A132 DEVELOPMENT OF THE TUMMY-CD, A SYMPTOMS-BASED DISEASE ACTIVITY PATIENT REPORTED OUTCOME (PRO) FOR PEDIATRIC CROHN’S DISEASE

In accordance with recent regulatory guidance which mandates patient reported outcomes (PRO) [without a proxy] for use in clinical trials, elucidating the signs and symptoms that children with Crohn’s disease deem important when referencing their level of disease activity is important. We now report the first stage in developing the TUMMY-CD index, a PRO for pediatric Crohn’s disease. Concept elicitation interviews were performed with children and teens who have Crohn’s disease and their caregivers in 9 international centers (Canada, USA, Ireland, UK, Israel). Interviews were recorded, transcribed verbatim, and analysed using qualitative analytic techniques. Items were ranked based on frequency of endorsement and rated level of importance according to participants. Items endorsed in over 6% of participants will be carried forward to Phase 2 of study. Disease activity was categorized using the Pediatric Crohn’s Disease Activity Index (PCDAI). A population of 59 children aged 4–17 years (52% male, 80% inactive disease, 14% mild disease, 6% moderate to severe disease), and 56 caregivers were interviewed. Of the signs and symptoms reported, 13 were endorsed in over 6% of participants in either the child or caregiver group. The items that will be explored in the next phase of this study, in decreasing order of endorsement are: Abdominal pain (100% child, 83% caregiver), Diarrhea (53% child, 54% caregiver), Fatigue (49% child, 63% caregiver), Appetite (25% child, 36% caregiver), Nausea +/- Vomiting (24% child, 20% caregiver), Bloody Stool (20% child, 34% caregiver), Joint Pain (14% child, 27% caregiver), Weight loss (9% child, 14% caregiver), Perianal Disease (7% child, 4% caregiver), Constipation (5% child, 7% caregiver), Mood (3% child, 13% caregiver), Pallor (3% child, 11% caregiver), and Mouth Sores (3% child, 9% caregiver). No significant difference was found in the symptoms reported by children experiencing higher or lower disease activity, complicated [penetrating and/or stricturing] versus inflammatory disease behavior, or among caregivers with varying levels of education. Children under 7 years had difficulty reporting symptoms, and therefore an Observer Reported Outcome (ObsRO) will be created for this group. From this first stage of the development of the TUMMY-CD Index, 13 signs and symptoms were significantly endorsed by children with Crohn’s disease and their caregivers. These items will be explored in a second phase, where vocabulary and response options will be determined. Once validated, the TUMMY-CD Index can supplement objective outcome measures in future clinical trials. NASPGHAN Foundation; Investigator-initiated Grant from AbbVie

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