Objective: To evaluate self- and caregiver-reported patient burden of Huntington disease (HD) chorea and the impact of deutetrabenazine (DTBZ) treatment on symptom management, quality of life (QoL), and patient-defined functional goals in a decentralized population.

Background: HD is a neurodegenerative disorder characterized by chorea that impairs daily functioning and QoL [1]. Burden is multifaceted for patients and caregivers, and understanding both perspectives is essential to characterizing real-world impact. Vesicular monoamine transporter 2 (VMAT2) inhibitors reduce chorea and may support meaningful functional improvement [2].

Method: Participants (aged ≥18 years) from the US self-reporting HD chorea (patients) or caring for someone with HD chorea (caregivers) completed a non-interventional online survey via the myHDStory platform. The survey assessed chorea-related burden across social, emotional, physical/activities of daily living (ADL), and vocational/recreational domains and current treatment. Results are presented for self- and caregiver-reported patient burden, stratified by treatment (DTBZ only, other VMAT2 inhibitor, or no treatment). DTBZ users completed additional questions on symptom change since initiation, including Patient Global Impression of Change (PGIC).

Results: Among 425 respondents, 192 were patients (mean age 32.5 years (SD, 8.4); 67.2% male) and 233 were caregivers reporting on patients (mean age 37.1 years (SD, 13.4); 54.5% male). Most patients and caregivers reported chorea interfered with social (patient, 63–92%; caregiver, 77–89%), emotional (61–84%; 67–95%), physical/ADL (63–87%; 77–92%), and vocational/recreational (63–94%; 77–97%) functioning and considered chorea important to manage. Among patients receiving DTBZ, 60–71% reported improvements across domains and 74% reported PGIC improvement. Caregiver-reported patient outcomes with DTBZ were similar or higher, with 80–86% reporting domain improvements and 91% reporting chorea improvement. Free-text responses described functional treatment goals.

Conclusion: In this decentralized real-world survey, patients and caregivers reported substantial multidimensional burden of HD chorea. Improvements with DTBZ were observed across domains from both perspectives, supporting inclusion of caregiver-reported patient outcomes in HD chorea research.

References: 1. van Lonkhuizen PJC, et al. Quality of life, health-related quality of life, and associated factors in Huntington’s disease: a systematic review. J Neurol. 2023;270(5):2416–37.
2. Baghaei A, et al. Safety and efficacy of VMAT2 inhibitors in Huntington Disease: A systematic review. Parkinsonism Relat Disord. 2026;28:108209.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/patient-and-caregiver-perspectives-on-patient-burden-of-huntington-disease-chorea-and-the-real-world-impact-of-deutetrabenazine-treatment/