The Journal of rheumatology · 2018
Efficacy of Continuous Interleukin 1 Blockade in Mevalonate Kinase Deficiency: A Multicenter Retrospective Study in 13 Adult Patients and Literature Review.
СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод пока не подготовлен
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Deshayes Samuel, Georgin-Lavialle Sophie, Hot Arnaud, Durel Cécile-Audrey, Hachulla Eric, Rouanes Nicolas, Audia Sylvain, Le Gallou Thomas, Quartier Pierre, Urbanski Geoffrey, Messer Laurent, Klein Stéphane, de Boysson Hubert, Bienvenu Boris, Grateau Gilles, Aouba Achille
Аннотация
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.
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.
Предметные рубрики MeSH
AdolescentAdultAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedAntirheumatic AgentsChemical and Drug Induced Liver InjuryChildChild, PreschoolDrug ToleranceFemaleFranceHumansInfectionsInjection Site ReactionInterleukin 1 Receptor Antagonist ProteinInterleukin-1betaMaleMevalonate Kinase DeficiencyPrecision MedicineRemission InductionRetrospective StudiesTreatment OutcomeWeight Gain