Dexamethasone Trial


Luciana Chessa, MD




This open label (non-placebo controlled) study was performed to assess the safety and tolerability of the EryDex System in patients with A-T, and examine the ability of the EryDex System to reduce the neurological symptoms associated with A-T.

EryDex System:

EryDex is dexamethasone sodium phosphate loaded patient erythrocytes (red blood cells or RBCs).  RBCs are removed from the patient, mechanically loaded with dexamethasone, then re-infused back into the patient. The EryDex System allows the slow release of dexamethasone, thus maintaining a sustained, low dose of drug in the blood stream for a one month period after loading.


The EryDex system significantly reduced the neurological symptoms associated with A-T as measured by the International Cooperative Ataxia Rating Scale (ICARS). No steroid side effects were observed.


Chessa, L., Leuzzi, V., Plebani, A., Soresina, A., Micheli, R., D’Agnano, D., Venturi, T., Molinaro, A., Fazzi, E., Marini, M., et al. (2014). Intra-erythrocyte infusion of dexamethasone reduces neurological symptoms in ataxia teleangiectasia patients: results of a phase 2 trial. Orphanet J Rare Dis 9, 5.

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