- Meeting abstract
- Open Access
PW02-041 - Canakinumab treatment regimens in CAPS-patients
© Hofer et al; licensee BioMed Central Ltd. 2013
- Published: 8 November 2013
- Dose Adjustment
- Partial Remission
- Dose Recommendation
- Severe Phenotype
Canakinumab is a recombinant monoclonal fully human antibody against Interleukin-1β and currently the only drug approved for the treatment of CAPS in Europe. Current dose recommendations are 150mg (body weight >40kg) respectively 2mg/kg bodyweight (15 to 40kg) every 8 weeks but yield insufficient response in some individuals, especially in children and patients with severe phenotypes .
In this study we analyzed the response to daily practice (in contrast to trial condition) canakinumab treatment regimens in CAPS-patients with focus on age, mutation and clinical presentation and the necessity and effect of dose adjustment.
An observational national multicenter study was conducted. CAPS-Patients were included if they received at least two doses of canakinumab. Data included information regarding demographics, treatment, clinical disease activity and inflammatory markers (including SAA, CRP, S100, ESR, IL-6). Response to treatment was assessed using CAPS-disease activity scores, CRP and/or SAA levels.
A cohort of 68 patients with CAPS was analyzed. At the beginning of treatment 27 patients had been younger than 18 years with a median age of 25.4 years (range 22 months to 73 years). The most frequent mutations were R260W, A439V, E311K, V198M, Q703K and most patients showed MWS or FCAS/MWS phenotype (3 patients with NOMID, 4 with MWS/NOMID). The median treatment duration was 855 days (range: 28-1973 days). In 57% (39) of patients full response was sustained until next scheduled drug application (34% (23) partial remission). With standard treatment 31% (21) of patients achieved full response. In 44% (30) of all patients canakinumab dose and/or application interval was increased above the standard regimen (2/3 NOMID, 3/4 MWS/NOMID). Two serious adverse events were reported (severe infection, osteonecrosis), mild and moderate adverse events were mostly upper respiratory tract infections but almost no injection site reactions.
Most CAPS-Patients achieve full remission with canakinumab. However, almost 50% of patients, particularly children, require dose adjustment. Dose increase was well tolerated and full remission was achieved without an increased rate of adverse events.
This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.