Effects Of N-Acetylcysteine Infusion In Patients With Sickle Cell Disease During Vaso-Occlusive Crises

Background: Our earlier studies showed that patients with sickle cell disease (SCD) have a high von Willebrand factor (VWF) activity index, which correlates with the extent of the hemolysis they experience [Chen et al Blood 2011; 117:3680‐3]. We are evaluating whether N-acetylcysteine (NAC), which dissociates VWF multimers, has a therapeutic role in SCD. Previously, we studied stable outpatients with SCD [Özpolat et al Blood Dec 2016, 128 (22) 1299]. In that study, we found that NAC infusion was safe and decreased red blood cell (RBC) fragments, dense cells, the size of VWF multimers, platelet activation, and the percentage of platelet-monocyte complexes. In addition, NAC infusion increased the total and free-thiol concentrations of cysteine and glutathione in blood, relieving oxidative stress in the patients at disease baseline. Given these findings, we have begun a pilot study of NAC in SCD patients admitted as inpatients with vaso-occlusive crisis (VOC) (NCT01800526) to examine the safety of NAC infusion and its effects on laboratory markers in that setting.