SOD3-Mimetic As a Complement for Genetic Therapy in Sickle Cell Disease

Conclusions:The present results support development of SanFlow, delivered through continuous infusion, for anemic SCD children to prevent the development of blood transfusion dependency in order to avoid stroke and painful vaso-occlusive crisis (VOC). These results also demonstrated that SanFlow can be used safely and effectively in the elimination of serious painful vaso-occlusive crisis and protect silent and major strokes. Clinical trials of SanFlow in SCD children, prior to their transfusion dependence, as well as in transfusion-dependent teenagers and adults with SCD patients are warranted. By extension, ß-Thalassemia patients can also be treated like SCD patients using SanFlow to relieve them from dependence on life-long blood exchange transfusion. However, these patients would be treated with SOD3-mimetic while waiting for genetic therapy.References:Thompson AA., et.al. Eng. J. Med 2018; 398: 1479-1493Brockman EC., et. al. 2017 Neurotrauma, 34(7):1337-50Cao S., et. al. 2017 J. Am Heart Assoc., 6(9):e006505.DisclosuresHsia: AntiRadical Therapeutics LLC: Employment.

Source: Blood - November 21, 2018 Category: Hematology Authors: Hsia, C. J., Ma, L. Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science: Poster II Source Type: research