Katchburian Lesley R, Oulton Kate, Main Eleanor, Morris Christopher, Carr Lucinda J
Neuroscience Unit,The Wolfson Neurodisability Movement Disorder Service, Great Ormond Street Hospital For Children, London, UK
Physiotherapy, UCL Great Ormond Street Institute of Child Health, London, UK.
BMJ Open. 2021 Apr 21;11(4):e049542. doi: 10.1136/bmjopen-2021-049542.
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).
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.
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.
肉毒杆菌神经毒素A（BoNT-A）是治疗儿童和青少年脑瘫（CYPwCP）患者肌张力亢进的一种公认治疗方式。然而，人们对BoNT-A的长期影响存在担忧，对于指导其使用的最有意义的疗效指标也缺乏共识。迄今为止，大多数证据基于短期疗效，与损伤水平（身体功能和结构受限）的变化有关，而非适应性技能（促进活动和参与）的变化。拟开展的研究旨在评估在12个月内接受下肢BoNT-A注射的能行走的CYPwCP患者在世界卫生组织《国际功能、残疾和健康分类》所有领域以及健康相关生活质量（HRQoL）方面的临床和患者报告的疗效。
这项务实的前瞻性纵向观察性研究将采用单组重复测量设计。将从英国伦敦一家成熟的运动障碍服务机构招募60名CYPwCP患者，年龄在4至18岁之间，分为粗大运动功能分类系统（GMFCS）I-III级。将在注射前以及注射后6周、6个月和长达12个月时收集《国际功能、残疾和健康分类》所有领域（身体结构和功能、活动（包括运动质量）、目标达成、参与和HRQoL）的标准化临床和患者报告的疗效指标。儿童和照料者的一个代表性亚组将参与研究的定性部分，探讨他们的BoNT-A治疗体验与临床疗效指标的关系。
伦敦中央研究伦理委员会已批准该研究的伦理申请（#IRAS 211617 #REC 17/LO/0579）。研究结果将以同行评审出版物、会议以及通过网络等多种可获取的形式，包括社交媒体，传播给参与者和相关利益攸关方。
