Huntington Disease May Be Undertreated and Current Monotherapies Inadequate

brain scan of someone with Huntington disease
brain scan of someone with Huntington disease
Investigators used data from the Enroll-HD registry to assess patterns of treatment for chorea in patients with Huntington disease.

The following article is a part of conference coverage from Psych Congress 2021 , held October 29th through November 1, 2021, in San Antonio, Texas. The team at Psychiatry Advisor will be reporting on the latest news and research conducted by leading experts in psychiatry. Check back for more from the Psych Congress 2021.


A minority of individuals with chorea are receiving anti-chorea medication, which suggests that Huntington disease may be undertreated, according to research recently presented at Psych Congress 2021, held from October 29 through November 1, 2021, in San Antonio, Texas.

Derived from data collected between June 2012 and October 2020 in a prospective, worldwide, observational study (Enroll-HD; Identifier: NCT01574053), this analysis included 2590 North American adults with a Unified Huntington Disease Rating Scale (UHDRS) diagnostic confidence level of 4.  The study researchers defined chorea as a UHDRS total maximal chorea score of at least 2. Participants were undergoing treatment with antipsychotic agents alone, vesicular monoamine transporter 2 inhibitors (VMAT2) alone, medication other than antipsychotic agents or VMAT2 (other), or a combination of 2 or more of these categories (combination).

Among the study population, 96.8% (n=2507) met the threshold for chorea during 96.5% (6678 of 6920) of visits. Anti-chorea medication was prescribed to 36.1% (n=906) of individuals with chorea at any visit. The most common treatments, in order of frequency prescribed, were VMAT2 (49.9%), antipsychotic agents (27.5%), other (18.7%), and combination (4%). Antipsychotic agents had the longest first-line therapy duration (41.2 months) and VMAT2 the shortest (28.8 months). Among individuals prescribed anti-chorea medication, 75.9% (n=688) continued the first medication prescribed to them (VMAT2, 42%; antipsychotic agents, 21.2%; other, 9.8%; and combination, 2.9%), and 16.2% (n=147) changed treatment. Second-line treatment was most commonly combination (72.1%). Medication discontinuation for more than 90 days occurred in 7.8% (n=71) of patients initially receiving anti-chorea medication, with a mean discontinuation time of 1 to 2 years; of those who switched to second-line therapies, 29.4% (n=64) eventually discontinued treatment or switched to third-line  therapies.

The study authors conclude that “96.8% of patients with manifest [Huntington disease] presented with chorea at any study visit, yet only 36.1% of patients were prescribed a medication to address it.” However, they advise that further research is necessary “to better understand optimal treatment patterns for chorea in patients with [Huntington disease] throughout the disease course.”

Disclosure: This clinical trial was supported by Neurocrine Biosciences, Inc. Please see the original reference for a complete list of authors’ disclosures.


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Furr-Stimming EE, Claassen DO, Sen GP, et al. Longitudinal treatment patterns for chorea in patients with Huntington disease: data from Enroll-HD. Presented at Psych Congress 2021; October 29-November 1, 2021; San Antonio, Texas. Poster 41.