Developing an Instrumented Measure of Upper Limb Function in Friedreich Ataxia

Area of Science:

• Neurology
• Biomedical Engineering
• Clinical Outcome Measures

Background:

• Upper limb function is crucial for daily activities in individuals with Friedreich ataxia (FA).
• Existing clinical measures inadequately assess FA-related upper limb impairments.
• There is a need for sensitive and functionally relevant outcome measures for FA clinical trials.

Purpose of the Study:

• To evaluate the Ataxia Instrumented Measure-Spoon (AIM-S) as a sensitive and functionally relevant clinical outcome measure for Friedreich ataxia.
• To assess the AIM-S's capacity to detect changes in upper limb function over time in individuals with FA.
• To compare the AIM-S with existing clinical assessments for upper limb function in FA.

Main Methods:

• Development of the AIM-S, utilizing a spoon with a BioKin wireless motion capture device and signal analysis algorithms.
• Prospective longitudinal study design to evaluate AIM-S sensitivity to change over 48 weeks.
• Assessment of Friedreich ataxia clinical severity, Nine-Hole Peg Test, Box and Block Test, and patient-reported outcomes.

Main Results:

• The AIM-S demonstrated greater sensitivity in detecting upper limb function deterioration compared to other measures.
• Patient-reported outcomes indicated the AIM-S reflects a daily activity and was more enjoyable than other assessments.
• The AIM-S proved to be a more accurate and less variable measure of upper limb function in FA.

Conclusions:

• The AIM-S is a superior measure of upper limb function in Friedreich ataxia compared to existing tools.
• The AIM-S is perceived as relevant to daily life by individuals with FA.
• The AIM-S facilitates the inclusion of non-ambulant individuals in future clinical trials, enhancing research accessibility.