POSB349 Work Productivity and Activity Impairment of Huntington's Disease Patients By Disease Stage in the US and EU5: Evidence from the Huntington's Disease Burden of Illness Study (HDBOI)

Santana, I Rodriguez; Frank, Samuel; Hamilton, Jamie; Hubberstey, Hayley; Arnesen, Astri; Ruiz, Leonardo; Willock, Robina Josiah; Doherty, Mary Ellen; Dolmetsch, Ricardo; Li, N; Ratsch, Sarah; Ali, TM

doi:10.1016/j.jval.2021.11.1116

Value in Health

2022

DOI: 10.1016/j.jval.2021.11.1116

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POSB349 Work Productivity and Activity Impairment of Huntington's Disease Patients By Disease Stage in the US and EU5: Evidence from the Huntington's Disease Burden of Illness Study (HDBOI)

I Rodriguez Santana

Samuel Frank

Jamie Hamilton

et al.

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2024

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Long-Term Health Outcomes of Huntington Disease and the Impact of Future Disease-Modifying Treatments

Guzauskas,

Tabrizi,

Long

et al. 2024

Neur Clin Pract

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Background and ObjectivesDisease-modifying treatments (DMTs) such as gene therapy are currently under investigation as a potential treatment for Huntington disease (HD). Our objective was to estimate the longterm natural history of HD progression and explore the potential efficacy impacts and value of a hypothetical DMT using a decision-analytic modeling framework. MethodsWe developed a health state transition model that separately analyzed 40-year-old individuals with prefunctional decline (PFD, HD Integrated Staging System [HD-ISS] stage <3, total functional score [TFC] 13), active functional decline Shoulson and Fahn category 1 (SF1, HD-ISS stage 3, TFC 13-11), and SF2 (HD-ISS stage 3, TFC 10-7). Three-year outcomes from the TRACK-HD longitudinal study were linearly extrapolated to estimate the long-term health outcomes and costs of each population. For PFD individuals, we used the HD-ISS to predict the onset of functional decline. HD costs and quality-adjusted life years (QALYs) were estimated over a lifetime horizon by applying health state-specific costs and utilities derived from a related HD burden-of-illness study. We then estimated the long-term health impacts of hypothetical DMTs that slowed or delayed onset of functional decline. We conducted sensitivity analyses to assess model uncertainties. ResultsThe expected life years for 40-year-old PFD, SF1, and SF2 populations were 20.46 (95% credible range [

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Long-Term Health Outcomes of Huntington Disease and the Impact of Future Disease-Modifying Treatments

Guzauskas,

Tabrizi,

Long

et al. 2024

Neur Clin Pract

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show abstract

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POSB349 Work Productivity and Activity Impairment of Huntington's Disease Patients By Disease Stage in the US and EU5: Evidence from the Huntington's Disease Burden of Illness Study (HDBOI)

Cited by 1 publication

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Long-Term Health Outcomes of Huntington Disease and the Impact of Future Disease-Modifying Treatments

Long-Term Health Outcomes of Huntington Disease and the Impact of Future Disease-Modifying Treatments

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