title: Long-term whole-body vibration training in two late-onset Pompe disease patients.
abstract: The treatment of late-onset Pompe disease (LOPD) relies on enzyme replacement therapy (ERT) and physiotherapy but the most appropriate exercise program is not yet established. Whole-body vibration training (WBVT) has showed promising results, improving motor performances in various populations. Our aim is to assess the effects of WBVT performed by two LOPD patients in addition to ERT and physiotherapy. A side-alternating WBVT lasting 2 years; clinical assessments included: manual muscle testing (MRC sumscore), knee extension and arm flection isometric strength (multi-muscle tester M3diagnos), timed function tests (10 m walking, standing-up from chair, ascending 4-steps), 6 min walking (6 MWT), motor disability (Walton Gardner-Medwin scale), pulmonary function. Follow-up evaluations performed for 9 years since ERT start (pre-WBVT and post-WBVT) are reported for comparison. MRC sumscore improved in both patients (Pt.1:41 → 48, Pt.2:42 → 47) as isometric strength of knee extension (Pt.1: + 62 %, Pt.2: + 26 %) and arm flection (Pt.1: + 88 %, Pt.2: + 66 %), 6 MWT improved in Pt.1 (+75 m). Timed function tests did not greatly change. Patients reported no significant CK elevation or WBVT-related complaints. WBVT may be safely used in LOPD and seems to moderately boost muscle strength in patients receiving ERT and physiotherapy for more than 3 years. Larger cohorts should be studied to better assess WBVT potential as adjunctive exercise tool in LOPD.
authors.affiliation: Department of Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians-University, Ziemssenstraße 1a, 80336, Munich, Germany. federica.montagnese@med.uni-muenchen.de.|Department of Neuroscience, University of Messina, Messina, Italy. federica.montagnese@med.uni-muenchen.de.
authors.forename: Federica
authors.initials: F
authors.lastname: Montagnese
authors.affiliation: Department of Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians-University, Ziemssenstraße 1a, 80336, Munich, Germany.
authors.forename: Simone
authors.initials: S
authors.lastname: Thiele
authors.affiliation: Department of Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians-University, Ziemssenstraße 1a, 80336, Munich, Germany.
authors.forename: Stephan
authors.initials: S
authors.lastname: Wenninger
authors.affiliation: Department of Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians-University, Ziemssenstraße 1a, 80336, Munich, Germany.
authors.forename: Benedikt
authors.initials: B
authors.lastname: Schoser
mesh_terms: D000328:Adult; D056947:Enzyme Replacement Therapy; D005080:Exercise Test; D005260:Female; D006009:Glycogen Storage Disease Type II / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D008137:Longitudinal Studies; D053580:Muscle Strength / Q000502:physiology*; D014732:Vibration / Q000627:therapeutic use*; D016138:Walking / Q000502:physiology; D055815:Young Adult
keywords: Enzyme replacement therapy; Late-onset Pompe disease; Side-alternating vibration; Whole-body vibration