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Efficacy and safety of anakinra therapy in pediatric and adult patients with the autoinflammatory Muckle-Wells syndrome
Arthritis & Rheumatism, 11/18/2010
Kuemmerle-Deschner JB et al. – Anakinra was a safe and effective treatment for severe MWS, leading to a significant improvement of disease activity at 2 weeks and long-term. Anakinra therapy therefore should be considered in children and adults with severe MWS disease requiring IL-1 blockade.
Methods- Single center observational study
- Standardized assessments included clinical features, MWS disease activity score (MWS-DAS), classical and novel inflammatory markers, and patient-derived measures of health
- Measures of MWS disease activity analyzed using descriptive statistics and paired comparative analysis
- MWS–DAS <10 at 2 weeks and at last follow-up
- Total of 12 patients with severe MWS (5 children, 7 adults) received anakinra for median time of 11months (5–14)
- Disease activity significantly lower in all patients at 2 weeks (p=0.0005)
- MWS organ manifestations improved, as did all patient-derived measures of health, inflammatory markers, and hearing loss in two patients
- Novel neutrophil activation biomarker S100A12 followed clinical disease activity
- Treatment well tolerated, and no serious adverse events observed
