IntroductionBotulinum neurotoxin-A (BoNT-A) is an accepted treatment modality for the management of hypertonia in children and young people with cerebral palsy (CYPwCP). Nevertheless, there are ...concerns about the long-term effects of BoNT-A, with a lack of consensus regarding the most meaningful outcome measures to guide its use. Most evidence to date is based on short-term outcomes, related to changes at impairment level (restrictions of body functions and structures), rather than changes in adaptive skills (enabling both activity and participation). The proposed study aims to evaluate clinical and patient reported outcomes in ambulant CYPwCP receiving lower limb BoNT-A injections over a 12-month period within all domains of the WHO’s International Classification of Functioning, Disability and Health and health-related quality of life (HRQoL).Methods and analysisThis pragmatic prospective longitudinal observational study will use a one-group repeated measures design. Sixty CYPwCP, classified as Gross Motor Function Classification System (GMFCS) levels I–III, aged between 4 and 18 years, will be recruited from an established movement disorder service in London, UK. Standardised clinical and patient reported outcome measures within all ICF domains; body structures and function, activity (including quality of movement), goal attainment, participation and HRQoL, will be collected preinjection and at 6 weeks, 6 months and up to 12 months postinjection. A representative subgroup of children and carers will participate in a qualitative component of the study, exploring how their experience of BoNT-A treatment relates to clinical outcome measures.Ethics and disseminationCentral London Research Ethics Committee has granted ethics approval (#IRAS 211617 #REC 17/LO/0579). Findings will be disseminated in peer-reviewed publications, conferences and via networks to participants and relevant stakeholders using a variety of accessible formats including social media.
Cerebral palsy (CP) is frequently characterized by hypertonia, impairing motor function and affecting the quality of life of children and young people with CP (CYPwCP). Botulinum Toxin A (BoNT-A) has ...emerged as a widely utilized treatment modality to manage lower limb hypertonia in ambulant CYPwCP. This paper explores the current evidence base and clinical practice surrounding the use of BoNT-A in this population. BoNT-A injections are indicated for the management of focal lower limb hypertonia, targeting specific muscles that contribute to functional limitations. Clinical assessment plays a pivotal role in determining the suitability of BoNT-A treatment, with emphasis placed on evaluating tone, functional impairments, and individualized treatment goals. Timing of treatment is crucial, with interventions typically initiated when dynamic tone significantly impacts motor function and mobility. Optimal treatment age remains a topic of debate, with studies indicating benefits across various age groups. Close monitoring and regular follow-up evaluations are essential components of BoNT-A treatment, ensuring timely adjustments to address changing needs. BoNT-A represents a valuable therapeutic option for managing lower limb hypertonia in ambulant CYPwCP. Clinical decision-making should be guided by comprehensive assessments, utilization of appropriate outcome measures, and consideration of individualized treatment goals. Further research is warranted to elucidate optimal treatment strategies, long-term efficacy, and potential adverse effects to optimize outcomes in this patient population.
ObjectivesThis study aimed to identify the ideas and preferences of children with cerebral palsy (CP) for planning physiotherapy, including their perceptions of the F-word framework (Rosenbaum, ...Gorter 2011).MethodsEthical approvals were granted from the HRA (IRAS 311409) and local R&D departments. Qualitative research was undertaken using semi-structured interviews with children attending Great Ormond Street botulinum toxin service.The initial interview format was devised in collaboration with young people with CP and their parents. Interviews were recorded, transcribed and anonymised. Reflexive analysis of data was used to identify themes, within a framework using the F-words (Braun and Clark 2012).ResultsFive children participated, aged 8-12 years, four girls, one boy. There were GMFCS levels I, II and IV. Interviews employed creative methods and strategies to ‘depersonalise’ discussion (Allard et al 2013). One interview was discontinued due to participant anxiety.Children stated that physiotherapists should be nice: ‘friendly, not too professional’.Thematic analysis identified the following understanding/ideas:‘Fitness’ – strengthening exercises, being challenged physically.‘Function’ – activities of daily living, mobility, hobbies. Use of equipment for independence.‘Friends’ – Social connection. Being accepted, sharing physical activity with friends, having friends with similar life experiences.‘Family’ – value of family involvement and encouragement.‘Fun’ – distraction from painful intervention, games to make sessions enjoyable. Signposting to fun accessible activities outside treatment.‘Future’ – developing independence, physiotherapy adapting to change, supporting aspirations.ConclusionsParticipants had their own ideas of what the F-words might represent, which fit well with the model’s ethos. Its effective use in community practice can be strengthened by understanding how children with CP perceive the model and which factors they feel should be included.Insight gained into the interviewing process will be used to develop resources to hear from more children including those with communication difficulties or learning disabled.Abstract 102 Figure 1International classification of functioning disability and health F-word frameworkAbstract 102 Figure 2Gross motor function levels in cerebral palsyAcknowledgements for funding or supportThis research was completed as part of an NIHR funded pre-doctoral clinical academic fellowship and supported by GOSH Centre for Outcomes and Experience Research in Children’s Health Illness and Disability (ORCHID)
Background: Botulinum 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. Aims: To 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. Method: A 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). Results: There 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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