Evidence Based Recommendations for the Management of Cryopyrin Associated Periodic Syndromes (CAPS)

Nienke ter Haar¹, Marlen Oswald², Luca Cantarini³, Marco Gattorno⁴, Michael Hofer⁵, Isabelle Kone-Paut⁶, Jordi Anton Lopez⁷, Karyl Barron⁸, Paul Brogan⁹, Joost Frenkel¹⁰, Caroline Galeotti¹¹, Gilles Grateau¹², Veronique Hentgen¹³, Tilmann Kallinich¹⁴, Helen Lachmann¹⁵, Huri Ozdogan¹⁶, Seza Ozen¹⁷, Ricardo Russo¹⁸, Anna Simon¹⁹, Yosef Uziel²⁰, Carine Wouters²¹, Brian Feldman²², Bas Vastert¹⁰, Nico Wulffraat²³, Susanne Benseler²⁴ and Jasmin Kuemmerle-Deschner²⁵, ¹Laboratory for Translational Immunology, University Medical Center Utrecht, Utrecht, Netherlands, ²University Hospital Tuebingen, Tuebingen, Germany, ³University of Siena, Siena, Italy, ⁴Pediatric Rheumatology, Instituto Giannina Gaslini, Genoa, Italy, ⁵Centre Multisite Romand de Rhumatologie Pediatrique, Lausanne, Switzerland, ⁶Department of Pediatric Rheumatology, Reference Centre for Autoinflammatory Disorders CEREMAI, Bicêtre Hospital, University of Paris SUD, Paris, France, ⁷pediatric Rheumatology, University Childrenxs Hospital, Barcelona, Spain, ⁸NIH, Bethesda, MD, ⁹Rheumatology Unit, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom, ¹⁰University Medical Center Utrecht, Utrecht, Netherlands, ¹¹Bicêtre Hospital, University of Paris SUD, Paris, France, ¹²Service De Médecine Interne, Hopital Tenon, Paris, France, ¹³Versailles Hospital, Le Chesnay Cedex, France, ¹⁴Charite, University Medicine Berlin, Berlin, Germany, ¹⁵UK National Amyloidosis Centre, University College London Medical School, London, United Kingdom, ¹⁶Division of Rheumatology, Department of Internal Medicine, Cerrahpasa Medical Faculty, University of Istanbul, Istanbul, Turkey, ¹⁷Hacettepe University, Ankara, Turkey, ¹⁸Immunology & Rheumatology, Hospital De Pediatria, Buenos Aires, Argentina, ¹⁹Radboudumc, Nijmegen, Netherlands, ²⁰Tel-Aviv University, Sackler School of Medicine, Tel-Aviv, Israel, ²¹University of Leuven, Laboratory of Pediatric Immunology, University Hospital Leuven, Leuven, Belgium, ²²Rheumatology, The Hospital for Sick Children, Toronto, ON, Canada, ²³Paediatric Immunology and Rheumatology, University Medical Center Utrecht, Utrecht, Netherlands, ²⁴The Hospital for Sick Children, Toronto, ON, Canada, ²⁵Pediatrics, University Hospital Tuebingen, Tuebingen, Germany

Meeting: 2014 ACR/ARHP Annual Meeting

Keywords: evidence-based practice and treatment, Muckle-Wells syndrome

Session Information

Title: Pediatric Rheumatology - Clinical and Therapeutic Aspects: Systemic Juvenile Idiopathic Arthritis, Spondyloarthropathy and Miscellaneous Pediatric Rheumatic Diseases

Session Type: Abstract Submissions (ACR)

Background/Purpose Cryopyrin-associated periodic syndromes (CAPS) is a group of rare monogenetic autoinflammatory disorders. Evidence-based guidelines are lacking and management is mostly based on physician’s experience. Consequently, treatment regimens differ throughout Europe. In 2012, a European initiative called SHARE (Single Hub and Access point for pediatric Rheumatology in Europe)was launched to optimize and disseminate diagnostic and management regimens in Europe for children and young adults with rheumatic diseases. One of the aims of SHARE was to provide evidence based recommendations for management (treatment and monitoring) of CAPS.

Methods Evidence based recommendations were developed using the European League Against Rheumatism (EULAR) standard operating procedure. An expert committee was instituted, consisting of pediatric and adult rheumatologists. The expert committee defined search terms for the systematic literature review. Two independent experts scored articles for validity and level of evidence. Recommendations derived from the literature were evaluated by an online survey. Those with less than 80% agreement on the online survey or with relevant comments of the experts were reformulated. Subsequently, all recommendations were discussed at a consensus meeting using Nominal Group Technique. Recommendations were accepted if more than 80% agreement was reached.

Results The literature search yielded 1698 articles, of which 25 papers on treatment were considered relevant and therefore scored for validity and level of evidence. Seventeen were scored valid and used in the formulation of the recommendations. Fifteen recommendations were suggested in the online survey and discussed during the consensus meeting. Six general recommendations on management, five for monitoring and four for treatment were accepted with more than 80% agreement. Topics covered are the following:

General recommendations: use of the multidisciplinary team, treatment goals, adjunctive therapies, psychosocial support and vaccinations
Monitoring: monitoring frequency, minimal assessments in all CAPS patients and monitoring of severe phenotypes
Treatment: IL-1 blockade, NSAIDs and/or glucocorticoids during attacks and DMARDS/biologicals other than IL-1 blockade

Conclusion The SHARE initiative provides recommendations for the management of CAPS and thereby facilitates improvement and uniformity of care throughout Europe.

Disclosure:

N. ter Haar,
None;

M. Oswald,
None;

L. Cantarini,

Novartis Pharma AG, SOBI,

M. Gattorno,
None;

M. Hofer,
None;

I. Kone-Paut,
None;

J. A. Lopez,

Abbvie, Novartis, Pfizer,

Novartis Pharmaceutical Corporation,

Abbvie, Novartis, Pfizer, Roche, SOBI,

K. Barron,
None;

P. Brogan,

Novartis, Roche,

Novartis Pharmaceutical Corporation,

J. Frenkel,

European Union ERANET,

Novartis Pharmaceutical Corporation,

SOBI,

C. Galeotti,

Novartis Pharmaceutical Corporation,

G. Grateau,
None;

V. Hentgen,

Novartis Pharmaceutical Corporation,

Novartis, Pfizer, Roche,

T. Kallinich,

Novartis, SOBI,

Novartis Pharmaceutical Corporation,

H. Lachmann,
None;

H. Ozdogan,
None;

S. Ozen,

Novartis Pharma AG,

Biovitrium-SOBI, Novartis Pharma AG,

R. Russo,
None;

A. Simon,

Servier,

Novartis, SOBI, Xoma,

Y. Uziel, Novartis Pharmaceutical Corporation, 2, Novartis Pharmaceutical Corporation, 5, Abbvie, Neopharm, Novartis, Roche, 8; C. Wouters,
None;

B. Feldman,
None;

B. Vastert,

Novartis Pharmaceutical Corporation,

N. Wulffraat,

Abbvie, GSK, Roche,

Genzyme, Novartis, Pfizer, Roche,

S. Benseler,
None;

J. Kuemmerle-Deschner,

Novartis Pharmaceutical Corporation,

SOBI,

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