OBJECTIVES: Given that clinical symptoms in ulcerative colitis (UC; abdominal pain, diarrhea, increased stool frequency, rectal bleeding) are important to patients, they are commonly evaluated in clinical trials. Nevertheless, there is limited evidence on the patient-reported outcome measures (PROMs) in real-world setting. We aimed to analyze the use of PROMs for advanced therapies in UC.

METHODS: A systematic literature review was conducted in Embase and MEDLINE to identify real-world studies reporting PROMs on advanced therapies (including small molecules and biologics) in moderate-to-severe UC patients. Studies published in English as full papers (Jan-2012–April-2023) or conference abstracts (Jan-2019–April-2023) were included.

RESULTS: Of the 4,896 articles retrieved, 246 studies had information on effectiveness/safety outcomes. Of note, only 22 studies reported PROMs and were included for analysis. Most studies were from Europe (n=13 [59%]) or America (n=6 [27%]) and of prospective design (n=17; [77%]). Eighteen studies (82%) were single-treatment (7 vedolizumab, 5 golimumab, 4 tofacitinib, 1 ustekinumab, and 1 adalimumab) and four comparative studies (one compared vedolizumab, ustekinumab and anti-TNFs; three compared between anti-TNFs). Fourteen studies (64%) reported at least one disease-specific PROMs, comprising Inflammatory Bowel Disease Questionnaire (IBDQ-32; n=3 [14%]), Short IBDQ (SIBDQ; n=5 [23%]), IBDQ – short form (IBDQ-9; n=1 [5%]), IBD – Disability Index (IBD-DI; n=1 [5%]), Rectal Blood Subscore (n=5 [23%]), Stool Frequency Subscore (n=4 [18%]), and Stool Urgency Subscore (n=1 [5%]). The commonly used generic PROMs were EQ-5D (n=5 [23%]), Short Health Scale (n=4 [18%]), and EQ-Visual Analog Scale (EQ-VAS, n=3 [14%]).

CONCLUSIONS: Only a few real-world studies of advanced therapies reported the use of PROMs in moderate-to-severe UC. The limited use could be ascertained to the limited guidelines on PROMs assessment in real-world setting. However, in routine clinical practice, combining these PROMs with physician’s assessment to integrate disease activity profile of patients might be helpful for precise decision-making in UC care.