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. 2015 Nov;42(9):1082-92.
doi: 10.1111/apt.13387. Epub 2015 Sep 3.

Randomised clinical study: discrepancies between patient-reported outcomes and endoscopic appearance in moderate to severe ulcerative colitis

B Jharap  1 , W J Sandborn  2 , W Reinisch  3   4 , G D'Haens  5 , A M Robinson  6 , W Wang  6 , B Huang  6 , A Lazar  7 , R B Thakkar  6 , J-F Colombel  1
Affiliations

Randomised clinical study: discrepancies between patient-reported outcomes and endoscopic appearance in moderate to severe ulcerative colitis

B Jharap et al. Aliment Pharmacol Ther. 2015 Nov.

Abstract

Background: Associations between patient-reported outcomes and mucosal healing have not been established in ulcerative colitis (UC).

Aim: To evaluate relationships of rectal bleeding and stool frequency with mucosal healing and quality of life (QoL) in patients with UC in two Phase 3 studies (ULTRA 1 and 2).

Methods: Associations of patient-reported rectal bleeding and stool frequency subscores with mucosal healing (Mayo endoscopy subscore = 0 or 0/1) and QoL [inflammatory bowel disease questionnaire (IBDQ)] were assessed in adalimumab-randomised patients (160/80 mg at Weeks 0/2 followed by 40 mg biweekly or weekly) at Weeks 8 (n = 433) and 52 (n = 299), and in patients with mucosal healing [endoscopy subscore = 0 (n = 17); 0/1 (n = 52)] at Weeks 8 and 52.

Results: At Week 8, the positive predictive values (PPVs) of rectal bleeding subscore = 0, stool frequency subscore = 0 or both scores = 0 for endoscopy subscore = 0/1 were 69%, 84% and 90% respectively; all proportions increased at Week 52. Equivalent PPVs for these subscores in patients with endoscopy subscore = 0 were 26%, 37% and 46% respectively. Among patients with endoscopy subscore = 0 at Week 8, 87% reported no rectal bleeding, while only 29% reported normal stool frequency; these proportions had increased to 94% and 41% respectively, at Week 52. Among patients with mucosal healing, IBDQ scores trended highest for patients with both rectal bleeding and stool frequency subscores = 0.

Conclusions: Absence of rectal bleeding and normal stool frequency are often predictive of mucosal healing and QoL, but complete normalisation of stool frequency is encountered rarely in patients with mucosal healing.

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Figures

Figure 1
Figure 1
Calculation of positive predictive value and negative predictive value of rectal bleeding and stool frequency for mucosal healing at Weeks 8 and 52 in patients randomised to adalimumab 160/80 mg. The positive predictive value and negative predictive value of rectal bleeding and stool frequency for mucosal healing were calculated separately for rectal bleeding subscore = 0, stool frequency subscore = 0, the combination of both rectal bleeding subscore = 0 and stool frequency subscore = 0 and either rectal bleeding or stool frequency subscore = 0. Adapted from Ref. 15. MH, mucosal healing; NPV, negative predictive value; PPV, positive predictive value; RBS, rectal bleeding subscore; SFS, stool frequency subscore.
Figure 2
Figure 2
Proportion of patients randomised to adalimumab 160/80 mg with (a) rectal bleeding and (b) stool frequency subscores of 0, 1, 2 or 3 per each endoscopy subscore value at Week 8. ES, endoscopy subscore; RBS, rectal bleeding subscore; SFS, stool frequency subscore.
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References

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