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Is intrathecal baclofen an effective treatment for children with hereditary spastic paraparesis?
  1. Ross Simon Pointon1,
  2. Hannah Whelan1,
  3. Rushna Raza2,
  4. Rajib Lodh2
  1. 1School of Medicine, University of Leeds, Leeds, UK
  2. 2Paediatric Neurorehabilitation, Leeds Children's Hospital, Leeds, UK
  1. Correspondence to Mr Ross Simon Pointon, University of Leeds, Leeds LS2 9JT, UK; ross_pointon{at}yahoo.co.uk

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Scenario

A 4-year-old boy has a diagnosis of hereditary spastic paraparesis (HSP) with no gene identified. He has weakness and spasticity of lower limbs which results in frequent, daily falls and limited stamina of gait. He is unable to tolerate splints and regularly complains of cramps in his calves causing difficulties with sleep and behaviour. He has had trials of oral medications (baclofen, gabapentin) and targeted botulinum toxin, with no significant benefits.

Structured clinical question

In this index case, would intrathecal baclofen (ITB) have the potential to improve quality of life (QoL)?

In ambulant children with HSP, does ITB therapy result in improvements to gait, stability, mobility and overall QoL when compared with standard care? Is ITB a safe treatment for children with HSP?

Search

Dates of search: from inception to December 2019. The relevant articles we identified as outlined below are described in table 1.

View this table:
Table 1

Summary

Primary sources

  • Ovid Medline database—keywords ‘Hereditary Spastic Paraplegia/Paraparesis’, ‘intrathecal baclofen’, ‘ITB’, ‘HSP’ and ‘Paraparesis’ → eight individual articles → two relevant.

  • PubMed database—keywords ‘Hereditary Spastic Paraplegia/Paraparesis’, ‘intrathecal baclofen’, ‘ITB’, ‘gait analysis’, ‘HSP’ and ‘Paraparesis’ → 18 …

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Footnotes

  • RSP and HW are joint first authors.

  • RR and RL are joint senior authors.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests None declared.

  • Patient consent for publication Not required.

  • Provenance and peer review Not commissioned; internally peer reviewed.

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