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Positive effect of erythrocyte-delivered dexamethasone in ataxia-telangiectasia.
Leuzzi, Vincenzo; Micheli, Roberto; D'Agnano, Daniela; Molinaro, Anna; Venturi, Tullia; Plebani, Alessandro; Soresina, Annarosa; Marini, Mirella; Ferremi Leali, Pierino; Quinti, Isabella; Pietrogrande, Maria C; Finocchi, Andrea; Fazzi, Elisa; Chessa, Luciana; Magnani, Mauro.
Afiliação
  • Leuzzi V; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Micheli R; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • D'Agnano D; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Molinaro A; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Venturi T; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Plebani A; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Soresina A; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Marini M; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Ferremi Leali P; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Quinti I; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Pietrogrande MC; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Finocchi A; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Fazzi E; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Chessa L; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
  • Magnani M; Department of Pediatrics, Child Neurology and Psychiatry (V.L., D.D., T.V.), Department of Molecular Medicine (I.Q.), and Department of Clinical and Molecular Medicine (L.C.), Sapienza University of Rome, Italy; Unit of Child Neurology and Psychiatry (R.M., E.F.), Department of Clinical and Experime
Neurol Neuroimmunol Neuroinflamm ; 2(3): e98, 2015 Jun.
Article em En | MEDLINE | ID: mdl-25884015
ABSTRACT

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.
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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Guideline Idioma: En Ano de publicação: 2015 Tipo de documento: Article
Texto completo
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PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Guideline Idioma: En Ano de publicação: 2015 Tipo de documento: Article