Presentation (CTI)

OBJECTIVES: Primary lateral sclerosis (PLS) is a rare neurodegenerative disorder with slow progression and heterogeneous symptoms, that may have substantial impact on health-related quality of life (HRQoL). Though a number of generic HRQOL and preference-based measures (PBMs) exist to measure HRQoL impact; they have not been assessed for their suitability in PLS. Therefore, the objective was to compare the validity, coverage of relevant domains, feasibility, and acceptability of those measurements for assessing the impact of PLS on the HRQoL of individuals.
METHODS: A targeted literature review was conducted to identify drivers of HRQoL among individuals with PLS. Two independent reviewers mapped HRQoL drivers against domains of generic HRQoL and PBM instruments. A gap analysis was performed to assess whether these instruments capture the primary signs and symptoms of PLS. Available data were tabulated, and overall recommendations for measure selection in PLS formulated.
RESULTS: Drivers of individuals HRQoL included physical function (e.g. upper and lower limb), emotion and mood, fatigue, speech and swallowing, cognitive impairment, and pain. None of the reviewed articles included a disease specific HRQoL or preference-based utility measure and no PBM had been validated in PLS. Of the generic PBMs assessed, HUI3 provides the strongest direct coverage of relevant HRQoL drivers, while EQ-5D and SF-6D may mainly capture relevant HRQoL divers indirectly through selected domains. The SF-12 and SF-36 provide broader coverage of relevant HRQoL drivers, with the SF-36 including domains related to upper limb function. These instruments can support derivation of utilities for economic evaluations via mapping.
CONCLUSIONS: Generic HRQoL instruments and PBMs with coverage of PLS-relevant constructs may provide useful insights into the disease burden. Further research is needed to inform the selection of outcome measures for HRQoL studies, health economic evaluations and clinical trials in PLS.