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International Retrospective Chart Review of Treatment Patterns in Severe Familial Mediterranean Fever, Tumor Necrosis Factor Receptor–Associated Periodic Syndrome, and Mevalonate Kinase Deficiency/Hyperimmunoglobulinemia D Syndrome

Ozen, S; Kuemmerle-Deschner, JB; Cimaz, R; Livneh, A; Quartier, P; Kone-Paut, I; Zeft, A; ... Lachmann, HJ; + view all (2017) International Retrospective Chart Review of Treatment Patterns in Severe Familial Mediterranean Fever, Tumor Necrosis Factor Receptor–Associated Periodic Syndrome, and Mevalonate Kinase Deficiency/Hyperimmunoglobulinemia D Syndrome. Arthritis Care & Research , 69 (4) pp. 578-586. 10.1002/acr.23120. Green open access

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Abstract

OBJECTIVES: Periodic fever syndromes (PFS) are characterised by recurrent attacks of fever and localised inflammation. This study examined the diagnostic pathway and treatments at tertiary centres for FMF, TRAPS and MKD/HIDS. METHODS: PFS specialists at medical centres in the USA, European Union and Eastern Mediterranean participated in a retrospective chart review, providing de-identified data in an electronic case report form. Patients were treated between 2008-2012 with at least 1 year of follow-up, all had clinical and/or genetically proven disease, and were on/eligible for biologic treatment. RESULTS: 134 patients were analysed in total: FMF (n=49), TRAPS (n=47), and MKD/HIDS (n=38). Fever was commonly reported as severe across all indications. Other frequent severe symptoms were serositis for FMF patients and elevated acute-phase reactants and gastro-intestinal upset for TRAPS and MKD/HIDS. A long delay from disease onset to diagnosis was seen within TRAPS and MKD/HIDS (5.8 years and 7.1 years, respectively) with a 1.8 year delay in FMF. An equal proportion of TRAPS patients first received anti-IL-1 and anti-TNF biologics whereas IL-1 blockade was the main choice for MKD/HIDS and FMF patients. For TRAPS patients, treatment with anakinra versus anti-TNF treatments as first biologic resulted in significantly higher clinical and biochemical responses (p=0.03 and p<0.01, respectively). No significant differences in responses were observed between biological agents among other cohorts. CONCLUSIONS: Referral patterns and diagnostic delays highlight the need for greater awareness and improved diagnostics for PFSs. This real-world treatment assessment supports the need for further refinement of treatment practices.

Type: Article
Title: International Retrospective Chart Review of Treatment Patterns in Severe Familial Mediterranean Fever, Tumor Necrosis Factor Receptor–Associated Periodic Syndrome, and Mevalonate Kinase Deficiency/Hyperimmunoglobulinemia D Syndrome
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1002/acr.23120
Publisher version: http://dx.doi.org/10.1002/acr.23120
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation
URI: https://discovery.ucl.ac.uk/id/eprint/1528208
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