Early Responses in Patient-reported Outcomes Are Predictors for Mucosal Healing in Patients with Moderately to Severely Active Ulcerative Colitis Treated with Golimumab: Results from PURSUIT SC Induction

Abstract Background Optimal management of patients with ulcerative colitis (UC) requires the accurate assessment of disease activity. Endoscopic evaluation is considered the gold standard approach, but it is invasive. We aimed to determine how strong patient reported outcomes, clinical scores and symptoms correlate with endoscopy for assessment of disease activity in UC patients. Methods 171 patients were included prospectively and consecutively (age: 49 (IQR: 38-61) years, duration 12 (4-19)years, 79 females (46.2%), 57.3% extensive disease, 42.7% on biologicals) at the time of the colonoscopy. The 2 item patient reported outcome (PRO), partial MAYO, Simple Clinical Colitis Activity Index (SCCAI), Mayo endoscopic subscore (MES), Baron and Ulcerative Colitis Endoscopic Index of Severity (UCEIS) scores were calculated. C reactive Protein (CRP) and fecal calprotectin (FCAL) was available in 83 and 45.6% of patients. 17.0% had clinical flare, treatment was escalated in 14.6% of patients. Sensitivity, specificity, PPV and NPV values were calculated, ROC analysis and K-statistics were performed. Results Rectal bleeding (RBS), stool frequency (SF) subscore of 0, or total PRO2 remission (RBS 0 and SF ≤1), partial MAYO (≤2) and SCCAI (≤2.5) remission were similarly associated to mucosal healing defined by MES (0 or ≤1) or Baron (0 or ≤1) scores (Table 1). PRO2 (AUCMES0/Baron0: 0.770/0.740, AUCMES0-1/Baron0-1: 0.868/0.858), SF (AUCMES0/Baron0:0.751/0.724, AUCMES0-1/Baron0-1:0842/0.820), RBS (AUCMES0/Baron0: 0.718/0.698, AUCMES0-1/Baron0-1: 0.814/0.845) partial Mayo (AUCMES0/Baron0: 0.823/0.788, AUCMES0-1/Baron0-1: 0.927/0.902) and SCCAI (AUCMES0/Baron0: 0.767/0.752, AUCMES0-1/Baron0-1:0.888/0.867) were similarly associated with mucosal healing in a ROC analysis. There was a strict association between MES 0 and Baron 0 (k=0.917) and UCEIS <4 and MES 0-1 (k=0.813), while moderate to fair agreement between UCEIS <4 and MES 0 (K=0.471) or Baron 0 (K=0.414)/Baron 0-1 (K=0.353), and between MES 0-1 and Baron 0-1 (K= 0.350) scores. Agreement between CRP and clinical remission or endoscopic healing (MES/Baron) was poor (K~0.2), while agreement between FCAL (>100 or >250) and RBS-PRO2 remission (K>100 or >250: 0.44-0.60) or pMAYO (K>100 or >250: 0.41-0.59) or MES/Baron 0 was moderate to good (K>100:0.53-0.52 and K>250:0.57-0.53). Conclusion We found no difference across accuracy of RBS, SF, PRO2, partial Mayo and SCCAI in predicting endoscopic healing. A strong association was found with high PPV for MES/Baron ≤1 and high NPV for MES/Baron 0. FCAL, but not CRP was associated to clinical and endoscopic remission.

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A comparison of physician-rated disease severity and patient reported outcomes in mild to moderately active ulcerative colitis

Journal of Crohn s and Colitis ◽

10.1016/j.crohns.2009.11.010 ◽

2010 ◽

Vol 4 (3) ◽

pp. 275-282 ◽

Cited By ~ 17

Author(s):

Chris D. Poole ◽

Mark P. Connolly ◽

Sandy Kildegaard Nielsen ◽

Craig J. Currie ◽

Philippe Marteau

Keyword(s):

Ulcerative Colitis ◽

Disease Severity ◽

Patient Reported Outcomes ◽

Active Ulcerative Colitis ◽

Patient Reported

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Mo1220 Patient-Reported Symptom Measures Differ in Their Association With Mucosal Healing in Adults With Moderately to Severely Active Ulcerative Colitis: Results From ULTRA 1 and 2

Gastroenterology ◽

10.1016/s0016-5085(14)62135-1 ◽

2014 ◽

Vol 146 (5) ◽

pp. S-589

Author(s):

Jean-Frederic Colombel ◽

William Sandborn ◽

Walter Reinisch ◽

Anne Robinson ◽

Wenrui Wang ◽

...

Keyword(s):

Ulcerative Colitis ◽

Mucosal Healing ◽

Active Ulcerative Colitis ◽

Patient Reported ◽

Symptom Measures

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Randomized trial of tofacitinib in active ulcerative colitis: analysis of efficacy based on patient-reported outcomes

BMC Gastroenterology ◽

10.1186/s12876-015-0239-9 ◽

2015 ◽

Vol 15 (1) ◽

Cited By ~ 52

Author(s):

Julián Panés ◽

Chinyu Su ◽

Andrew G Bushmakin ◽

Joseph C Cappelleri ◽

Carla Mamolo ◽

...

Keyword(s):

Ulcerative Colitis ◽

Randomized Trial ◽

Patient Reported Outcomes ◽

Active Ulcerative Colitis ◽

Patient Reported

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P773 Patient reported outcomes, partial MAYO score and SCCAI are equally accurate in predicting mucosal healing in UC: Preliminary results from a prospective study

Journal of Crohn s and Colitis ◽

10.1093/ecco-jcc/jjz203.901 ◽

2020 ◽

Vol 14 (Supplement_1) ◽

pp. S613-S614

Author(s):

P Golovics ◽

L Gonczi ◽

J Reinglass ◽

C Verdon ◽

W Afif ◽

...

Keyword(s):

Ulcerative Colitis ◽

Disease Activity ◽

Patient Reported Outcomes ◽

Roc Analysis ◽

Activity Index ◽

Strong Association ◽

Fecal Calprotectin ◽

Mucosal Healing ◽

Patient Reported ◽

Assessment Of Disease Activity

Abstract Background Optimal management of patients with ulcerative colitis (UC) requires the accurate assessment of disease activity. Endoscopic evaluation is considered the gold standard approach, but it is invasive. We aimed to determine how strong patient reported outcomes, clinical scores and symptoms correlate with endoscopy for assessment of disease activity in UC patients. Methods One hundred and thirty-six patients were included prospectively (age: 48 (IQR: 38–61) years, duration 12 (4–19) years, 63 females, 53.7% extensive disease, 40.4% on biologicals) at the time of the colonoscopy. The 2 item patient reported outcome (PRO), partial MAYO, Simple Clinical Colitis Activity Index (SCCAI), Mayo endoscopic subscore (MES), Baron and Ulcerative Colitis Endoscopic Index of Severity (UCEIS) scores were calculated. C reactive Protein (CRP) and fecal calprotectin (FCAL) was available in 58.1 and 33.8% of patients. 20.7% had clinical flare, treatment was escalated in 17.8% of patients. Sensitivity, specificity, PPV and NPV values were calculated, ROC analysis and K-statistics were performed. Results Rectal bleeding(RBS), stool frequency(SF) subscore of 0, or total PRO2 remission(RBS0 and SF≤1), partial MAYO(≤2) and SCCAI(≤2.5) remission were similarly associated to mucosal healing defined by MES(0 or ≤1) or Baron (0 or ≤1) scores (Table 1). PRO2 remission (AUCMES0/Baron0:0.747/0.715, AUCMES0-1/Baron0-1:0.867/0.863), SF AUCMES0/Baron0:0.731/0.703, AUCMES0-1/Baron0-1:0851/0.839), RBS(AUCMES0/Baron0:0.708/0.685, AUCMES0-1/Baron0-1:0.828/0.835) partial Mayo (AUCMES0/Baron0:0.792/0.755, AUCMES0-1/Baron0-1:0.917/0.903) and SCCAI (AUCMES0/Baron0:0.738/0.724, AUCMES0-1/Baron0-1:0.908/0.880) were similarly associated with mucosal healing in a ROC analysis. There was a string association between MES and Baron (k=0.798), while moderate agreement between UCEIS and MES (K=0.451) or Baron (K=0.499) scores. Agreement between CRP and clinical remission or endoscopic healing (MES/Baron) was poor (K~0.2), while agreement between FCAL (>100 or >250) and RBS-PRO2 remission (K>250:0.56–0.61) or MES/Baron 0 was moderate to good(K>100:0.54-0.53 and K>250:0.50–0.54) Conclusion We found no difference across accuracy of RBS, SF, PRO2, partial Mayo and SCCAI in predicting endoscopic healing. A strong association was found with high PPV for MES/Baron ≤1 and high NPV for MES/Baron 0. FCAL, but not CRP was associated to clinical and endoscopic remission.

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