Sjogren's syndrome: abatacept flunks phase 3 ASAP-III trial

  • The Lancet Rheumatology

  • curated by Miriam Davis, PhD
  • Clinical Essentials
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Takeaway

  • In a highly anticipated study, abatacept was not superior in efficacy to placebo in the phase 3 randomized controlled trial (RCT) ASAP-III, despite prior small, open-label studies finding superiority.

Why this matters

  • Study authors recommend against using abatacept as a standard of care, but point to the need for more directed research in patients with specific symptom clusters.
  • The method of evaluating efficacy, improvement in the European League Against Rheumatism Sjögren's Syndrome Disease Activity Index (ESSDAI), may not be conducive to detecting symptom improvement.

Study design

  • A single-center, double-blind, placebo-controlled, phase 3 RCT (N=80) of weekly at-home abatacept or placebo injections (125 mg) for 24 weeks.
  • Primary outcome was improvement in ESSDAI.
  • Funding: Bristol-Myers Squibb.

Key results

  • No difference between abatacept and placebo in ESSDAI (adjusted mean difference, −1.3; 95% CI, −4.1 to 1.6).
  • No deaths or treatment-related serious adverse events (AEs) were recorded.
  • 103 AEs occurred, involving 95% of abatacept recipients (including 46 infections) vs 87 AEs involving 95% of placebo recipients.

Limitations

  • Single-center study.
  • Small numbers.