Biologics and JAK inhibitors for the treatment of monogenic systemic autoinflammatory diseases in children - 03/03/23
Abstract |
Systemic autoinflammatory diseases (SAIDs) are caused by aberrant activation of 1 or more inflammatory pathways in an antigen-independent manner. Monogenic forms of SAIDs typically manifest during childhood, and early treatment is essential to minimize morbidity and mortality. On the basis of the mechanism of disease and the dominant cytokine(s) that propagates inflammation, monogenic SAIDs can be grouped into major categories including inflammasomopathies/disorders of IL-1, interferonopathies, and disorders of nuclear factor-κB and/or aberrant TNF activity. This classification scheme has direct therapeutic relevance given the availability of biologic agents and small-molecule inhibitors that specifically target these pathways. Here, we review the experience of using biologics that target IL-1 and TNF as well as using Janus kinase inhibitors for the treatment of monogenic SAIDs in pediatric patients. We provide an evidence-based guide for the use of these medications and discuss their mechanism of action, safety profile, and strategies for therapeutic monitoring.
Le texte complet de cet article est disponible en PDF.Key words : Systemic autoinflammatory disease, autoinflammation, inflammasome, interferon, NF-κB, IL-1, TNF, biologics, JAK inhibitors
Abbreviations used : AGS, CAPS, DADA2, DIRA, DMARD, FMF, HA20, HIDS, HOIL-1, IFN-I, JAK, Jakinib, JIA, LoE, MAS, MKD, NF-κB, NLRC4, NLRP3, NOD, NOMID, OTULIN, SAID, STAT, TNFi, TRAPS
Plan
| This work was supported by the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases (grant no. K08-AR074562 to P.Y.L. and grant nos. R01-AR065538, R01-AR073201, R01-AR075906, and P30-AR070253 to P.A.N.), the Fundación Bechara (P.A.N.), the Arbuckle Family Foundation for Arthritis Research (P.A.N.), the Samara Jan Turkel Clinical Center at Boston Children's Hospital (P.A.N.), the Rheumatology Research Foundation Investigator Award and K Supplement Award (P.Y.L.), the Charles H. Hood Foundation Child Health Research Award (P.Y.L.), and the Arthritis National Research Foundation All Arthritis Grant (P.Y.L.). |
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| Disclosure of potential conflict of interest: P. A. Nigrovic is a consultant for BMS, Brickell Bio, Exo Therapeutics, Novartis, Pfizer, and Sobi; receives sponsored research from BMS and Pfizer; and receives royalties from the American Academy of Pediatrics and UpToDate. F. Dedeoglu is a consultant for Novartis and receives royalties from UpToDate. P. Y. Lee is a consultant for Brickell Bio and Exo Therapeutics and receives royalties from UpToDate. The rest of the authors declare that they have no relevant conflicts of interest. |
Vol 151 - N° 3
P. 607-618 - mars 2023 Retour au numéro
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