Medication Use and Treatment Indications in Huntington's Disease; Analyses from a Large Cohort

Background: Huntington's Disease is a rare neurodegenerative disorder in which appropriate medication management is essential. While many medications are prescribed based on expert knowledge, overviews of actual medication use in HD are sparse.

Objectives: We provide a detailed overview of medication use and associated indications across HD disease stages, considering sex and regional differences.

Methods: Data from the largest observational HD study, ENROLL-HD, were used. We created HD-related medication and indication classes to identify medication trends in manifest, premanifest and control subjects. We studied medication use in adult, childhood- and adolescent-onset HD, incorporating disease stage (including phenoconverters), sex and regional differences.

Results: In 8546 manifest HD patients, 84.6% used medication (any type), with the average number of medications per user rising from 2.5 in premanifest HD to 5.2 in end stage disease. Antipsychotics (29.2%), SSRIs (27.5%) and painkillers (21.8%) were most often used. Medication use varied with disease progression. Several differences were observed between the sexes, and notably between Europe and Northern America as well. Medication use increased after phenoconversion (from 64.8% to 70.6%, P < 0.05), with the largest difference in antipsychotic use (4.4%-7.8%, P < 0.05). Medication patterns were different in childhood-onset HD, with no use of painkillers, less use of anti-chorea and antidepressant drugs, and more for aggression and irritability.

Conclusions: Medication use in HD increases with disease progression, with varying types of medications prescribed based on disease stage, sex, and region of living. Recognizing these medication trends is vital for further personalized HD management.