RT Journal Article
SR Electronic
T1 Efficacy of Continuous Interleukin 1 Blockade in Mevalonate Kinase Deficiency: A Multicenter Retrospective Study in 13 Adult Patients and Literature Review
JF The Journal of Rheumatology
JO J Rheumatol
FD The Journal of Rheumatology
SP jrheum.170684
DO 10.3899/jrheum.170684
A1 Samuel Deshayes
A1 Sophie Georgin-Lavialle
A1 Arnaud Hot
A1 Cécile-Audrey Durel
A1 Eric Hachulla
A1 Nicolas Rouanes
A1 Sylvain Audia
A1 Thomas Le Gallou
A1 Pierre Quartier
A1 Geoffrey Urbanski
A1 Laurent Messer
A1 Stéphane Klein
A1 Hubert de Boysson
A1 Boris Bienvenu
A1 Gilles Grateau
A1 Achille Aouba
YR 2018
UL http://www.jrheum.org/content/early/2018/01/05/jrheum.170684.abstract
AB 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.