BACKGROUND: Action observation treatment (AOT) is an innovative therapeutic approach consisting in the observation of actions followed by their subsequent repetition. The standard version of AOT consists in the observation/imitation of a typically developed individual, which is proposed as model (TDM-AOT). AIM: This study aims to compare the effectiveness of AOT based on a pathological ameliorative model (PAM-AOT) versus TDM-AOT in improving upper limb ability in children with unilateral cerebral palsy (UCP). DESIGN: The study consists in a prospective randomized controlled, evaluator-blinded trial (RCT), with two active arms, designed to evaluate the effectiveness of AOT based on pathological model (PAM-AOT) as compared to a standard AOT based on TDM (TDM-AOT). SETTING: The 3-week AOT program was administered in a clinical setting. For some patients, the treatment was delivered at participant's home with the remote support of the physiotherapist (tele-rehabilitation). POPULATION: Twenty-six children with UCP (mean age 10.5 +/- 3.09 years; 14 females) participated in the study, with the experimental group observing a pathological model and the control group observing a typically developed model. METHODS: Motor assessments included unimanual and bimanual ability measures conducted at T0 (baseline, before the treatment), T1 (3 weeks after T0), T2 (8 to 12 weeks after treatment) and T3 (24 to 28 weeks after treatment); a subset of 16 patients also underwent fMRI motor assessment. Generalized Estimating Equations models were used for statistical analysis. RESULTS: Both groups showed significant improvement in bimanual function (GEE, Wald 106.16; p < 0.001) at T1 (p < 0.001), T2 (p < 0.001), and T3 (p < 0.001). Noteworthy, the experimental group showed greater improvement than the control group immediately after treatment (p < 0.013). Both groups exhibited similar improvement in unimanual ability (GEE, Wald 25.49; p < 0.001). The fMRI assessments revealed increased activation of ventral premotor cortex after treatment in the experimental compared with control group (GEE, Wald 6.26; p < 0.012). CONCLUSIONS: Overall, this study highlights the effectiveness of PAM-AOT in achieving short-term improvement of upper limb ability in children with UCP.

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