Non-cancerous Blood Disorders


Non-cancerous Blood Disorders

One of the oldest programs dedicated to the diagnosis and treatment of hemophilia

Hemophilia and Other Bleeding Disorders

More than Half a Century of Experience

NewYork-Presbyterian has exceptional expertise in the care of people with hemophilia and other bleeding disorders. The internationally recognized Comprehensive Hemophilia Treatment Center at NewYork-Presbyterian/Weill Cornell Medical Center was founded more than 50 years ago. Today, young patients with hemophilia and other bleeding disorders have access to this renowned program at NewYork-Presbyterian Komansky Children's Hospital, which is designated as a federally funded Hemophilia Treatment Center (HTC). An HTC brings together doctors, nurses, and other healthcare professionals who are experienced in treating people with bleeding disorders, and focuses on providing education and support to help patients and families stay healthy.

Types of bleeding disorders

Hemophilia is a bleeding problem caused by the lack of functioning blood clotting factors VIII (causing hemophilia A) or IX (causing hemophilia B or "Christmas disease"). It is usually inherited and most often occurs in males. People with hemophilia are at risk for bleeding more easily and for a longer period of time than other people. Other types of bleeding disorders that we treat include:

  • Von Willebrand disease (VWD), the most common type of inherited bleeding disorder. People with this bleeding problem have less of a blood clotting factor called Von Willebrand factor, or it does not work well enough to help form a blood clot. 
  • Factor XI deficiency (hemophilia C) is a rare bleeding disorder that is seen more commonly in individuals of Ashkenazi Jewish descent. Other rare bleeding disorders are caused by deficiencies in fibrinogen and blood clotting factors II, V, VII, X, or XIII
  • Acquired bleeding disorders may be caused by other medical problems, such as an infection or injury, liver disease, vitamin K deficiency, or autoimmune diseases. 

Symptoms of hemophilia

Common signs of hemophilia and other bleeding disorders include:

  • Easy bruising
  • Prolonged bleeding after losing a tooth or having surgery or circumcision
  • Oral bleeding or nose bleeds that are frequent and hard to stop
  • Bleeding into the joints or soft tissues, causing swelling and pain in the knees, elbows, and ankles
  • Blood in the stool or urine

How we diagnose bleeding disorders

We take a thorough patient and family history of bleeding episodes and perform a physical exam. In addition, at New York Presbyterian, we have a specialized coagulation laboratory to perform testing to see if the blood is clotting properly and determine if there is a deficiency. These tests help us identify the cause of the bleeding disorder and assess how severe it is. Since hemophilia and other bleeding disorders are often hereditary, we can also perform genetic testing and offer genetic counseling to interested family members at our comprehensive center.

Our Approach to Care

Doctor putting band-aid on patientBleeding disorders are highly complex and require the expertise of a team of professionals to provide the most effective care. Our care teams include pediatric hematologists with years of experience treating hemophilia, other bleeding disorders, and their possible complications, as well as nurses and nurse practitioners, physical therapists, social workers, genetic counselors, and special laboratory personnel who perform blood clotting tests. When an adolescent approaches young adulthood, we provide transitional care to encourage them to become independent advocates of their own health. Our goal is to empower children, adolescents, and young adults with bleeding disorders to manage and prevent their bleeding symptoms independently and to lead normal healthy lives.

Treatment of hemophilia and other bleeding disorders

There have been many advances in the treatment of bleeding disorders. We customize your child's treatment based on the type and severity of hemophilia or other bleeding disorders and create a plan to prevent and treat bleeding symptoms.

Factor replacement therapy. The most effective treatment is replacement of the missing blood clotting factor. This helps the blood clot properly. The treatment is given intravenously (through a vein) using medications called "factor concentrates." Patients and caregivers of children with hemophilia can learn how to perform these infusions themselves to independently manage bleeding episodes. Our team of hemophilia experts can help you learn how to do this. Giving factor treatment products at home allows you to treat bleeding episodes more quickly, resulting in less serious bleeding and fewer side effects. Infusions of replacement blood clotting factors can be given regularly (an approach called prophylaxis) to prevent most bleeding episodes. 

Non-factor therapy. Emicizumab (Hemlibra®) is a newer medication to prevent bleeding episodes in people with hemophilia A. It works by replacing the function of factor VIII, rather than replacing the missing clotting factor VIII directly. It is given by injection under the skin rather than into a vein.

Desmopressin acetate (DDAVP) is similar to a naturally occurring hormone. It releases factor VIII from where it is stored in the body's tissues and makes it more available in the body to assist with blood clotting. It can be given through a vein or via a nasal spray.

Anti-fibrinolytic therapy prevents blood clots from breaking down, resulting in a firmer clot. It is often used for bleeding in the mouth because it blocks a substance in saliva that breaks down clots. It can be given through a vein or by mouth (as a pill or a liquid).

Gene therapy is a promising investigational treatment being assessed in clinical trials. It involves trying to add a gene to increase a missing or defective blood clotting factor to decrease or eliminate the need for factor replacement therapy.

Why Choose Us

The best care for a child with a bleeding disorder comes from a hospital that not only provides diagnostic and treatment services, but all of the care and support a child and family living with a bleeding disorder may need. That is the approach we take at NewYork-Presbyterian, where you have access to all of the specialists your child may require, all under one roof. In addition, your child may be offered participation in a clinical trial of a promising new therapy. From diagnosis through adolescence and into adulthood, our patients can receive all the care they need from our specialized teams. Contact us to make an appointment for a consultation.

Contact us

Weill Cornell Medicine Comprehensive Hemophilia Treatment Center

Columbia Blood Disorders Care