Memorial Sloan Kettering experts are leaders in diagnosing and treating pediatric blood disorders, including inherited bone marrow failure syndromes, acquired aplastic anemia, and hemoglobinopathies. These diseases involve damage to blood-forming stem cells, known as hematopoietic stem cells.
Though many inherited blood disorders themselves cannot be cured, our team of experts has designed a treatment approach that can effectively cure the bone marrow failure associated with many of these disorders. Since the mid-1980s, doctors in our Department of Pediatrics have been among the pioneers in the treatment of bone marrow failure, and today we care for patients who come from around the country and the world to receive treatment.
One of our approaches includes performing allogeneic hematopoietic stem cell transplantation (HSCT), or the replenishing of blood-forming stem cells from a donor. Our approach does not require total-body irradiation to destroy the body’s damaged stem cells prior to the transplant. Instead, we use low doses of chemotherapy to accomplish the same goal.
We also perform transplants without white blood cells known as T cells. A T-cell depleted transplant reduces the risk of graft-versus-host disease, in which the transplanted cells recognize the patient’s own cells as foreign and produce an inflammatory reaction.
In addition, we have managed and treated patients with aplastic anemia since the late 1970s. Today, we employ immunotherapy, hematopoietic growth factors, or HSCT, and our results are among the top in the country. (1) We have also performed dozens of transplants for people with the inherited blood disorders beta-thalassemia and sickle cell disease.
Research to Improve Treatments
In the laboratory, our researchers are studying gene therapy for pediatric blood disorders, and we are pioneering a clinical trial to deliver a new stem-cell-based gene therapy to people with beta-thalassemia. In the future, doctors may be able to use a similar type of therapy to treat sickle cell disease and other bone marrow failure syndromes. To continue developing new treatment approaches and state-of-the-art medical services for blood and immune disorders affecting children, Memorial Sloan Kettering became one of the founding members of the New York Tri-Institutional Consortium for Inborn Errors of Hematopoiesis and Immunity. The consortium was established in 2011 and includes the Weill Cornell Medical College and The Rockefeller University.
Learn more about how we diagnose and treat pediatric blood disorders.
- Inherited Bone Marrow Failure Syndromes
Though inherited bone marrow failure syndromes themselves cannot be cured, the failure of bone marrow to produce the required blood cells can be treated and cured, most often with allogeneic stem cell transplantation.
- Acquired Severe Aplastic Anemia
For patients with acquired severe aplastic anemia, our team of experts develops a treatment plan designed to cure the bone marrow failure if possible, address any additional symptoms, and improve your child’s quality of life.
- Paroxysmal Nocturnal Hemoglobinuria
There are two major treatment options for this type of aplastic anemia: a recently developed monoclonal antibody called eculizumab or an allogeneic stem cell transplant.
Patients with beta-thalassemia and sickle cell disease can receive supportive care, and in some cases, a cure through stem cell transplantation.