Rehabilitation in childhood-onset hyperkinetic movement disorders including dystonia: Treatment change in outcomes across the ICF and feasibility of outcomes for full trial evaluation

TY - JOUR

T1 - Rehabilitation in childhood-onset hyperkinetic movement disorders including dystonia

T2 - Treatment change in outcomes across the ICF and feasibility of outcomes for full trial evaluation

AU - Gimeno, Hortensia

AU - Polatajko, Helene J.

AU - Cornelius, Victoria

AU - Lin, Jean Pierre

AU - Brown, Richard G.

N1 - Funding Information: We would like to thank the children, young people, and their families that participated in the study. We would also like to thank the blind raters, Professor Gill Chard from AMPS UK and Ireland; Allied Health Professionals from the Complex Motor Disorders Service, Kylee Tustin, Erin Morton, Lesley Baker, Laura Ainaga and Emily Fleischfresser. Hortensia Gimeno is funded by a National Institute for Health Research (NIHR/HEE Clinical Doctoral Research Fellowship, CDRF-2013-04-039 ). This paper represents independent research part funded by the National Institute for Health Research (NIHR) (HG), and the NIHR Biomedical Research Centre at South London and Maudsley NHS Foundation Trust and King's College London (RB). The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health. Funding Information: We would like to thank the children, young people, and their families that participated in the study. We would also like to thank the blind raters, Professor Gill Chard from AMPS UK and Ireland; Allied Health Professionals from the Complex Motor Disorders Service, Kylee Tustin, Erin Morton, Lesley Baker, Laura Ainaga and Emily Fleischfresser. Hortensia Gimeno is funded by a National Institute for Health Research (NIHR/HEE Clinical Doctoral Research Fellowship, CDRF-2013-04-039). This paper represents independent research part funded by the National Institute for Health Research (NIHR) (HG), and the NIHR Biomedical Research Centre at South London and Maudsley NHS Foundation Trust and King's College London (RB). The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health. Publisher Copyright: © 2021 Copyright: Copyright 2021 Elsevier B.V., All rights reserved.

PY - 2021/7

Y1 - 2021/7

N2 - Background: Childhood-onset hyperkinetic movement disorders (HMD), including dystonia are notoriously difficult to treat and there are limited studies showing successful medical, surgical or non-pharmacological interventions. Methods: This prospective study used grouped data (n = 22) from two studies of the Cognitive Orientation to daily Occupational Performance (CO-OP) Approach for patient-selected goals. Eligibility included aged 6–21 years, deep brain stimulation in place, with manual ability classification system level I-IV. Outcome was assessed on a range of patient-reported and clinician-rated measures across the International Classification of Function at end-treatment (10 weekly sessions) (series 1 and 2) and 3-month follow-up (series 1). Feasibility of outcomes to be used in a full trial were explored. Findings: Nineteen participants completed the intervention and were included in the analysis. Of the primary outcome measures, the self-reported Canadian Occupational Performance Measure showed improvement in goal performance (mean change 4.08, 95% CI [3.37,4.79] post-; 4.18 [5.10,5.26] follow-up), and satisfaction (4.03 [3.04,5.03) post-; 4.44 [3.07,5.82] follow-up]. The Assessment of Motor and Process Skills showed improved motor score (0.52 [0.01,1.03] at follow-up only, while the process score did not change. Objective blind-rated pooled data using the Performance Quality Rating Scale-individualized indicated significant change for trained goals (3.79 [3.37,4.21] post-; (4.01,5.10) follow-up] and untrained goals (1.90 [1.24,2.55] post 1.91 [0.23,3.60] follow-up]. Motor impairment assessed by the Burke-Fahn Motor Disability Rating Scale was unchanged (−3.26 [-6.62,0.09] post-; −1.11 [-8.05,5.82] follow-up). Improvement was also observed in self-efficacy (0.97 [0.47,1.47] post-; 1.37 [1.91–0.83] follow-up) and Quality of Life (0.12 [0.03–0.22] follow-up). Goal improvement; self-efficacy and quality of life captured significant change post-intervention. This improvement was shown despite no change on impairment-related measures and were shown to be feasible measures to use in a larger study of CO-OP for this population.

AB - Background: Childhood-onset hyperkinetic movement disorders (HMD), including dystonia are notoriously difficult to treat and there are limited studies showing successful medical, surgical or non-pharmacological interventions. Methods: This prospective study used grouped data (n = 22) from two studies of the Cognitive Orientation to daily Occupational Performance (CO-OP) Approach for patient-selected goals. Eligibility included aged 6–21 years, deep brain stimulation in place, with manual ability classification system level I-IV. Outcome was assessed on a range of patient-reported and clinician-rated measures across the International Classification of Function at end-treatment (10 weekly sessions) (series 1 and 2) and 3-month follow-up (series 1). Feasibility of outcomes to be used in a full trial were explored. Findings: Nineteen participants completed the intervention and were included in the analysis. Of the primary outcome measures, the self-reported Canadian Occupational Performance Measure showed improvement in goal performance (mean change 4.08, 95% CI [3.37,4.79] post-; 4.18 [5.10,5.26] follow-up), and satisfaction (4.03 [3.04,5.03) post-; 4.44 [3.07,5.82] follow-up]. The Assessment of Motor and Process Skills showed improved motor score (0.52 [0.01,1.03] at follow-up only, while the process score did not change. Objective blind-rated pooled data using the Performance Quality Rating Scale-individualized indicated significant change for trained goals (3.79 [3.37,4.21] post-; (4.01,5.10) follow-up] and untrained goals (1.90 [1.24,2.55] post 1.91 [0.23,3.60] follow-up]. Motor impairment assessed by the Burke-Fahn Motor Disability Rating Scale was unchanged (−3.26 [-6.62,0.09] post-; −1.11 [-8.05,5.82] follow-up). Improvement was also observed in self-efficacy (0.97 [0.47,1.47] post-; 1.37 [1.91–0.83] follow-up) and Quality of Life (0.12 [0.03–0.22] follow-up). Goal improvement; self-efficacy and quality of life captured significant change post-intervention. This improvement was shown despite no change on impairment-related measures and were shown to be feasible measures to use in a larger study of CO-OP for this population.

KW - Cerebral palsy

KW - CO-OP Approach

KW - Dystonia

KW - Participation

KW - Rehabilitation

UR - http://www.scopus.com/inward/record.url?scp=85107454656&partnerID=8YFLogxK

U2 - 10.1016/j.ejpn.2021.04.009

DO - 10.1016/j.ejpn.2021.04.009

M3 - Article

C2 - 34052114

AN - SCOPUS:85107454656

SN - 1090-3798

VL - 33

SP - 159

EP - 167

JO - European Journal of Paediatric Neurology

JF - European Journal of Paediatric Neurology

ER -