Real-life effectiveness of canakinumab in cryopyrin-associated periodic syndrome

被引：49

作者：

Kuemmerle-Deschner, Jasmin B. ^{[1
]}

Hofer, Ferdinand ^{[1
]}

Endres, Theresa ^{[1
]}

Kortus-Goetze, Birgit ^{[2
]}

Blank, Norbert ^{[3
]}

Weissbarth-Riedel, Elisabeth ^{[4
]}

Schuetz, Catharina ^{[5
]}

Kallinich, Tilmann ^{[6
]}

Krause, Karoline ^{[7
]}

Rietschel, Christoph ^{[8
]}

Horneff, Gerd ^{[9
]}

Benseler, Susanne M. ^{[1
,10
]}

机构：

[1] Univ Childrens Hosp Tuebingen, Dept Pediat, Div Pediat Rheumatol, Hoppe Seyler Str 1, D-72076 Tubingen, Germany

[2] Univ Marburg, Div Nephrol, D-35032 Marburg, Germany

[3] Univ Heidelberg Hosp, Haematol Oncol & Rheumatol, Heidelberg, Germany

[4] Univ Hosp Eppendorf, Pediat Rheumatol Clin, Hamburg, Germany

[5] Univ Hosp Ulm, Dept Pediat & Adolescents Med, Ulm, Germany

[6] Charite Campus Virchow, Rheumatol Sect, Childrens Hosp, Berlin, Germany

[7] Charite Campus Mitte, Dept Dermatol, Allergie Ctr Charite, Berlin, Germany

[8] Clementine Childrens Hosp, Dept Rheumatol, Frankfurt, Germany

[9] Asklepios Klin Sankt Augustin, Ctr Pediat Rheumatol Sankt Augustin, St Augustin, Germany

[10] Univ Calgary, Rheumatol, Alberta Childrens Hosp, Calgary, AB T2N 1N4, Canada

来源：

RHEUMATOLOGY | 2016年 / 55卷 / 04期

关键词：

canakinumab; cryopyrin-associated periodic syndrome; CAPS; treat-to-target; effectiveness; outcome; Muckle-Wells syndrome; FCAS; MWS; NOMID; CINCA; MONOCLONAL-ANTIBODY ACZ885; MUCKLE-WELLS-SYNDROME; OPEN-LABEL; EFFICACY; INTERLEUKIN-1; ANAKINRA; SAFETY; INFLAMMATION; MULTICENTER; ARTHRITIS;

D O I：

10.1093/rheumatology/kev416

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Methods. A multi-centre study of consecutive children and adults with CAPS treated with canakinumab was performed. Demographics, CAPS phenotype and disease activity, inflammatory markers and canakinumab treatment strategy were recorded. Treatment response was assessed using CAPS disease activity scores, CRP and/or serum amyloid A levels. Comparisons between age groups, CAPS phenotypes and centres were conducted. Results. A total of 68 CAPS patients at nine centres were included. All CAPS phenotypes were represented. Thirty-seven (54%) patients were females, the median age was 25 years and 27 (40%) were children, and the median follow-up was 28 months. Overall, complete response (CR) was seen in 72% of CAPS patients, significantly less often in severe (14%) than in mild CAPS phenotypes (79%). Only 53% attained CR on standard dose canakinumab. Dose increase was more commonly required in children (56%) than in adults (22%). Centres with a treat-to-target approach had significantly higher CR rates (94 vs 50%). Conclusion. Real-life effectiveness of canakinumab in CAPS was significantly lower than in controlled trials. Treat-to-target strategies may improve the outcome of children and adults living with CAPS.

引用

页码：689 / 696

页数：8

共 21 条

[1] The human anti-IL-1β monoclonal antibody ACZ885 is effective in joint inflammation models in mice and in a proof-of-concept study in patients with rheumatoid arthritis [J].

Alten, Rieke ;

Gram, Hermann ;

Joosten, Leo A. ;

van den Berg, Wim B. ;

Sieper, Joachim ;

Wassenberg, Siegfrid ;

Burmester, Gerd ;

van Riel, Piet ;

Diaz-Lorente, Maria ;

Bruin, Gerardus Jm ;

Woodworth, Thasia G. ;

Rordorf, Christiane ;

Batard, Yannik ;

Wright, Andrew M. ;

Jung, Thomas .

ARTHRITIS RESEARCH & THERAPY, 2008, 10 (03)

[2] The schedule of administration of canakinumab in cryopyrin associated periodic syndrome is driven by the phenotype severity rather than the age [J].

Caorsi, Roberta ;

Lepore, Loredana ;

Zulian, Francesco ;

Alessio, Maria ;

Stabile, Achille ;

Insalaco, Antonella ;

Finetti, Martina ;

Battagliese, Antonella ;

Martini, Giorgia ;

Bibalo, Chiara ;

Martini, Alberto ;

Gattorno, Marco .

ARTHRITIS RESEARCH & THERAPY, 2013, 15 (01)

[3] Treating inflammation by blocking interleukin-1 in humans [J].

Dinarello, Charles A. ;

van der Meer, Jos W. M. .

SEMINARS IN IMMUNOLOGY, 2013, 25 (06) :469-484

[4] Current Status of Understanding the Pathogenesis and Management of Patients With NOMID/CINCA [J].

Goldbach-Mansky, Raphaela .

CURRENT RHEUMATOLOGY REPORTS, 2011, 13 (02) :123-131

[5] Spectrum of clinical features in Muckle-Wells syndrome and response to anakinra [J].

Hawkins, PN ;

Lachmann, HJ ;

Aganna, E ;

McDermott, MF .

ARTHRITIS AND RHEUMATISM, 2004, 50 (02) :607-612

[6] Efficacy and safety of rilonacept (Interleukin-1 Trap) in patients with cryopyrin-associated periodic syndromes [J].

Hoffman, Hal M. ;

Throne, Martin L. ;

Amar, N. J. ;

Sebai, Mohamed ;

Kivitz, Alan J. ;

Kavanaugh, Arthur ;

Weinstein, Steven P. ;

Belomestnov, Pavel ;

Yancopoulos, George D. ;

Stahl, Neil ;

Mellis, Scott J. .

ARTHRITIS AND RHEUMATISM, 2008, 58 (08) :2443-2452

[7] Genetic and Molecular Basis of Inflammasome-mediated Disease [J].

Hoffman, Hal M. ;

Brydges, Susannah D. .

JOURNAL OF BIOLOGICAL CHEMISTRY, 2011, 286 (13) :10889-10896

[8] Mutation of a new gene encoding a putative pyrin-like protein causes familial cold autoinflammatory syndrome and Muckle-Wells syndrome [J].

Hoffman, HM ;

Mueller, JL ;

Broide, DH ;

Wanderer, AA ;

Kolodner, RD .

NATURE GENETICS, 2001, 29 (03) :301-305

[9] Two-year results from an open-label, multicentre, phase III study evaluating the safety and efficacy of canakinumab in patients with cryopyrin-associated periodic syndrome across different severity phenotypes [J].

Kuemmerle-Deschner, Jasmin B. ;

Hachulla, E. ;

Cartwright, R. ;

Hawkins, P. N. ;

Tran, T. A. ;

Bader-Meunier, B. ;

Hoyer, J. ;

Gattorno, M. ;

Gul, A. ;

Smith, J. ;

Leslie, K. S. ;

Jimenez, S. ;

Morell-Dubois, S. ;

Davis, N. ;

Patel, N. ;

Widmer, A. ;

Preiss, R. ;

Lachmann, H. J. .

ANNALS OF THE RHEUMATIC DISEASES, 2011, 70 (12) :2095-2102

[10]

Lachmann H, 2008, CLIN EXP RHEUMATOL, V26, P181

← 1 2 3 →