Presentation (CTI)

OBJECTIVES: Primary lateral sclerosis (PLS) is a rare neurodegenerative disorder characterised by slow progression and heterogeneous symptoms, that may substantially impact on health-related quality of life (HRQoL). Previous research suggests that generic preference-based measures (PBMs) may inadequately capture important dimensions of HRQoL. This study evaluated the ability of the EQ-5D to capture utility relevant disease impacts across early vs late PLS by mapping EQ-5D domains against key disease features identified in the literature.
METHODS: Two hypothetical PLS profiles representing distinct disease stages were constructed using literature-derived disease characteristics and medical expert input. HRQoL drivers for each profile were mapped against EQ-5D domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) by two independent reviewers to assess the extent to which the instrument captures key impacts across disease stages.
RESULTS: Key HRQoL drivers identified in the literature included physical function (e.g. upper and lower limb impairment), emotion and mood, fatigue, speech and swallowing difficulties, cognitive impairment, and pain. Mapping suggested several gaps in the relevance and comprehensiveness of the EQ-5D across disease stages, particularly for impacts related to speech, swallowing, and cognition, which are not directly assessed within EQ-5D domains. These findings suggest that the EQ-5D may more readily capture overt physical decline in late disease, while underrepresenting important HRQoL impacts experienced in early disease progression.
CONCLUSIONS: The EQ-5D may inadequately capture important HRQoL impacts across disease stages in PLS. As a result, utility estimates derived from generic PBMs may underrepresent treatment-related benefits, especially earlier in disease progression. Alternative approaches, including disease-specific assessment tools and vignette-based utility estimation, may warrant further exploration to better characterise burden and utility in PLS.