Universität zu Köln

Henes, Joerg C., Saur, Sebastian ORCID: 0000-0001-7259-1207, Kofler, David M., Kedor, Claudia ORCID: 0000-0001-9361-9213, Meisner, Christoph ORCID: 0000-0002-4802-3100, Schuett, Marion, Krusche, Martin ORCID: 0000-0002-0582-7790, Koetter, Ina, Xenitidis, Theodoros, Schulze-Koops, Hendrik ORCID: 0000-0002-1681-491X and Feist, Eugen ORCID: 0000-0002-9872-5282 (2022). Tocilizumab for the Treatment of Familial Mediterranean Fever-A Randomized, Double-Blind, Placebo-Controlled Phase II Study. J. Clin. Med., 11 (18). BASEL: MDPI. ISSN 2077-0383

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Abstract

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's 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 >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 <= 2 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.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Henes, Joerg C. UNSPECIFIED UNSPECIFIED UNSPECIFIED Saur, Sebastian UNSPECIFIED orcid.org/0000-0001-7259-1207 UNSPECIFIED Kofler, David M. UNSPECIFIED UNSPECIFIED UNSPECIFIED Kedor, Claudia UNSPECIFIED orcid.org/0000-0001-9361-9213 UNSPECIFIED Meisner, Christoph UNSPECIFIED orcid.org/0000-0002-4802-3100 UNSPECIFIED Schuett, Marion UNSPECIFIED UNSPECIFIED UNSPECIFIED Krusche, Martin UNSPECIFIED orcid.org/0000-0002-0582-7790 UNSPECIFIED Koetter, Ina UNSPECIFIED UNSPECIFIED UNSPECIFIED Xenitidis, Theodoros UNSPECIFIED UNSPECIFIED UNSPECIFIED Schulze-Koops, Hendrik UNSPECIFIED orcid.org/0000-0002-1681-491X UNSPECIFIED Feist, Eugen UNSPECIFIED orcid.org/0000-0002-9872-5282 UNSPECIFIED
URN:	urn:nbn:de:hbz:38-691299
DOI:	10.3390/jcm11185360
Journal or Publication Title:	J. Clin. Med.
Volume:	11
Number:	18
Date:	2022
Publisher:	MDPI
Place of Publication:	BASEL
ISSN:	2077-0383
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language COLCHICINE-RESISTANT; ANAKINRA Multiple languages Medicine, General & Internal Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/69129