Objective: To evaluate the effectiveness and safety of deutetrabenazine (DTBZ) in Chinese patients with Huntington’s Disease (HD)-associated chorea.

Background: Chorea is a key symptom of HD that gradually spreads to all body regions, raising injury risk and leading to full dependency and death [1]. DTBZ, a selective VMAT2 inhibitor, is approved for HD-associated chorea and has shown efficacy and tolerability in trials [2-3]. Evidence in Asian population is limited.

Method: This was a multicenter, prospective, interventional Phase 4 study across 3 sites in China (ChiCTR2300077341). Adults with HD-associated chorea and baseline total maximal chorea (TMC) score ≥8 were enrolled and followed up to Week 16. DTBZ was titrated then maintained at physician-determined doses and durations. Primary endpoint was TMC score change from baseline to Week 16 in patients receiving DTBZ ≥24 mg/day for ≥4 weeks. Secondary endpoints were TMC change in all patients and safety; change in <24 mg/day group was exploratory. Descriptive analyses and paired t-tests were conducted.

Results: Fifty patients were enrolled and included in the effectiveness and safety analyses. The mean (SD) age of all patients was 48.9 (10.8) years, 50.0% were male, and the mean (SD) chorea duration was 5.6 (3.2) years (Table 1). All patients had motor symptoms, 52.0% had psychiatric symptoms, and 28.0% had cognitive impairment. The mean (SD) CAG repeat length was 44.9 (3.9). The mean (SD) treatment duration was 15.9 (0.7), 15.9 (0.9), and 15.9 (1.6) weeks in ≥24 mg/day (N=42), all dosage (N=50), and <24 mg/day (N=8) groups, respectively. Mean TMC scores significantly reduced by 4.20 (SD=2.59, p<0.01) in ≥24 mg/day group (Figure 1). A similar reduction was shown in all dosage group (-3.88, SD=2.90, p<0.01), a non-significant decrease was shown in <24 mg/day (-2.25, SD=3.96, p=0.15). Twenty-one patients (42.0%) had 36 adverse events (AEs), 88.9% of which were grade 1-2 (mild to moderate) (Table 2). Treatment-related AEs occurred in 12 patients (24.0%). Two patients (4.0%) had 3 serious AEs (acute pancreatitis, pneumonia, pneumothorax); none were treatment related. No patients discontinued treatment due to AEs.

Conclusion: DTBZ showed significant symptom improvement in Chinese patients with HD-associated chorea in both ≥24 mg/day and all dosage. The safety profile was as expected and consistent with prior clinical trials.

Table 1

Table 2

Figure 1

References: [1] Roos RA. Huntington’s Disease: A Clinical Review. Orphanet journal of rare diseases. Dec 20 2010;5:40. doi:10.1186/1750-1172-5-40
[2] Huntington Study G, Frank S, Testa CM, et al. Effect of Deutetrabenazine on Chorea Among Patients With Huntington Disease: A Randomized Clinical Trial. JAMA. Jul 5 2016;316(1):40-50. doi:10.1001/jama.2016.8655
[3] Frank S, Stamler D, Kayson E, et al. Safety of Converting From Tetrabenazine to Deutetrabenazine for the Treatment of Chorea. JAMA neurology. Aug 1 2017;74(8):977-982. doi:10.1001/jamaneurol.2017.1352

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