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

AbstractOBJECTIVE:
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.
AuthorsSamuel Deshayes, Sophie Georgin-Lavialle, Arnaud Hot, Cécile-Audrey Durel, Eric Hachulla, Nicolas Rouanes, Sylvain Audia, Thomas Le Gallou, Pierre Quartier, Geoffrey Urbanski, Laurent Messer, Stéphane Klein, Hubert de Boysson, Boris Bienvenu, Gilles Grateau, Achille Aouba
JournalThe Journal of rheumatology (J Rheumatol) Vol. 45 Issue 3 Pg. 425-429 (03 2018) ISSN: 0315-162X [Print] Canada
PMID29335349 (Publication Type: Journal Article, Multicenter Study)
Chemical References
  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Antirheumatic Agents
  • IL1B protein, human
  • Interleukin 1 Receptor Antagonist Protein
  • Interleukin-1beta
  • canakinumab
Topics
  • Adolescent
  • Adult
  • Antibodies, Monoclonal (pharmacology, therapeutic use)
  • Antibodies, Monoclonal, Humanized
  • Antirheumatic Agents (pharmacology, therapeutic use)
  • Chemical and Drug Induced Liver Injury
  • Child
  • Child, Preschool
  • Drug Tolerance
  • Female
  • France
  • Humans
  • Infections (chemically induced)
  • Injection Site Reaction (etiology)
  • Interleukin 1 Receptor Antagonist Protein (adverse effects, pharmacology, therapeutic use)
  • Interleukin-1beta (antagonists & inhibitors)
  • Male
  • Mevalonate Kinase Deficiency (drug therapy)
  • Precision Medicine
  • Remission Induction
  • Retrospective Studies
  • Treatment Outcome
  • Weight Gain (drug effects)

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