Intrathecal Baclofen in Hereditary Spastic Paraparesis

Abstract

Introduction: Treatment with intrathecal baclofen (ITB) is a therapeutic option in the management of severe spasticity in patients with hereditary spastic paraparesis (HSP). However, information on the impact of ITB on the natural course of disease, especially the effect of ITB on functional parameters over time is limited. Materials and Methods: We evaluated seven patients with HSP retrospectively who were treated with an ITB device. The following parameters were measured before (pre-implantation) and after implantation (post-implantation) of the ITB device at steady state dosage of ITB and annually until last follow-up: modified Ashworth Scale, Reflex Scale, modified Rankin Scale, and Rivermead Mobility Index. The ITB dosages were assessed after reaching steady state as well as annually until last follow-up. Results: The ITB device was implanted 13 ± 6 (range 9–16) years after diagnosis of HSP on average. Severe spasticity was controlled in all patients by a mean baclofen dosage of 188 ± 60 (range 145–230) μg per day at steady state post-implantation. The modified Ashworth Scale improved significantly from 3 (interquartile range [IQR] 3–3.25) to 1 (IQR 1–1.25; p = 0.046), as did the Reflex Scale from 5 (IQR 4.75–5) to 3 (IQR 2.75–3; p = 0.046) at steady state dosage of ITB. The modified Rankin Scale improved from 2 (IQR 2–2) to 1 (IQR 1–1.5; p = 0.083) and the Rivermead Mobility Index remained 14 (IQR 13.5–14 pre-implantation, IQR 14–14 post-implantation; p = 0.18). Post-implantation, spasticity improved for 2–3 years, followed by a stable phase of ambulatory and other mobility functions for 4–5 years. Thereafter, the maintenance or progressive loss of mobility depended on individual courses of the disease. No ITB-related severe side effects occurred. Discussion: Our data further support the role of ITB in the treatment of severe spasticity in patients with deteriorated walking performance suffering HSP. ITB therapy may initially improve spasticity and stabilize mobility functions for the first 6–8 years in patients with HSP.

Intrathecal baclofen for the management of hereditary spastic paraparesis: a systematic review

“…A total of 6 articles reporting the results of ITB for treatment of HSP were included in this review. Two studies were retrospective published in 2015 [14] and 2019 [13], 2 were case studies published in 2005 [16] and 2015 [17], and the other 2 were prospective published in 2007 [18] and 2014 [19] (Fig. 1 and Tables 1 and 2).…”

“…On the other hand, in the study with the longest follow-up spasticity was controlled by a mean baclofen dosage of 188 μg per day (range 145–230) at steady state. After 20 years of ITB treatment, the mean ITB dosage was 122 ± 28 (range 95–150) μg per day [13].…”

“…The most used scale to measure spasticity was the MAS. The improvement in spasticity ranged on average between 1.9 [19] and 2 [13]. In the case study by Heetla et al .…”

An efficient multidisciplinary approach in a pregnant patient with hereditary spastic paraparesis treated by intrathecal baclofen therapy: A case report

“…The treatment of spasticity includes administration of baclofen, a gamma-aminobutyric acid agonist that inhibits mono-and polysynaptic spinal reflexes. For patients who are unresponsive to or experience side-effects with oral anti-spastic medication, ITB is a highly effective treatment [2,3]. The treatment goal is to reduce spasticity while preserving and/or improving mobility functions.…”

Is intrathecal baclofen an effective treatment for children with hereditary spastic paraparesis?

“…In patients with severe spasticity, use of enteral baclofen, either alone or in combination with other oral antispastic drugs, often fails to achieve the desired spasticity control or side effects limit dosing 4. Baclofen has been successfully used via an intrathecal pump in much smaller doses and fewer systemic side effects for management of hypertonia 1 5–10. Patients with HSP are often able to walk even in advanced stages of their disease.…”