Overview

In addition to caring for young people with cancer, MSK Kids is known for expertise in the diagnosis and treatment of blood and immune disorders that affect children, adolescents, and young adults. We are experts in treating conditions such as severe combined immunodeficiency, Fanconi anemia, and sickle cell disease, among other noncancerous potentially life- threatening conditions. 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, transplant and gene therapy experts, pharmacists, nurse practitioners, psychologists, social workers, and patient care coordinators, among others. Because pediatric blood disorders can affect multiple organs and organ systems in the body, our hematologist-oncologists work with experts in genetics/genomics, endocrinology, head and neck oncology, neurology and neuropsychology, cardiology, pulmonology, gynecology, pediatric anesthesia, critical care, 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. We also know that these illnesses can affect children, adults, and families emotionally. We provide many services to support all those who walk through our doors.  

Types of Disorders We Treat in Children

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

We care for young patients with:

• Fanconi anemia and other inherited bone marrow failure syndromes
• Aplastic anemia and other acquired bone marrow failure syndromes
• Sickle cell disease, thalassemia, and other inherited hemoglobin disorders
• Primary immunodeficiencies and white blood cell disorders
• Autoimmune blood cell disorders
• Inherited metabolic disorders

How We Treat Pediatric Blood and Immune Disorders

Treating children with pediatric blood and immune disorders is very complex. We put together the most effective treatment plan for your child, customized to their 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 Cancer Center 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.

Learn more about pediatric blood disorders and immune disorders