Environmental modifiers: Prospects for rehabilitation in Huntington s disease

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Environmental modifiers: Prospects for rehabilitation in Huntington s disease Jan Frich Oslo University Hospital / University of Oslo EHDN 8th Plenary Meeting, Sept 19, 2014

Background Growing interest in studying effects of exercise and rehabilitation in patients with Huntington s disease (HD) Programs may have different formats: Home-based exercise programs Community-based exercise/rehabilitation programs Inpatient (intensive/multidisciplinary) rehabilitation programs (For example: Khalil et al, 2013; Busse et al 2013; Zinzi et al 2007; Piira et al 2013)

Activities (Photo: North Norway Rehabilitation Center)

Research Target groups: Early- to mid-stage HD Observational studies indicate positive effects of multidisciplinary rehabilitation on physical function/balance, swallowing, independence, mood and social relationships (Zinzi et al, Clin Rehab 2007; Ciancarelli et al, Eur J Phys Rehabil Med, 2013; Thompson et al Eur J Neurol 2013) A randomized study of a 12-week community based program found that the program was safe, feasible, acceptable and suggests beneficial effects of rehabilitation (Busse et al, JNPT, 2013)

The Norwegian Project Intensive rehabilitation programs for patients with HD was funded by the Norwegian Directorate of Health in 2009 The initiative was inspired by the results from a landmark observational study (Zinzi et al, Clin Rehab, 2007) The aim of the mixed-method evaluation was to assess the feasibility and the effects of participating in a one-year multidisciplinary rehabilitation program Ethical approval by Norwegian Social Science Data Services (ref. 26587) and The Regional Ethics Committee, Health Region South-East (ref. 2010/1026-1)

Inclusion Participants Clinical diagnosis of Huntinton s disease Early- to midphase (Shoulson & Fahn, stage I-III) Age > 18 years Exclusion Serious psychiatric conditions No apparent severe impairment in general cognitive function at the time of first admission Piira A, van Welsam MR, Mikalsen G, Nilsen KH, Knutsen S, Frich JC. Effects of a one year intensive multidisciplinary rehabilitation program for patients with Huntington s disease: a prospective intervention study. PLOS Curr 2013. doi: 10.1371/currents.hd.9504af71e0d1f87830c25c394be47027

One year program: 3 x 3 weeks stay + evaluation stay Stay 1 (3 weeks) Stay 2 (3 weeks) Stay 3 (3 weeks) Evaluation stay 3 months (95.4 days (SD ±34.2) after discharge of the last stay North Norway Rehabilitation Center Vikersund

Start of each stay Cognitive function (MSSE), depression (HADS), motor function, balance, gait (ABC, 6 Min. Walk Test ) ADL (Barthel) End of each stay Motor function, balance, gait Three weeks stay - 8 hours of various activities 5 days a week - Groups of 4-6 - Physiotherapy / exercise - Group meetings, trips, making food, etc 3 months

The multidisciplinary team Physician/neurologist Nurses Physical therapist Occupational therapist Speech therapist Dietician Social worker Psychologist The institutions are specialised regional rehabilitation centres (stroke rehabilitation etc.)

Characteristics of the sample (baseline), n = 37 Variables Male N = 18 (48,6%) Female N = 19 (51,4%) Age 52,4 SD = 13,1 Symptom duration 7,2 år SD = 5,7 Total Functional Capacity (0-13) 8,9 SD = 2,3 Stage (Shoulson & Fahn) I 9 (24,3%) II 21 (56,8%) III 7 (18,9%) UHDRS motor 36,6 16,8 UHDRS behavior 9,2 8,5 Piira A, et al PLOS Curr 2013

Clinical measures at baseline, n = 37 Clinical measures Mean SD MMSE 25,4 3,5 BMI 22,8 3,2 Activities of Balance Confidence (ABC) 72,6 23,9 Bergs Balance Scale (BBS) 52,1 3,8 Timed up and og (TUG) 8,1 3,1 10 Meter Walk test (10MWT) 6,8 2,6 6 Min Walk test (6MWT) 484,9 147,5 Barthel index 19,2 1,3 HADS 8,2 8,7 SF-12 (physical health) 43,9 9,4 SF-12 (mental health) 52,2 11,2 Piira A, et al PLOS Curr 2013

31 out of 37 completed the one-year program as planned (83,8 %)

Score Fig 1. Mean change in health-related quality of life (SF-12) 60 50 ns p=0,000 40 30 20 Baseline Evaluation stay 10 0 Mental health Physical health Piira A, et al PLOS Curr 2013

Meter Fig 2) Mean change in 6 Minutes Walk Test (meters) 580 560 p=0,000 540 520 500 Baseline Evaluation stay 480 460 440 6 Minute Walk Test Piira A, et al PLOS Curr 2013

M/S or seconds Fig 3) Change in gait function 9 8 7 p=0,000 6 5 4 3 2 p=0,000 Timed-up-and-go test (Seconds) 10 Meter Walk Test (m/s) 1 0 Baseline Evaluation stay Timed-up-and-go and 10 Meter Walk Test Piira A, et al PLOS Curr 2013

Scores Fig 4) Mean change in balance: Berg Balance Scale (BBS) and Activities of Balance Confidence scale (ABC) 78 ns 74 70 66 62 58 p= 0.032 Baseline Evaluation stay 54 50 BBS ABC Piira A, et al PLOS Curr 2013

Score Fig 5) Mean change in BMI, HADS and Barthel Index 25 p=,013 20 ns 15 10 p=,004 Baseline Evaluation stay 5 0 HADS BMI Barthel index Piira A, et al PLOS Curr 2013

Score Fig 6) Mean change in cognitive scores 70 ns 60 50 ns Baseline Evaluation stay 40 30 ns ns p=0,019 ns 20 10 0 Stroop word Stroop colour Stroop interference Verbal fluency Symbol digit modality test MMSE Piira A, et al PLOS Curr 2013

Main findings Significant improvements were observed in gait function, balance, in physical quality of life, anxiety and depression, as well as in BMI ADL-function remained stable with no significant decline One cognitive measure (SDMT) showed significant decline, while no decline was observed for the remaining cognitive measures Piira A, et al PLOS Curr 2013

Feasibility? A qualitative, explorative study In-depth interviews with 11 patients and 9 family caregivers Focus groups with 15 health professionals Research questions: How did participants experience the structure and content? What outcomes did patients experience? What challenges and success factors did health professionals report? Frich JC, Røthing M, Berge AR. Participants, caregivers, and professionals experiences with a group-based rehabilitation program for Huntington s disease: a qualitative study. BMC Health Serv Res 2014; 14: 395.

Participants experiences Some had difficulties defining individual rehabilitation goals: Goal setting for individuals with HD is not necessarily a straightforward process. Perhaps that s exactly what they need to work on then need to find out what they need to work on (interview with health professional) Written individualised plans and schedules were appreciated Being member of an HD-group was valuable, though there could be tensions and conflicts in groups Frich JC et al 2014

Participants experiences Participants typically reported improved gait and balance, increased self-confidence, and social outcomes: I have become more open, in a way, and if I fall, I will get back on my feet again this is not how it used to be. I used to be afraid of walking around, in case I would fall (interview with participant) The intensive schedule was acceptable for most participants Adjustments had been made to allow for more time between sessions Frich JC et al 2014

Participants experiences Health professionals - success factors were Assigning every patient with a contact person Using clinical test results as motivation Supervising health professionals in patients local municipalities Frich JC et al 2014

Conclusions Intensive multidisciplinary rehabilitation for individuals with early- to mid-stage HD is feasible and associated with improved motor function and physical quality of life Participants emphasise mental and social outcomes in addition to physical outcomes An individually tailored plan, a contact person, a peer group approach and communication between institutions and primary health professionals could be mediators of outcomes

Conclusions Randomised controlled trials are needed to study effects of various interventions, as well as cost-benefit analysis of interventions Future research should aim at studying effects of specific components in rehabilitation programs Tensions between a standardised intervention and an individually tailored approach? Busse, Multidisciplinary rehabilitation: what future? EHDN News, 2014

Acknowledgements The research team: Anu Piira, Jan Frich, Marleen van Walsem, Synnøve F. Knutsen, Geir Mikalsen, Kjell Havik Nilsen, Alf Reiar Berge, Merete Røthing, Lars Øie Thanks to: Patients, families and The Norwegian Association for HD North Norway Rehabilitation Center Vikersund Rehabilitation Center Centre for Rare Disorders, Oslo University Hospital The Norwegian Directorate of Health Bodil Stokke & Arvid Heiberg