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Use of continuous intrathecal baclofen in hereditary spastic paraplegia

Authors Khera J, Andrew NE, Cadilhac DA, Purvis T, Fahey MC, Rawicki HB

Received 25 June 2015

Accepted for publication 27 October 2015

Published 15 December 2015 Volume 2015:7 Pages 19—26

DOI https://doi.org/10.2147/CA.S91140

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Shanmugasundaram Rajkumar

Peer reviewer comments 4

Editor who approved publication: Professor Marietta Stanton

Jessie Khera,1 Nadine E Andrew,1 Dominique A Cadilhac,1,2 Tara Purvis,1 Michael C Fahey,3,4 Hyam Barry Rawicki1

1Department of Medicine, Faculty of Medicine, Nursing and Health Sciences, Monash University, Clayton, 2Stroke Division, Florey Institute of Neurosciences and Mental Health, Heidelberg, 3Department of Paediatrics, Faculty of Medicine, Nursing and Health Sciences, Monash University, Clayton, 4Department of Medicine, Melbourne University, Parkville, VIC, Australia

Objective: Hereditary spastic paraplegia (HSP) is a rare progressive disorder with few treatment options. We aim to describe the effect of continuous intrathecal baclofen (ITB) pump therapy on the clinical and functional outcomes of patients with HSP.
Methods: This is a retrospective study, using medical record audit data. Adult patients with HSP who had received ITB trial or therapy and had pre- and post-ITB assessment data available were eligible for inclusion. A purposefully designed audit tool was used. Patients with a successful trial received an ITB implantable SynchroMed® II pump. Demographic, clinical, and outcome data were obtained pre- and post-pump trial and pump insertion. Functional, spasticity, and mobility measures were compared pre- and post-ITB trial and pre- and post-ITB pump insertion.
Results: Data for nine patients were available. Six were male and the median age was 55 years (Q1, Q3: 46, 55). All received an ITB trial, and those who responded favorably (n=8) had an ITB pump inserted. Following ITB therapy, improvements were demonstrated for rectus femoris (P=0.04) and gastrocnemius spasticity measures (P=0.03). All patients reported subjective improvements in function, and three of the four with pre- and post-pump assessments, demonstrated clinically meaningful improvements in mobility. Side effects were minimized with appropriate dose titrations.
Conclusion: This is the largest retrospective patient study in the field. The potential benefits of ITB in selected patients with HSP were demonstrated.

Keywords: baclofen, intrathecal baclofen, hereditary spastic paraplegia, gait analysis

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