European Quality of Life Five Dimension (EQ-5D) Utility Values for Clinical Health States in Fabry Disease (FD): Insights from Pegunigalsidase Alpha Clinical Trials
Author(s)
Azimpour K1, Dorling P1, Koulinska I1, Lan Z2, Poritz J3, Tremblay G2, Raad A4
1Chiesi, Boston, MA, USA, 2Cytel Inc., Waltham, MA, USA, 3Cytel Inc., Houston, TX, USA, 4Cytel Inc., Toronto, ON, Canada
Presentation Documents
OBJECTIVES: FD is a progressive, rare X‑linked lysosomal storage disorder caused by GLA gene mutations, resulting in α-galactosidase A enzyme deficiency. The safety and efficacy of pegunigalsidase alfa (Elfabrio®) in FD patients previously treated with Enzyme replacement therapies (ERT) was evaluated in phase III BALANCE, BRIDGE, and BRIGHT trials. The objective of this study was to estimate the utility values of patients from the BALANCE, BRIDGE and BRIGHT trials.
METHODS:
Individual patient-level data for EQ-5D-5L from all three trials were included in the analysis. Of the 129 participants across the three trials, 124 had at least one EQ-5D-5L follow-up measurement. Data from both treatment arms of the BALANCE trial were included in the analysis. EQ-5D-5L was mapped to EQ-5D-3L, as recommended by NICE, using the “eq5dmap” function within the “eq5d” R package. Health states were based on patient experience of one or more types of Fabry clinical events (FCE) or pain-related adverse event (AE). The impact of covariates including study, age, sex, treatment arm, estimated glomerular filtration rate, baseline EQ-5D-3L, serious AE, pain-related AE, FCE (renal, cardiac, or cerebrovascular) were modelled via mixed-effects linear regression. Best fit was obtained through backward-stepwise model selection.RESULTS: Total mean (standard deviation) utility values across three trials were 0.778 (0.230) at baseline and ranged from 0.808 (0.201) to 0.742 (0.261) at Weeks 26–104 of follow-up. In the mixed-effect model, baseline EQ-5D-3L significantly impacted follow-up utility values (p<0.0001), whereas all other variables were not significant. Final model-derived utilities were 0.800 for no FCE/no pain-related AE, 0.774 for pain-related AE, 0.719 for cardiac FCE, 0.792 for cerebrovascular FCE, and 0.688 for renal FCE.
CONCLUSIONS:
This study utilized data from the BALANCE, BRIDGE, and BRIGHT trials to generate EQ-5D-3L utility values in FD. The resulting utility values were generally consistent with previously published values.Conference/Value in Health Info
Value in Health, Volume 26, Issue 11, S2 (December 2023)
Code
PCR143
Topic
Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Health State Utilities, PRO & Related Methods
Disease
Neurological Disorders, Rare & Orphan Diseases