Cheney J G Drew1, Lori Quinn2, Katy Hamana3, Rhys Williams-Thomas1, Lucy Marsh1, Polyxeni Dimitropoulou1, Rebecca Playle1, Beth Ann Griffin4, Mark Kelson5, Robin Schubert6, Lisa Muratori7, Ralf Reilmann8, Anne Rosser9, Monica Busse10. 1. Centre for Trials Research, Cardiff University, South Glamorgan, United Kingdom. 2. Centre for Trials Research, Cardiff University; and Teachers College, Columbia University, New York, New York. 3. School of Health Care Sciences, Cardiff University. 4. RAND Corporation, Arlington, Virginia. 5. Department of Mathematics, University of Exeter, Exeter, United Kingdom. 6. George Huntington Institute and Institute for Clinical Radiology, University of Münster, Münster, Germany. 7. George Huntington Institute and Institute for Clinical Radiology, University of Münster; and School of Health Technology and Management, Stony Brook University, Stony Brook, New York. 8. George Huntington Institute and Institute for Clinical Radiology, University of Münster; and Department of Neurodegenerative Diseases and Hertie-Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany. 9. School of Bioscience, Cardiff University. 10. Centre for Trials Research, Cardiff University, 4th Floor, Neuadd Meirionydd Health Park, Cardiff, South Glamorgan CF14 7YS, United Kingdom.
Abstract
BACKGROUND: Exercise is emerging as an important aspect in the management of disease-related symptoms and functional decline in people with Huntington disease (HD). Long-term evaluation of physical activity and exercise participation in HD has yet to be undertaken. OBJECTIVE: The objective is to investigate the feasibility of a nested randomized controlled trial (RCT) alongside a longitudinal observational study of physical activity and exercise outcomes in people with HD. DESIGN: This will be a 12-month longitudinal observational study (n = 120) with a nested evaluation of a physical activity intervention (n = 30) compared with usual activity (n = 30) using a "trial within a cohort" design. SETTING: The study will take place in HD specialist clinics in Germany, Spain, and the United States, with intervention delivery in community settings. PARTICIPANTS: The participants will have early-mid-stage HD and be participating in the Enroll-HD study. INTERVENTION: This will be a 12-month physical activity behavioral change intervention, delivered by physical therapists in 18 sessions, targeting uptake of aerobic exercise and increased physical activity. MEASUREMENTS: All participants (n = 120) will complete Enroll-HD assessments (motor, cognitive, behavioral, and quality of life) at baseline and at 12 months. Additional Physical ACtivity and Exercise Outcomes in Huntington Disease (PACE-HD) assessments include fitness (predicted maximal oxygen uptake [V o2max]), self-reported and quantitative measures of physical activity, disease-specific symptoms, and walking endurance. RCT participants (n = 60) will complete an additional battery of quantitative motor assessments and a 6-month interim assessment. Enroll-HD data will be linked to PACE-HD physical activity and fitness data. LIMITATIONS: The limitations include that the embedded RCT is open, and assessors at RCT sites are not blinded to participant allocation. CONCLUSION: PACE-HD will enable determination of the feasibility of long-term physical activity interventions in people with HD. The novel "trial within a cohort" design and incorporation of data linkage have potential to reduce participant burden. This design could be applied to other neurological diseases and movement disorders where recruitment and retention are challenging.
BACKGROUND: Exercise is emerging as an important aspect in the management of disease-related symptoms and functional decline in people with Huntington disease (HD). Long-term evaluation of physical activity and exercise participation in HD has yet to be undertaken. OBJECTIVE: The objective is to investigate the feasibility of a nested randomized controlled trial (RCT) alongside a longitudinal observational study of physical activity and exercise outcomes in people with HD. DESIGN: This will be a 12-month longitudinal observational study (n = 120) with a nested evaluation of a physical activity intervention (n = 30) compared with usual activity (n = 30) using a "trial within a cohort" design. SETTING: The study will take place in HD specialist clinics in Germany, Spain, and the United States, with intervention delivery in community settings. PARTICIPANTS: The participants will have early-mid-stage HD and be participating in the Enroll-HD study. INTERVENTION: This will be a 12-month physical activity behavioral change intervention, delivered by physical therapists in 18 sessions, targeting uptake of aerobic exercise and increased physical activity. MEASUREMENTS: All participants (n = 120) will complete Enroll-HD assessments (motor, cognitive, behavioral, and quality of life) at baseline and at 12 months. Additional Physical ACtivity and Exercise Outcomes in Huntington Disease (PACE-HD) assessments include fitness (predicted maximal oxygen uptake [V o2max]), self-reported and quantitative measures of physical activity, disease-specific symptoms, and walking endurance. RCT participants (n = 60) will complete an additional battery of quantitative motor assessments and a 6-month interim assessment. Enroll-HD data will be linked to PACE-HD physical activity and fitness data. LIMITATIONS: The limitations include that the embedded RCT is open, and assessors at RCT sites are not blinded to participant allocation. CONCLUSION: PACE-HD will enable determination of the feasibility of long-term physical activity interventions in people with HD. The novel "trial within a cohort" design and incorporation of data linkage have potential to reduce participant burden. This design could be applied to other neurological diseases and movement disorders where recruitment and retention are challenging.
Authors: Swati Sathe; Jen Ware; Jamie Levey; Eileen Neacy; Robi Blumenstein; Simon Noble; Alzbeta Mühlbäck; Anne Rosser; G Bernhard Landwehrmeyer; Cristina Sampaio Journal: Front Neurol Date: 2021-08-18 Impact factor: 4.086