The use of intrathecal baclofen for management of spasticity in hereditary spastic paraparesis: A case series

• Published in: European journal of paediatric neurology 2022-01, Vol.36, p.14-18
• Main Authors: Pointon, Ross, Whelan, Hannah, Raza, Rushna, Peacock, Sharron, Wilsmore, Catherine, Mulkeen, Andie, Goodden, John, Lodh, Rajib
• Format: Article
• Language: English
• Subjects: Adolescent; Baclofen - therapeutic use; Child; Hereditary spastic paraparesis; Humans; Infusion Pumps, Implantable; Injections, Spinal; Intrathecal baclofen; Muscle Relaxants, Central - therapeutic use; Muscle Spasticity - drug therapy; Muscle Spasticity - etiology; Paraparesis, Spastic - drug therapy; Quality of Life
• ISSN: 1090-3798; 1532-2130
• DOI: 10.1016/j.ejpn.2021.11.003

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. •To date, this is the first study to evaluate the use of intrathecal baclofen in children with hereditary spastic paraparesis.•Intrathecal baclofen was not linked with any long-term adverse effects in the children presented in this study over 3 years.•Intrathecal baclofen may improve spasticity, goal attainment & quality of life in paediatric hereditary spastic paraparesis.