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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
Institution:1. Department of Pediatrics, Albert Einstein College of Medicine, Bronx, NY, United States;2. Department of Cell Biology, Albert Einstein College of Medicine, Bronx, NY, United States;3. Department of Epidemiology and Population Health, Albert Einstein College of Medicine, Bronx, NY, United States;4. Department of Anesthesiology, Albert Einstein College of Medicine, Bronx, NY, United States;5. New York Blood Center, New York, United States;6. Department of Medicine, Albert Einstein College of Medicine, Bronx, NY, United States;7. Ruth L. and David S. Gottesman Institute for Stem Cell and Regenerative Medicine Research, Albert Einstein College of Medicine, Bronx, NY, United States;1. Department of Integrated Chinese and Western Medicine, Yuhuangding Hospital Affiliated to Qingdao University, Yantai, 264000, China;2. Centre of Integrated Chinese and Western Medicine, School of Basic Medicine, Qingdao University, Qingdao, 266071, China;3. Department of Clinical Medicine, Weifang Medical University, Weifang, 261000, China;4. Emergency Center, The Affiliated Yantai Yuhuangding Hospital of Qingdao University, Yantai, Shandong, 264000, China;1. Department of Obstetrical, Penglai People''s Hospital, Penglai, Shandong, 265600, China;2. Department of Gynecologic, The General Hospital of Ningxia Medical University, Yinchuan, Ningxia, 750004, China;3. Department of Obstetrical, Guicheng Hospital, Nanhai District, Foshan City, Guangdong Province, 528200, China;4. Department of Obstetrical, Jinan Maternal and Child Healthcare Hospital, Jinan, Shandong, 250001, China;5. UKM Medical Molecular Biology Institute, Universiti Kebangsaan Malaysia, Kuala Lumpur, Malaysia;6. Department of Radiology, Taleghani Hospital, Student Research Committee, Shahid Beheshti University of Medical Sciences, Tehran, Iran;7. Department of Obstetrics, Dongying People''s Hospital of Shandon, Dongying, Shandong, 257091, China;1. Ruth L. and David S. Gottesman Institute for Stem Cell and Regenerative Medicine Research, Albert Einstein College of Medicine, Bronx, NY;;2. Program in Cellular and Molecular Medicine, Boston Children’s Hospital, Boston, MA, and Department of Pediatrics, Harvard Medical School, Boston, MA;;3. Department of Pediatrics, Albert Einstein College of Medicine, Bronx, NY; and;4. Division of Hematology/Oncology, Boston Children’s Hospital, Boston, MA;1. Departmentof Endocrinology, Shangrao People’s Hospital, No.86 Shuyuan Road, Shangrao, Jiangxi, 334000, China;2. Internal Medicine, Ji''an Detachment Health Team, Chinese Pepople’s Armed Police Force, Ji''an, 343000, China;3. Internal Medicine, Mobile Detachment Medical Team of Jiangxi Provincial General Corps, Chinese Pepople’s Armed Police Force, Anyi, 330500, China;4. Department of Emergency, Guangfeng People''s Hospital, Yongfeng South Street, Shangrao, Jiangxi, 334600, China;5. School of Medicine, Federal University of Uberlandia (UFU), Uberlandia, Minas Gerais, Brazil;6. Department of Radiology, Taleghani Hospital, Student Research Committee, Shahid Beheshti University of Medical Sciences, Tehran, Iran;7. Department of Pharmacy, Serdang Hospital, Selangor, Malaysia;8. Department of Emergency, Minhang Hospital, Fudan University, No. 170 Xinsong Road, Minhang District, Shanghai, 201199, China
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.
Keywords:Immune globulin  IVIG  Sickle
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