Dataset Information

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.

ABSTRACT:

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.

SUBMITTER: Milne SC

PROVIDER: S-EPMC7747606 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature

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Publications

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.

Milne Sarah C SC Corben Louise A LA Roberts Melissa M Szmulewicz David D Burns J J Grobler Anneke C AC Williams Shannon S Chua Jillian J Liang Christina C Lamont Phillipa J PJ Grootendorst Alison C AC Massey Libby L Sue Carolyn C Dalziel Kim K LaGrappe Desiree D Willis Liz L Freijah Aleka A Gerken Paul P Delatycki Martin B MB

BMJ open 20201217 12

<h4>Introduction</h4>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-base ...[more]

PMID: 33334834

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Project description:Clinicoanatomic correlation in the spinocerebellar ataxias (SCA) and Friedreich's ataxia (FRDA) is difficult as these diseases differentially affect multiple sites in the central and peripheral nervous systems. A new way to study cerebellar ataxia is the systematic analysis of the "reciprocal cerebellar circuitry" that consists of tightly organized reciprocal connections between Purkinje cells, dentate nuclei (DN), and inferior olivary nuclei (ION). This circuitry is similar to but not identical with the "cerebellar module" in experimental animals. Neurohumoral transmitters operating in the circuitry are both inhibitory (γ-aminobutyric acid in corticonuclear and dentato-olivary fibers) and excitatory (glutamate in olivocerebellar or climbing fibers). Glutamatergic climbing fibers also issue collaterals to the DN. The present study applied five immunohistochemical markers in six types of SCA (1, 2, 3, 6, 7, 17), genetically undefined SCA, FRDA, and FRDA carriers to identify interruptions within the circuitry: calbindin-D28k, neuron-specific enolase, glutamic acid decarboxylase, and vesicular glutamate transporters 1 and 2. Lesions of the cerebellar cortex, DN, and ION were scored according to a guide as 0 (normal), 1 (mild), 2 (moderate), and 3 (severe). Results of each of the five immunohistochemical stains were examined separately for each of the three regions. Combining scores of each anatomical region and each stain yielded a total score as an indicator of pathological severity. Total scores ranged from 16 to 38 in SCA-1 (nine cases); 22 to 39 in SCA-2 (six cases); 9 to 15 in SCA-3 (four cases); and 13 and 25 in SCA-6 (two cases). In single cases of SCA-7 and SCA-17, scores were 16 and 31, respectively. In two genetically undefined SCA, scores were 36 and 37, respectively. In nine cases of FRDA, total scores ranged from 11 to 19. The low scores in SCA-3 and FRDA reflect selective atrophy of the DN. The FRDA carriers did not differ from normal controls. These observations offer a semiquantitative assessment of the critical role of the DN in the ataxic phenotype of SCA and FRDA while other parts of the circuitry appear less important.

Project description:ObjectivesTo evaluate whether a home-based rehabilitation programme for people assessed as being at risk of a poor outcome after knee arthroplasty offers superior outcomes to traditional outpatient physiotherapy.DesignA prospective, single-blind, two-arm randomised controlled superiority trial.Setting14 National Health Service physiotherapy departments in the UK.Participants621 participants identified at high risk of a poor outcome after knee arthroplasty using a bespoke screening tool.InterventionsA multicomponent home-based rehabilitation programme delivered by rehabilitation assistants with supervision from qualified therapists versus usual care outpatient physiotherapy.Main outcome measuresThe primary outcome was the Late-Life Function and Disability Instrument (LLFDI) at 12 months. Secondary outcomes were the Oxford Knee Score (a disease-specific measure of function), Knee injury and Osteoarthritis Outcome Score Quality of Life subscale, Physical Activity Scale for the Elderly, 5 dimension, 5 level version of Euroqol (EQ-5D-5L) and physical function assessed using the Figure of 8 Walk test, 30 s Chair Stand Test and Single Leg Stance.Results621 participants were randomised between March 2015 and January 2018. 309 were assigned to CORKA (Community Rehabilitation after Knee Arthroplasty) home-based rehabilitation, receiving a median five treatment sessions (IQR 4-7). 312 were assigned to usual care, receiving a median 4 sessions (IQR 2-6). The primary outcome, LLFDI function total score at 12 months, was collected for 279 participants (89%) in the home-based CORKA group and 287 participants (92%) in the usual care group. No clinically or statistically significant difference was found between the groups (intention-to-treat adjusted difference=0.49 points; 95% CI -0.89 to 1.88; p=0.48). There were no statistically significant differences between the groups on any of the patient-reported or physical secondary outcome measures at 6 or 12 months.There were 18 participants in the intervention group reporting a serious adverse event (5.8%), only one directly related to the intervention, all other adverse events recorded throughout the trial related to underlying chronic medical conditions.ConclusionsThe CORKA intervention was not superior to usual care. The trial detected no significant differences, clinical or statistical, between the two groups on either primary or secondary outcomes. CORKA offers an evaluation of an intervention utilising a different service delivery model for this patient group.Trial registration numberISRCTN13517704.

Project description:IntroductionThe REFORM (REhabilitation FOR Musculoskeletal conditions) trial is a non-inferiority randomised controlled trial (n=210) designed to determine whether a supported home exercise programme is as good or better than a course of face-to-face physiotherapy for the management of some musculoskeletal conditions. The trial is currently being conducted across Sydney government hospitals in Australia. This process evaluation will run alongside the REFORM trial. It combines qualitative and quantitative data to help explain the trial results and determine the feasibility of rolling out supported home exercise programmes in settings similar to the REFORM trial.Methods and analysisTwo theoretical frameworks underpin our process evaluation methodology: the Realist framework (context, mechanism, outcomes) considers the causal assumptions as to why a supported home exercise programme may be as good or better than face-to-face physiotherapy in terms of the context, mechanisms and outcomes of the trial. The RE-AIM framework describes the Reach, Effectiveness, Adoption, Implementation and Maintenance of the intervention. These two frameworks will be broadly used to guide this process evaluation using a mixed-methods approach. For example, qualitative data will be derived from interviews with patients, healthcare professionals and stakeholders, and quantitative data will be collected to determine the cost and feasibility of providing supported home exercise programmes. These data will be analysed iteratively before the analysis of the trial results and will be triangulated with the results of the primary and secondary outcomes.Ethics and disseminationThis trial will be conducted in accordance with the National Health and Medical Research Council National Statement on Ethical Conduct in Human Research (2018) and the Note for Good Clinical Practice (CPMP/ICH-135/95). Ethical approval was obtained on 17 March 2017 from the Northern Sydney Local Health District Human Research Ethics Committee (trial number: HREC/16HAWKE/431-RESP/16/287) with an amendment for the process evaluation approved on 4 February 2020. The results of the process evaluation will be disseminated through publications in peer-reviewed journals and presentations at scientific conferences.Trial registration numberACTRN12619000065190.

Project description:IntroductionExercise, support and advice are considered core components of management for most musculoskeletal conditions and are typically provided by physiotherapists through regular face-to-face treatments. However, exercise can be provided remotely as part of a home exercise programme, while support and advice can be provided over the telephone. There is initial evidence from trials and systematic reviews to suggest that remotely provided physiotherapy can be used to manage a variety of musculoskeletal conditions safely and effectively.Methods and analysisThe aim of this single-blind randomised controlled non-inferiority trial is to determine whether a supported home exercise programme is as good as or better than face-to-face physiotherapy for the treatment of musculoskeletal conditions. Two hundred and ten participants will be recruited from five public hospitals in Sydney, Australia. Participants will be randomised to either the supported home exercise group or the face-to-face physiotherapy group. Participants allocated to the supported home exercise group will initially receive one face-to-face session with the trial physiotherapist and will then be managed remotely for the next 6 weeks. Participants allocated to the face-to-face physiotherapy group will receive a course of physiotherapy as typically provided in Sydney government hospitals. The primary outcome is function measured by the Patient Specific Functional Scale at 6 weeks. There will be nine secondary outcomes measured at 6 and 26 weeks. Separate analyses will be conducted on each outcome, and all analyses will be conducted on an intention-to-treat basis. A health economic evaluation will be conducted from a health funder plus patient perspective.Ethics and disseminationEthical approval was obtained on the 17 March 2017 from the Northern Sydney Local Health District HREC, trial number HREC/16HAWKE/431-RESP/16/287. The results of this study will be submitted for publication to peer-reviewed journals and be presented at national and international conferences. Recruitment commenced in March 2019, and it is anticipated that the trial will be completed by December 2021. This trial will investigate two different models of physiotherapy care for people with musculoskeletal conditions.Trial registration numberCPMP/ICH-135/95.Protocol versionThe most recent version of the protocol is V.1.2 dated November 2019.

Project description:ObjectiveFor people with ataxia, there are validated outcome measures to address body function and structure (BFS) impairments and participation; however, no outcome measure exists for upper extremity (UE) activity level in this population. The purpose of this study was to determine whether the action research arm test (ARAT), a measure of UE activity validated for other neurological conditions, might be a useful outcome measure for capturing UE activity limitations in ataxia.MethodsA total of 22 participants with ataxia were evaluated to assess construct validity of the ARAT; 19 of the participants were included in the interrater reliability assessment. Participants received a neurologic examination and completed a battery of outcome measures, including the ARAT. ARAT performance was video recorded and scored by 4 additional raters.ResultsFor construct validity, Spearman rho showed a significant moderate relationship between the ARAT and BSF outcome measures. A small, nonsignificant relationship was noted for the ARAT and the participation measure. For interrater reliability, Spearman rho showed a large, significant relationship among all raters for the ARAT (range = .87-.94). High reliability was demonstrated using the intraclass correlation coefficient ([2,1] = .97).ConclusionThe ARAT is moderately correlated with ataxia BFS outcome measures, but not with participation scores. The ARAT is a measure of UE activity, which is different from BFS and participation outcome measures. The ARAT was identified to have strong interrater reliability among raters with varying amounts of experience administering the ARAT. Thus, for the ataxic population, the ARAT may be useful for assessing UE activity limitations.ImpactAtaxia can negatively affect reaching tasks; therefore, it is important to assess UE activity level in people with ataxia. Until this study, no outcome measure had been identified for this purpose.Lay summaryPeople with ataxia may have difficulty with daily tasks that require reaching. The ARAT is an outcome measure that clinicians can use to assess UE activity limitations to help design a treatment program.

Project description:ObjectivesAlthough several rehabilitation programmes following hip arthroscopy for femoracetabular impingement (FAI) syndrome have been described, there are no clinical trials evaluating whether formal physiotherapy-prescribed rehabilitation improves recovery compared with self-directed rehabilitation. The objective of this study was to evaluate the efficacy of adding a physiotherapist-prescribed rehabilitation programme to arthroscopic surgery for FAI syndrome.DesignRandomised controlled trial.MethodsPeople aged ?16 years with FAI syndrome scheduled for hip arthroscopy were recruited and randomly allocated to physiotherapy (PT) or control. The PT group received seven PT sessions (one preoperative and six postoperative) incorporating education, manual therapy and a progressive rehabilitation programme of home, aquatic and gym exercises while the control group did not undertake PT rehabilitation. Measurements were taken at baseline (2 weeks presurgery) and 14 and 24?weeks postsurgery. The primary outcomes were the International Hip Outcome Tool (iHOT-33) and the sport subscale of the Hip Outcome Score (HOS) at week 14.ResultsDue to slower than expected recruitment and funding constraints, recruitment was ceased after 23 months. Thirty participants (14 PT and 16 control) were randomised and 28 (14 PT and 14 control; 93%) and 22 (11 PT and 11 control; 73%) completed week 14 and 24 measurements, respectively. For the 14-week primary outcomes, the PT group showed significantly greater improvements on the iHOT-33 (mean difference 14.2 units; 95%?CI 1.2 to 27.2) and sport subscale of the HOS (13.8 units; 95%?CI 0.3 to 27.3). There were no significant between-group differences at week 24.ConclusionsAn individual PT treatment and rehabilitation programme may augment improvements in patient-reported outcomes following arthroscopy for FAI syndrome. However, given the small sample size, larger trials are needed to validate the findings.Trial registration numberTrial registered with the Australian New Zealand Clinical Trials Registry :ACTRN12613000282785, Results.

Dataset Information

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.

Introduction

Methods and analysis

Ethics and dissemination

Trial registration number

Publications

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.

Similar Datasets

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