University of Leeds Medical School, Worsley Building, Woodhouse, Leeds LS2 9NL, UK.
Leeds General Infirmary, Great George St, Leeds LS1 3EX, UK.
Eur J Paediatr Neurol. 2022 Jan;36:14-18. doi: 10.1016/j.ejpn.2021.11.003. Epub 2021 Nov 11.
Hereditary Spastic Paraparesis (HSP) causes lower limb spasticity, pain and limits ambulation resulting in a negative impact on an individual's quality of life. This case series evaluates the use of Intra-thecal Baclofen (ITB) on 5 ambulant children with HSP. Our results suggest ITB is associated with a reduction in spasticity and a trend towards improvement in patient-reported quality of life and achievement of personalised goals. This was evidenced with lower Modified Ashworth Scale (MAS) scores and increasing values using the Cerebral Palsy Quality of Life (CPQoL) tool and Goal Attainment Scale (GAS). ITB was not associated with any major immediate or longer-term adverse effects. Overall, our study supports the role of ITB, used in a goal-directed manner, in the management of children and young people with HSP where other standard treatment options have been unsuccessful.
遗传性痉挛性截瘫 (HSP) 可引起下肢痉挛、疼痛,并限制活动能力,从而对个人的生活质量产生负面影响。本病例系列评估了鞘内注射巴氯芬 (ITB) 在 5 名可走动的 HSP 患儿中的应用。我们的结果表明,ITB 与痉挛的减轻以及患者报告的生活质量和个性化目标的实现方面的改善趋势相关。这一点通过较低的改良 Ashworth 量表 (MAS) 评分以及使用脑瘫生活质量 (CPQoL) 工具和目标实现量表 (GAS) 得到了证明,这些工具的评分值在增加。ITB 与任何重大的即时或长期不良影响无关。总的来说,我们的研究支持在其他标准治疗方法无效的情况下,以目标为导向使用 ITB 治疗 HSP 儿童和青少年的作用。