Autoinflammatory & rare systemic diseases
Clinical Characteristics of Cryopyrin‐Associated Periodic Syndrome and Long‐Term Real‐World Efficacy and Tolerability of Canakinumab in Japan: Results of a Nationwide Survey
Authors' conclusion
Although early initiation of anti-IL-1 treatment with canakinumab is beneficial for improving disease prognosis, some patients do not achieve remission despite a high serum concentration of canakinumab. Moreover, IBD may develop in CAPS after canakinumab treatment.
Study context
- Population
- Patients with cryopyrin-associated periodic syndrome (CAPS) in Japan treated with IL-1 inhibitors, primarily canakinumab
- Participants
- 101 patients
- Exposure
- Germline vs Somatic mosaic
- Germlinen = 86
- Somatic mosaicn = 15
- Outcome
- Complete remission with canakinumab
Methodological detailscohort study, real-world
- Cohort study
- Real-world
Main finding
As reported in the abstract
- Complete remission, all patients
- 73.7%
- Complete remission, familial cold autoinflammatory syndrome
- 100%
- Complete remission, Muckle-Wells syndrome
- 79.6%
- Complete remission, chronic infantile neurological cutaneous articular syndrome/neonatal onset multisystem inflammatory disease
- 44.4%
- No inflammatory remission, with crucial deterioration of organ damage
- 23.7%
Observational data: associations, not effects.
Further findings
Mutation types identified
- Mutation types
- 39
Auditory outcomes
- Early therapeutic intervention associated with better auditory outcomes
Poor response vs canakinumab concentration
- Poor response not related to canakinumab shortage
Deaths
- Deaths
- 6
- Proportion of all patients
- 5.9%
- Deaths caused by CAPS-associated symptoms
- 4
- Deaths while receiving high-dose canakinumab (nonremitters)
- 2
Safety
Inflammatory bowel disease (IBD)-unclassified during canakinumab treatment in inflammatory nonremitters
- Patients developing IBD-unclassified
- 4
Original publication
Clinical Characteristics of Cryopyrin‐Associated Periodic Syndrome and Long‐Term Real‐World Efficacy and Tolerability of Canakinumab in Japan: Results of a Nationwide Survey. Arthritis Rheumatol. Published 7 March 2024. doi:10.
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