Blood Cancers

Specialized Treatment for Sickle Cell Disease and Its Complications

With decades of experience, NewYork-Presbyterian has the expertise to provide state-of-the-art care for adults with sickle cell disease from diverse walks of life. We are here to help you live your best life possible. Our goal is to decrease the risk of complications associated with sickle cell disease through early intervention. We offer a range of FDA-approved medical treatments, stem cell transplantation (bone marrow transplant), and clinical trial therapies, such as gene therapy.

Sickle cell disease can affect any organ in the body. NewYork-Presbyterian offers access to physicians from every medical specialty. This includes neurology and neurosurgery, orthopedic surgery, general surgery, pulmonology, cardiology, nephrology, ophthalmology, and gastroenterology—to manage the complications associated with sickle cell disease.

About our program

Your multidisciplinary team includes hematologists, nurse practitioners and nurses, and any other specialists you may need. Each team member gets to know you well so that you see the same familiar faces each time you visit us. Many of our patients have been treated at NewYork-Presbyterian since they were infants, and we can access their medical records going back to the earliest days of their sickle cell disease treatment. When adolescents approach adulthood, we provide transitional care to ensure they are equipped with the tools to transfer their care to an adult hematologist and other adult care providers.

What is sickle cell disease? 

Sickle cell disease is a disorder that affects hemoglobin, the iron-containing protein in red blood cells that carries oxygen throughout the body. The abnormal hemoglobin changes the shape of red blood cells from a standard disc shape to a crescent, or sickle, shape. The sickled cells can get stuck in blood vessels, causing a vaso-occlusive crisis—resulting in pain. If the blockage is in a vessel supplying blood to an organ, it can block blood flow to that organ, causing complications such as a stroke. 

People with sickle cell disease break down their red blood cells faster than they can produce new ones, which is why the condition is also called "sickle cell anemia." They may experience:

• Sudden, severe pain (pain crises)
• Fatigue (feeling tired)
• Swelling of the hands and feet
• Jaundice (yellowish color) in the skin or "scleral icterus" (yellowing of the eyes)

What causes sickle cell disease? 

Sickle cell disease is caused by a mutation in the gene that makes hemoglobin. To develop the disease, a person has to inherit two sickle cell genes, one from each parent. People with one gene have a "sickle cell trait" and are generally in good health but can pass the gene to their children. 

Sickle cell disease is diagnosed by testing blood for abnormal hemoglobin. In the United States, testing blood for sickle cell disease is part of the routine health screening of every newborn. Finding it early allows doctors to start treatment sooner to prevent long-term organ damage. 

Medical treatments for sickle cell disease

In addition to hydroxyurea and blood transfusions, we use the newest U.S. Food and Drug Administration-approved therapies available for sickle cell. These include: 

• Voxelotor (Oxbryta®) comes in tablets taken by mouth or mixed with water to make a solution for pediatric patients. It holds onto oxygen by binding to hemoglobin. This prevents red blood cells from sickling and improves anemia. 
• Crizanlizumab (Adakveo®) is given to prevent pain and vaso-occlusive crises, particularly in patients whose symptoms are not relieved by hydroxyurea. It reduces the ability of red blood cells to stick to blood vessels by binding to a protein called P-selectin. It is given intravenously (by vein) in the infusion center once a month.
• L-glutamine (Endari®) is used in some patients to reduce the acute complications of sickle cell disease. It is a powder that is mixed with food or a beverage.

Stem cell transplantation to cure sickle cell disease

Bone marrow transplantation is the only cure for sickle cell disease. It replaces sickle cell-forming "stem" cells with healthy cells from a donor. We provide bone marrow transplantation to appropriate candidates through the stem cell transplantation program at NewYork-Presbyterian/Columbia University Irving Medical Center. 

Clinical trials of new therapies for sickle cell disease

New treatments for sickle cell disease have emerged in the last several years due to advances in research. Clinical trials offer new and innovative treatment options for patients living with sickle cell disease. You may ask your care team if you are eligible for one of our clinical trials. 

Special services for adults with sickle cell disease

In addition to sickle cell disease treatments, we provide:

• Direct access to your healthcare provider via MyConnect to ensure you can receive our services seamlessly.
• Preconception counseling, genetic counseling and testing, and maternal-fetal medicine (specialized care for complex pregnancies) for people with sickle cell disease or sickle cell trait.
• Guidance from patient navigators to help you take care of your health, such as keeping up with vaccines and appointments with other specialists you may need.