Background: Under the qualification program of the FDA and EMA, we aimed to develop a Patient Reported Outcome measure of signs and symptoms for pediatric ulcerative colitis (UC) (i.e. the TUMMY-UC index) for using in trials and clinical practice. We previously selected items to the index via concept elicitation qualitative interviews.
Methods: Trained personnel performed Cognitive debriefing Interviews in five countries with UC children 2-18 years and, in order to develop an Observer Reported Outcome (obsRO) for those ≤8 years, also their caregivers. We compiled contending scales for each item and explored the vocabulary children prefer. Clinical data were recorded. Weights were assigned to each item based on ranking of importance.
Results: Interviews were performed with 34 children (age 10.6± 4 years; 28% with moderate-severe disease; 83% with extensive disease) and 13 caregivers (age 37±5 years, 23% males). . 88% of children understood the meaning of “last 24-hours” as a recall period. Only three (≤12YO) children had difficulties recalling the last 24-hours. The exact response options were amended based on the obtained feedback; Rank order of the items was: rectal bleeding-amount (4.5), rectal bleeding-frequency (4.3), stool frequency (4.2), abdominal pain (4), urgency (3.8), stool consistency (3.7), nocturnal stools (3.5) and weakness (3.2).
Conclusions: In this phase 2B, the exact wording, response options and weighting of the eight TUMMY-UC items were finalized. An obsRO version of the TUMMY-UC has been developed for the younger children. The TUMMY-UC will be now validated and evaluated for cutoff scores in a phase 3 study.
