Abstract
Day-to-day functioning is a component of health-related quality of life and is an important end point for therapies to treat Huntington Disease (HD). Specific areas of day-to-day function changes have not been reported for prodromal or very early stages of HD. An exploratory self-report telephone interview was conducted with sixteen people with prodromal HD or early HD who met criteria designed to capture research participants most near to motor diagnosis. All completed semi-structured interviews on function in nine aspects of day-to-day life. Out of 16, 14 reported changes in at least one area. All day-to-day function areas were endorsed by at least one participant with driving being the most common area endorsed by 11/16. Changes in ability to perform some day-to-day tasks are experienced by people who are close to the time of clinical diagnosis for HD. Functional ability is likely to be an important component of outcome assessments of clinical trials and in ongoing clinical management.
Highlights
Huntington disease (HD) is an autosomal dominant, progressive neurodegenerative disorder in which losses of neurologic function continue until the end of the person’s life
These global measures of functioning may not be sensitive to subtle changes in functional abilities that occur in prodromal HD
This study examined self-reports of losses of abilities to perform day-to-day tasks in a small cohort of people in the prodromal HD or very early phase of HD and for whom scores on the UHRDS motor scale indicated a significant increase of motor decline
Summary
Huntington disease (HD) is an autosomal dominant, progressive neurodegenerative disorder in which losses of neurologic function continue until the end of the person’s life. The FDA description of patient-reported outcomes in clinical trials includes measures of any aspect of a patient’s health status that document the effect of a disease on a person’s quality of life, which in turn may include the person’s ability to perform daily activities (FDA, 2006). The FAS[10] is a questionnaire of tasks related to occupation, finances, activities of daily living, domestic chores, level of care, and physical abilities These global measures of functioning may not be sensitive to subtle changes in functional abilities that occur in prodromal HD. Findings have suggested that the currently available measures lack sensitivity to prodromal and very early diseases This limitation is expected since most functional scales were initially developed for neurodegenerative diseases to track stages of dementia. In an effort to maximize the data obtained through the qualitative interviews, we created an algorithm to recruit prodromal research volunteers who are considered most close to receiving a formal motor diagnosis and who were most likely to experience early functional decline
Sign-in/Register to view highlights & summary Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
