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P174. Is there a difference between self-assessed patient-reported outcome and physician evaluation in ulcerative colitis? Results from a study with interferon-β-1a

P. Mannon1, W. Reinisch2, P. Miner3, Y. Xi4, A. McAllister4, F. Cataldi4

1UAB, Birmingham, AL, United States; 2Vienna University, Vienna, Austria; 3OFDR, Oklahoma-City, OK, United States; 4Biogen, Cambridge, MA, United States

Aim: Advances in immunology and pharmaceutical science have resulted in a large number of new potential treatments of ulcerative colitis (UC). Clinical development of these drugs requires objective outcome measures to evaluate efficacy combining findings from endoscopy and clinical characteristics of the patients. In a placebo-controlled study of interferon-β-1a (IFN) in moderately to severely active UC, we evaluated the efficacy outcome between the two scoring systems used in the trial: the Mayo score and the Simple Clinical Colitis Activity Index (SCCAI). We then conducted a post-hoc analysis comparing the differences in improvement between the Physician Global Assessment (PGA) of the Mayo score and the General Well Being (GWB) of the SCCAI in order to assess their correlation.

Methods: 123 adult patients were randomized to receive IFN 30 μg IM (62) or placebo (P) (61) twice a week, for 12 weeks. The primary endpoint was clinical response at Week 8, defined as a decrease from baseline in the total Mayo score of at least 3 points. The secondary endpoint was the percentage of subjects with a decrease in the SCCAI score of ≥3 points at Week 8. The Mayo score collects stool frequency (SF); rectal bleeding (RB); PGA; endoscopy. SCCAI: SF day and night; urgency; RB; GWB; extra intestinal manifestations. The PGA is a 4-point-scale: 0 = normal; 1 = mild disease; 2 = moderate disease; 3 = severe disease. GWB is a patient-self-reported 5-point scale of symptoms collected over the prior 24-hour: 0 = very-well; 1 = slightly-below-par; 2 = Poor; 3 = very-poor; 4 = terrible.

Results: At week 8, 53% of patients on IFN vs. 44% on P (p = 0.35) were in clinical response based on the total Mayo score; 64% on IFN vs. 46% on P (p = 0.05) based on the SCCAI. 54 patients (44%) were considered to have improved from baseline (defined as at least a 1-point drop) by both PGA and GWB and 34 (28%) were considered not to have improved by both measures. 25 patients (20%) were considered to have improved by PGA but not by GWB, 9 (7%) to have improved by GWB but not PGA. Lastly, the improvement from baseline between treatments was compared for each of these measures at week 8 and demonstrated that more patients on IFN improved (Figure).

Conclusions: In this study we had the opportunity to compare the response measured by two different scores and found a major discordance in the efficacy results between the Mayo score and SCCAI. There was also a certain degree of disagreement between PGA of the Mayo score and the GWB of the SCCAI. The difference in perception of symptoms between patients and physicians should be considered in clinical scoring of patients within trials for UC.

Figure: Improvement from baseline at Week 8 in PGA-Mayo vs. GBW-SCCAI. Improvement is a reduction from baseline of 1 point or more.