Comprehensive hemoglobinopathy programs

The Division of Hematology/Oncology and Stem Cell Transplantation at Cohen Children’s has been designated by New York state as a Comprehensive Hemoglobinopathy Center since 2013. As a result, we are able to provide children and young adults with hemoglobinopathy disorders top-notch care through the following programs: 

Our Sickle Cell Program serves over 500 children and adolescents with sickle cell disease and is one of the largest programs in the tri-state area. 

Sickle cell disease is an inherited red blood cell disorder affecting nearly 100,000 people in the United States. Normal red blood cells have hemoglobin A, which helps keep red blood cells round and soft so they flow easily through small blood vessels. People with sickle cell disease have mostly hemoglobin S in their red blood cells, which can form hard fibers inside the red cells, causing them to take the sickle shape.

These abnormally shaped sickle cells cannot move through blood vessels easily and at times are blocked from delivering oxygen to some body tissues. They also do not live as long as normal red blood cells do. As a result, sickle cell disease may lead to anemia (low hemoglobin level); jaundice (yellowing of the eyes); weakened immune system against certain bacterial infections; enlarged spleen; stroke; and effects on the brain, eyes, lungs and kidneys. Children with sickle cell disease are typically diagnosed at birth by universal newborn screening programs. A complete family history and additional blood tests may also be required for diagnosis.

Program goals

• Improving the health and well-being of our patients with personalized, coordinated, multidisciplinary care
• Educating patients, families, healthcare professionals and school personnel about sickle cell disease
• Providing referrals to specialists as needed
• Offering psychosocial counseling as needed
• Offering opportunities to participate in research to improve the lives of people with sickle cell disease

Our expertise

Our team is made up of pediatric hematologists, nurse practitioners, dedicated sickle cell nurse, social workers and a genetic counselor. Because sickle cell disease can affect nearly every major organ system, patients also receive evaluation and care from other specialty physicians at Cohen Children’s Medical Center including cardiologists, pulmonologists, nephrologists, neurologists, ophthalmologists, radiologists and surgeons.

Our program offers age-specific sickle cell clinics three days a week. Emphasizing a prevention-centered treatment plan, our providers work to minimize children’s symptoms and avoid complications. A typical plan may include infection prevention with penicillin until age 5, completion of all routine childhood vaccines and additional sickle cell-specific vaccinations, and maintaining vigilance regarding fevers.

We also treat sickle cell disease with hydroxyurea therapy, an oral medication that causes the body to produce fetal hemoglobin, which can significantly reduce complications of the disease; blood transfusions to prevent complications such as stroke; iron chelation to prevent iron toxicity related to chronic or recurrent blood transfusions; and stem cell transplantation, which is a curative treatment for sickle cell disease.

In addition to routine health maintenance visits in the clinic, patients with fever or pain crisis can be treated in the Pediatric Ambulatory Chemotherapy and Transfusion Center as outpatients. Patients requiring hospitalization are admitted to Cohen Children’s Medical Center. When it’s time for patients to transition to adult sickle cell care, we continue to provide support through our affiliation with Long Island Jewish Hospital.

Research

Our Sickle Cell Program is actively involved in both institutional and national studies that are investigating sickle cell disease and its management in an effort to improve care. The current areas of research include hemoglobin F-inducing treatments, hydroxyurea therapy, stem cell transplantation and the effects of sickle cell disease on the heart and the lungs.

Our Thalassemia Program enables families to receive specialized care for their children with thalassemia starting in infancy to prevent complications. We currently serve many children and adults with beta thalassemia major and other forms of thalassemia.

Thalassemia is not a single disorder, but a group of inherited diseases of the blood with similar clinical effects. It affects the ability of the body to make hemoglobin - the part of the red blood cell that carries oxygen from the lungs throughout the body - resulting in anemia. Normal hemoglobin is made up of two proteins, alpha and beta.

There are two main types of thalassemia, beta thalassemia and alpha thalassemia. The most severe type of thalassemia is beta thalassemia major. Children with this form of thalassemia usually require frequent blood transfusions and medical care to survive. Without treatment, the spleen, liver and heart become enlarged, bones become deformed and growth is stunted. Frequent blood transfusions cause iron overload. A drug called chelation is often given to remove the excess iron. 

Complications from the disease and its treatment can include heart problems, liver damage, diabetes, infertility, growth failure and thin or deformed bones.

Program goals

• Improving the health and well-being of our patients with personalized, coordinated, multidisciplinary care
• Educating patients, families, healthcare professionals about thalassemia
• Providing referrals to specialists as needed
• Offering psychosocial counseling as needed
• Offering opportunities to participate in research to improve the lives of people with thalassemia

Our expertise

Our team is made up of pediatric hematologists, nurse practitioners, social workers and a genetic counselor. Because thalassemia and transfusion therapy with resultant iron overload can affect nearly every major organ system, patients also receive evaluation and care from other specialty physicians at Cohen Children’s Medical Center including cardiologists, endocrinologists, radiologists and surgeons.

Our program offers chronic transfusion therapy, iron chelation and stem cell transplantation, which is a curative treatment for thalassemia. In addition to routine health maintenance visits in the clinic, patients receive their transfusions in the Pediatric Ambulatory Chemotherapy and Transfusion Center as an outpatient. Patients requiring hospitalization are admitted to Cohen Children’s Medical Center.