DI-080 Study of the use of canakinumab in muckle–wells syndrome in a tertiary hospital

Abstract

BackgroundCanakinumab is a IgG1 anti-interleukin-1β monoclonal antibody indicated in the treatment of Muckle–Wells syndrome (MWS). It is an autosomal dominant congenital disease that is considered a rare disease. Hives, joint pains, conjunctivitis, deafness, amyloidosis and fever are its symptoms. For years, the only available treatments were non-steroidal anti-inflammatory drugs (NSAIDs) and systemic corticoids.PurposeThe objective of this study was to determine the effectiveness of canakinumab in the treatment of MWS in patients who have failed treatment with NSAIDs and systemic corticoids.Material and methodsAn observational retrospective study was carried out in a tertiary care hospital from February 2011 to October 2016. Patients who were treated with canakinumab during this period were selected. The data was obtained from the electronic software used in the hospital (Landtools). Indication, posology, duration of treatment, previous therapy, adverse effects and C reactive protein (CRP) levels were collected from patients’ digital history. Suspension of treatment with canakinumab was also registered. Remission was defined as clinical improvement plus normal CRP (<6 mg/L). Available treatments were NSAIDs and systemic corticoids.Results6 patients were selected; median age 52 years (36–66 years). The indication for treatment with canakinumab was MWS. Patients received canakinumab 150 mg subcutaneously every 8 weeks (n=5) or 12 weeks (n=1) for a median of 47 months (range 24–70). All patients had received corticoids and NSAIDs with no suitable response. Other previous therapies were antihistamines, methotrexate, colchicine, infliximab, etanercept and hydroxychloroquine. Canakinumab was well tolerated; 1 patient experienced an injection site reaction.Treatment with canakinumab caused a significant reduction in clinical disease activity and CRP levels (average before treatment of 40.7 mg/L (3.7–81.2 mg/L) versus average after treatment of 19.27 mg/L (0.45–86.03 mg/L)). 50% (n=3) of canakinumab treated patients achieved remission. All patients are currently receiving treatment.ConclusionCanakinumab is an effective therapeutic alternative for the treatment of MKS if poor effects have been achieved on other therapies. The observed evolution was favourable, being safe and well tolerated.References and/or acknowledgementsOrphanet guide.No conflict of interest

Systematic literature review of efficacy/effectiveness and safety of current therapies for the treatment of cryopyrin-associated periodic syndrome, hyperimmunoglobulin D syndrome and tumour necrosis factor receptor-associated periodic syndrome

“…Ten observational studies reported the effectiveness of canakinumab in patients with different CAPS phenotypes and of various age groups. 34 39– 41 48 52 59 66 82 92 Among the studies reporting CR/PR, 93% of canakinumab-treated MWS patients achieved a CR at short-term follow-up. 59 Patients who achieved a CR with canakinumab ranged from 62% 48 to 93% 59 at mid-term follow-up, and 50% 34 52 to 100% 82 at long-term follow-up (online supplementary figure 2A–B).…”

“…34 39– 41 48 52 59 66 82 92 Among the studies reporting CR/PR, 93% of canakinumab-treated MWS patients achieved a CR at short-term follow-up. 59 Patients who achieved a CR with canakinumab ranged from 62% 48 to 93% 59 at mid-term follow-up, and 50% 34 52 to 100% 82 at long-term follow-up (online supplementary figure 2A–B). Other observational studies have also shown the effectiveness of canakinumab in improving the Disease Activity Score (DAS), 57 65 Autoinflammatory Disease Activity Index (AIDAI) score, 36 83 complete/partial resolution of symptoms, 38 45 88 improvement in physician/patient assessed PROs 36 65 85 and the Child Health Assessment Questionnaire (CHAQ).…”

“…The conventional treatments such as corticosteroids, colchicine, NSAIDs and methotrexate were also used in few studies of CAPS and provided either PR or no response (online supplementary figure 2A–B). 44 52 68 92…”