Rehabilitation management of children with hereditary spastic paraplegia: a retrospective cohort study

Treatment discontinuation rates and reasons were recorded. Treatment profiles were compared between pure and complex HSP phenotypes.</p> <p>Thirty-one children were included (pure HSP <i>n</i> = 16; complex HSP <i>n</i> = 15; 48.4% female). Motor decline was observed in both pure and complex HSP phenotypes (22.6%, <i>n</i> = 7 children).

BoNT-A was administered to 77.4% (<i>n</i> = 24) of the cohort, with a median of 4.5 (interquartile range 2–9) treatment episodes; however, 75% of treated children (<i>n</i> = 18) ceased BoNT-A, half (<i>n</i> = 9) due to perceived inefficacy and half (<i>n</i> = 9) due to escalation to surgery or intrathecal baclofen. Oral baclofen was prescribed to 67.7% (<i>n</i> = 21) of the cohort with 47.6% (<i>n</i> = 10) discontinuing, predominantly due to side effects or perceived inefficacy.

Orthopedic surgery was undertaken in 45.2% of the cohort. Treatment profiles were broadly similar between pure and complex phenotypes.</p> <p>In this tertiary rehabilitation setting, spasticity interventions were commonly used in children with HSP but were frequently discontinued due to limited perceived benefit or tolerability, or escalation to orthopedic surgery or intrathecal baclofen. These findings highlight the need for cautious extrapolation from non-progressive conditions, systematic review of treatment response over time, and a rehabilitation approach that accounts for the progressive nature of pediatric HSP.</p>