Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis.

Deepa Manwani, Chunliang Xu, Sung Kyun Lee, George Amatuni, Hillel W Cohen, Veronica Carullo, Kerry Morrone, Jennifer Davila, Patricia Ann Shi, Karen Ireland, Janine Keenan, Paul S Frenette

Journal: Complementary therapies in medicine 2021;52():102481

PMID: 32951731

Full Text on Elsevier Science View on PubMed

Abstract

Sickle Cell Disease (SCD) is a chronic hemolytic disorder associated with frequent pain episodes, end organ damage and a shortened lifespan. Currently there exist no disease specific targeted therapies for the treatment of acute vaso-occlusive crisis (VOC) and management with analgesics and hydration is purely supportive. Improvement in understanding of disease pathophysiology has resulted in a great interest in disease modifying novel therapies and many are being evaluated in clinical trials. Here we report the results from the pre-specified mid-point analysis of the Phase 2 study of Intravenous Gamma Globulin (IVIG) for the treatment of acute VOC in patients with SCD and lessons learned.

Address: Department of Pediatrics, Albert Einstein College of Medicine, Bronx, NY, United States. Electronic address: [email protected].; Department of Cell Biology, Albert Einstein College of Medicine, Bronx, NY, United States.; Department of Epidemiology and Population Health, Albert Einstein College of Medicine, Bronx, NY, United States.; Department of Anesthesiology, Albert Einstein College of Medicine, Bronx, NY, United States.; Department of Pediatrics, Albert Einstein College of Medicine, Bronx, NY, United States.; New York Blood Center, New York, United States.; Department of Cell Biology, Albert Einstein College of Medicine, Bronx, NY, United States; Department of Medicine, Albert Einstein College of Medicine, Bronx, NY, United States; Ruth L. and David S. Gottesman Institute for Stem Cell and Regenerative Medicine Research, Albert Einstein College of Medicine, Bronx, NY, United States.

DOI: 10.1016/j.ctim.2020.102481

Link outs

Free resources

Medical:

Subscription / membership required

Full Text Sources:

MeSH Terms: Adolescent, Adult, Anemia, Sickle Cell, Child, Double-Blind Method, Female, Humans, Immunoglobulins, Intravenous, Immunologic Factors, Male, Pain Management, Young Adult, gamma-Globulins