Objective: To characterize willingness among people with premanifest Huntington’s disease (PwPHD) to consider early-intervention therapies if available, the minimally acceptable delay in HD onset/progression, and healthcare professionals’ (HCP) perspectives on initiating treatment in early disease.

Background: Limited empirical evidence exists regarding how PwPHD and treating clinicians evaluate the potential benefits of early-intervention therapies. Quantifying what PwPHD and HCPs consider clinically meaningful delay in onset/progression is important for informing clinical development of early interventions.

Method: In the Pre-HDBOI study, a multinational, cross-sectional survey conducted across the US and EU-5, 2 PwPHD subgroups (PwPHD confirmed or at-risk) provided their preferences related to preventative treatment, perceived meaningful benefits, and acceptability of different therapeutic modalities. HD specialists completed a survey evaluating readiness to prescribe early-intervention therapies and their expectations for clinically relevant benefit. All analyses were descriptive.

Results: Among 153 PwPHD, over 90% reported willingness to consider an early-intervention therapy, including gene therapy, aimed at delaying disease onset or slowing disease progression. Approximately 30% of all PwPHD indicated that a 6–12-month delay in onset/progression would represent a minimally acceptable level of clinical benefit. Of those expressing hesitation toward gene therapy (30%), most (72%) cited a desire for more information, rather than categorical reluctance toward the modality. Across 21 HCPs, all indicated willingness to prescribe an early-intervention therapy, if available. Among them, 24% and 38% reported that a 6–12-month delay in onset or progression, respectively, would be sufficient to initiate treatment, aligning with patient expectations. HCPs also emphasized the need for earlier psychological support and more systematic recognition of emerging symptoms in PwPHD.

Conclusion: PwPHD and HD specialists showed strong openness toward early-intervention therapies; even 6-12 month delays in onset or progression were viewed as clinically meaningful. These findings provide important context for defining meaningful benefit, shaping intervention strategies, and informing endpoint selection in HD early-intervention trials.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/perception-of-early-intervention-therapies-and-clinically-meaningful-delays-in-huntingtons-disease-onset-and-progression-the-pre-hdboi-study/