Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger.

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Version: Final published version
License: CC BY-NC-ND 4.0
Serval ID
serval:BIB_3992E8E3EF64
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger.
Journal
Arthritis & rheumatology
Author(s)
Brogan P.A., Hofer M., Kuemmerle-Deschner J.B., Koné-Paut I., Roesler J., Kallinich T., Horneff G., Calvo Penadés I., Sevilla-Perez B., Goffin L., Lauwerys B.R., Lachmann H.J., Uziel Y., Wei X., Laxer R.M.
ISSN
2326-5205 (Electronic)
ISSN-L
2326-5191
Publication state
Published
Issued date
11/2019
Peer-reviewed
Oui
Volume
71
Number
11
Pages
1955-1963
Language
english
Notes
Publication types: Clinical Trial, Phase III ; Journal Article ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Abstract
To assess long-term efficacy and safety of canakinumab and the response to vaccination in children ages ≤5 years with cryopyrin-associated periodic syndrome (CAPS).
CAPS patients (ages ≤5 years) received 2 mg/kg canakinumab subcutaneously every 8 weeks; patients with neonatal-onset multisystem inflammatory disease (NOMID) received a starting dose of 4 mg/kg in this open-label trial. Efficacy was evaluated using physician global assessment of disease activity and serum levels of C-reactive protein (CRP) and amyloid A (SAA). Adverse events (AEs) were recorded. Vaccination response was evaluated using postvaccination antibody titers at 4 and 8 weeks after immunization.
Of the 17 patients enrolled, 12 (71%) had Muckle-Wells syndrome, 4 (24%) had NOMID, and 1 (6%) had familial cold autoinflammatory syndrome. All 17 patients had a complete response to canakinumab. Disease activity improved according to the physician global assessment, and for 65% of the patients autoinflammatory disease was characterized as "absent" at the end of the study. Median CRP levels decreased over time. No such change was evident in SAA levels. During the extension study, postvaccination antibody titers increased above protective levels in 16 (94%) of 17 assessable vaccinations. Ten of the patients (59%) had AEs suspected to be related to canakinumab; 8 (47%) experienced at least 1 serious AE (SAE). None of the AEs or SAEs required interruption of canakinumab therapy.
Our findings indicate that canakinumab effectively maintains efficacy through 152 weeks and appears to have no effect on the ability to produce antibodies against standard childhood non-live vaccines. The safety profile of canakinumab was consistent with previous studies, supporting long-term use of canakinumab for CAPS in children ≤5 years of age.
Keywords
Antibodies, Monoclonal, Humanized/therapeutic use, Antibody Formation/immunology, C-Reactive Protein/metabolism, Child, Preschool, Cryopyrin-Associated Periodic Syndromes/drug therapy, Cryopyrin-Associated Periodic Syndromes/immunology, Cryopyrin-Associated Periodic Syndromes/metabolism, Diarrhea/chemically induced, Female, Fever/chemically induced, Humans, Infant, Infant, Newborn, Male, Nasopharyngitis/chemically induced, Respiratory Tract Infections/epidemiology, Respiratory Tract Infections/etiology, Serum Amyloid A Protein/metabolism, Treatment Outcome, Vaccines/therapeutic use
Pubmed
Web of science
Open Access
Yes
Create date
17/06/2019 16:29
Last modification date
15/01/2021 7:08
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