Hyperpigmentation of Skin

Abstract

<h3>Background</h3> High-frequency, remote assessments of cognitive and motor symptoms in neurodegenerative diseases using smartphones have the potential to monitor disease progression and therapeutic interventions. Here, we report the digital EQ-5D-5L survey results from GENERATION HD1 (NCT03761849). <h3>Aim</h3> To compare changes in self-reported QoL between tominersen treatment arms (120 mg every 8 [Q8W] and 16 weeks [Q16W]) versus placebo using the EQ-5D-5L survey from the Roche HD digital monitoring platform. <h3>Methods/Techniques</h3> Survival analysis of QoL progression was performed using the time of sustained reported worsening as events up until Week 69. Survival curves were compared between Q8W, Q16W and placebo arms, which were split into low- versus high-age (&lt;48 vs. ≥48 years), and low- or high-CAG-age product (CAP) score (&lt;500 vs. ≥500) subgroups. For the visual analogue scale (VAS) score, a worse response was defined as a 10-point decrease. <h3>Results/Outcome</h3> A consistent favourable trend was observed across motor-related EQ-5D-5L items, as well as the index and VAS score, in the Q16W low-age subgroup compared with placebo. The effect was more pronounced in the Q16W low-age, low-CAP subgroup, was substantially weaker in the Q8W low-age, low-CAP subgroup and was not observed in any high-age subgroups. <h3>Conclusions</h3> High-frequency self-reporting of QoL enables sensitive survival analyses of treatment effects. Individuals in the Q16W low-age, low-CAP subgroups appeared to sustain their QoL for longer than placebo, consistent with findings from an exploratory post hoc analysis showing that tominersen may have potential benefits in this subgroup.¹ <h3>Disclosures Statement</h3> GENERATION HD1 was funded by F. Hoffmann-La Roche Ltd. Medical writing support was provided by Chrysalis Medical Communications and funded by F. Hoffmann-La Roche Ltd. <h3>Reference</h3> McColgan P, et al. <i>17^th Annual HD Therapeutics Conference</i>. 2022.