Autoinflammatory & rare systemic diseases
Refining treat-to-target strategies in cryopyrin-associated periodic syndromes: the role of inflammatory markers
Authors' conclusion
Guidance for refining real-life T2T strategies in CAPS cohorts can be drawn from serial assessments of PGA and PPGA, reliably reflecting changes in disease activity. Individual parameters including age and NLRP3 gene variants are important predictors, while the sensitivity of inflammatory markers is limited due to the confounding anti-IL-1 therapy.
Study context
- Population
- Consecutive children and adults diagnosed with cryopyrin-associated periodic syndromes (CAPS) and treated with anti-IL-1 therapy
- Participants
- 54 children759 follow-up visits
- Outcome
- Effectiveness of anti-IL-1 treat-to-target (T2T) strategies
Methodological detailssingle-centre, consecutive patients, Children 31/54 (57%); median follow-up 108 months (12-620), Moderate CAPS phenotype 89%; pathogenic/likely pathogenic NLRP3 variants 59%
- Single-centre
- Consecutive patients
- Children 31/54 (57%); median follow-up 108 months (12-620)
- Moderate CAPS phenotype 89%; pathogenic/likely pathogenic NLRP3 variants 59%
Main finding
As reported in the abstract
- Patients achieving remission at last follow-up
- 96%
- Significant improvement in both PGA/PPGA throughout the study
- P < 0.01
- Visits with therapy adjustments (35 escalations, 15 reductions)
- 50/759
- Therapy escalation visits for children
- 74%
- Moderate clinical disease activity at time of visit
- 63%
- Severe clinical disease activity at time of visit
- 37%
Observational data: associations, not effects.
Original publication
Refining treat-to-target strategies in cryopyrin-associated periodic syndromes: the role of inflammatory markers. Rheumatology (Oxford). Published 5 July 2024. doi:10.
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