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

Samuel 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

doi:10.3899/jrheum.170684

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

J Rheumatol. 2018 Mar;45(3):425-429. doi: 10.3899/jrheum.170684. Epub 2018 Jan 15.

Authors

Samuel Deshayes^{1

2}, Sophie Georgin-Lavialle^{1

2}, Arnaud Hot^{1

2}, Cécile-Audrey Durel^{1

2}, Eric Hachulla^{1

2}, Nicolas Rouanes^{1

2}, Sylvain Audia^{1

2}, Thomas Le Gallou^{1

2}, Pierre Quartier^{1

2}, Geoffrey Urbanski^{1

2}, Laurent Messer^{1

2}, Stéphane Klein^{1

2}, Hubert de Boysson^{1

2}, Boris Bienvenu^{1

2}, Gilles Grateau^{1

2}, Achille Aouba^{3

4}

Affiliations

¹ From the Department of Internal Medicine, Université Caen Normandie, Medical School, CHU de Caen, Caen; Department of Internal Medicine, Tenon Hospital, University Pierre and Marie Curie-Paris 6; Department of Pediatric Immunology, Hematology and Rheumatology, Hôpital Necker, Paris; Department of Internal Medicine, Edouard Herriot Hospital, Lyon; Department of Internal Medicine, Claude Huriez Hospital, University of Lille, Lille; Department of Internal Medicine, CH de Périgueux, Périgueux; Department of Internal Medicine and Clinical Immunology, CHU de Dijon, Dijon; Department of Internal Medicine, CHU de Rennes, Rennes; Department of Internal Medicine and Vascular Diseases, CHU d'Angers, Angers; Department of Rheumatology, CH de Colmar, Colmar, France.
² S. Deshayes, MD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; S. Georgin-Lavialle, MD, PhD, Tenon Hospital, Department of Internal Medicine, University Pierre and Marie Curie-Paris 6; A. Hot, MD, PhD, Edouard Herriot Hospital, Department of Internal Medicine; C.A. Durel, MD, Edouard Herriot Hospital, Department of Internal Medicine; E. Hachulla, MD, PhD, Claude Huriez Hospital, Department of Internal Medicine, University of Lille; N. Rouanes, MD, CH de Périgueux, Department of Internal Medicine; S. Audia, MD, PhD, CHU de Dijon, Department of Internal Medicine and Clinical Immunology; T. Le Gallou, MD, CHU de Rennes, Department of Internal Medicine; P. Quartier, MD, PhD, Hôpital Necker, Department of Pediatric Immunology, Hematology and Rheumatology; G. Urbanski, MD, CHU d'Angers, Department of Internal Medicine and Vascular Diseases; L. Messer, MD, CH de Colmar, Department of Rheumatology; S. Klein, MD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; H. de Boysson, MD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; B. Bienvenu, MD, PhD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; G. Grateau, MD, PhD, Tenon Hospital, Department of Internal Medicine, University Pierre and Marie Curie-Paris 6; A. Aouba, MD, PhD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School.
³ From the Department of Internal Medicine, Université Caen Normandie, Medical School, CHU de Caen, Caen; Department of Internal Medicine, Tenon Hospital, University Pierre and Marie Curie-Paris 6; Department of Pediatric Immunology, Hematology and Rheumatology, Hôpital Necker, Paris; Department of Internal Medicine, Edouard Herriot Hospital, Lyon; Department of Internal Medicine, Claude Huriez Hospital, University of Lille, Lille; Department of Internal Medicine, CH de Périgueux, Périgueux; Department of Internal Medicine and Clinical Immunology, CHU de Dijon, Dijon; Department of Internal Medicine, CHU de Rennes, Rennes; Department of Internal Medicine and Vascular Diseases, CHU d'Angers, Angers; Department of Rheumatology, CH de Colmar, Colmar, France. [email protected].
⁴ S. Deshayes, MD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; S. Georgin-Lavialle, MD, PhD, Tenon Hospital, Department of Internal Medicine, University Pierre and Marie Curie-Paris 6; A. Hot, MD, PhD, Edouard Herriot Hospital, Department of Internal Medicine; C.A. Durel, MD, Edouard Herriot Hospital, Department of Internal Medicine; E. Hachulla, MD, PhD, Claude Huriez Hospital, Department of Internal Medicine, University of Lille; N. Rouanes, MD, CH de Périgueux, Department of Internal Medicine; S. Audia, MD, PhD, CHU de Dijon, Department of Internal Medicine and Clinical Immunology; T. Le Gallou, MD, CHU de Rennes, Department of Internal Medicine; P. Quartier, MD, PhD, Hôpital Necker, Department of Pediatric Immunology, Hematology and Rheumatology; G. Urbanski, MD, CHU d'Angers, Department of Internal Medicine and Vascular Diseases; L. Messer, MD, CH de Colmar, Department of Rheumatology; S. Klein, MD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; H. de Boysson, MD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; B. Bienvenu, MD, PhD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School; G. Grateau, MD, PhD, Tenon Hospital, Department of Internal Medicine, University Pierre and Marie Curie-Paris 6; A. Aouba, MD, PhD, CHU de Caen, Department of Internal Medicine, Université Caen Normandie, Medical School. [email protected].

PMID: 29335349
DOI: 10.3899/jrheum.170684

Abstract

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.

Keywords: ANAKINRA; CANAKINUMAB; HEREDITARY AUTOINFLAMMATORY DISEASES; INTERLEUKIN 1 RECEPTOR ANTAGONIST PROTEIN; MEVALONATE KINASE DEFICIENCY.

Publication types

Multicenter Study

MeSH terms

Adolescent
Adult
Antibodies, Monoclonal / pharmacology
Antibodies, Monoclonal / therapeutic use*
Antibodies, Monoclonal, Humanized
Antirheumatic Agents / pharmacology
Antirheumatic Agents / 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
Interleukin 1 Receptor Antagonist Protein / pharmacology
Interleukin 1 Receptor Antagonist Protein / therapeutic use*
Interleukin-1beta / antagonists & inhibitors
Male
Mevalonate Kinase Deficiency / drug therapy*
Precision Medicine
Remission Induction
Retrospective Studies
Treatment Outcome
Weight Gain / drug effects

Substances

Antibodies, Monoclonal
Antibodies, Monoclonal, Humanized
Antirheumatic Agents
IL1B protein, human
Interleukin 1 Receptor Antagonist Protein
Interleukin-1beta
canakinumab