Objective: To assess if deutetrabenazine treatment of chorea associated with HD resulted in changes in QoL, Patient Global Impression of Change (PGIC), or Clinical Global Impression of Change (CGIC).

Background: In First-HD, a double-blind, placebo-controlled, parallel-group study in which 90 patients with HD were randomized 1:1 to deutetrabenazine or placebo, 12 weeks of deutetrabenazine was well tolerated and improved Unified Huntington Disease Rating Scale total maximal chorea (TMC) score (primary endpoint; P<.0001 vs placebo). Additional scales were used to investigate the clinical utility of reducing TMC, which can provide insight into the larger clinical impact of therapeutic interventions. SF-36 is a recommended QoL measure in HD patients; global outcomes provide insight into patients’ and clinicians’ views on overall patient status and may be especially useful to gauge improvement over time.

Methods: First-HD secondary endpoints included change in the SF-36 physical functioning scale PF-10 (items 3a to 3j) from baseline (BL) to Week 12 and PGIC and CGIC assessed at Weeks 4, 9, and 12, with improvement defined as a rating of “much improved” or “very much improved” compared with BL. Adverse events (AEs) were monitored throughout the study. The Hospital Anxiety and Depression Scale (HADS) was used to assess neuropsychiatric safety.

Results: Deutetrabenazine-treated patients reported an improvement in mean [SD] SF-36 PF-10 score compared to a worsening with placebo at Week 12 (0.74 [9.77] vs –3.61 [9.67]; P=.03). There were no depression- or anxiety-related safety signals detected with deutetrabenazine, as measured by the HADS or AE reporting. Deutetrabenazine resulted in improvement on the PGIC vs placebo at Week 9 (P=.0008), which was maintained at Week 12 (P=.0002). This was consistent with the improvement on the CGIC in deutetrabenazine patients vs placebo at Weeks 9 (P=.0006) and 12 (P=.0014).

Conclusions: Deutetrabenazine was generally well tolerated and resulted in a significant improvement in QoL, as measured by SF-36 PF-10, and global impression of overall HD symptoms, as reported by both patients and clinicians, compared with placebo. HD patients were able to provide meaningful input on the overall impact of a new therapy in a blinded clinical trial setting.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/evaluation-of-quality-of-life-qol-patient-and-clinician-reported-outcomes-with-deutetrabenazine-treatment-of-chorea-in-huntington-disease-in-first-hd/