Abstract
BackgroundDespite their limited licensed indications, anti–interleukin-1 (anti–IL-1) agents are often used in clinical practice for an increasing number of auto-inflammatory diseases. We conducted a national cross-sectional observational study from January 2011 to January 2013 to record the off-label use of such agents in France. We aimed to estimate the off-label use of anti–IL-1 treatments in France, assess their efficacy in rare diseases, and increase the reporting of their possible side effects.MethodsPhysicians answered a questionnaire that covered patient and disease data, anti–IL-1 agent use, efficacy and adverse events. The study involved adult or paediatric patient who had received an anti–IL-1 agent after January 2005 in France.ResultsIn total, 189 patients from 38 centres were included. The main diseases were adult-onset Still’s disease (AOSD) (35), gout (28), systemic juvenile idiopathic arthritis (27), cryopyrin-associated periodic syndrome (CAPS) (21), familial Mediterranean fever (14) and mevalonate kinase deficiency (12). The main off-label used agent was anakinra, used at least once for 185 patients, with canakinumab used for 25. Anakinra was effective in most patients (90%), with higher complete clinical response rates for Schnitzler’s syndrome, gout, CAPS and AOSD. Overall, 58% of patients showed at least one adverse event, mainly minor injection-site reactions. The main reported serious adverse event was severe infection. Injection-site reactions and liver toxicity were significantly more frequent in children than adults. The main non-cutaneous adverse event was liver toxicity, significantly associated with treatment duration. Weight gain was reported in about 10% of patients and was associated with treatment duration and CAPS. Canakinumab was rarely used and showed better cutaneous tolerance than anakinra but similar rates of non-cutaneous and severe adverse events.ConclusionsAnakinra was well tolerated and effective in most patients with various inflammatory diseases. The main adverse events were mild injection-site reactions, especially in children. The survey allowed for collecting limited information on the off-label use of canakinumab.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-015-0228-7) contains supplementary material, which is available to authorized users.
Highlights
Despite their limited licensed indications, anti–interleukin-1 agents are often used in clinical practice for an increasing number of auto-inflammatory diseases
Anakinra was administered as on-demand treatment (
Only the association with treatment duration persisted, and we found a significant trend for increased odds of overall non-cutaneous adverse events (AE) with increased quartile of treatment duration (p = 0.05)
Summary
Despite their limited licensed indications, anti–interleukin-1 (anti–IL-1) agents are often used in clinical practice for an increasing number of auto-inflammatory diseases. We aimed to estimate the off-label use of anti–IL-1 treatments in France, assess their efficacy in rare diseases, and increase the reporting of their possible side effects. Levels of acute-phase reactants are markedly elevated during flares and classically disappear (or are markedly reduced) in between flares. Other polygenic diseases such as adult-onset Still’s disease (AOSD), systemic juvenile idiopathic arthritis (sJIA), Beçhet’s disease and gout, belong to the AIDs, in that deregulation of IL-1 is involved, at least in part, in their pathogenesis [3]. The use of anti–IL-1 treatments for these conditions is continually increasing, most often in an off-label setting and without standardized monitoring. 0.27 (0.11 to 0.64) 0.003 0.72 (0.33 to 1.59) 0.42 Corticosteroids
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