Please use this identifier to cite or link to this item:
http://hdl.handle.net/1942/29127
Title: | Anakinra as a diagnostic challenge and treatment option for systemic autoinflammatory disorders of undefined etiology. | Authors: | Harrison, Stephanie R. McGonagle, Dennis Nizam, Sharmin Jarrett, Stephen VAN DER HILST, Jeroen McDermott, Michael F. Savic, Sinisa |
Issue Date: | 2016 | Source: | JCI Insight, 1(6) (Art N° e86336) | Abstract: | BACKGROUND. Some adult patients presenting with unexplained pyrexia, serositis, skin rashes, arthralgia, myalgia, and other symptoms commonly found in autoinflammatory disorders may not fit a specific diagnosis, either because their clinical phenotype is nondiagnostic or genetic tests are negative. We used the term undifferentiated systemic autoinflammatory disorder (uSAID) to describe such cases. Given that well-defined autoinflammatory diseases show responses to IL-1 blockade, we evaluated whether anakinra was useful for both diagnosing and treating uSAID patients. METHODS. We performed a retrospective analysis of consecutive patients presenting with uSAID between 2012–2015 who were treated with the recombinant IL-1 receptor antagonist anakinra. uSAID was diagnosed after excluding malignancy, infection, and pathogenic mutations in known hereditary fever syndromes (HFS) genes and where clinical criteria for adult onset Still’s disease (AOSD) were not met. RESULTS. A total of 11 patients presented with uSAID (5 males and 6 females), with a mean time to diagnosis of 3.5 years (1–8 years). Patients were unresponsive or only partially controlled on disease-modifying antirheumatic drug (DMARD)/steroid treatment. Anakinra controlled symptoms within 4–6 weeks of starting treatment in 9 of 11 cases. Two patients discontinued therapy — one due to incomplete response and another due to severe injection-site reactions. CONCLUSION. This retrospective case series demonstrates that the spectrum of poorly defined autoinflammatory disorders that show responsiveness to anakinra is considerable. Anakinra seems a viable treatment option for these patients, who are unresponsive to standard steroid/DMARD treatments. Moreover, given the mechanisms of action, response to anakinra implicates underlying IL-1 dysregulation in the disease pathogenesis of responding uSAIDs patients | Keywords: | Clinical Medicine; Immunology; Inflammation | Document URI: | http://hdl.handle.net/1942/29127 | e-ISSN: | 2379-3708 | DOI: | 10.1172/jci.insight.86336 | ISI #: | 000387111300005 | Category: | A1 | Type: | Journal Contribution | Validations: | ecoom 2021 |
Appears in Collections: | Research publications |
Files in This Item:
File | Description | Size | Format | |
---|---|---|---|---|
Article PDF.pdf Restricted Access | Published version | 1.44 MB | Adobe PDF | View/Open Request a copy |
Anakinra as a diagnostic challenge and treatment option for systemic autoinflammatory disorders of undefined etiology.pdf | Peer-reviewed author version | 161.3 kB | Adobe PDF | View/Open |
WEB OF SCIENCETM
Citations
43
checked on Oct 18, 2024
Page view(s)
100
checked on Sep 6, 2022
Download(s)
86
checked on Sep 6, 2022
Google ScholarTM
Check
Altmetric
Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.