Defining Remission Using Patient-reported Outcome Components of the Crohnʼs Disease Activity Index: Post-hoc Analyses of Vedolizumab Data from the GEMINI 2 Study

Introduction: Clinical trials in Crohn's disease (CD) are evolving toward including patient (pt)-reported outcomes (PROs) to evaluate drug efficacy; however, no validated PROs are available. In post hoc analyses of data from the GEMINI 2 study,1 we evaluated different definitions of vedolizumab (VDZ)-induced remission using PRO components of the CD Activity Index (CDAI).Table 1: Definitions of RemissionMethods: Pts with moderately to severely active CD (CDAI 220-450) who responded to 6 weeks (wks) of VDZ induction therapy were re-randomized to maintenance therapy with placebo (PBO) or VDZ for 46 wks. CDAI stool frequency (SF), abdominal pain (AP), and general well-being (GWB) subscores were evaluated alone and in combination (PRO-2 [SF + AP] and PRO-3 [SF + AP + GWB]) at wks 0, 6, and 52, and their contributions to baseline and mean change from baseline CDAI total scores were analyzed. The sensitivity of PRO-based remission—defined by unweighted combined daily (PRO-2 [SF ≤3 + AP ≤1]) and weighted 7-day (PRO-2 ≤75 or PRO-3 ≤80) mean scores—to detect clinical remission (CDAI =150), their agreement with CDAI ≤150, and the percentage of GEMINI 2 pts who met PRO-based entry criteria (SF ≥4 and/or AP ≥2) were evaluated. Results: The SF, AP, and GWB subscores contributed 25%, 21%, and 31%, respectively, to the mean baseline CDAI score. PRO-2 and PRO-3 accounted for, respectively, 53% and 91% of the mean change from baseline CDAI score at wk 6, and 47% and 82% at wk 52. The PRO-based definition with the best agreement with CDAI remission at wk 6 was PRO-3 ≤80 and at wk 52 was PRO-2 ≤75 (Table). PRObased entry criteria of SF ≥4 and/or AP ≥2 were met by 86% of GEMINI 2 pts; 42% of patients had both SF ≥4 and AP ≥2. Conclusion: Of CDAI components, the PROs contributed most to baseline and treatment-induced changes in CDAI score. Different PRO-based definitions of remission (PRO-2 ≤75 or PRO-3 ≤80) had the best agreement with clinical remission at wks 6 and 52. Interpretations of these post hoc analyses are limited, but applying cutoffs used here to other data sets could clarify the reliability and clinical meaningfulness of PROs as outcome measures.

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