Sjögren's disease & overlap diseases
Development and testing of an alternative responder definition for EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI)
Authors' conclusion
Completing a trial with an improvement of ≥1.5 points compared with baseline and an ESSPRI score of ≤3 points is a relevant RD for moderate-to-severe systemic Sjögren's and reduces placebo rates.
Study context
- Population
- Individuals with moderate-to-severe systemic primary Sjögren's; predominantly female (>90%), white (90%), mean age 50 yearsexperts N=3
- Participants
- 190 vs 126
- Comparison
- Revised ESSPRI responder definition vs original
- Phase IIb trial datan = 190
- Pooled phase II trialsn = 126
- Outcome
- Alternative ESSPRI responder definition thresholds (MCII and low/minimal symptom severity)
Methodological detailsanchor-based and distribution-based methods, derivation in phase IIb trial data, confirmation in pooled data from three early phase II trials, expert triangulation
- Anchor-based and distribution-based methods
- Derivation in phase IIb trial data
- Confirmation in pooled data from three early phase II trials
- Expert triangulation
Performance
As reported in the abstract
ESSPRI responder definition thresholds
ROC estimates supported an Minimal Clinically Important Improvement (MCII) threshold in the phase II data
1.5-1.6
Pooled data: MCII
1.5-2.1
Pooled data: low/minimal symptom severity
2.7-3.7
Further findings
Placebo responder rates, revised vs original responder definition (RD) (unblinded analyses)
- Revised RD reduced placebo rates
Original publication
Development and testing of an alternative responder definition for EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI). RMD Open. Published 17 March 2023. doi:10.
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