RT Journal Article
SR Electronic
T1 Positive effect of erythrocyte-delivered dexamethasone in ataxia-telangiectasia
JF Neurology - Neuroimmunology Neuroinflammation
JO Neurol Neuroimmunol Neuroinflamm
FD Lippincott Williams & Wilkins
SP e98
DO 10.1212/NXI.0000000000000098
VO 2
IS 3
A1 Leuzzi, Vincenzo
A1 Micheli, Roberto
A1 D'Agnano, Daniela
A1 Molinaro, Anna
A1 Venturi, Tullia
A1 Plebani, Alessandro
A1 Soresina, Annarosa
A1 Marini, Mirella
A1 Ferremi Leali, Pierino
A1 Quinti, Isabella
A1 Pietrogrande, Maria C.
A1 Finocchi, Andrea
A1 Fazzi, Elisa
A1 Chessa, Luciana
A1 Magnani, Mauro
YR 2015
UL http://nn.neurology.org/content/2/3/e98.abstract
AB 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 autologous 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 adaptive behavior (by Vineland Adaptive Behavior Scales) and clinical and laboratory tests revealing treatment- and steroid-dependent adverse effects, if present.Results: Patients in the extended study experienced a continuous neurologic improvement with respect to their pretreatment status, whereas controls showed a progressive neurologic deterioration (according to the natural history of the disease) after the discontinuation of the treatment. The delivery system we adopted proved to be safe and well-tolerated, and none of the side effects usually associated with the chronic administration of corticosteroids were observed during the entire trial.Conclusions: These promising preliminary results call for a large-scale controlled study on protracted treatment of patients with AT with dexamethasone-loaded erythrocytes.AT=ataxia-telangiectasia; ATM=ataxia telangiectasia mutated; DSP=dexamethasone phosphate; ICARS=International Cooperative Ataxia Rating Scale; VABS=Vineland Adaptive Behavior Scales