Understanding Clinical and Patient Reported Response of Children and Young People with Cerebral Palsy to Botulinum Toxin A: A Longitudinal Observational Study (The Toxin Study)

Background \nBotulinum Toxin A (BoNT-A) is an established treatment for focal spasticity in children and young people with spastic cerebral palsy (CYPwCP). A systematic review of the available literature within this thesis highlighted that the published evidence for BoNT-A effectiveness is mostly related to short term outcomes focused on impairment level, relating to restriction of body functions and structures, rather than more meaningful measures of activity and participation. \nAims \nTo determine the effect of lower limb BoNT-A on ambulant CYPwCP by evaluating outcome across the WHO’s International Classification of Functioning and Disability (ICF) domains of body structure and function, activity and participation and change in movement quality over a 12-month period and investigate whether clinical outcomes reflect children and families’ experience of BoNT-A treatment. \nMethod \nA prospective observational mixed methods longitudinal study used a one group repeated measures design conducted in two phases. In Phase I the Quality Function Measure and the Canadian Occupational Performance Measure were used to evaluate change in movement quality and evaluate goal attainment following lower limb BoNT-A treatment. Change was also evaluated throughout the ICF domains of body structure and function, activity and participation, using a number of secondary outcome measures (64 CYPwCP). In Phase II semi-structured interviews with a subgroup of families from Phase I explored CYPwCP and parents experience of BoNT-A treatment (Phase II: 18 CYPwCP). \nResults \nThere was a significant improvement in movement quality and goal attainment across the 12 months following BoNT-A. Spasticity was significantly reduced at 6 weeks with mixed results at 6 and 12 months, dependent on the muscles injected. Functional balance and gait improvements, although improved at 6 weeks, only reached clinical significance at 6 and 12 months, respectively. However, clinically significant improvement in motor function and participation outcomes were seen at 6 weeks post BoNT-A and these were maintained across 12 months. CYPwCP and their families described improvements in movement quality and short term reduced stiffness following injections which were associated with increased activity, improved participation opportunities and increased confidence and self esteem.

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