Outcomes and Barriers to Use of Novel Sickle Cell Therapeutic Agents in a Community Health Center

Abstract

Sickle cell disease is genetic red blood cell disorder transmitted via an autosomal recessive mutation due to valine replacing glutamicacid on the beta globulin chain of the hemoglobin molecule. The disease impacts millions of people worldwide majority living in sub-Saharan Africa and India and impacts approximately 100,000 Americans mostly those of African descent. [2-3] In 2019, two novel treatment agents for sickle cell anemia, crizanlizumab (Adakveo) and voxelotor (Oxbryta) were approved by the United States Food and Drug Administration (US FDA) [7, 8]. Both medications offer sickle cell patients improved control of their disease by reducing sickling of the red blood cells (voxelotor) and the painful effects of vaso-occlusive crises, (crizanlizumab). We studied the effects of crizanlizumab and voxelotor on a population of patients in a sickle cell clinic. Fifty-two charts were reviewed for inclusion in the study; 12 patients were using crizanlizumab and 12 patients were using voxelotor. Eight patients met criteria for evaluation of crizanlizumab and 7 patients for voxelotor. Of all data collected, the only significant difference between baseline measures and post-therapy measures was for voxelotor and hemoglobin levels at baseline and at 3 or more months post therapy. This was a small study which reflects the experience of one clinic; sickle cell providers must continue to address the social determinants of health, psychosocial and psychological needs of their patients in addition to prescribing these novel medications.