F101 Huntington’s disease progression and health economics: insights from the enroll-hd database analysis

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Background The Enroll-HD database provides a valuable resource for understanding the long-term progression of Huntington’s disease (HD), which is crucial for developing effective treatments and exploring their health economic implications. Aims Our study aimed to analyze how disease progression varies based on patients‘ age at database entry and their initial scores on HD clinical, cognitive and motor measures. The findings of this analysis were used to validate our health economic modeling approach to HD natural history, in which we estimated the rate of HD progression over a remaining lifetime to calculate expected healthcare costs and quality-adjusted survival. Methods/Techniques: We calculated the average scores over time, depending on their initial scores, utilizing longitudinal data from the Enroll-HD database. We focused on Total Functional Capacity (TFC), Symbol Digit Modalities Test (SMDT), Stroop Word Reading (SWR), Total Motor Score (TMS), and Independence Scale (IS) scores. Results/Outcome All progression score trajectories were near-linear for the large-N visits. However, results became more uncertain over time due to fewer patients and/or loss to follow-up. The progression trajectory of HD patients remained similar regardless of age at database entry or initial scores, indicating that the rate of HD progression is relatively constant over time in most patients. Conclusions These results, in tandem with our health economic model findings, underscore the importance of early intervention and monitoring in HD management. Targeting future treatments at earlier stages of HD progression will likely be more cost-effective by delaying progression to HD stages with a greater disease burden and associated costs.

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