The treatments we offer for pediatric blood disorders are based on the latest medical findings. We are also conducting clinical trials of promising new therapies.
Leadership in the Treatment of Bone Marrow Failure Syndromes
If your child has an inherited or acquired bone marrow failure syndrome, he or she has low blood counts due to problems with the bone marrow — the spongy tissue inside bones that produces stem cells, which mature to become various types of functional blood cells in the body: oxygen-rich red blood cells, infection-fighting white blood cells, and platelets to help stop bleeding. MSK Kids experts have helped develop new treatments for bone marrow failure syndromes such as Fanconi anemia. We are also well-known for our expertise in treating acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria.
There are international registries for some inherited bone marrow failure syndromes. They pool together information on all of the people with that syndrome, with the goal of better understanding and treating each one. MSK Kids is in close contact with these registries as well as with researchers around the world to stay up-to-date about the latest studies and treatments for these disorders. You can rest assured that your child is receiving the most advanced therapies available.
The Latest Treatments for Hemoglobin Disorders
Hemoglobinopathies are the most common genetic disorders of the blood, affecting millions of people around the world who are born with them. The two most common hemoglobinopathies are thalassemia (alpha and beta thalassemia) and sickle cell disease (SCD). MSK Kids offers comprehensive care for children with hemoglobin disorders, with special expertise in stem cell transplantation and opportunities to participate in clinical trials of new approaches, such as gene therapy.
Treatment of Rare Immune System Disorders
People from around the world come to MSK Kids to receive care for pediatric blood disorders that many other doctors rarely see — such as those that cause the number of infection-fighting white blood cells to plummet, which weakens the body’s immune defense. We also treat patients with the opposite problem: autoimmune disorders where the immune system goes into overdrive and does not function normally. These disorders not only impair health but also quality of life. Our team does everything possible to support children with immune deficiencies and autoimmune disorders, and in some cases are able to achieve a cure.
Care for Children with Metabolic Disorders
The loss of a single vital enzyme or a defect in a critical protein can cause rare, potentially life-threatening inborn errors of metabolism. In some cases, early diagnosis is critical for any chance of a successful outcome. The MSK Kids team recognizes how important it is to identify and treat these children early in life, and quickly. We are here for you and will see you as soon as possible to make an accurate diagnosis, identify your child’s most pressing needs, and create a care plan to designed to improve your child’s quality of life.