{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T20:10:20Z","timestamp":1787947820653,"version":"build-2784847793"},"reference-count":17,"publisher":"MDPI AG","issue":"18","license":[{"start":{"date-parts":[[2022,9,13]],"date-time":"2022-09-13T00:00:00Z","timestamp":1663027200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Roche\/Chugai"},{"name":"\u201cangewandte klinische Forschung\u201d program of the University of Tuebingen"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background: The purpose of this trial was to evaluate the effectiveness and safety of the IL-6 receptor antibody Tocilizumab (TCZ) in the treatment of Familial Mediterranean Fever (FMF). Methods: This was a randomized, double-blinded, placebo-controlled phase II trial in adult patients with active FMF and an inadequate response or intolerance to colchicine (crFMF). The physician\u2019s global assessment of disease activity (PGA), based on a five-point scale for six symptoms, was used as a clinical score, which had to be &gt;2 at screening, together with elevated c-reactive protein (CRP) or erythrocyte sedimentation rate (ESR) and serum amyloid A (SAA) levels, to be eligible for inclusion. Patients were randomized 1:1 to either receive monthly TCZ or a placebo over a period of 24 weeks. The primary endpoint was the number of patients achieving an adequate response to treatment at week 16, defined as a PGA of \u22642 and normalized ESR or CRP and normalized SAA. Results: We randomized 25 patients with a median age of 31 years. At week 16, an adequate treatment response was achieved by two patients in the TCZ and none of the patients in the placebo arm (p = 0.089). SAA levels normalized with TCZ, but not with the placebo (p = 0.015). Conclusion: In this first randomized, placebo-controlled study in patients with active crFMF, more patients in the TCZ arm experienced a response to treatment in comparison to those receiving the placebo. As the prevention of amyloidosis is a major treatment goal in FMF, the normalization of SAA in TCZ-treated patients is essential. These findings have to be confirmed in a larger trial.<\/jats:p>","DOI":"10.3390\/jcm11185360","type":"journal-article","created":{"date-parts":[[2022,9,13]],"date-time":"2022-09-13T22:37:28Z","timestamp":1663108648000},"page":"5360","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Tocilizumab for the Treatment of Familial Mediterranean Fever\u2014A Randomized, Double-Blind, Placebo-Controlled Phase II Study"],"prefix":"10.3390","volume":"11","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8385-6861","authenticated-orcid":false,"given":"Joerg C.","family":"Henes","sequence":"first","affiliation":[{"name":"Centre for Interdisciplinary Clinical Immunology, Rheumatology and Auto-Inflammatory Diseases and Department of Internal Medicine II (Oncology, Hematology, Immunology and Rheumatology), University Hospital Tuebingen, 72076 Tuebingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7259-1207","authenticated-orcid":false,"given":"Sebastian","family":"Saur","sequence":"additional","affiliation":[{"name":"Centre for Interdisciplinary Clinical Immunology, Rheumatology and Auto-Inflammatory Diseases and Department of Internal Medicine II (Oncology, Hematology, Immunology and Rheumatology), University Hospital Tuebingen, 72076 Tuebingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6164-3980","authenticated-orcid":false,"given":"David M.","family":"Kofler","sequence":"additional","affiliation":[{"name":"Division of Rheumatology and Clinical Immunology, Department I of Internal Medicine, University Hospital Cologne, 50441 Cologne, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9361-9213","authenticated-orcid":false,"given":"Claudia","family":"Kedor","sequence":"additional","affiliation":[{"name":"Institute of Medical Immunology, Charit\u00e9 Universitaetsmedizin Berlin, 10115 Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4802-3100","authenticated-orcid":false,"given":"Christoph","family":"Meisner","sequence":"additional","affiliation":[{"name":"Study Center, Robert Bosch Hospital, 70376 Stuttgart, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marion","family":"Schuett","sequence":"additional","affiliation":[{"name":"Institute for Clinical Epidemiology and Applied Biometry, University Hospital Tuebingen, University Tuebingen, 72076 Tuebingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0582-7790","authenticated-orcid":false,"given":"Martin","family":"Krusche","sequence":"additional","affiliation":[{"name":"Institute of Medical Immunology, Charit\u00e9 Universitaetsmedizin Berlin, 10115 Berlin, Germany"},{"name":"University Hospital Hamburg Eppendorf, 20095 Hamburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ina","family":"Koetter","sequence":"additional","affiliation":[{"name":"University Hospital Hamburg Eppendorf, 20095 Hamburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Theodoros","family":"Xenitidis","sequence":"additional","affiliation":[{"name":"Centre for Interdisciplinary Clinical Immunology, Rheumatology and Auto-Inflammatory Diseases and Department of Internal Medicine II (Oncology, Hematology, Immunology and Rheumatology), University Hospital Tuebingen, 72076 Tuebingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1681-491X","authenticated-orcid":false,"given":"Hendrik","family":"Schulze-Koops","sequence":"additional","affiliation":[{"name":"Division of Rheumatology and Clinical Immunology, Department of Internal Medicine IV, Ludwig-Maximilians-University of Munich, 80539 Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9872-5282","authenticated-orcid":false,"given":"Eugen","family":"Feist","sequence":"additional","affiliation":[{"name":"Helios Department of Rheumatology, Cooperation Partner of the Otto-von-Guericke University, 39245 Gommern, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2022,9,13]]},"reference":[{"key":"ref_1","first-page":"355","article-title":"Quality of life of patients with familial Mediterranean fever","volume":"15","author":"Buskila","year":"1997","journal-title":"Clin. 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