Journal
BMJ OPEN
Volume 11, Issue 4, Pages -Publisher
BMJ PUBLISHING GROUP
DOI: 10.1136/bmjopen-2021-049542
Keywords
paediatric neurology; developmental neurology & neurodisability; rehabilitation medicine
Categories
Funding
- National Institute for Health Research (NIHR) [ICA-CDRF-2015-01-037]
- NIHR GOSH BRC at UCL GOSH Institute of Child Health
- National Institutes of Health Research (NIHR) [ICA-CDRF-2015-01-037] Funding Source: National Institutes of Health Research (NIHR)
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This study aims to evaluate clinical and patient reported outcomes in ambulant children and young people with cerebral palsy receiving lower limb BoNT-A injections over a 12-month period, exploring the relationship between treatment outcomes and health-related quality of life.
Introduction Botulinum 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 analysis This 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 dissemination Central 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.
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