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.
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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 > 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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