title: Intrathecal Baclofen Therapy for the Treatment of Spasticity in Sjögren-Larsson Syndrome.
abstract: Intrathecal baclofen therapy is widely accepted as a treatment option for patients with severe spasticity. The current treatment of spasticity in patients with Sjögren-Larsson syndrome is largely symptomatic, given that no effective causal therapy treatments are available. We report the outcome of 2 patients with Sjögren-Larsson syndrome who had pump implantation for intrathecal baclofen. We observed a positive response, with a decrease of spasticity, reflecting in the Modified Ashworth Scale, and parents and caregivers observed a functional improvement in both patients. One patient experienced skin irritation 15 months after surgery, necessitating pump repositioning. No infection occurred. Our report shows that intrathecal baclofen therapy can have a positive therapeutic effect on spasticity in patients with Sjögren-Larsson syndrome, and therefore may be a promising addition to current treatments.
authors.affiliation: 1 Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, NY, USA.
authors.forename: Eveline Teresa
authors.initials: ET
authors.lastname: Hidalgo
authors.affiliation: 1 Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, NY, USA.
authors.forename: Cordelia
authors.initials: C
authors.lastname: Orillac
authors.affiliation: 1 Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, NY, USA.
authors.forename: Andrew
authors.initials: A
authors.lastname: Hersh
authors.affiliation: 1 Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, NY, USA.
authors.forename: David H
authors.initials: DH
authors.lastname: Harter
authors.affiliation: 2 Department of Pediatrics, University of Nebraska Medical Center, Nebraska Medical Center, Omaha, NE, USA.
authors.forename: William B
authors.initials: WB
authors.lastname: Rizzo
authors.affiliation: 1 Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, NY, USA.
authors.forename: Howard L
authors.initials: HL
authors.lastname: Weiner
mesh_terms: D001418:Baclofen / Q000008:administration & dosage* / Q000009:adverse effects; D002675:Child, Preschool; D005260:Female; D006801:Humans; D015918:Infusion Pumps, Implantable; D007278:Injections, Spinal; D008297:Male; D009125:Muscle Relaxants, Central / Q000008:administration & dosage* / Q000009:adverse effects; D009128:Muscle Spasticity / Q000188:drug therapy* / Q000209:etiology / Q000503:physiopathology; D016111:Sjogren-Larsson Syndrome / Q000150:complications / Q000000981:diagnostic imaging* / Q000503:physiopathology; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D009125:Muscle Relaxants, Central; D001418:Baclofen
keywords: Modified Ashworth Scale; Sjögren-Larsson syndrome; baclofen pump; intrathecal baclofen therapy; spasticity