Tofacitinib Salvage Therapy for Children Hospitalized for Corticosteroid- and Biologic-Refractory Ulcerative Colitis

Constant, Brad D.; Baldassano, Robert N.; Kirsch, Jacqueline; Mitchel, Elana B.; Stein, Ronen; Albenberg, Lindsey

doi:10.1097/mpg.0000000000003616

Cited by 14 publications

(10 citation statements)

References 41 publications

(57 reference statements)

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“…So far, only few adult and pediatric patients have been reported to receive TFB treatment with rescue therapy indication. 18-20 …”

Section: Discussionmentioning

confidence: 99%

Real-Life Efficacy of Tofacitinib in Various Situations in Ulcerative Colitis: A Retrospective Worldwide Multicenter Collaborative Study

Resál,

Bacsur,

Keresztes

et al. 2023

Inflammatory Bowel Diseases

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Background and Aims Tofacitinib (TFB) appears to be effective in the treatment of ulcerative colitis (UC); however, available real-world studies are limited by cohort size. TFB could be an option in the treatment of acute severe ulcerative colitis (ASUC). We aimed to investigate efficacy and safety of TFB in moderate-to-severe colitis and ASUC. Methods This retrospective, international cohort study enrolling UC patients with ≥6-week follow-up period was conducted from February 1 to July 31, 2022. Indications were categorized as ASUC and chronic activity (CA). Baseline demographic and clinical data were obtained. Steroid-free remission (SFR), colectomy, and safety data were analyzed. Results A total of 391 UC patients (median age 38 [interquartile range, 28-47] years; follow-up period 26 [interquartile range, 14-52] weeks) were included. A total of 27.1% received TFB in ASUC. SFR rates were 23.7% (ASUC: 26.0%, CA: 22.8%) at week 12 and 41.1% (ASUC: 34.2%, CA: 43.5%) at week 52. The baseline partial Mayo score (odds ratio [OR], 0.850; P = .006) was negatively associated with week 12 SFR, while biologic-naïve patients (OR, 2.078; P = .04) more likely achieved week 52 SFR. The colectomy rate at week 52 was higher in ASUC group (17.6% vs 5.7%; P < .001) and decreased with age (OR, 0.94; P = .013). A total of 67 adverse events were reported, and 17.9% resulted in cessation of TFB. One case of thromboembolic event was reported. Conclusions TFB is effective in both studied indications. TFB treatment resulted in high rates of SFR in the short and long terms. Higher baseline disease activity and previous biological therapies decreased efficacy. No new adverse event signals were found.

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“…So far, only few adult and pediatric patients have been reported to receive TFB treatment with rescue therapy indication. 18-20 …”

Section: Discussionmentioning

confidence: 99%

Real-Life Efficacy of Tofacitinib in Various Situations in Ulcerative Colitis: A Retrospective Worldwide Multicenter Collaborative Study

Resál,

Bacsur,

Keresztes

et al. 2023

Inflammatory Bowel Diseases

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“…To our knowledge, there is only one published series of 11 pediatric patients treated with tofacitinib as salvage therapy for refractory UC, of which eight had ASC. 31 They suggest that tofacitinib may be used as a bridge therapy allowing for nutritional rehabilitation before colectomy. We believe that it may have a role in two scenarios.…”

Section: Discussionmentioning

confidence: 99%

The Role of Tofacitinib in the Treatment of Acute Severe Colitis in Children

Costaguta,

Girard,

Groleau

et al. 2023

Journal of the Canadian Association of Gastroenterology

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Objectives Acute severe colitis (ASC) occurs in up to 15 percent of children with ulcerative colitis, with a high index of morbidity and mortality. Treatment includes high-dose steroids, infliximab, and salvage therapies. Unfortunately, up to 20 percent of patients may need an urgent colectomy due to treatment failure. We report our experience using tofacitinib for the treatment of six patients. Methods A retrospective review of our medical electronic records was conducted. We included every patient with ASC and treatment failure, in whom tofacitinib was used as a salvage therapy. Response, complications, and disease course were noted. Results Six patients were included with Pediatric Ulcerative Colitis Activity Index (PUCAI) scores ranging from 65 to 85 on admission, and 35 to 85 before tofacitinib was started (P 0.07). Median response time was 72 h. A median decrease of 40 points in PUCAI was noted (P 0.00001). Mean length of stay was 18 days with discharge 9 days after tofacitinib introduction. Haemoglobin, albumin, fecal calprotectin, and CRP improved after tofacitinib (P 0.02, P 0.02, P 0.025, and P 0.01, respectively). The mean follow-up was 8.5 months, four patients achieved complete remission and only one had a recrudescence of symptoms (P 0.01). One patient had a systemic Epstein-Barr virus infection prior to tofacitinib therapy, which resolved with rituximab treatment. No other complications were noted. Conclusions Tofacitinib response is rapid and impressive in children suffering from ASC, and the safety profile appears comparable to or better than other available treatments. In the future, tofacitinib should be integrated into pediatric protocols.

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“…The treatment courses and colectomy-free survival among 11 pediatric patients treated with tofacitinib while hospitalized for refractory ulcerative colitis from 2018 to 2021 was studied (28). All patients demonstrated nonresponse to both intravenous corticosteroids and anti-tumor necrosis factor (TNF) therapy prior to tofacitinib initiation.…”

Section: Inflammatory Bowel Diseasementioning

confidence: 99%