Abstract

Tofacitinib in Patients with Ulcerative Colitis: Inflammatory Bowel Disease Questionnaire Items in Phase 3 Randomized Controlled Induction Studies

Inflamm Bowel Dis. 2020 Aug 14;izaa193. doi: 10.1093/ibd/izaa193. Online ahead of print.

Marla C Dubinsky 1, Marco DiBonaventura 2, Haiyun Fan 3, Andrew G Bushmakin 4, Joseph C Cappelleri 4, Eric Maller 3, Andrew J Thorpe 3, Leonardo Salese 3, Julian Panés 5

 
     

Author information

  • 1Icahn School of Medicine at Mount Sinai, New York, New York, USA.
  • 2Pfizer Inc., New York, New York, USA.
  • 3Pfizer Inc., Collegeville, Pennsylvania, USA.
  • 4Pfizer Inc., Groton, Connecticut, USA.
  • 5Inflammatory Bowel Diseases Unit, Hospital Clínic de Barcelona, IDIBAPS, CIBERehd, Barcelona, Spain.

Abstract

Background: Tofacitinib is an oral, small molecule Janus kinase inhibitor for the treatment of ulcerative colitis (UC). We examined the effect of tofacitinib induction treatment on Inflammatory Bowel Disease Questionnaire (IBDQ) items in adults with moderate to severe UC.

Methods: Data were pooled from the randomized, 8-week, double-blind, phase 3 OCTAVE Induction 1 and 2 studies. The IBDQ was self-administered by patients at baseline, week 4, and week 8, with higher scores indicating better health-related quality of life (HRQoL). Change from baseline in IBDQ items was analyzed for 10 mg of tofacitinib twice daily (BID) vs placebo using a linear mixed-effects model, with no multiplicity adjustment performed. Effect sizes were calculated. Subgroup analyses by tumor necrosis factor inhibitor (TNFi) experience were performed.

Results: Significant improvements (nominal P < 0.05) were observed in all IBDQ items with 10 mg of tofacitinib BID vs placebo at weeks 4 and 8. For the overall population, the largest treatment differences across all items were reported for "bowel movements been loose" at weeks 4 and 8, and "problem with rectal bleeding" at week 8 (mean treatment differences all 1.1; both in bowel symptoms domain). These items also showed the largest effect sizes. Treatment benefits were generally slightly numerically higher in TNFi-experienced vs TNFi-naïve patients.

Conclusions: Tofacitinib induction therapy improved all IBDQ items vs placebo in patients with UC, reflecting improvements in HRQoL, with greatest benefits reported in bowel symptoms domain items (Funded by Pfizer Inc; OCTAVE Induction 1 and OCTAVE Induction 2; ClinicalTrials.gov, NCT01465763 and NCT01458951, respectively).

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