Περίληψη:
Objective: To assess the efficacy and safety of the IL-1b inhibitor canakinumab in all adults with refractory Still's disease identified from the National Organization For Medicines for off-label drug use. Methods:: In a retrospective longitudinal multicenter cohort of 50 patients (median age 39 years) with active Still's disease despite treatment with corticosteroids (n = 11), conventional and synthetic (n = 34) and/or biologic disease modifying anti-rheumatic drugs (n = 30), we assessed the efficacy of canakinumab 150–300 mg administered every 4 (n = 47) or 8 weeks (n = 3) as combination therapy or monotherapy (n = 7) during a median follow-up of 27 (3–84) months. Results: Α complete response was initially observed in 78% of patients within 3 months (median), irrespective of age at disease onset. A partial response was evident in 20%. One patient had resistant disease. Treatment de-escalation was attempted in 15 of 39 complete responders and a complete drug discontinuation in 21 patients for 8 months (median). Eleven patients (22%) relapsed during treatment, one during de-escalation process, and 11 after treatment discontinuation. Overall, 9 of 11 relapses were successfully treated with canakinumab treatment intensification or re-introduction. At last visit, 18% of patients were off treatment due to remission and 26% due to disease activity. Canakinumab had a significant corticosteroid sparing effect allowing weaning in 21 of 41 cases. Infections (20%, severe 4%) and leucopenia (6%) led to treatment cessation in one patient. Conclusion: High rates of sustained remission were observed in this, largest so far, real-life cohort of adult patients with refractory Still's disease treated with canakinumab. © 2020
Συγγραφείς:
Laskari, K.
Tektonidou, P.M.G.
Katsiari, P.C.
Athanassiou, P.
Dimopoulou, D.
Gerodimos, C.
Salamaliki, C.
Papagoras, P.C.
Settas, P.L.
Vassilopoulos, P.D.
Voulgari, P.P.V.
Zakalka, M.
Georgiadis, A.
Gkoni, G.
Daoussis, P.D.
Dimitroulas, P.T.
Iliou, C.
Kallitsakis, I.
Grika, E.P.
Mavragani, P.C.
Pikazis, P.D.
Raftakis, J.
Sarikoudis, T.
Kougkas, N.
Soukera, D.
Theodorou, E.
Tsatsani, P.
Tsiakou, E.
Vlachoyiannopoulos, P.P.
Vosvotekas, G.
Sfikakis, P.P.P.
Λέξεις-κλειδιά:
abatacept; anakinra; canakinumab; corticosteroid; immunoglobulin; methotrexate; methylprednisolone; tocilizumab; tumor necrosis factor inhibitor; antirheumatic agent; biological product; canakinumab; monoclonal antibody, adolescent; adult; adult onset Still disease; aged; Article; clinical article; cohort analysis; disease activity; disease resistance; drug dose intensification; drug efficacy; drug megadose; drug safety; drug withdrawal; female; follow up; genital tract infection; human; leukopenia; longitudinal study; macrophage activation syndrome; male; monotherapy; mouth infection; multicenter study; onset age; pneumonia; priority journal; relapse; remission; respiratory tract infection; retrospective study; skin infection; soft tissue infection; treatment outcome; treatment response; urinary tract infection; clinical trial; off label drug use; treatment outcome, Adult; Antibodies, Monoclonal, Humanized; Antirheumatic Agents; Biological Products; Humans; Off-Label Use; Retrospective Studies; Still's Disease, Adult-Onset; Treatment Outcome
