Efficacy analysis assessing differences in overall response rate, ≥VGPR and ≥CR in the intervention and placebo arms of the BELLINI trial by IRC and the dR algorithm using 50% of all available laboratory measurements and 25% of all urine M protein measurements.
MINOR
*Nominal p-values without multiple hypotheses correction are shown for completeness and serve to compare the estimated treatment effect between dR and IRC.CI, confidence interval; CMH, Cochran-Mantel-Haenszel; CR, complete response; dR, derived response; IRC, independent review committee; M, monoclonal; ORR, overall response rate; VGPR, very good partial response.For consistency, we tested the algorithm on the same datasets with missing data, but excluded the cases with insufficient laboratory measurements from the statistical analysis. The exclusion resulted in minor reduction of the Cohen’s Kappa statistic to 0.71 for both datasets with missing data when responders and non-responder assignments were compared and to 0.45 and 0.34 when multiple depths of response were considered in the 50% and 75% missing dataset, respectively.