Menu Back to Poster-Presentations-Details

S101: Analysis of Quality of Life Patient-Reported Outcomes in Clinical Trials for Huntington’s Disease

Poster Presenter

James Kim

Undergraduate Student Researcher
University of Southern California
United States

Objectives

Examine the use of Patient-Reported Outcomes (PRO) and biomarkers in Huntington’s Disease (HD) clinical trials (CT) and how they compare to what was important to patients’ from Patient-Focused Drug Design meeting (PFDD)

Method

Conducted literature review on 1. background of HD, 2. what symptoms and outcomes are most important to patients, including the PFDD meeting for HD. Utilizing Clinicaltrials.gov, trials for HD were examined to assess the number of trials conducted, what outcome measures were used, and funding types.

Results

From the PFDD meeting, the symptoms patients were most interested in were psychiatric/behavioral, cognitive, and motor issues. From Clinicaltrials.gov, 239 total trials for HD were filtered based on study type and status, and resulted in 139 trials. The criteria for being filtered out were observational trials, and studies that were suspended, terminated, unknown status, or withdrawn. 62 different outcome measure tools were used throughout the 139 trials. Of the 62 outcome measure tools, 9 were PROs (14.5%). From the outcome measures, 5 different symptoms, or clinical features, were identified: Motor, Cognitive, Behavioral/Psychiatric, QoL, and others. 14 were Cognitive (22.6%), 14 were Behavioral (22.6%), 12 were Motor (19.35%), 10 were QoL (16.1%), and 12 were Others (19.35%). Of the outcome measure tools used, the Unified Huntington’s Disease Rating Scale - Motor Assessment (UHDRS-MA) was the most common, used 44 times (15.1%). The UHDRS Functional Capacity (FC) was the second most used and measures QoL. It was used 23 times (7.9%). The Columbia Suicide Severity Rating Scale (C-SSRS).

Conclusion

There were a variety of outcome measures that were used (62 so far) in the clinical trials studied. Asides from the UHDRS and a couple others, many of them were not disease-specific. Less than 15% of the outcome measures used were PROs which is fairly low. Amongst them, only 1 was found to be HD-related so far: the HD-QoL. This brings into light the necessity and widespread use of a PRO that is validated for HD. In the PFDD meeting for HD, patients voiced other concerns other than their symptoms. Many of them focused on the impact of the disease on daily life, such as a loss of functional capacity and becoming more dependent on others. Very few, only 16.1% of the outcome measures looked at QoL. A possible reason for the many outcome measures used is that because of the standardization of the UHDRS, there weren’t many outcome measures that were sensitive to a symptom, which led to the use of other outcome measures specific to the symptoms trials were focused on. It could also explain the spread in proportion of the symptoms measured. Patients also brought up the necessity of earlier detection, which can be shown by the large amount of biomarker studies.