TY - CHAP M1 - Book, Section TI - Sickle Cell Disease A1 - Chan, C. Y. Jennifer A1 - Frei-Jones, Melissa A2 - DiPiro, Joseph T. A2 - Talbert, Robert L. A2 - Yee, Gary C. A2 - Matzke, Gary R. A2 - Wells, Barbara G. A2 - Posey, L. Michael PY - 2017 T2 - Pharmacotherapy: A Pathophysiologic Approach, 10e AB - Content UpdateNovember 26, 2019Voxelotor Approved for Treatment of Sickle Cell Disease: Voxelotor (Oxbryta®) is the first hemoglobin S polymerization inhibitor approved by the FDA and is indicated for adults and children older than 12 for the treatment of sickle cell disease. Sickle cell disease affects approximately 100,000 individuals in the US and is a hereditary condition which results in the production of sickle, or abnormally, shaped hemoglobin and has limited treatment options. In a phase 3 randomized, placebo controlled, double blind trial, two doses of voxelotor were compared to placebo. Those treated with the 1500mg dose had a significantly higher hemoglobin response compared to placebo, 51% (95% CI, 41 - 61) versus 7% (95% CI, 1 - 12). Adverse effects were similar across the study groups and the most common being headache and diarrhea. SN - PB - McGraw-Hill Education CY - New York, NY Y2 - 2024/03/28 UR - accesspharmacy.mhmedical.com/content.aspx?aid=1168070625 ER -