Session Information
Session Type: Abstract Submissions (ACR)
Background/Purpose:
Cryopyrin-associated periodic syndrome (CAPS) is an extremely rare autoinflammatory disorder associated with overproduction of interleukin-1β (IL-1β). Canakinumab, a fully human, selective, anti-IL-1β monoclonal antibody, is approved for the treatment of CAPS (including familial cold autoinflammatory syndrome [FCAS] and Muckle-Wells syndrome [MWS]). In order to enhance long term data, the β-Confident Registry, a global, prospective, observational study, is monitoring safety and disease progression in patients treated with canakinumab over a 5-year period.
Methods:
CAPS patients receiving canakinumab as part of their routine medical care are included in the registry. Data are collected during routine clinical assessments (no mandatory visits), and the registry is updated every 6 months. Assessments include adverse events (AEs); physician’s global assessment of autoinflammatory disease activity; and C-reactive protein (CRP) and serum amyloid A (SAA) levels. Data reported here are adverse events through 18 months of follow-up (cut-off date, March 2012). Additional safety data will be updated with the presentation, as available.
Results:
Since December 2009, 229 patients with CAPS and other autoinflammatory diseases have enrolled. Selected baseline characteristics are shown in the Table. Overall, 59 AEs (25.8%) were reported in 29 patients (12.7%). Infections were the most common AE, with 12 AEs (5.2%) reported in 8 patients (3.5%), and upper respiratory type infections accounted for most. CNS disorders were the second most common AE, with 7 AEs (3.1%) reported in 5 patients (2.2%), and headache accounted for most. 11 Serious AEs (4.8%) were reported in 8 patients (3.5%), including 1 malignancy (rectal adenocarcinoma in a 76 yo MWS patient) and 2 infections (pneumonia in a 40 yo female CAPS patient with later hospital discharge; aseptic meningitis in a 25 yo female CINCA patient with recovery). There was only 1 permanent canakinumab discontinuation, due to patient preference.
Conclusion:
Consistent with the earlier 12-month assessment, the safety of canakinumab treatment at 18 months was maintained and no unexpected safety signals were reported.
|
Table: Baseline characteristics of enrolled patients |
|||||
|
|
Overall (N=229) |
FCAS (n=35) |
MWS (n=135) |
NOMID (n=18) |
Others (n=29) |
|
Age groups: |
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|
< 18y |
74 |
7 |
33 |
11 |
18 |
|
≥ 18y |
155 |
28 |
102 |
7 |
11 |
|
Sex: |
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|
Male |
107 |
12 |
68 |
10 |
15 |
|
Female |
122 |
23 |
67 |
8 |
14 |
|
NLRP3 mutation, n(%) |
|||||
|
Yes |
183 (79.9) |
35 (100.0) |
126 (93.3) |
15 (83.3) |
7 (24.1) |
|
No |
16 (7.0) |
0 |
3 (2.2) |
1 (5.6) |
12 (41.4) |
|
Unknown/missing |
30 (13.1) |
0 |
6 (4.4) |
2 (11.1) |
10 (34.5) |
|
Mean disease duration, months |
312 |
403 |
316 |
154 |
228 |
|
History of rash/ arthralgia/ headache/ conjunctivitis, % |
79/81/55/58 |
100/94/54/63 |
81/80/59/67 |
89/94/89/67 |
76/93/38/28 |
|
Prior canakinumab treatment, n |
191 |
28 |
117 |
17 |
29 |
|
Prior treatment duration, Median (range), wk. |
87.3 (8.9–139.4) |
85.1 (8.9–126.3) |
87.3 (8.9–139.4) |
82.9 (43.9–122.0) |
65.4 (13.3–139.4) |
|
Prior number of injections/patients, mean±SD |
6.4±4.4 |
4.3±2.7 |
6.8±3.9 |
8.6±5.2 |
6.1±6.2 |
|
FCAS, familial cold autoinflammatory syndrome; MWS, Muckle-Wells syndrome; NOMID, neonatal-onset multisystem inflammatory disease |
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Disclosure:
H. Hoffman,
Novartis,
5,
Regeneron,
5,
Sobi Biovitrum,
5;
J. B. Kuemmerle-Deschner,
Novartis,
2,
Norartis,
5,
Novartis,
7;
P. Hawkins,
None;
T. van der Poll,
Novartis,
5;
U. A. Walker,
Novartis ,
5;
B. Rauer,
Novartis ,
3;
J. M. Nebesky,
Novartis ,
3;
H. Tilson,
Bio Soteria,
5,
Bristol-Myers Squibb,
5,
Gilead,
5,
GlaxoSmithKline,
5,
HealthCore ,
5,
Kendle ,
5,
Merck ,
5,
Novartis ,
5,
Glaxo SmithKline,
1,
Procter & Gamble ,
1,
Other non-pharmaceutical holdings,
1.
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