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, Désirée Van Der Heijde

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4 Scopus citations

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. Trial registration number NCT03502616

Original languageEnglish (US)
Pages (from-to)1004-1013
Number of pages10
JournalAnnals of the rheumatic diseases
Volume80
Issue number8
DOIs
StatePublished - Aug 1 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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