Samuel Deshayes1,2, Sophie Georgin-Lavialle1,2, Arnaud Hot1,2, Cécile-Audrey Durel1,2, Eric Hachulla1,2, Nicolas Rouanes1,2, Sylvain Audia1,2, Thomas Le Gallou1,2, Pierre Quartier1,2, Geoffrey Urbanski1,2, Laurent Messer1,2, Stéphane Klein1,2, Hubert de Boysson1,2, Boris Bienvenu1,2, Gilles Grateau1,2, Achille Aouba3,4. 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. aouba-a@chu-caen.fr. 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. aouba-a@chu-caen.fr.
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.
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.
Authors: Susanne M Benseler; Jasmin B Kuemmerle-Deschner; Tatjana Welzel; Anna L Wildermuth; Norbert Deschner Journal: Pediatr Rheumatol Online J Date: 2021-09-14 Impact factor: 3.054