Sickle Cell Disease
Sickle cell disease is caused by an abnormal type of hemoglobin, a protein inside red blood cells that carries oxygen. The abnormal hemoglobin, called hemoglobin S, causes red blood cells to become fragile and look like crescents or sickles. The abnormal red blood cells deliver less oxygen to tissues in the body and can get stuck in small blood vessels, interrupting healthy blood flow. Sickle cell disease is inherited from both parents. It is more common in people of African and Mediterranean descent.
Our sickle cell program offers comprehensive care and the most advanced breakthroughs in treatments that dramatically improve outcomes for patients. Our program is part of the New York Implementation Science Consortium – a program that is supported by funding by the National Heart, Lung, Blood Institute. We strive to join clinical care and scientific research along with psychosocial services to provide the best care available anywhere. Our team includes dedicated physicians, advanced practice providers, and social workers who specialize in sickle cell disease and who expertly manage both the acute and chronic complications that sickle cell disease may cause. Our social workers strive to partner with patients to manage the psychosocial complexities that may also occur while living with sickle cell disease.
Research and Clinical Trials
Our research program offers patients the opportunity to participate in several translational research studies aimed at improving patient outcomes and the quality of life for people living with sickle cell disease. We are investigating the role of adhesion in the pathophysiology of sickle cell disease and the development of therapeutic approaches that target cell adhesion. We are also conducting research to help develop gene therapy as a way of curing the disease.
We collaborate with leading organizations to offer innovative clinical trials to our sickle cell disease patients.
Current and upcoming trials:
GBT440-039, A Phase 4, multicenter, open-label study to evaluate the treatment effect of voxelotor on physical activity in adolescents and adults with sickle cell disease (Industry sponsor: Global Blood Therapeutics Inc.)
BO42451, A randomized double-blind Phase 2A study evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of crovalimab as adjunct treatment in prevention of acute vaso-occlusive episodes (VOE) in sickle cell disease (Industry sponsor: F. Hoffmann-La Roche)
BO42452, A Phase 1B randomized, placebo-controlled study evaluating the safety, pharmacokinetics, pharmacodynamics, and efficacy of crovalimab for the management of acute vaso-occlusive episode (VOE) in patients with sickle cell disease (Industry sponsor: F. Hoffmann-La Roche)
NN7533-4470 (ASCENT1), A multicenter trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (EPI01) in patients with sickle cell disease (Industry sponsor: Novo Nordisk)
Care for Pediatric Patients
We provide specialized care for children with sickle cell disease and are equipped to treat complications such as stroke and acute chest syndrome in collaboration with Mount Sinai’s Pediatric Intensive Care Unit. We work closely with your child’s pediatrician to diagnose and treat sickle cell disease and to provide patient and family education. We also provide opportunities to participate in support groups, camps, and other extracurricular activities.