Abstract
BackgroundHuntington’s disease (HD) is a multifaceted neurodegenerative disorder characterized by involuntary movements, specifically chorea, as well as behavioral and psychiatric disturbance, and cognitive dysfunction. Tetrabenazine was the first approved treatment for chorea, although tolerability concerns exist.ObjectivesTo characterize demographic and clinical characteristics of HD patients with chorea based on tetrabenazine use and examine treatment persistence with tetrabenazine in a real-world setting.MethodsPatients with a claim for HD-associated chorea (ICD-9-CM code 333.4) between 1/1/08 and 9/30/15 were selected from the MarketScan® Commercial and Medicare Supplemental databases. The first diagnosis date during the study period was considered the index date, with ≥6 months of continuous medical and prescription coverage before and after the index date. Treatment persistence was defined as the number of days from initiation to discontinuation or end of follow-up period. Discontinuation was defined as a gap in therapy of ≥60 days.Results1644 patients met selection criteria (mean age ± standard deviation: 54.5 ± 15.5), of which 151 (9.2%) were treated with tetrabenazine during the study period. The average (median) daily dose of tetrabenazine during the treatment period was 45.5 (42.3) mg/day. A total of 41.8% (59/141) of HD patients who initiated tetrabenazine experienced a ≥60-day gap in tetrabenazine therapy, with a median time to discontinuation of 293.5 days. During the 6-month post-index period after HD diagnosis, HD patients incurred higher all-cause healthcare costs ($20 204) vs the 6-month pre-index period ($6057), driven by higher hospitalization and pharmacy costs.ConclusionsA small percentage of HD patients with chorea were treated with tetrabenazine and discontinuation rates were high among those receiving treatment, with a median time to discontinuation of 9 months.
Highlights
Huntington’s disease (HD) is a multifaceted neurodegenerative disorder characterized by involuntary movements, chorea, as well as behavioral and psychiatric disturbance, and cognitive dysfunction
Tetrabenazine, a Vesicular monoamine transporter 2 (VMAT2) inhibitor that modulates dopamine, was approved by the US Food and Drug Administration (FDA) as a treatment option for chorea associated with HD in 2008.8,9 In a randomized, double-blind, placebo-controlled study of tetrabenazine in HD patients (TETRA-HD), tetrabenazine significantly improved chorea, but its use was associated with an increased risk of neurologic and psychiatric adverse events (AEs), including drowsiness/somnolence, insomnia, fatigue, fall, depression, agitation, and anxiety.[10]
The primary objective of this study was to characterize demographic and clinical characteristics of HD patients with chorea based on tetrabenazine use utilizing real-world data from the MarketScan® Commercial and Medicare Supplemental Databases
Summary
Huntington’s disease (HD) is a multifaceted neurodegenerative disorder characterized by involuntary movements, chorea, as well as behavioral and psychiatric disturbance, and cognitive dysfunction. Vesicular monoamine transporter 2 (VMAT2) is responsible for the storage and release of dopamine from synaptic vesicles in the brain.[6,7] Tetrabenazine, a VMAT2 inhibitor that modulates dopamine, was approved by the US Food and Drug Administration (FDA) as a treatment option for chorea associated with HD in 2008.8,9 In a randomized, double-blind, placebo-controlled study of tetrabenazine in HD patients (TETRA-HD), tetrabenazine significantly improved chorea, but its use was associated with an increased risk of neurologic and psychiatric adverse events (AEs), including drowsiness/somnolence, insomnia, fatigue, fall, depression, agitation, and anxiety.[10] These neuropsychiatric AEs, likely due to high peak concentrations and plasma fluctuations, cause tolerability concerns that may limit the real-world use of tetrabenazine.[10,11]
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