Blood Disorders in Children

Blood Disorders in Children

Pictured: Farid Boulad

Dedicated doctors: Pediatric bone marrow transplant specialist Farid Boulad talks with a patient.

In addition to caring for young people with cancer, MSK Kids is known for expertise in the diagnosis and treatment of children, adolescents, and young adults with blood disorders, such as Fanconi anemia and sickle cell disease. Although they are not cancers, they can be life-threatening if they’re not diagnosed early or left untreated. These are generally rare conditions, but our team sees a higher number of them than most hospitals. This gives us extensive experience that benefits your child and your family. Our doctors have been among the pioneers in treating these illnesses, and today we care for patients who come to us from around the world.

Our Team Approach

Your child’s care team works together to create a treatment plan that aims to cure the blood and bone marrow complications of these diseases whenever possible. Their plans are also designed to relieve symptoms and improve quality of life. Our team includes pediatric hematologist-oncologists, gene therapy experts, nurse practitioners, psychologists, social workers, and patient care coordinators. Because pediatric blood disorders can affect multiple organs and organ systems in the body, our hematologists-oncologists work with experts in endocrinology, head and neck oncology, neurology and neuropsychology, cardiology, pulmonology, gynecology, and orthopedics to monitor each patient’s health. Together, they ensure our patients benefit from the expertise of specialists who have advanced training in multiple areas of medicine. This gives your child the best chance of success.

What is hematology?

Hematology is the study of blood and blood diseases. At MSK Kids, every one of our doctors is a pediatric hematologist/oncologist. This gives them unique expertise in developing the best, most personalized care plan for your child.

This information is about blood disorders in children. Read about blood disorders in adults.

Who do we treat?

There are many types of noncancerous blood disorders. At MSK Kids, our team has seen young people with virtually every type of blood disorder. That gives us the experience and knowledge to treat them with the greatest level of expertise.

We care for young patients with:

Red blood cell and iron disorders

  • Childhood red blood cell disorders
  • Sickle cell disease
  • Thalassemia
  • Childhood anemia
  • Iron deficiency anemia
  • Iron-refractory iron deficiency anemia (IRIDA)
  • Congenital sideroblastic anemia
  • Congenital dyserythropoietic anemia
  • Megaloblastic anemia (including pernicious anemia)
  • Hemolytic anemia
  • Hemolytic disease of the newborn
  • Hydrops fetalis
  • Spherocytosis
  • Hemochromatosis

Bleeding disorders

  • Hemophilia
  • von Willebrand disease
  • Thrombocytopenia
  • Platelet function disorders
  • Hypofibrinogenemia and dysfibrinogenemia

Thrombosis and anticoagulation disorders

  • Thrombosis
  • Stroke
  • Factor V Leiden
  • Prothrombin gene mutation
  • Protein C deficiency
  • Protein S deficiency
  • Antithrombin deficiency

Autoimmune blood cell disorders

  • Immune thrombocytopenia (ITP)
  • Evans syndrome
  • Autoimmune hemolytic anemia

Bone marrow failure syndromes

  • Aplastic anemia
  • Congenital amegakaryocytic thrombocytopenia
  • Diamond-Blackfan anemia
  • Dyskeratosis congenita
  • Fanconi anemia
  • Myelodysplastic syndrome (MDS)
  • Shwachman-Diamond syndrome
  • Thrombocytopenia absent radius

White blood cell disorders

  • Childhood white blood cell disorders
  • Chronic granulomatous disease
  • Severe congenital neutropenia (Kostmann syndrome)
  • Cyclical neutropenia
  • Leukocyte adhesion deficiency
  • Myeloperoxidase deficiency

Other blood disorders

  • Polycythemia/Erythrocytosis

How We Treat Pediatric Blood Disorders

Treating children with pediatric blood disorders is very complex. We put together the most effective treatment plan for your child, customized to his or her needs and designed to achieve the highest possible level of success.

MSK Kids is renowned for its expertise in stem cell transplantation, which for many pediatric blood disorders is the only cure. Our team has refined the procedure to reduce its intensity, increase its effectiveness, lower the risk of complications, and make it accessible to more patients. Our clinical trial researchers are also studying new gene therapy approaches for several of these diseases.

Regardless of which therapy we think is best for your child, all patients benefit from supportive care. This includes medications, enzyme replacement therapies, blood transfusions, lifelong monitoring, and psychosocial support.

Advancing the Field of Children’s Hematology through Research

Every day, we’re learning about pediatric blood disorders and how to cure them. In addition to providing traditional treatments, MSK Kids team members conduct pioneering research. Before, during, and after a patient’s diagnosis, we bank and study blood samples closely to understand the root causes of pediatric blood disorders. This gives us insight into how we can develop better ways to treat them. Through our collaboration with experts across Memorial Sloan Kettering and in partnership with the Leukodystrophy Center at Weill Cornell Medicine, we are well-positioned to make headway against these illnesses and contribute to the field in ways that could benefit children everywhere.