PurposeThe purpose of this study is to explore the association of P300 components with clinical characteristics and efficacy of pharmacotherapy in alcohol use disorder (AUD).MethodsOne hundred fifty-one AUD patients and 96 healthy controls were recruited and evaluated for the symptoms of depression, anxiety, sleep, and cognitive function by the Alcohol Use Disorders Identification Test (AUDIT), the 9-item Patient Health Questionnaire (PHQ-9), the 7-item Generalized Anxiety Disorder scale (GAD-7), the Pittsburgh Sleep Quality Index (PSQI), Digit Symbol Substitution test (DSST), and event-related potential P300, which is one of the averaged scalp electroencephalography responses time-locked to specific events. Among the AUD group, 101 patients finished an 8-week pharmacotherapy and were evaluated for the above data at post-intervention.Results1. At baseline, AUD patients had higher scores of AUDIT, PHQ-9, GAD-7, PSQI, and P300 latency at Cz, Pz, and Fz and lower DSST score and smaller P300 amplitudes at Fz, Cz, and Pz compared with controls. P300 components correlated significantly with alcohol dose and score of AUDIT, PHQ-9, GAD-7, PSQI, and DSST. 2. After 8 weeks' treatment, there were significant changes for the P300 components; alcohol dose; and score of AUDIT, PHQ-9, GAD-7, PSQI, and DSST. Variables at baseline, including P300 amplitudes at Fz, Cz, and Pz; latency of Fz and Pz; alcohol dose; and scores of PHQ-9, GAD-7, PSQI, and DSST, were significantly associated with changes of reduction rate of AUDIT scores. However, P300 amplitudes at Fz, Cz, and Pz in AUD patients after 8-week treatment were still significantly shorter than healthy controls (HCs), and P300 latencies at Fz, Cz, and Pz were significantly longer than HCs. 3. When validated area under the receiver operating characteristic curve (AUC) was over 0.80, the baseline variables including amplitudes at Cz and Pz, alcohol dose, and scores of PSQI could predict the changes of reduction rate of AUDIT score.ConclusionP300 amplitudes and latencies at Fz, Cz, and Pz could be used as biological markers for evaluating the clinical characters and severity of AUD. P300 amplitudes at Cz and Pz, sleep condition, and cognitive function at baseline could predict the efficacy of pharmacotherapy for AUD patients.