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Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised Phase 3 CLUSTER trial.

Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised Phase 3 CLUSTER trial.

Authors :
Jeyaratnam, Jerold
Simon, Anna
Calvo, Inmaculada
Constantin, Tamas
Shcherbina, Anna
Hofer, Michael
Gattorno, Marco
Martini, Alberto
Bader-Meunier, Brigitte
Vastert, Bas
Levy, Jeremy
Dekker, Elise
Benedetti, Fabrizio de
Frenkel, Joost
Source :
Rheumatology. May2022, Vol. 61 Issue 5, p2088-2094. 7p.
Publication Year :
2022

Abstract

Objectives To evaluate the long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency during the open label extension (weeks 41–113) of the randomized controlled CLUSTER trial. Methods During a 72-week period, patients received open-label canakinumab 150 or 300 mg, every 4 or 8 weeks. The disease activity was evaluated every 8 weeks using physician global assessment and counting the number of flares. Concentrations of CRP and serum amyloid A protein were measured. The safety was studied by determination and classification of observed adverse events. The safety and efficacy were analysed separately in three subgroups of patients receiving a cumulative dose of less than <35 mg/kg, ≥35 to <70 mg/kg or ≥70 mg/kg. Results Of the 74 patients who started the CLUSTER study, 66 entered Epoch 4 and 65 completed it. During the 72-week period, 42 (64%) patients experienced no flares, while 13 (20%) had one flare, as compared with a median of 12 flares per year reported at baseline. Low physician global assessment scores were seen at the end of the study for all groups with >90% reporting minimal disease activity or none at all. Median CRP concentrations were consistently equal or lower than 10 mg/l, while median serum amyloid A concentrations remained only slightly above the normal range of 10 mg/l. The study showed no new or unexpected adverse events. Conclusion Canakinumab proved effective to control disease activity and prevent flares in mevalonate kinase deficiency during the 72-week study period. No new safety concerns were reported. Trial registration NCT02059291. https://clinicaltrials.gov. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
14620324
Volume :
61
Issue :
5
Database :
Academic Search Index
Journal :
Rheumatology
Publication Type :
Academic Journal
Accession number :
156714366
Full Text :
https://doi.org/10.1093/rheumatology/keab696