Effect of present state bias on minimal important change estimates: a simulation study.

Abstract

Purpose: The minimal important change (MIC) in a patient-reported outcome measure is often estimated using patient-reported transition ratings as anchor. However, transition ratings are often more heavily weighted by the follow-up state than by the baseline state, a phenomenon known as "present state bias" (PSB). It is unknown if and how PSB affects the estimation of MICs using various methods.

Methods: We simulated 3240 samples in which the true MIC was simulated as the mean of individual MICs, and PSB was created by basing transition ratings on a "weighted change", differentially weighting baseline and follow-up states. In each sample we estimated MICs based on the following methods: mean change (MC), receiver operating characteristic (ROC) analysis, predictive modeling (PM), adjusted predictive modeling (APM), longitudinal item response theory (LIRT), and longitudinal confirmatory factor analysis (LCFA). The latter two MICs were estimated with and without constraints on the transition item slope parameters (LIRT) or factor loadings (LCFA).

Results: PSB did not affect MIC estimates based on MC, ROC, and PM but these methods were biased by other factors. PSB caused imprecision in the MIC estimates based on APM, LIRT and LCFA with constraints, if the degree of PSB was substantial. However, the unconstrained LIRT- and LCFA-based MICs recovered the true MIC without bias and with high precision, independent of the degree of PSB.

Conclusion: We recommend the unconstrained LIRT- and LCFA-based MIC methods to estimate anchor-based MICs, irrespective of the degree of PSB. The APM-method is a feasible alternative if PSB is limited.