International retrospective chart review of treatment patterns in severe FMF, TRAPS and MKD/HIDS

Seza Ozen; Jasmin B Kuemmerle-Deschner; Rolando Cimaz; Avi Livneh; Pierre Quartier; Isabelle Kone-Paut; Andrew Zeft; Steve Spalding; Ahmet Gul; Veronique Hentgen; Sinisa Savic; Ivan Foeldvari; Joost Frenkel; Luca Cantarini; Dony Patel; Jeffrey Weiss; Nina Marinsek; Ravi Degun; Kathleen G Lomax; Helen J Lachmann

doi:10.1002/acr.23120

International retrospective chart review of treatment patterns in severe FMF, TRAPS and MKD/HIDS

Seza Ozen, Jasmin B Kuemmerle-Deschner, Rolando Cimaz, Avi Livneh, Pierre Quartier, Isabelle Kone-Paut, Andrew Zeft, Steve Spalding, Ahmet Gul, Veronique Hentgen, Sinisa Savic, Ivan Foeldvari, Joost Frenkel, Luca Cantarini, Dony Patel, Jeffrey Weiss, Nina Marinsek, Ravi Degun, Kathleen G Lomax, Helen J Lachmann

Research output: Contribution to journal › Article › Academic › peer-review

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. This article is protected by copyright. All rights reserved.

Original language	English
Pages (from-to)	578-586
Number of pages	8
Journal	Arthritis care and research
DOIs	https://doi.org/10.1002/acr.23120
Publication status	Published - Apr 2017

Access to Document

10.1002/acr.23120

Cite this

Ozen, S., Kuemmerle-Deschner, J. B., Cimaz, R., Livneh, A., Quartier, P., Kone-Paut, I., Zeft, A., Spalding, S., Gul, A., Hentgen, V., Savic, S., Foeldvari, I., Frenkel, J., Cantarini, L., Patel, D., Weiss, J., Marinsek, N., Degun, R., Lomax, K. G., & Lachmann, H. J. (2017). International retrospective chart review of treatment patterns in severe FMF, TRAPS and MKD/HIDS. Arthritis care and research, 578-586. https://doi.org/10.1002/acr.23120

@article{36566ddd85db48c99b5b77579747a9fe,

title = "International retrospective chart review of treatment patterns in severe FMF, TRAPS and MKD/HIDS",

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. This article is protected by copyright. All rights reserved.",

author = "Seza Ozen and Kuemmerle-Deschner, {Jasmin B} and Rolando Cimaz and Avi Livneh and Pierre Quartier and Isabelle Kone-Paut and Andrew Zeft and Steve Spalding and Ahmet Gul and Veronique Hentgen and Sinisa Savic and Ivan Foeldvari and Joost Frenkel and Luca Cantarini and Dony Patel and Jeffrey Weiss and Nina Marinsek and Ravi Degun and Lomax, {Kathleen G} and Lachmann, {Helen J}",

note = "{\textcopyright} 2016, American College of Rheumatology.",

year = "2017",

month = apr,

doi = "10.1002/acr.23120",

language = "English",

pages = "578--586",

journal = "Arthritis care and research",

issn = "2151-464X",

publisher = "John Wiley & Sons Inc.",

}

Ozen, S, Kuemmerle-Deschner, JB, Cimaz, R, Livneh, A, Quartier, P, Kone-Paut, I, Zeft, A, Spalding, S, Gul, A, Hentgen, V, Savic, S, Foeldvari, I, Frenkel, J, Cantarini, L, Patel, D, Weiss, J, Marinsek, N, Degun, R, Lomax, KG & Lachmann, HJ 2017, 'International retrospective chart review of treatment patterns in severe FMF, TRAPS and MKD/HIDS', Arthritis care and research, pp. 578-586. https://doi.org/10.1002/acr.23120

TY - JOUR

T1 - International retrospective chart review of treatment patterns in severe FMF, TRAPS and MKD/HIDS

AU - Ozen, Seza

AU - Kuemmerle-Deschner, Jasmin B

AU - Cimaz, Rolando

AU - Livneh, Avi

AU - Quartier, Pierre

AU - Kone-Paut, Isabelle

AU - Zeft, Andrew

AU - Spalding, Steve

AU - Gul, Ahmet

AU - Hentgen, Veronique

AU - Savic, Sinisa

AU - Foeldvari, Ivan

AU - Frenkel, Joost

AU - Cantarini, Luca

AU - Patel, Dony

AU - Weiss, Jeffrey

AU - Marinsek, Nina

AU - Degun, Ravi

AU - Lomax, Kathleen G

AU - Lachmann, Helen J

PY - 2017/4

Y1 - 2017/4

N2 - 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. This article is protected by copyright. All rights reserved.

AB - 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. This article is protected by copyright. All rights reserved.

U2 - 10.1002/acr.23120

DO - 10.1002/acr.23120

M3 - Article

C2 - 27723279

SN - 2151-464X

SP - 578

EP - 586

JO - Arthritis care and research

JF - Arthritis care and research

ER -

International retrospective chart review of treatment patterns in severe FMF, TRAPS and MKD/HIDS

Abstract

Access to Document

Fingerprint

Cite this