Positive effect of erythrocyte-delivered dexamethasone in ataxia-telangiectasia

Neurology: Neuroimmunology and NeuroInflammation

Volumn 2, Issue 3, 2015, Pages e98-

  Leuzzi, Vincenzo ^a   Micheli, Roberto ^d   D'Agnano, Daniela ^a   Molinaro, Anna ^e   Venturi, Tullia ^a   Plebani, Alessandro ^d,e   Soresina, Annarosa ^d,e   Marini, Mirella ^d,e   Leali, Pierino Ferremi ^d,e   Quinti, Isabella ^b   Pietrogrande, Maria C ^f   Finocchi, Andrea ^g   Fazzi, Elisa ^d   Chessa, Luciana ^c   Magnani, Mauro ^h,i  

^a Child Neurology and Psychiatry ^*  (Italy)

^b SAPIENZA UNIVERSITY OF ROME   (Italy)

^c SAPIENZA UNIVERSITY OF ROME   (Italy)

^d Maternal Infantile Department Pediatrics Clinic   (Italy)

^e UNIVERSITY OF BRESCIA   (Italy)

^f UNIVERSITY OF MILAN   (Italy)

^g UNIVERSITY OF ROME TOR VERGATA   (Italy)

^h UNIVERSITY OF URBINO   (Italy)

ⁱ EryDel SpA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

DEXAMETHASONE;

ABSENCE OF SIDE EFFECTS; ADAPTIVE BEHAVIOR; ADOLESCENT; ADULT; ARTICLE; ATAXIA TELANGIECTASIA; CHILD; CLINICAL ARTICLE; CLINICAL OUTCOME; CONTROLLED STUDY; CORTICOSTEROID THERAPY; DRUG DELIVERY SYSTEM; ERYTHROCYTE; FEMALE; HUMAN; INTERNATIONAL COOPERATIVE ATAXIA RATING SCALE; LABORATORY TEST; MALE; PRIORITY JOURNAL; RATING SCALE; VINELAND ADAPTIVE BEHAVIOR SCALE;

EID: 84949558806     PISSN: None     EISSN: 23327812     Source Type: Journal    
DOI: 10.1212/NXI.0000000000000098     Document Type: Article

References (10)

1. Sedgwick O, Boder E. Ataxia-teleangiectasia. In: Vinken PJ, Bruyn GW, editors. Handbook of Clinical Neurology. Vol 14. Amsterdam: Elsevier; 1972: 267-239.
2. Ataxia-Teleangiectasia; AT, #208900. Online Mendelian Inheritance in Man (OMIM), Center for Medical Genetics, John Hopkins University, Baltimore, MD, and the National Center for Biotechnology Information, National Library of Medicine, Bethesda, MD. Available at: http://omim.org/entry/208900. Accessed October 24, 2012.
3. Buoni S, Zannolli R, Sorrentino L, Fois A. Betamethasone and improvement of neurological symptoms in ataxia tel-angiectasia. Arch Neurol 2006;63:1469-1482.
4. Broccoletti T, Del Giudice E, Cirillo E, et al. Efficacy of very-low-dose betamethasone on neurological symp-toms in ataxia-telangiectasia. Eur J Neurol 2011;18: 564-570.
5. Zannolli R, Buoni S, Betti G, et al. A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia. Mov Disord 2012;27:1312-1316.
6. Chessa L, Leuzzi V, Plebani A, et al. Intra-erythrocyte infusion of dexamethasone reduces neurological symptoms in ataxia teleangiectasia patients: results of a phase 2 trial. Orphanet J Rare Dis 2014;9:5.
7. Magnani M, Rossi L, D'ascenzo M, Panzani I, Bigi L, Zanella A. Erythrocyte engineering for drug delivery and targeting. Biotechnol Appl Biochem 1998;28:1-6.
8. Trouillas P, Takayanagi T, Hallett M, et al. International Cooperative Ataxia Rating Scale for pharmacological assessment of the cerebellar syndrome. The Ataxia Neuro-pharmacology Committee of the World Federation of Neurology. J Neurol Sci 1997;145:205-211.
9. Van Duijn G, Dijkxhoorn Y, Noens I, Scholte E, van Berckelaer-Onnes I. Vineland Screener 0-12 years research version (NL). Constructing a screening instrument to assess adaptive behaviour. Int J Methods Psychiatr Res 2009;18:110-117.
10. Moxley RT III, Ashwal S, Pandya S, et al; Quality Standards Subcommittee of the American Academy of Neurology, Practice Committee of the Child Neurology Society. Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005;64:13-20.