| Autor: |
Theresa A. Zesiewicz, Mary Danoudis, Sarah C Milne, Ritchie Malapira, Jane Larkindale, Geneieve Tai, Tyagi Ramakrishnan, Nellie Georgiou-Karistianis, Seok Hun Kim, Jessica D. Shaw, Fatemeh Rasouli, Louise A. Corben, Eppie M. Yiu, Anna Murphy, Jennifer M. Farmer, Martin B. Delatycki |
| Rok vydání: |
2021 |
| Předmět: |
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| DOI: |
10.1101/2021.03.18.434657 |
| Popis: |
ObjectiveTo identify gait and balance measures that are responsive to change during the timeline of a clinical trial in Friedreich ataxia (FRDA) we administered a battery of potential measures three times over a 12-month period.MethodsSixty-one ambulant individuals with FRDA underwent assessment of gait and balance at baseline, six months and 12 months. Outcomes included: GAITRite® spatiotemporal gait parameters; Biodex Balance System Postural Stability Test (PST) and Limits of Stability; Berg Balance Scale (BBS); Timed 25 Foot Walk Test; Dynamic Gait Index (DGI); SenseWear MF Armband step and energy activity; and the Friedreich Ataxia Rating Scale Upright Stability Subscale (FARS USS). The standardised response mean (SRM) or correlation coefficients were reported as effect size indices for comparison of internal responsiveness. Internal responsiveness was also analysed in subgroups.ResultsSenseWear Armband daily step count had the largest effect size of all the variables over six months (SRM=-0.615), while the PST medial-lateral index had the largest effect size (SRM=0.829) over 12 months. The FARS USS (SRM=0.824) and BBS (SRM=-0.720) were the only outcomes able to detect change over 12 months in all subgroups. The DGI was the most responsive outcome in children, detecting a mean change of −2.59 (95% CI −3.52 to −1.66, pConclusionsThe FARS USS and BBS are highly responsive and can detect change in a wide range of ambulant individuals with FRDA. However, therapeutic effects in children may be best measured by the DGI. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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