An open-label, add-on trial of cetirizine for neuromyelitis optica (P2.334)

Objective: To preliminarily study the efficacy and safety of cetirizine for NMO Background: NMO is a severe inflammatory disease affecting the CNS with the potential to cause serious neurological disability. Granulocyte infiltration with subsequent neutrophil and eosinophil degranulation are critical to the ultimate destruction of astrocytes, demyelination, and neuronal/axonal loss. This trial was conducted to examine the efficacy and tolerability of cetirizine, an eosinophil-stabilizer, as an add-on to currently-available therapies for NMO. Design/Methods: Eligible patients met Wingerchuk 2006 criteria or had a single episode with positive NMO IgG, and were stable on DMT for 3 months. Subjects were followed clinically and with serum/CSF analysis for cytokines/chemokines associated with eosinophil activity for 1 year. Planned endpoints included relapse rates, tolerability with particular attention to drowsiness, and changes in immunological parameters. Results: 16 subjects were enrolled between April 2014 and February 2015. 15 were female, 7 were black, with median age 36.5. 13 were NMO IgG positive. 8 were on rituximab, 7 on mycophenolate, and 1 on azathioprine. 7 had previously experienced a total of 9 relapses while on the current treatment. There was 1 mild confirmed relapse during the study. The pre- and post-study ARR differences were statistically significantly different from zero (p=0.047). Median EDSS was 3.0 at baseline and at study end. Mean Epworth Sleepiness Scale score was 6.5 ± 5.33 at baseline and 6.9 ± 4.50 at conclusion (p=0.74). There were no significant drug-related adverse events. Analysis of immunological profiling is in process. Conclusions: Eosinophil-stabilizing properties and favorable safety profile make cetirizine an attractive add-on therapy for NMO. In our pilot study the drug was very well-tolerated with no significant increase in sleepiness. The very low pre-study relapse rates in our patient population preclude definitive conclusions related to cetirizine’s effect on relapse rates and severity, though results are interesting and warrant further study. Study Supported by: The Guthy Jackson Charitable Foundation Disclosure: Dr. Katz Sand has nothing to disclose. Dr. Telford has nothing to disclose. Dr. Fabian has nothing to disclose. Dr. Kraus has nothing to disclose. Dr. Farrell has nothing to disclose. Dr. Phelps has nothing to disclose. Dr. Chehade has nothing to disclose. Dr. Masilamani has nothing to disclose. Dr. Riffle has nothing to disclose. Dr. Moran has nothing to disclose. Dr. Cook has nothing to disclose. Dr. Lubin has received personal compensation for activities with Abbott, Acorda, Actelion, Allozyne, Avanir, Bayer HealthCare, Biogen Idec, Celgene, EMD Serono, Genmab, Johnson u0026 Johnson, Medicinova, MorphoSys, Novartis, Pfizer, Questcor, Roche, Sanofi and Teva Neuros for consulting or serving on an advisory board.