Introduction: Tofacitinib (tofa) is an oral small molecule JAK inhibitor for the treatment of ulcerative colitis (UC). We performed a retrospective cohort study to assess clinical outcomes up to 78 weeks after tofa initiation for UC in a real-world setting.
Methods: This study included adults initiating tofa therapy between 5/1/18-4/1/21 at a large, US academic medical center. Electronic health records were manually reviewed for clinical data. The primary outcome was steroid-free clinical remission (SFCR; i.e. simple clinical colitis activity index <2 or per provider global assessment and no use of oral/IV corticosteroids for >30 days) at 12, 52, and 78 (+/-4) weeks. Secondary outcomes were endoscopic response (decrease in Mayo endoscopic subscore by >1 pt) and remission (Mayo endoscopic subscore 0) at >8 weeks, biochemical response (improvement in elevated C-reactive protein [CRP] or fecal calprotectin [FC] by >25% from baseline) and remission (normalized CRP or FC) at >8 weeks, and dose de-escalation, improvement in arthralgia, colectomy, hospitalization, adverse events (AEs), and treatment discontinuation during follow-up. Continuous data were reported as medians with interquartile range (IQR) due to normality and categorical data were reported as proportions.
Results: 73 patients initiated tofa with median follow-up of 88 weeks (IQR 40.9-151.0 weeks). 60.2% were female, 54.7% had prior exposure to >2 anti-tumor necrosis factor agents, 74.0% had prior exposure to vedolizumab, and 54.7% were receiving concomitant oral/IV steroids (Table 1). Among patients with available data, 31/60 (51.7%) were in SFCR at 78 weeks, 39/47 (83.0%) achieved endoscopic response, and 21/47 (44.7%) achieved endoscopic remission (median time to endoscopy 58.1 weeks, IQR 30.7-103.6 weeks after initiation). Other outcomes are presented in Fig 1A. 31/73 (42.5%) discontinued tofa after a median of 616 days (IQR 286-1057 days) primarily due to non-response (14/31) or colectomy for refractory disease (11/31) and dysplasia (2/31). 15/73 (20.5%) experienced potential AEs during follow-up (Fig 1B), of which 1 (6.7%) required discontinuation (elevated liver enzymes). 38/73 (52.1%) underwent dose de-escalation, among which 18/32 (56.3%) with adequate follow-up were in SFCR at 52 weeks after de-escalation (Fig 1C).
Discussion: In a real-world refractory UC population, tofa was effective for the majority of patients through 78 weeks. Due to a limited sample size, larger real-world studies are needed to corroborate these findings.
