Rehabilitation for ataxia study: protocol for a randomised controlled trial of an outpatient and supported home-based physiotherapy programme for people with hereditary cerebellar ataxia
Carolyn Sue,
Martin B Delatycki,
Kim Dalziel,
Anneke C Grobler,
Phillipa J Lamont,
Sarah C Milne,
Louise A Corben,
Melissa Roberts,
David Szmulewicz,
J Burns,
Shannon Williams,
Jillian Chua,
Christina Liang,
Alison C Grootendorst,
Libby Massey,
Desiree LaGrappe,
Liz Willis,
Aleka Freijah,
Paul Gerken
Affiliations
Carolyn Sue
3Kolling Institute, St Leonards, NSW, Australia
Martin B Delatycki
Victorian Clinical Genetics Services, Murdoch Children`s Research Institute, Melbourne, Victoria, Australia
Kim Dalziel
6 Center for Health Policy, Melbourne School of Population and Global Health, University of Melbourne, Melbourne, Victoria, Australia
Anneke C Grobler
Clinical Epidemiology and Biostatistics Unit, Murdoch Childrens Research Institute, Parkville, Victoria, Australia
Phillipa J Lamont
Department of Neurology, Royal Perth Hospital, Perth, Western Australia, Australia
Sarah C Milne
Department of Paediatrics, The University of Melbourne, Parkville, Victoria, Australia
Louise A Corben
Bruce Lefroy Centre for Genetic Health Research, Murdoch Children’s Research Institute, Parkville, Victoria, Australia
Melissa Roberts
Bruce Lefroy Centre for Genetic Health Research, Murdoch Children’s Research Institute, Parkville, Victoria, Australia
David Szmulewicz
Balance Disorders & Ataxia Service, Royal Victorian Eye and Ear Hospital, East Melbourne, Victoria, Australia
J Burns
University of Sydney School of Health Sciences, Faculty of Medicine and Health & Children`s Hospital at Westmead, Sydney, New South Wales, Australia
Shannon Williams
Neurogenetic Clinic and Physiotherapy Department, Royal Perth Hospital, Perth, Western Australia, Australia
Jillian Chua
Physiotherapy Department, Ryde Hospital, Eastwood, New South Wales, Australia
Christina Liang
1Royal North Shore Hospital, St Leonards, NSW, Australia
Alison C Grootendorst
MJD Foundation, Darwin, Northern Territory, Australia
Libby Massey
MJD Foundation, Darwin, Northern Territory, Australia
Desiree LaGrappe
Judith Lumley Centre, NHMRC Centre of Research Excellence in Sexual and Reproductive Health for Women in Primary Care (SPHERE), Melbourne, Victoria, Australia
Liz Willis
MJD Foundation, Darwin, Northern Territory, Australia
Aleka Freijah
Rehabilitation Services, Royal Darwin and Palmerston Hospitals, Darwin, Northern Territory, Australia
Paul Gerken
Rehabilitation Services, Royal Darwin and Palmerston Hospitals, Darwin, Northern Territory, Australia
Introduction Emerging evidence indicates that rehabilitation can improve ataxia, mobility and independence in everyday activities in individuals with hereditary cerebellar ataxia. However, with the rarity of the genetic ataxias and known recruitment challenges in rehabilitation trials, most studies have been underpowered, non-randomised or non-controlled. This study will be the first, appropriately powered randomised controlled trial to examine the efficacy of an outpatient and home-based rehabilitation programme on improving motor function for individuals with hereditary cerebellar ataxia.Methods and analysis This randomised, single-blind, parallel group trial will compare a 30-week rehabilitation programme to standard care in individuals with hereditary cerebellar ataxia. Eighty individuals with a hereditary cerebellar ataxia, aged 15 years and above, will be recruited. The rehabilitation programme will include 6 weeks of outpatient land and aquatic physiotherapy followed immediately by a 24- week home exercise programme supported with fortnightly physiotherapy sessions. Participants in the standard care group will be asked to continue their usual physical activity. The primary outcome will be the motor domain of the Functional Independence Measure. Secondary outcomes will measure the motor impairment related to ataxia, balance, quality of life and cost-effectiveness. Outcomes will be administered at baseline, 7 weeks, 18 weeks and 30 weeks by a physiotherapist blinded to group allocation. A repeated measures mixed-effects linear regression model will be used to analyse the effect of the treatment group for each of the dependent continuous variables. The primary efficacy analysis will follow the intention-to-treat principle.Ethics and dissemination The study has been approved by the Monash Health Human Research Ethics Committee (HREC/18/MonH/418) and the Human Research Ethics Committee of the Northern Territory Department of Health and Menzies School of Health Research (2019/3503). Results will be published in peer-reviewed journals, presented at national and/or international conferences and disseminated to Australian ataxia support groups.Trial registration number ACTRN12618000908235.
