walking pattern, lesser tripping and falling, and improved endurance.
“In conclusion, this is the first placebo-controlled study to demonstrate that single injections of [Dysport] significantly improve the ability of pediatric patients with cerebral palsy to achieve their functional goals that are important to the patients and their families,” wrote Dr. Ann Tilton and colleagues. Tilton is a professor of clinical neurology at Louisiana State University in New Orleans and the study’s first author.
“Goal Attainment Scaling is a useful measure to include in the routine clinical assessment and management of children with cerebral palsy and has the potential to improve current practice,” the team concluded. “Realistic goals and expectations should be established with the patient and family before treatment, and the therapy should be tailored to their individual needs.”