P02-008 - Dramatic response to canakinumab in MKD
نویسندگان
چکیده
Introduction Mevalonate kinase deficiency-associated periodic fever syndrome (MKD) is a systemic autoinflammatory disease caused by mutations in the mevalonate kinase gene (MVK), previously named “hyper-IgD syndrome” due to its characteristic increase in serum IgD level. The patients suffer recurrent fever attacks every 2-8 weeks beginning from infancy, often precipitated by immunizations, infections or emotional stress. Fever lasts 2-7 days and can be accompanied by malaise, headache, diarrhea, abdominal pain, vomiting, skin rashes, arthralgia, arthritis, tender lymphadenopathy and hepatosplenomegaly. Fever attacks usually respond to the administration of steroids. However, increasing frequency of fever episodes with steroid use and the natural chronic disease course may require a continuous long-term treatment. Colchicine, cyclosporine, thalidomide and statins are not effective. A TNF-a blocking agent etanercept and an IL-1 blocking agent anakinra have been demonstrated to reduce the frequency of fever attacks in MKD. Canakinumab is a human monoclonal antibody targeted at interleukin-1 beta. Here, we report a 6-year-old boy with MKD who had a dramatic response to canakinumab.
منابع مشابه
P02-026 - Model-based characterization of the PKPD relationship for canakinumab in CAPS: a step towards personalized
Objectives The objectives of the study were to describe the kinetics of canakinumab and dynamics of binding IL-1b in CAPS patients; to determine if these are different in 2and 3-year-old children versus older children and adults; and to explore the impact of CAPS phenotype (Muckle-Wells Syndrome [MWS], Familial Cold Autoinflammatory Syndrome [FCAS], Neonatal-Onset Multisystem Inflammatory Disea...
متن کاملEfficacy of interleukin-1-targeting drugs in mevalonate kinase deficiency.
OBJECTIVE To describe the efficacy and safety of IL-1-targeting drugs, anakinra and canakinumab, in patients with mevalonate kinase deficiency (MKD). METHODS A questionnaire was sent to French paediatric and adult rheumatologists to retrospectively collect information on disease activity before and after treatment with IL-1 antagonists from genetically confirmed MKD patients. We assessed the ...
متن کاملP02-027 - Quality of life in CAPS treated by Canakinumab
Methods Patients were those first included in the pivotal (D2304) and in the extension (D2306) Canakinumab/CAPS study [1] and followed in the reference center for autoinflammatory diseases at Bicêtre Hospital (Paris). All carried a heterozygous mutation in the NLPR3 gene and had received Canakinumab (dose of 150 mg every 8 weeks or 2mg/kg for patient weight under 40kg). All patients had been fo...
متن کاملP02-015 - A novel MVK mutation in a child with AA amyloid
Introduction AA amyloidosis may develop as a consequence of chronic inflammatory conditions including inherited periodic fever syndromes. Mevalonate-kinase (MVK) deficiency (MKD) appears to be the least frequent underlying condition after FMF, TRAPS and CAPS. Moreover, amyloidosis rarely manifests during childhood. We report a case of a small child in whom renal biopsy performed because of the ...
متن کاملTherapy of autoinflammatory diseases: a review of the literature
Results RCTs provide evidence for rilonacept and canakinumab in CAPS, colchicine in FMF and (adeno)tonsillectomy in PFAPA syndrome. For Behçet disease, RCTs have been conducted on the effect of several drugs, including colchicine, azathioprine, cyclosporine A, interferon alfa, etanercept, sucralfate suspension and pimecrolimus. Descriptive studies suggest that NSAIDs and corticosteroids are hig...
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