Tofacitinib for the treatment of ankylosing spondylitis: A phase III, randomised, double-blind, placebo-controlled study

Atul Deodhar, Paula Sliwinska-Stanczyk, Huji Xu, Xenofon Baraliakos, Lianne S. Gensler, Dona Fleishaker, Lisy Wang, Joseph Wu, Sujatha Menon, Cunshan Wang, Oluwaseyi Dina, Lara Fallon, Keith S. Kanik, Désireé Van Der Heijde

Research output: Contribution to journalArticlepeer-review

Abstract

Objective: To assess the efficacy/safety of tofacitinib in adult patients with active ankylosing spondylitis (AS). Methods: This phase III, randomised, double-blind, placebo-controlled study enrolled patients aged ≥18 years diagnosed with active AS, meeting the modified New York criteria, with centrally read radiographs, and an inadequate response or intolerance to ≥2 non-steroidal anti-inflammatory drugs. Patients were randomised 1:1 to receive tofacitinib 5 mg two times per day or placebo for 16 weeks. After week 16, all patients received open-label tofacitinib until week 48. The primary and key secondary endpoints were Assessment of SpondyloArthritis international Society ≥20% improvement (ASAS20) and ≥40% improvement (ASAS40) responses, respectively, at week 16. Safety was assessed throughout. Results: 269 patients were randomised and treated: Tofacitinib, n=133; placebo, n=136. At week 16, the ASAS20 response rate was significantly (p<0.0001) greater with tofacitinib (56.4%, 75 of 133) versus placebo (29.4%, 40 of 136), and the ASAS40 response rate was significantly (p<0.0001) greater with tofacitinib (40.6%, 54 of 133) versus placebo (12.5%, 17 of 136). Up to week 16, with tofacitinib and placebo, respectively, 73 of 133 (54.9%) and 70 of 136 (51.5%) patients had adverse events; 2 of 133 (1.5%) and 1 of 136 (0.7%) had serious adverse events. Up to week 48, with tofacitinib, 3 of 133 (2.3%) patients had adjudicated hepatic events, 3 of 133 (2.3%) had non-serious herpes zoster, and 1 of 133 (0.8%) had a serious infection; with placebo→tofacitinib, 2 (1.5%) patients had non-serious herpes zoster. There were no deaths, malignancies, major adverse cardiovascular events, thromboembolic events or opportunistic infections. Conclusions: In adults with active AS, tofacitinib demonstrated significantly greater efficacy versus placebo. No new potential safety risks were identified.

Original languageEnglish (US)
Article number2020219601
JournalAnnals of the rheumatic diseases
DOIs
StateAccepted/In press - 2021

Keywords

  • ankylosing
  • antirheumatic agents
  • spondylitis
  • therapeutics

ASJC Scopus subject areas

  • Rheumatology
  • Immunology and Allergy
  • Immunology
  • Biochemistry, Genetics and Molecular Biology(all)

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