F098 Introducing a new study: quantifying the clinical, humanistic and economic burden of pre-manifest Huntington’s disease (pre-HDBOI)
Year: 2024
Type: conference-paper
Abstract: Background There is not enough evidence in the literature on the multi-dimensional burden in Persons with Pre-motor manifest Huntington’s Disease (PwPHD) from a multinational perspective. Aims The aim of the Pre-HDBOI is to explore the clinical, humanistic and economic burden in PwPHD in the USA and across Europe. Methods The Pre-HDBOI will be descriptive in nature, and will consist of two data collection instruments: a PwPHD Survey (PS), completed individually by PwPHD, and a Health Care Professional Survey (HCP-S), completed by medical specialists. Both surveys will be administered via bespoke weblinks. Figure 1 illustrates the Pre-HDBOI study design. To ensure the study design robustness, we will be guided throughout by a panel of Key Opinion Leaders and representatives from Patient Advocacy Groups. Results The PS will collect data on the clinical profile, medical and non-medical resource use, and impact of symptoms on the quality of life of PwPHD and work/school impairment. The HCP-S is a unique survey per physician, to provide general information about the PwPHD at their practice, their clinical burden, identified unmet needs, etc. The fieldwork will start in Q3 2024 and study report to be completed by Q1 2025. Conclusion This study aims to examine the burden in PwPHD, building upon insights from the Huntington Disease Burden of Illness study (HDBOI), conducted in 2020-2021 and centered on HD motor-manifest population. The Pre-HDBOI study will further our understanding of the multi-dimensional burden experienced by people with Huntington’s disease as they move through the different stages of their illness.
Language: English
Subfield: Cellular and Molecular Neuroscience
Field: Neuroscience
Domain: Life Sciences
Open Access status: gold
OpenAlex ID: W4402374800