Abstract Background Post hoc analyses and small real-world case series have suggested potential clinical benefit of tofacitinib in selected patients with CD. Robust real-world data remain limited. This study evaluated the clinical effectiveness and safety of tofacitinib in patients with moderate-to-severe CD. Methods We conducted a retrospective cohort study of adults with active CD who initiated tofacitinib. Baseline demographics, disease phenotype, and prior therapeutic exposures were recorded. Clinical outcomes at Weeks 16 and 48 included clinical response, clinical remission, steroid-free remission, surgery-free and hospitalization-free survival, and endoscopic remission (when available). Clinical response was defined as a reduction in the Harvey–Bradshaw Index (HBI) by at least 3 points from baseline, while clinical remission was defined as achieving an HBI score of 4 or less. Predictors of Week-16 clinical response were assessed using univariate and multivariate analyses. Results A total of 92 patients (median age, 43 years; 43 females 46.7%) received tofacitinib for CD that was refractory to conventional therapy or biologics, or characterized by steroid dependence. Patients with perianal fistula were excluded. Prior treatment exposures included corticosteroids in 80/92 (86.9%), thiopurines in 43/92 (46.7%), and anti–TNF agents in 23/92 (25%). At Week 16, clinical response, clinical remission, and steroid-free remission were achieved in 44/92 (47.8%), 34/92 (36.9%), and 28/92 (30.4%) patients, respectively. Hospitalization-free survival was observed in 70/92 (76.1%), and surgery-free survival in 74/92 (80.4%). Endoscopic remission was achieved in 1/7 (14.2%) of patients undergoing endoscopic reassessment. Clinical response at Week 16 was numerically higher in colonic CD compared with ileal/ileocolonic CD (19/34 55.9% vs. 25/58 43.1%, p=0.23). At Week 48, clinical response, clinical remission, and steroid-free remission were observed in 42/92 (45.6%), 38/92 (41.3%), and 31/92 (33.7%), respectively, with endoscopic remission in 13/26 (50%) of those reassessed. At week 16, patients with isolated colonic disease (OR 3.14; 95% CI 1.07–9.99; p = 0.04) and prior corticosteroid exposure (OR 4.18; 95% CI 1.05–23.64; p = 0.04) were more likely to respond to tofacitinib. Adverse events were infrequent, with no major cardiovascular or thromboembolic events recorded. The most common adverse events included follicular acne in 6/92 (6.5%), nasopharyngitis in 4/92 (4.3%), and herpes zoster in 1/92 (1.1%). Conclusion Tofacitinib was associated with clinically meaningful improvements in symptoms and endoscopic activity, with an acceptable safety profile. Conflict of interest: Dr. Singh, Arshdeep: No conflict of interest Bhardwaj, Arshia: No conflict of interest Mahajan, Ramit: No conflict of interest Singh, Dharmatma: No conflict of interest Upneja, Raghav: No conflict of interest Sharma, Nishant: No conflict of interest Midha, Vandana: No conflict of interest Sood, Ajit: Ajit Sood received honorarium for speaker events from Pfizer India and Takeda India.
Singh et al. (Thu,) studied this question.