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Updated: Jun 10, 2025

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Published on: June 9, 2018
Medication Use and Treatment Indications in Huntington's Disease; Analyses from a Large Cohort
Stephanie Feleus1,2, Lara E M Skotnicki1, Raymund A C Roos1
1Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
Insights
Medication use in Huntington's Disease (HD) increases with disease stage. Prescription patterns vary by age of onset, sex, and region, highlighting the need for personalized treatment strategies.
Area of Science:
- Neuroscience
- Pharmacology
- Genetics
Background:
- Huntington's Disease (HD) is a rare neurodegenerative disorder requiring careful medication management.
- Current understanding of actual medication use patterns in HD is limited.
Purpose of the Study:
- To provide a comprehensive overview of medication use in HD.
- To analyze medication indications across different HD disease stages.
- To explore variations in medication use based on sex and geographic region.
Main Methods:
- Utilized data from the ENROLL-HD observational study, the largest of its kind.
- Developed medication and indication classes to identify trends in premanifest, manifest, and control subjects.
- Examined medication use in adult, childhood- and adolescent-onset HD, considering disease stage, phenoconversion, sex, and region.
Main Results:
- 84.6% of 8546 manifest HD patients used medication, with average prescriptions increasing from 2.5 to 5.2 from premanifest to end-stage disease.
- Most common medications included antipsychotics (29.2%), SSRIs (27.5%), and painkillers (21.8%).
- Medication use rose post-phenoconversion, with notable differences observed between sexes and regions (Europe vs. Northern America). Childhood-onset HD showed distinct patterns, including no painkiller use and increased medication for aggression.
Conclusions:
- Medication use in Huntington's Disease escalates with disease progression.
- Prescribed medications differ based on disease stage, patient sex, and geographic location.
- Understanding these medication trends is crucial for tailoring personalized management strategies for HD patients.
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.
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