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By Vincenzo Leuzzi Md, Roberto Micheli Md, Daniela D’agnano Md, Anna Molinaro Pt, Tullia Venturi Pt, Alessandro Plebani Md, Annarosa Soresina Md, Mirella Marini Md, Pierino Ferremi Leali, Isabella Quinti Md, Maria C. Pietrogrande, Elisa Fazzi Md and Mauro Magnani Md


Positive effect of erythrocyte-delivered dexamethasone in ataxia-telangiectasia Objective: Ataxia-telangiectasia (AT) is a rare, devastating neurodegenerative disease presenting with early-onset ataxia, oculocutaneous telangiectasia, immunodeficiency, radiosensitivity, and proneness to cancer. In a previous phase 2 study, we showed that 6 monthly infusions of autol-ogous erythrocytes loaded with dexamethasone (EryDex; EryDel, Urbino, Italy) were effective in improving neurologic impairment in young patients with AT. The present article reports the results of the extension of this study for an additional 24-month period. Methods: After the end of the first trial, 4 patients continued to be treated with monthly EryDex infusions for an additional 24 months, and their clinical outcome was compared with that of 7 age-matched patients who stopped the treatment after the first 6 infusions. The protocol included serial assessment of ataxia (by International Cooperative Ataxia Rating Scale) and adap-tive behavior (by Vineland Adaptive Behavior Scales) and clinical and laboratory tests revealing treatment- and steroid-dependent adverse effects, if present

Year: 2016
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