Randomized, double‐blind, placebo‐controlled study of safety and efficacy of miltefosine in antihistamine‐resistant chronic spontaneous urticaria. (29th August 2012)

• Record Type: Journal Article
• Authors: Magerl, M.; Rother, M.; Bieber, T.; Biedermann, T.; Brasch, J.; Dominicus, R.; Hunzelmann, N.; Jakob, T.; Mahler, V.; Popp, G.; Schäkel, K.; Schlingensiepen, R.; Schmitt, J.; Siebenhaar, F.; Simon, J.C.; Staubach, P.; Wedi, B.; Weidner, C.; Maurer, M.
• Is Part Of: Journal of the European Academy of Dermatology and Venereology. Volume 27:Number 3(2013:Mar.)
• Page Start: e363
• Page End: e369
• Publication Date: 2012-08-29
• Subjects: Dermatology -- Periodicals; Sexually transmitted diseases -- Periodicals; 616.5
• DOI: 10.1111/j.1468-3083.2012.04689.x
• Languages: English
• ISSNs: 0926-9959

Abstract

<abstract abstract-type="main" xml:lang="en"> <title>Abstract</title> <p> <bold>Background </bold> 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 <italic>in vivo</italic>.</p> <p> <bold>Objective </bold> To study the safety and efficacy of systemic miltefosine treatment in CSU patients resistant to standard‐dosed antihistamines.</p> <p> <bold>Methods </bold> 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 (<italic>n </italic>= 47) or placebo (<italic>n </italic>= 26). Disease activity was assessed using the urticaria activity score. Safety and tolerability of miltefosine were also assessed.</p> <p> <bold>Results </bold> 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; <italic>P</italic> = 0.05). Also, the number of weals, but not the intensity of pruritus, was significantly reduced in miltefosine‐treated patients vs. placebo‐treated patients (<italic>P </italic>= 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.</p> <p> <bold>Conclusions </bold> 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.</p> </abstract>