The off-label use of anakinra in pediatric systemic autoinflammatory diseases.
anakinra
anti-IL-1 inhibitors
autoinflammatory diseases
children
interleukin-1
Journal
Therapeutic advances in musculoskeletal disease
ISSN: 1759-720X
Titre abrégé: Ther Adv Musculoskelet Dis
Pays: England
ID NLM: 101517322
Informations de publication
Date de publication:
2020
2020
Historique:
received:
22
04
2020
accepted:
26
08
2020
entrez:
5
11
2020
pubmed:
6
11
2020
medline:
6
11
2020
Statut:
epublish
Résumé
Interleukin 1 (IL-1), a central mediator of innate immunity, is considered a master cytokine of local and systemic inflammation. IL-1 has emerged as pivotal in the pathogenesis of autoinflammatory diseases (AIDs), and blockade of its pathway has become a crucial target for therapy. Anakinra (ANA), a recombinant IL-1β receptor antagonist, was the first anti-IL-1 agent employed in clinical practice. ANA is currently approved for the treatment of rheumatoid arthritis, systemic juvenile idiopathic arthritis, adult-onset Still's disease, and cryopyrin-associated autoinflammatory syndrome. It has also been successfully used for off-label treatment of various monogenic, polygenic, or undefined etiology systemic AIDs. This review describes currently available evidence for the off-label use of ANA in pediatric rheumatologic diseases. Specifically, the use of ANA in Kawasaki disease, idiopathic recurrent pericarditis, Behçet disease, monogenic AIDs, undifferentiated AIDs, chronic non-bacterial osteomyelitis, macrophage activation syndrome, and febrile infection-related epilepsy, in terms of its safety and efficacy. In selected pediatric rheumatic disorders, the off-label administration of ANA appears to be effective and safe. In order to control severe and/or relapsing disease, ANA should be considered as a valuable treatment option in children suffering from rare inflammatory diseases. However, currently available data consist of retrospective studies and short case series; thus, randomized controlled trials and larger series with long-term follow up are mandatory to better assess the efficacy and cost effectiveness of ANA in these challenging patients.
Identifiants
pubmed: 33149772
doi: 10.1177/1759720X20959575
pii: 10.1177_1759720X20959575
pmc: PMC7580132
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
1759720X20959575Informations de copyright
© The Author(s), 2020.
Déclaration de conflit d'intérêts
Conflict of interest statement: The authors declare that there is no conflict of interest.
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