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Senicapoc was safe and well tolerated in the study. Ten patients, five of whom were in the placebo group, discontinued the study early. Three patients dropped out due to adverse events: one in the low-dose group for weakness and shortness of breath, and two in the high-dose group for pain crises and acute chest syndrome. The most common serious adverse effect (SAE) was pain crisis, followed by pneumonia and acute chest syndrome, with a similar incidence across the active and placebo arms. None of the SAEs were thought attributable to senicapoc.

"The results we saw in this study provide further evidence that blocking the Gardos channel reduces dehydration of the sickle erythrocytes and thus supports the hypothesis that this type of approach may lead to an amelioration of the anemic state in patients with sickle cell anemia," said Dr. Ataga. "As noted in the paper, a Phase III study of senicapoc in SCD patients was recently terminated due to the low probability of achieving a reduction in crisis rate, the primary endpoint of that study. Clearly, further work is necessary to understand how the reduction in RBC dehydration and hemolysis demonstrated from the results of this Phase II study impacts the complex pathophysiology of sickle cell disease."

The American Society of Hematology (http://www.hematology.org) is the world's largest professional society concerned with the causes and treatment of blood disorders. Its mission is to further the understanding, diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems, by promoting research, clinical care, education, training, and advocacy in hematology.