1,259
Views
0
CrossRef citations to date
0
Altmetric
Research Article

Meaningful within-patient change for clinical outcome assessments: model-based approach versus cumulative distribution functions

, , , , , & show all
Received 23 Sep 2022, Accepted 31 Oct 2023, Published online: 20 Nov 2023

Figures & data

Figure 1. eCDF/ePDF curves of target COA (WOMAC pain subscale) change from baseline by anchor change.

eCDF empirical cumulative distribution function, ePDF empirical probability density function, COA clinical outcome assessment.
Figure 1. eCDF/ePDF curves of target COA (WOMAC pain subscale) change from baseline by anchor change.

Table 1. Descriptive statistics of the change from baseline in WOMAC pain subscale for each category of the anchor change.

Figure 2. Estimates of within-patient change in target COA (WOMAC pain subscale) by model-based and eCDF methods.

COA clinical outcome assessment, eCDF empirical cumulative distribution function, eCDF_mean_visit mean of eCDF-derived means by visit, eCDF_median_visit median of eCDF medians by visit, PRO patient-reported outcome, RMM_cat/RMM_con repeated measures model with a categorical/continuous anchor change.
Figure 2. Estimates of within-patient change in target COA (WOMAC pain subscale) by model-based and eCDF methods.

Table 2. Estimates of within-patient change in WOMAC pain subscale by model-based and eCDF methods in the real-life data set.

Figure 3. Within-patient change in target COA (WOMAC pain subscale) given 1-level anchor change (bootstrapping and parametric model results).

COA clinical outcome assessment, eCDF empirical cumulative distribution function, PRO patient-reported outcome, RMM repeated measures model. The yellow dot and gray line indicate a point estimate and corresponding 95% confidence interval, respectively.
Figure 3. Within-patient change in target COA (WOMAC pain subscale) given 1-level anchor change (bootstrapping and parametric model results).

Table 3. Estimates of within-patient change in WOMAC pain subscale by model-based and eCDF methods in the simulated data set.