M Magerl1, M Rother, T Bieber, T Biedermann, J Brasch, R Dominicus, N Hunzelmann, T Jakob, V Mahler, G Popp, K Schäkel, R Schlingensiepen, J Schmitt, F Siebenhaar, J C Simon, P Staubach, B Wedi, C Weidner, M Maurer. 1. Department of Dermatology and Allergy; Allergie-Centrum-Charité; Charité- Universitätsmedizin Berlin, Berlin, Germany Department Clinical Operations, X-pert Med GmbH, Gräfelfing, Germany Department of Dermatology and Allergy, University of Bonn, Bonn, Germany Department of Dermatology, Eberhard Karls University Tübingen, Tübingen, Germany Department of Dermatology and Allergy, UK-SH, Campus Kiel, Kiel, Germany Praxisklinik und Gemeinschaftspraxis, Dülmen, Germany Department of Dermatology and Allergy, Universitätsklinikum Köln, Cologne, Germany Department of Dermatology and Allergy, Universitäts-Hautklinik Freiburg, Freiburg, Germany Department of Dermatology, University Hospital Erlangen, Erlangen, Germany Licca Clinical Research Institute, Augsburg, Germany Department of Dermatology, Universitätsklinikum Heidelberg, Heidelberg, Germany Jado Technologies GmbH, Dresden, Germany Department of Occupational and Social Medicine, Medizinische Fakultät Carl Gustav Carus, TU Dresden, Germany Department of Dermatology, Venerology and Allergy, Universitätklinikum Leipzig, Leipzig, Germany Department of Dermatology and Allergy, Universitätsmedizin Mainz, Mainz, Germany Department of Dermatology and Allergy, Medizinische Hochschule Hannover, Hannover, Germany X-pert Med GmbH, Jena, Germany.
Abstract
BACKGROUND:Chronic spontaneous urticaria (CSU), a mast cell-driven condition, is debilitating, common, and hard to treat. Miltefosine, a lipid raft modulator, can inhibit mast cell responses in vivo. OBJECTIVE: To study the safety and efficacy of systemic miltefosine treatment in CSU patients resistant to standard-dosed antihistamines. METHODS: In this investigator-initiated multicentre, randomized, double-blind, placebo-controlled study, CSU patients were treated for 4 weeks with daily doses of up to 150-mg miltefosine (n = 47) or placebo (n = 26). Disease activity was assessed using the urticaria activity score. Safety and tolerability of miltefosine were also assessed. RESULTS: After 4 weeks of treatment, Urticaria Activity Score (UAS7) levels were substantially more reduced in miltefosine-treated patients (-6.3 vs. -3.5 in placebo-treated patients; P = 0.05). Also, the number of weals, but not the intensity of pruritus, was significantly reduced in miltefosine-treated patients vs. placebo-treated patients (P = 0.02). In general, adverse events were frequent in both groups (miltefosine: 88%, placebo: 65% of patients) but mostly mild to moderate in severity. We did not observe any serious adverse events. CONCLUSIONS: The results of this study indicate that miltefosine is an effective and safe treatment option for CSU patients who do not respond to standard-dosed antihistamines.
RCT Entities:
BACKGROUND: Chronic spontaneous urticaria (CSU), a mast cell-driven condition, is debilitating, common, and hard to treat. Miltefosine, a lipid raft modulator, can inhibit mast cell responses in vivo. OBJECTIVE: To study the safety and efficacy of systemic miltefosine treatment in CSU patients resistant to standard-dosed antihistamines. METHODS: In this investigator-initiated multicentre, randomized, double-blind, placebo-controlled study, CSU patients were treated for 4 weeks with daily doses of up to 150-mg miltefosine (n = 47) or placebo (n = 26). Disease activity was assessed using the urticaria activity score. Safety and tolerability of miltefosine were also assessed. RESULTS: After 4 weeks of treatment, Urticaria Activity Score (UAS7) levels were substantially more reduced in miltefosine-treated patients (-6.3 vs. -3.5 in placebo-treated patients; P = 0.05). Also, the number of weals, but not the intensity of pruritus, was significantly reduced in miltefosine-treated patients vs. placebo-treated patients (P = 0.02). In general, adverse events were frequent in both groups (miltefosine: 88%, placebo: 65% of patients) but mostly mild to moderate in severity. We did not observe any serious adverse events. CONCLUSIONS: The results of this study indicate that miltefosine is an effective and safe treatment option for CSU patients who do not respond to standard-dosed antihistamines.
Authors: Antoinette I M van Laarhoven; Ineke M van der Sman-Mauriks; A Rogier T Donders; Mathilde C Pronk; Peter C M van de Kerkhof; Andrea W M Evers Journal: J Invest Dermatol Date: 2014-12-01 Impact factor: 8.551
Authors: Sara Botschuijver; Sophie A van Diest; Isabelle A M van Thiel; Rafael S Saia; Anne S Strik; Zhumei Yu; Daniele Maria-Ferreira; Olaf Welting; Daniel Keszthelyi; Gary Jennings; Sigrid E M Heinsbroek; Ronald P Oude Elferink; Frank H J Schuren; Wouter J de Jonge; René M van den Wijngaard Journal: Sci Rep Date: 2019-08-29 Impact factor: 4.379