A systematic literature review of efficacy, effectiveness and safety of biologic therapies for treatment of familial Mediterranean fever.

Abstract

OBJECTIVES:

To identify and summarize the existing evidence on the efficacy, effectiveness and safety of biologic therapies used, either as indicated or off-label, in the treatment of FMF.

METHODS:

A systematic literature review was conducted using Embase®, MEDLINE®, MEDLINE®-In Process, and Cochrane databases to identify randomized/non-randomized controlled trials (RCTs/non-RCTs) and real-world observational studies of FMF published as full-text articles (2000-September 2017) or conference abstracts (2014-September 2017). Studies with data for ≥1 biologic were included. Studies with <5 patients were excluded.

RESULTS:

Of the 3342 retrieved records, 67 publications, yielding 38 unique studies, were included. All studies were published after the year 2010, and the majority (21) were full-text articles. Most studies (33/38) were prospective/retrospective observational; three were double-blind, placebo-controlled RCTs (one each of anakinra, canakinumab and rilonacept); and two were non-RCTs (both canakinumab). Anakinra (26), canakinumab (21) and etanercept (6) were the most frequently used biologics across studies, whereas use of adalimumab, tocilizumab, rilonacept and infliximab was limited (1-2 studies). The available evidence suggested benefits of anakinra and canakinumab in FMF.

CONCLUSION:

Anti-IL-1 therapies (i.e. anakinra and canakinumab) appear to be effective and safe options in the treatment of overall FMF, including patients with colchicine resistance and FMF-related amyloidosis. There is a need for properly designed prospective or controlled studies to conclude the superiority of one anti-IL-1 therapy over another. Evidence on the use of TNF-α and IL-6 inhibitors is limited, and further research is suggested.

• Authors: Jasmin B Kuemmerle-Deschner, Raju Gautam, Aneesh T George, Syed Raza, Kathleen G Lomax, Peter Hur
• Journal: Rheumatology (Oxford, England) (Rheumatology (Oxford)) Vol. 59 Issue 10 Pg. 2711-2724 (10 01 2020) ISSN: 1462-0332 [Electronic] England
• PMID: 32533192 (Publication Type: Journal Article, Meta-Analysis, Research Support, Non-U.S. Gov't, Systematic Review)
• Copyright: © The Author(s) 2020. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: [email protected].

Chemical References

• Antibodies, Monoclonal, Humanized
• Antirheumatic Agents
• Interleukin 1 Receptor Antagonist Protein
• Interleukin-1
• Recombinant Fusion Proteins
• Tumor Necrosis Factor-alpha
• canakinumab
• rilonacept
• Infliximab
• Adalimumab
• tocilizumab

Topics

• Adalimumab (therapeutic use)
• Adolescent
• Adult
• Amyloidosis (complications)
• Antibodies, Monoclonal, Humanized (therapeutic use)
• Antirheumatic Agents (therapeutic use)
• Biological Therapy (methods)
• Child
• Familial Mediterranean Fever (epidemiology, therapy)
• Humans
• Infliximab (therapeutic use)
• Interleukin 1 Receptor Antagonist Protein (therapeutic use)
• Interleukin-1 (antagonists & inhibitors)
• Middle Aged
• Non-Randomized Controlled Trials as Topic
• Observational Studies as Topic
• Prospective Studies
• Randomized Controlled Trials as Topic
• Recombinant Fusion Proteins (therapeutic use)
• Retrospective Studies
• Safety
• Treatment Outcome
• Tumor Necrosis Factor-alpha (antagonists & inhibitors)