Study Supports Safety, Efficacy of Tofacitinib in the Treatment of Ankylosing Spondylitis

Tofacitinib is safe and effective for the treatment of adult patients with ankylosing spondylitis (AS), according to study results presented at the American College of Rheumatology (ACR) Convergence 2020, held virtually from November 5 to 9, 2020.

The current phase 3, randomized, double-blind, placebo-controlled study (ClinicalTrials.gov Identifier: NCT03502616) included adult patients with active AS who had an inadequate or intolerant response to prior treatment with 2 or more nonsteroidal anti-inflammatory drugs (NSAIDs). Patients were randomly assigned 1:1 to receive tofacitinib 5 mg twice daily or placebo for 16 weeks. After the 16-week double-blind period, all patients received open-label tofacitinib until week 48. The primary outcome measure was treatment response at week 16, according to the Assessment of SpondyloArthritis International Society (ASAS) criteria; specifically, the percentage of patients achieving a 20% improvement in ASAS response criteria (ASAS20) was recorded. Researchers also determined the percentage of patients achieving ASAS40 response. Adverse events were monitored throughout the trial.

The study cohort included 269 adults with AS, among whom 133 received tofacitinib and 136 received placebo. At week 16, 56.4% of the tofacitinib group vs 29.4% of the placebo group achieved ASAS20 (P <.0001). The percentage of ASAS40 responders at week 16 was also significantly greater in the tofacitinib vs placebo group (40.6% vs 12.5%; P <.001). Improvements in specific ASAS components were significantly greater with tofacitinib vs placebo, including total back pain, morning stiffness, patient’s global assessment of disease activity, and Bath Ankylosing Spondylitis Functional Index score.

During the first 16 weeks of the study, adverse events were reported by 72 (54.1%) and 70 (51.5%) patients in the tofacitinib and placebo groups, respectively. The most common adverse events in the tofacitinib group were upper respiratory tract infection (10.5%), nasopharyngitis (6.8%), diarrhea (4.5%), increased alanine aminotransferase (3.0%), arthralgia (0.8%), and headache (1.5%). Serious adverse events were reported in 2 patients who received tofacitinib (1 hepatic event and 1 serious infection); however, there were no deaths. Safety trends were similar until week 48.

Researchers concluded, “[Patients] with active AS had a rapid clinical response to tofacitinib. Efficacy was significantly greater with tofacitinib vs [placebo] for primary and secondary end points. [Adverse events] were more frequent with tofacitinib vs [placebo]; there were no new safety risks.”

Disclosure: This study was sponsored by Pfizer Inc. Please see the original reference for a full list of authors’ disclosures.

Reference

Deodhar A, Sliwinska-Stanczyk P, Xu H, et al. Tofacitinib for the treatment of adult patients with ankylosing spondylitis: primary analysis of a phase 3, randomized, double-blind, placebo-controlled study. Presented at: ACR Convergence 2020; November 5-9, 2020. Abstract L11.