Sickle Cell Disease

Pediatrics

Sickle Cell Disease

Medical treatment, stem cell transplantation, and comprehensive support for sickle cell disease

Diagnosis and Treatment

Our approach to care

Our teams include pediatric hematologists, nurse practitioners and nurses, coordinators, social workers, child life specialists, and psychosocial support experts who work closely with patients with sickle cell disease and their families. We have excellent blood banks as a resource for transfusions. Teachers, interpreters, fertility specialists, and genetic counselors are also key members of our team and help families live the best lives they can live despite a diagnosis of sickle cell disease.

Sickle cell disease is not limited to the blood, but can affect the entire body. At our children's hospitals, patients have access to physicians from every discipline — including neurologists and neurosurgeons, orthopedic surgeons, pediatric surgeons, pulmonologists, cardiologists, nephrologists, liver experts, and gastroenterologists — who specialize in pediatric conditions and help treat complications of sickle cell disease. We educate children and their families about sickle cell disease and what they can do to maintain their child's health. When an adolescent nears young adulthood, we provide care to transition to NewYork-Presbyterian's programs for adults with sickle cell disease, ensuring seamless continuity of care.

Medical treatments for sickle cell disease

Our doctors offer hydroxyurea treatment for infants with sickle cell disease as young as 9 months of age. If possible, we prefer to start this therapy before the first sickle cell episode has occurred, with most patients receiving their first dose of medication before age 2. Blood transfusions with healthy, non-sickled blood cells can be used to help relieve symptoms and lower the risk of stroke. Children can receive transfusions in our comfortable and cheerful pediatric infusion suites. In recent years, new drugs for sickle cell disease have been approved by the U.S. Food and Drug Administration, such as:

  • Voxelotor (Oxbryta®). We use this medication for patients ages 4 and older. It comes in tablets that can be taken by mouth or mixed with water to make a solution that can be easier for some patients to take. It works by binding to hemoglobin at the cellular level and keeping it in a state where it maintains oxygen. This prevents red blood cells from sickling and improves anemia.
  • Crizanlizumab (Adakveo®). This medication is designed to reduce red blood cell sickling by binding to a protein called P-selectin. It is given intravenously (by vein) in our infusion centers to patients age 16 and older.

L-glutamine (Endari®) is another medication used to reduce the acute complications of sickle cell disease in adults and children 5 years of age and older.

Stem cell transplantation to cure sickle cell disease

Bone marrow transplantation works to cure sickle cell disease by replacing unhealthy blood-forming "stem" cells with healthy cells. The Sickle Cell Transplant Program at NewYork-Presbyterian Morgan Stanley Children's Hospital is one of the largest in the tri-state area offering stem cell transplantation. Our doctors order immunological typing for all sickle cell patients and their full siblings early in life. All patients are referred for a consultation with our stem cell transplant professionals so a child can begin stem cell transplantation as early as desired, if a matched donor is identified.

  • Finding a donor. Finding a match can be difficult, because many children have siblings who also have sickle cell disease, making them unable to donate. Our team can provide help finding a suitable donor through the National Marrow Donor Program.
  • The transplant process. The transplant begins by giving the patient chemotherapy, which eliminates the stem cells that produce sickle cells. Our institution developed a low-intensity conditioning regimen to decrease long-term side effects. The patient then receives bone marrow from the donor, which contains healthy blood stem cells that do not sickle. The bone marrow cells from the donor, which are given intravenously (by vein), know where they belong in the body and settle in the bone marrow — where they grow and produce healthy new red blood cells, white blood cells, and platelets. This process is called "engraftment." Patients can expect to stay in the hospital for 8-12 weeks to receive the chemotherapy and the bone marrow transplant.
  • Recovering from the transplant. Patients may leave the hospital once doctors see that the bone marrow has been accepted and the patient has recovered from the side effects of the chemotherapy. We continue to monitor and support patients on an outpatient basis for an additional 6-12 months. We provide care to reduce the risk of infections and complications after transplantation. For most children, the return to regular activity and school takes approximately one year, though every child is different.

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NewYork-Presbyterian/Columbia

Sickle Cell Disease Program

NewYok-Presbyterian/Weill Cornell Pediatrics

Hematology/Oncology

NewYork-Presbyterian Brooklyn Methodist Hospital