Universität zu Köln

Brogan, Paul A., Hofer, Michael, Kuemmerle-Deschner, Jasmin B., Kone-Paut, Isabelle, Roesler, Joachim, Kallinich, Tilmann, Horneff, Gerd, Calvo Penades, Inmaculada, Sevilla-Perez, Belen, Goffin, Laurence, Lauwerys, Bernard R., Lachmann, Helen J., Uziel, Yosef, Wei, Xiaoling and Laxer, Ronald M. (2019). Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger. Arthritis Rheumatol., 71 (11). S. 1955 - 1964. HOBOKEN: WILEY. ISSN 2326-5205

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Abstract

Objective 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). Methods 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. Results 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. Conclusion 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.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Brogan, Paul A. UNSPECIFIED UNSPECIFIED UNSPECIFIED Hofer, Michael UNSPECIFIED UNSPECIFIED UNSPECIFIED Kuemmerle-Deschner, Jasmin B. UNSPECIFIED UNSPECIFIED UNSPECIFIED Kone-Paut, Isabelle UNSPECIFIED UNSPECIFIED UNSPECIFIED Roesler, Joachim UNSPECIFIED UNSPECIFIED UNSPECIFIED Kallinich, Tilmann UNSPECIFIED UNSPECIFIED UNSPECIFIED Horneff, Gerd UNSPECIFIED UNSPECIFIED UNSPECIFIED Calvo Penades, Inmaculada UNSPECIFIED UNSPECIFIED UNSPECIFIED Sevilla-Perez, Belen UNSPECIFIED UNSPECIFIED UNSPECIFIED Goffin, Laurence UNSPECIFIED UNSPECIFIED UNSPECIFIED Lauwerys, Bernard R. UNSPECIFIED UNSPECIFIED UNSPECIFIED Lachmann, Helen J. UNSPECIFIED UNSPECIFIED UNSPECIFIED Uziel, Yosef UNSPECIFIED UNSPECIFIED UNSPECIFIED Wei, Xiaoling UNSPECIFIED UNSPECIFIED UNSPECIFIED Laxer, Ronald M. UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-141618
DOI:	10.1002/art.41004
Journal or Publication Title:	Arthritis Rheumatol.
Volume:	71
Number:	11
Page Range:	S. 1955 - 1964
Date:	2019
Publisher:	WILEY
Place of Publication:	HOBOKEN
ISSN:	2326-5205
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language COLD AUTOINFLAMMATORY SYNDROME; OPEN-LABEL; ANAKINRA; MUTATION; RECOMMENDATIONS; INTERLEUKIN-1; MANAGEMENT; URTICARIA; CIAS1; GENE Multiple languages Rheumatology Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/14161