Opioid therapy is the standard treatment for children and young adults managing acute pain caused by sickle cell disease (SCD) due to associated blood vessel blockages. However studies conducted at Montefiore Health System and Albert Einstein College of Medicine suggest there could be a promising new treatment.

Investigators at the Children’s Hospital of Montefiore (CHAM) have received a four-year, $1.6 million grant from the U.S. Food and Drug Administration to evaluate the effectiveness and safety of a specific form of intravenous immune globulin (IVIG), a biologic medicine that may significantly  reduce pain and hospitalizations for children and young adults (ages 8-21 years) with SCD. If positive, the outcome of this Phase 2 research will be an appropriately powered Phase 3 study, a crucial step towards development of a novel therapy for SCD-related vaso occlusive crises (VOC) the medical term for the blood vessel blockages associated with the acute pain of SCD.

“For approximately 16 years, no new treatments for sickle cell disease have reached the marketplace,” said Deepa G. Manwani, M.D., director, Hematology Program, and attending physician, pediatric hematology/oncology, CHAM and associate professor of clinical pediatrics, Einstein. “We are thrilled that the FDA is underwriting this important clinical trial of IVIG which could have the potential to improve standard of care for these underserved patients.”

SCD, one the most common hematologic diseases in the world, is estimated to affect between 90,000 and 100,000 Americans, primarily of African or Hispanic descent. Current treatment strategies for the acute pain are purely symptomatic, and opioid therapy, even when administered by experts in pain management, has significant adverse effects. In addition to VOC, people with SCD are at risk for becoming very ill from common illnesses and require frequent hospitalization.  A U.S. government study estimated the cost for SCD hospitalizations in 2004 was $488 million and another study from 2009 estimates that the lifetime cost of care for someone with SCD averages around $460,000.

“It is our hope that the study will demonstrate a reduction in the length of these painful episodes of VOC and other improved outcomes,” said Dr. Manwani. “If successful, this study will set the stage for better treatments and cost savings of this devastating, and life-shortening disease. Given the lasting duration of IVIG, it is also possible that the study drug will decrease the 30 day readmission rate which has been reported to be as high as 30 to 40% among SCD patients.”

The study will also evaluate biomarkers of adhesion and inflammation (pre and post administration of IVIG) as well as secondary clinical endpoints including total opioid use, length of hospitalization and change in hemoglobin and LDH, a general indicator of tissue and cellular damage.