CLINICAL TRIAL: TREATMENT OF SICKLE CELL VASO-OCCLUSIVE CRISIS WITH HIGH DOSE IV

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Sickle cell anemia is one of the most common hematologic disease in the world. The most common clinical manifestation of this disease is the recurrence of vascular occlusive episodes ("crises") for which there is currently no therapy other than supportive care and analgesics. Our recent studies using a mouse model of sickle cell disease suggest that leukocytes (WBCs) that are adherent to the vessel wall of post-capillary and collecting venules play a key role in vascular occlusion by interacting with sickle eythrocytes (RBCs)1,2. In search for the molecular mechanisms responsible for these interactions, we found that IVIG had a profound effect on the interactions among RBCs, WBCs, and the endothelium, improved blood flow in venules of the cremaster muscle and, most importantly, impacted on the overall survival of sickle cell mice. We therefore want to evaluate high-dose intravenous immune globulin (IVIG) in a pilot phase I/II clinical study in sickle cell disease patients that are admitted to Mount Sinai Medical Center for vaso-occlusive crisis. Hypothesis: 1) IVIG is tolerated by patients with sickle cell disease admitted with vaso-occlusive crisis. 2) IVIG can be effective in ameliorating vaso-occlusive crisis in patients with sickle cell disease.