P477 Clinical remission by legacy vs. FDA definitions: definition justification and results from UNIFI Study
W. J. Sandborn*1, R. Strauss2, H. Zhang2, J. Johanns2, P. Szapary2, C. Marano2, S. Danese3
1University of California San Diego, La Jolla, USA, 2Janssen Research and Development, LLC, Spring House, USA, 3Humanitas Research Hospital, Milan, Italy
Ustekinumab (UST), an interleukin-12/23 blocker, was evaluated as induction and maintenance for moderate–severe ulcerative colitis (UC). Clinical remission was analysed using a US-specific definition (FDA) excluding the PGA (Physician’s Global Assessment) and the legacy definition which includes the PGA to accommodate regional regulatory preference.
Patients (pts) were randomised to receive a UST intravenous (IV) induction dose (either 130 mg [
The FDA definition demonstrated high concordance, specificity and sensitivity with the legacy definition with a similar treatment effect, and defined patients who had clinically meaningful benefit. In the UNIFI study, Week 8 clinical remission rates among patients receiving IV UST at either 130 mg or ~6 mg/kg were significantly higher than PBO patients by both legacy (15.6%, 15.5%, and 5.3%, respectively
Using either legacy or FDA remission definition, IV UST induced remission and SC UST maintained remission in UST induction responders with moderately-to-severely active UC. Importantly, the FDA definition with absolute stool number ≤3 is clinically meaningful, easily understood by physicians and patients, and is not based on patients’ distant recall of normal stool pattern.