Efficacy of Continuous Interleukin 1 Blockade in Mevalonate Kinase Deficiency: A Multicenter Retrospective Study in 13 Adult Patients and Literature Review

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Deshayes, Samuel | Georgin-Lavialle, Sophie | Hot, Arnaud | Durel, Cecile-Audrey | Hachulla, Eric | Rouanes, Nicolas | Audia, Sylvain | Le Gallou, Thomas | Quartier, Pierre | Urbanski, Geoffrey | Messer, Laurent | Klein, Stephane | de Boysson, Hubert | Bienvenu, Boris | Grateau, Gilles | Aouba, Achille

Edité par CCSD ; Internet Scientific Publications, LLC -

International audience. Objective. To report efficacy and tolerance of interleukin 1 blockade in adult patients with mevalonate kinase deficiency (MKD).Methods. We retrospectively collected data on 13 patients with MKD who had received anakinra (n = 10) and canakinumab (n = 7).Results. Anakinra resulted in complete or partial remission in 3/10 and 5/10 patients, respectively, and no efficacy in 2/10, but a switch to canakinumab led to partial remission. Canakinumab resulted in complete or partial remission in 3/7 and 4/7 patients, respectively.Conclusion. These data support frequent partial responses, showing a better response with canakinumab. The genotype and therapeutic outcomes correlation should help in the personalization of treatment.

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