New Information about Treatment for Pediatric Blood Disorders at Memorial Sloan Kettering

Pictured: Farid Boulad

Pediatric Bone Marrow Transplant specialist Farid Boulad interacts with a patient.

Memorial Sloan Kettering is one of the largest referral centers in the country for children, adolescents, and young adults who suffer from inherited bone marrow failure syndromes, acquired aplastic anemia, and red blood cell disorders such as beta-thalassemia and sickle cell disease.

These pediatric blood disorders are not cancerous, but can dramatically affect functioning and quality of life and, in certain cases, may be life-threatening. When a young person with a blood disorder comes to us, a dedicated team of multidisciplinary experts see him or her through the entire treatment and follow-up process – an integration of care personalized to each child’s and family’s needs. Learn more about our team of experts and state-of-the-art care in our new guide to pediatric blood disorders.

Innovative Therapies, Research to Advance Treatments

Although many inherited blood disorders themselves cannot be cured, our team of experts has designed innovative approaches that can treat the bone marrow failure syndromes associated with these disorders, and many of our patients have experienced long-term survival without recurrence of disease. Our physicians have been among the pioneers in this field, and today care for young people who come from around the country and the world to receive treatment.

One approach to our care involves allogeneic hematopoietic stem cell transplantation, which is the replenishing of blood-forming stem cells using cells obtained from a donor. Our physicians also perform transplants in which white blood cells known as T cells have been removed. A T cell-depleted transplant reduces the risk of graft versus host disease, a dangerous inflammatory response that can occur if the transplanted cells recognize the patient’s own cells as foreign.

If your child has been diagnosed with acquired severe aplastic anemia, our team of experts can develop a treatment plan designed to cure the bone marrow failure, address any additional symptoms, and improve your child’s quality of life. Our cure rate of bone marrow failure in children with this illness is about 90 percent.

In addition, we treat many young people with paroxysmal nocturnal hemoglobinuria, a type of aplastic anemia, with a recently developed monoclonal antibody called eculizumab, or an allogeneic stem cell transplant. Our team has performed numerous successful transplants using siblings or unrelated donors who are a bone marrow match.

To advance the development of new treatments, our researchers are studying gene therapy for pediatric blood disorders, and are initiating a clinical trial to deliver a new stem cell-based gene therapy to people with beta-thalassemia. In the future, physicians may be able to use a similar type of therapy to treat sickle cell disease and other bone marrow failure syndromes.

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Supportive Care

Recognizing that living with a blood disorder can be challenging for both children and their families, our experts are skilled in identifying and responding to the various symptoms and possible side effects associated with treatment. We offer an array of support programs and services designed to help the whole family cope with psychosocial issues. We also offer nutrition counseling and physical and occupation therapy.

In addition, the Department of Pediatrics makes their school program a top priority. We are one of 40 hospitals participating in a New York City Department of Education program called Hospital Schools, and several full-time teachers play active roles in educating pediatric patients. They work closely with each child’s school or home instructor to enable students to return to class after treatment with little or no loss of educational standing.

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We received fantastic care, and were blessed with fantastic results after t-cell depleted transplant. My son had severe aplastic anemia/ mylodysplastic syndrome. Today he is living a healthy life, thanks to Dr. Small and the BMT team.

Great to hear! Thank you for sharing.

I was contacted by a 23 yo woman with severe aplastic anemia and she is looking for an option as her doctor told her they can not help her. She didn't find any match for a bone marrow transplant. Is there any clinical trial available for her? Thank you