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Orphanet Journal of Rare Diseases

, 10:19

First Online: 15 February 2015Received: 29 October 2014Accepted: 18 January 2015DOI: 10.1186-s13023-015-0228-7

Cite this article as: Rossi-Semerano, L., Fautrel, B., Wendling, D. et al. Orphanet J Rare Dis 2015 10: 19. doi:10.1186-s13023-015-0228-7

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.

KeywordsAuto-inflammatory diseases IL-1 Anakinra Canakinumab Tolerance Off-label AbbreviationsAEadverse events

AIDsauto-inflammatory diseases

AOSDadult onset Still’s disease

CAPScryopyrin-associated periodic syndrome

CeRéMAIFrench National Reference Centre for Auto-inflammatory Diseases

CIconfidence interval

CINCAChronic Infantile Neurological Cutaneous and Articular syndrome

CRIClub rhumatisme et inflammation

CRPC-reactive protein

DITRAdeficiency of the IL-36 receptor antagonist

DMARDsdisease-modifying anti-rheumatic drugs

EMAEuropean Medicines Agency

FCASFamilial Cold Autoinflammatory syndrome

FMFfamilial Mediterranean fever

GPPgeneralized pustular psoriasis

IL-1interleukin-1

IL-1RAIL-1 receptor antagonist

IQRinterquartile range

MASmacrophage activation syndrome

MKDmevalonate kinase deficiency

MWSMuckle-Wells syndrome

NAPS12NLRP12-associated periodic syndrome

NSAIDsnon-steroidal anti-inflammatory drugs

RArheumatoid arthritis

SAEssevere adverse events

SDstandard deviation

sJIAsystemic juvenile idiopathic arthritis

TRtotal range

TRAPStumor necrosis factor receptor-associated periodic syndrome

Electronic supplementary materialThe online version of this article doi:10.1186-s13023-015-0228-7 contains supplementary material, which is available to authorized users.

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Autor: Linda Rossi-Semerano - Bruno Fautrel - Daniel Wendling - Eric Hachulla - Caroline Galeotti - Luca Semerano - Isabelle Touit

Fuente: https://link.springer.com/







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