Real-world Effectiveness of Tofacitinib for Moderate to Severe Ulcerative Colitis: A Multicentre UK Experience

TY - JOUR

T1 - Real-world Effectiveness of Tofacitinib for Moderate to Severe Ulcerative Colitis

T2 - A Multicentre UK Experience

AU - Honap, Sailish

AU - Chee, Desmond

AU - Chapman, Thomas P.

AU - Patel, Mehul

AU - Kent, Alexandra J.

AU - Ray, Shuvra

AU - Sharma, Esha

AU - Kennedy, James

AU - Cripps, Sarah

AU - Walsh, Alissa

AU - Goodhand, James R.

AU - Ahmad, Tariq

AU - Satsangi, Jack

AU - Irving, Peter M.

AU - Kennedy, Nicholas A.

AU - Begum, Yesmina

AU - O'Neill, Rhona

AU - Mawdsley, Joel

AU - Samaan, Mark

AU - Anderson, Simon

AU - Sanderson, Jeremy

AU - Gordon, Claire

AU - Prasad, Shyam

AU - Lin, Simeng

AU - Chanchlani, Neil

AU - Elworthy, Claire

AU - Cairnes, Vida

AU - Travis, Simon

AU - Brain, Oliver

AU - Palmer, Rebecca

AU - Ambrose, Timothy

AU - Dubois, Patrick

AU - Hayee, Bu' Hussain

AU - Chung-Faye, Guy

AU - Medcalf, Lucy

PY - 2020/10/1

Y1 - 2020/10/1

N2 - Background: Tofacitinib is a partially selective Janus kinase inhibitor approved for the treatment of refractory moderate to severe ulcerative colitis [UC]. We sought to define the effectiveness and adverse effects of tofacitinib in a real-world cohort. Methods: We conducted a retrospective observational cohort study of 134 patients with UC [64% male; median age 37 years [range 16-81]; 83% of patients had previously received at least one biologic] treated with tofacitinib from October 2018 to October 2019 in four UK centres. Disease activity was assessed using the Simple Clinical Colitis Activity Index [SCCAI] or partial Mayo score [PMS], depending on study site. Response and remission were defined as a reduction in SCCAI or PMS of ≥3and SCCAI ≤2 or a PMS ≤1, respectively. Results: Overall, 74% (88/119; 95 confidence interval [CI] 65-81%] patients responded to tofacitinib at Week 8 and steroid-free remission was observed in 44% [47/108; 95% CI 3453%] patients at Week 26. Primary non-response was independently associated with younger age [p = 0.014] and higher C-reactive protein [CRP] levels at baseline [p = 0.004]. Only 23% [3/13] of patients who continued tofacitinib in the setting of primary non-response were in steroid-free remission at Week 26. Prior biologic exposure did not influence response or remission rates. Dose escalation, however, recaptured response in approximately half of patients who had lost response. Dyslipidaemia was observed in 20% [27/134; 95% CI 1428%] of patients, but adverse events necessitating drug withdrawal were uncommon and no venous thromboembolic events occurred. Conclusions: In this multicentre real-world cohort, tofacitinib was well tolerated and clinically effective in a treatment-refractory UC population.

AB - Background: Tofacitinib is a partially selective Janus kinase inhibitor approved for the treatment of refractory moderate to severe ulcerative colitis [UC]. We sought to define the effectiveness and adverse effects of tofacitinib in a real-world cohort. Methods: We conducted a retrospective observational cohort study of 134 patients with UC [64% male; median age 37 years [range 16-81]; 83% of patients had previously received at least one biologic] treated with tofacitinib from October 2018 to October 2019 in four UK centres. Disease activity was assessed using the Simple Clinical Colitis Activity Index [SCCAI] or partial Mayo score [PMS], depending on study site. Response and remission were defined as a reduction in SCCAI or PMS of ≥3and SCCAI ≤2 or a PMS ≤1, respectively. Results: Overall, 74% (88/119; 95 confidence interval [CI] 65-81%] patients responded to tofacitinib at Week 8 and steroid-free remission was observed in 44% [47/108; 95% CI 3453%] patients at Week 26. Primary non-response was independently associated with younger age [p = 0.014] and higher C-reactive protein [CRP] levels at baseline [p = 0.004]. Only 23% [3/13] of patients who continued tofacitinib in the setting of primary non-response were in steroid-free remission at Week 26. Prior biologic exposure did not influence response or remission rates. Dose escalation, however, recaptured response in approximately half of patients who had lost response. Dyslipidaemia was observed in 20% [27/134; 95% CI 1428%] of patients, but adverse events necessitating drug withdrawal were uncommon and no venous thromboembolic events occurred. Conclusions: In this multicentre real-world cohort, tofacitinib was well tolerated and clinically effective in a treatment-refractory UC population.

KW - JAK inhibitors

KW - tofacitinib

KW - Ulcerative colitis

UR - http://www.scopus.com/inward/record.url?scp=85090098668&partnerID=8YFLogxK

U2 - 10.1093/ecco-jcc/jjaa075

DO - 10.1093/ecco-jcc/jjaa075

M3 - Article

C2 - 32280965

AN - SCOPUS:85090098668

SN - 1873-9946

VL - 14

SP - 1385

EP - 1393

JO - Journal of Crohn's and Colitis

JF - Journal of Crohn's and Colitis

IS - 10

ER -