Objective: To investigate the preliminary safety and efficacy of apremilast, an oral phosphodiesterase 4 inhibitor, for atopic dermatitis. Design: This investigator-initiated, open-label pilot study evaluated 2 doses of apremilast in patients with atopic dermatitis. Differential gene analysis was performed from peripheral whole blood using data before and after treatment. Setting: University-based dermatology clinical research unit. Patients: Sixteen adult patients with atopic dermatitis. Intervention: A specific phosphodiesterase 4 inhibitor, apremilast. Main Outcome Measures: The primary outcome was incidence of adverse events. Secondary outcomes included the differences in pruritus, Dermatology Life Quality Index (DLQI), and Eczema Area and Severity Index (EASI) scores between the baseline visit and end-of-study visit for each cohort. Results: The group receiving apremilast, 20 mg twice daily, displayed a significant reduction from baseline of pruritus (P =.02) and the DLQI (P =.003) at 3 months. The group receiving apremilast, 30 mg twice daily, displayed a significant reduction of the EASI (P =.008) and the DLQI (P =.01) at 3 months. At 6 months, there was a significant reduction of the EASI (P =.002), the visual analog scale (P =.03), and the DLQI (P =.03). Gene ontologic analyses comparing baseline with samples during treatment revealed alterations in immune response pathways, especially those related to cyclic adenosine monophosphate-mediated signaling. Conclusions: These results support further development of apremilast for treatment of atopic dermatitis. Larger randomized controlled studies are needed to more adequately evaluate both safety and efficacy. Limitations include the small sample size and absence of a control. Trial Registration: clinicaltrials.gov Identifier: NCT01393158.
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