Goal: Under the qualification program of the FDA and EMA, we aimed to develop a Patient Reported Outcome (PRO) measure of signs and symptoms for pediatric ulcerative colitis (UC) (i.e. the TUMMY index).
Background: The TUMMY will be used as an outcome measure in pediatric clinical trials - composite with endoscopic assessment i.
Methods: We performed concept elicitation qualitative interviews with 47 children with UC (age 12.2 ± 3.6, range 4-18 years; 47% males; 23% with moderate-severe disease) and 30 caregivers, in Israel, England, Ireland, Canada and the USA, thus ensuring cultural diversity. Interviews were centered at exploring signs and symptoms reflecting the colitis and which are important to patients. Items were rank ordered by the interviewees according to the frequency of endorsement and importance, graded on a 1-5 scale.
Results: There was a general agreement between the scoring of children and their caregivers. The following items were identified in decreasing order of weights (importance X frequency): abdominal pain (3.9), rectal bleeding (3.6), stool frequency (3.0), stool consistency (3.0), general well-being (2.9), urgency (1.9), and nocturnal stools (1.6). Children 13-18 years comprehended adult vocabulary, 8-12 years comprehended simple vocabulary and younger children had poor understanding and thus their disease may be more accurately scored by a caregiver-reported questionnaire.
Conclusions: In this first stage of the TUMMY development, items were generated and ranked by input purely from patients. These items are now being explored for optimal vocabulary and response options.
