EQ-5D-3L full health state discriminates between drug and placebo in clinical trials of systemic lupus erythematosus
Abstract Objectives To investigate the discriminative ability of EQ-5D-3L full health state (FHS) in clinical trials of SLE, and identify factors associated with FHS after treatment. Methods Data from the BLISS-52 (NCT00424476) and BLISS-76 (NCT00410384) trials of belimumab (N = 1684) were utilised. FHS was defined as a response of no problems in all five EQ-5D-3L dimensions, yielding an index score of 1. The Pearson’s chi-square or Fisher’s exact test was employed for comparisons, and logistic regression for adjustments and assessment of independence. Results We demonstrated higher EQ-5D-3L FHS frequencies among patients given standard therapy (ST) plus the licensed belimumab dose versus ST alone (26.1% versus 19.4%; P = 0.001; week 52), and within SRI-4 responders versus non-responders (27.0% versus 19.8%; P < 0.001; week 52) from week 36 to 52. In multivariable regression analysis, SLEDAI-2K (OR: 0.90; 95% CI: 0.87 − 0.94; P < 0.001) and SLICC/ACR Damage Index (OR: 0.79; 95% CI: 0.69 − 0.91; P = 0.001) scores were independently associated with lower FHS frequencies at week 52, while adding monthly infusions of belimumab 10 mg/kg to ST favoured FHS perception (OR: 1.60; 95% CI: 1.15 − 2.24; P = 0.006). Add-on belimumab 10 mg/kg yielded higher FHS frequencies in antimalarial users versus non-users (29.9% versus 20.1%; P = 0.011), and in anti-dsDNA and anti-Sm positive versus negative patients (31.4% versus 13.4%; P < 0.001 and 33.0% versus 22.6%; P = 0.010, respectively), whereas no significant differences were observed in patients given ST alone. Conclusion EQ-5D-3L FHS distinguished belimumab from placebo and responders from non-responders, and exhibited known-group validity in subgroup analysis. FHS may prove a useful patient-reported outcome in SLE studies.