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. 2025 Mar 13;19(4):jjaf040. doi: 10.1093/ecco-jcc/jjaf040

Table 3.

Studies of biologic and small molecule therapies using novel patient-reported outcomes in UC.

Study Treatment Key study details and findings
Rectal bleeding plus stool frequency (PRO2)
Post hoc analysis of GEMINI 1, GEMINI 2, and GEMINI 389 Vedolizumab

PRO2 clinical remission = RB of 0 and SF ≤ 1A significantly higher proportion of patients achieved PRO2 clinical remission in the vedolizumab group (n = 225) than the placebo group (n = 149) at weeks 2, 4, and 6 overall and in anti-TNFα-naive patient subgroups vedolizumab-treated (n = 130) and placebo-treated (n = 76). No treatment differences among anti-TNFα-experienced patients.Vedolizumab vs placebo, overall

  • Week 2: 19.1% vs 10.1%, difference adjusted change 9.0% (95% CI: 2.0-16.1)

  • Week 4: 28.0% vs 14.8%, difference adjusted change 13.2% (95% CI: 5.1-21.4)

  • Week 6: 33.8% vs 16.8% difference adjusted change 17.0% (95% CI: 8.4-25.6)
Post hoc analysis of UNIFI90 Ustekinumab PRO2 clinical remission = RB of 0 and SF ≤ 1At week 2, ustekinumab-treated patients in the 130 mg IV (n = 320) and ~6 mg/kg IV (n = 322) dose groups achieved a significantly higher rate of PRO2 clinical remission (20.0%; P = .015) and (20.2%; P = .012) compared with (12.9%) for patients in the placebo group (n = 319)The percentage of patients achieving PRO2 clinical remission increased from baseline through week 16 for both ustekinumab groups
Post hoc analysis SELECTION trial91 Filgotinib PRO2 clinical remission = RB of 0 and SF ≤ 1PRO2 clinical remission was significantly higher in patients treated filgotinib 200 mg (n = 262) vs placebo (n = 142) by day 9 in biologic-naive patients (18.8% vs 9.5%; P = .0144) and by day 7 in biologic-experienced patients (10.7% vs 4.2%; P = .0155)
Post hoc analysis of OCTAVE clinical program92 Tofacitanib PRO2 clinical remission = RB of 0 and SF ≤ 1After 52-week maintenance tofacitinib 5 mg twice daily treatment in the OCTAVE Sustain study, 172 patients in remission continued treatment in the 4-year OCTAVE Open study; 84/172 (48.0%) were in PRO2 clinical remission by month 48
Prospective cohort study93 Vedolizumab and tofacitinib PRO2 clinical remission = RB of 0 and SF ≤ 1 as a secondary endpointNo difference between vedolizumab-treated (n = 72) and tofacitinib-treated (n = 33) patients achieving PRO2 remission at month 6
Bowel urgency
Post hoc analysis of induction study U-ACHIEVE94 Upadacitinib

Bowel urgency was recorded daily by the patient via electronic diary reporting on the previous 24 hoursAt week 8, a higher proportion of patients who received upadacitinib 15-45 mg vs placebo-treated patients reported no bowel urgency

  • Greatest improvement for patients receiving upadacitinib 45 mg QD (n = 56); 46.4% reporting no bowel urgency at week 8 compared with 8.7% for placebo (n = 46), a 37.7%; difference (95% CI, 18.1-54.0); P ≤ 0.001
Post hoc analysis of U-ACHIEVE and U-ACCOMPLISH95 Upadacitinib

Bowel urgency was recorded daily by the patient via electronic diary reporting on the previous 24 hoursA larger proportion of upadacitinib-treated patients (n = 660) reported no bowel urgency compared with the placebo group (n = 328) at weeks 2-8 and at week 52Percent of patients reporting no bowel urgency induction treatment, upadacitinib vs placebo

  • Baseline: ~8% both groups

  • Week 2: 35.8% vs 13.7%; P < .001

  • Week 4: 44.3% vs 16.5%; P < .001

  • Week 6: 46.9% vs 19.0%; P < .001

  • Week 8: 51.1% vs 23.8%; P < .001

Maintenance treatment upadacitinib 15 mg and 30 mg vs placebo

  • Week 8: 64.9% and 64.3% vs 49.7%; P < .01

  • Week 52: 56.1% and 63.6% vs 17.4%; P < .001
LUCENT I and LUCENT II96 Mirikizumab Bowel urgency measured on the UNRS from daily patient-recorded information on the severity of bowel urgencyAt week 12, reduction from baseline UNRS (least squares mean ± SE) was −2.59 ± 0.08 in mirikizumab-treated patients (n = 868) vs −1.63 ± 0.1 for the placebo group (n = 294); P < .001At week 52, mirikizumab induction responders re-randomized to mirikizumab (n = 365) or placebo (n = 179) reported a significantly greater mean UNRS change from induction baseline in mirikizumab-treated patients −3.80 ± 0.14 than the placebo group −2.74 ± 0.20; P < .001
Subset of patients with proctitis in ELEVATE UC 52 and ELEVATE UC 1297 Etrasimod Bowel urgency measured on the UNRS in patients with isolated proctitisAt week 12, reduction from baseline UNRS (least squares mean) was −2.76 in etrasimod-treated patients (n = 35) vs −0.16 in the placebo group (n = 16), difference −2.60 (95% CI: −4.47-0.73); P < .007. No treatment-related differences at week 52

Abbreviations: IV, intravenous; QD, once daily; RB, rectal bleeding; SF, stool frequency; UNRS, Urgency Numerical Rating Scale.