Childhood BMT/Cell Therapy

About Cell Therapy

Bone marrow contains stem cells, which are immature cells that develop into the various types of mature blood cells: red blood cells, which carry oxygen; platelets, which help the blood clot; and white blood cells, which help fight infection. A bone marrow transplant replaces defective or damaged cells in patients whose normal blood cells have been crowded out by cancerous cells. Transplants can also be used to treat hereditary disorders such as sickle cell anemia, or to help patients recover from or better tolerate cancer treatment.

Bone marrow transplants, or "stem cell therapy" are being constantly improved. At the Children's Cancer Hospital at MD Anderson, we offer a state-of-the-art program to use immune cells in addition to stem cells to improve the chance of treatment success. For example, cell therapy infusing T cells and NK cells can be performed on children, adolescents and young adults whose cancer has not responded to chemotherapy and/or radiation treatment, or whose cancer has come back. However, at MD Anderson, we believe the best time for cell therapy is before a recurrent cancer gets out of control, not as a "last resort."

The Children's Cancer Hospital's Cell Therapy team is closely integrated with MD Anderson's Department of Stem Cell Transplantation and Cell Therapy for adult patients. This collaboration gives child, adolescent and young adult patients access to additional clinical trials and expertise. MD Anderson's pediatric and adult cell therapy program is accredited by the Foundation for Accreditation of Cellular Therapies and is the world's largest stem cell transplantation program.

Clinical Trials for Childhood Bone Marrow Transplantation

The most innovative treatments for childhood stem cell transplants are offered as part of clinical trials, which are closely monitored studies to test the safety and effectiveness of new treatments. Go to a current list of MD Anderson's cell therapy clinical trials to see if your child might be eligible.

Diseases Treated with Cell Therapy

  • Acute Lymphoblastic Leukemia (ALL) 
  • Acute Myelogenous Leukemia (AML)
  • Chronic Myelogenous Leukemia (CML)
  • Myelodysplasia
  • Lymphoma
  • Neuroblastoma

MD Anderson also offers cell therapy for other life-threatening childhood disorders:

  • Severe aplastic anemia
  • Wiskott-Aldrich syndrome
  • Severe combined immunodeficieny
  • Sickle cell anemia
  • Thalassemia
  • Amegakaryocytic thrombocytopenia
  • Inborn errors of metabolism 

What to Expect in a Cell Transplant

The transplant process is physically and psychologically demanding for both the patient and family members. Bone marrow transplants occur in three stages and require a hospital stay that averages 30 to 60 days: 

  • In the pre-transplant phase, the patient is given high-dose chemotherapy or radiation to destroy existing cancer cells, eliminate the body’s ability to reject donor cells, and make room in the marrow for donor cells. This phase takes about seven to ten days.
  • The transplant phase involves the actual process of transplanting donor cells into the patient through an intravenous (IV) tube, which takes about two hours.
  • In the post-transplant phase, the patient’s bone marrow and immune system recover. The first 30 to 40 days are spent in the hospital. In this phase, we wait for the patient’s blood counts to come back up. The white blood cells come back first and can take anywhere from 12-42 days. The red blood cells generally come back next, followed by the platelets (anywhere from 28-90 days). However, even when blood counts are fully recovered, the patient’s immune system will not function normally until at least six to 18 months after the transplant.

Complications of Cell Therapy

The high doses of chemotherapy and/or radiation can lead to serious complications both during and after transplantation. Many of these complications require close monitoring, which may require intensive care provided by a specially trained team of doctors and nurses together with the transplant team. Each patient is unique and not everyone will experience side effects, but many can be treated with medications.

  • Infections: After the transplant, the immune system will be very weak. This means that the patient will be at risk for developing bacterial, fungal or viral infections, which may be life-threatening. These can be treated with antibiotics and other medications until the white blood cell count starts to increase. MD Anderson has world-reknowned infectious disease specialists who can help manage these infections. Moreover, new antibiotics that are not yet widely available can be given in certain circumstances.
  • Loss of appetite: Typically, patients may not be able or willing to eat during or after transplant. Our clinical nutrition specialists provide tips on nutritional supplementation and support. Some patients may require nutrition through their central line (total parenteral nutrition, TPN). 
  • Pain: Pre-transplant therapy causes sores throughout the gastrointestinal tract (mucositis). Patients may experience pain in the mouth and throat or when swallowing, stomach cramps or diarrhea. Medications are given so that the pain will disappear. Pain management specialists are consulted when needed for both acute and chronic pain.
  • Anxiety/Frustration/Fear: The transplant process puts a significant emotional burden on the daily lives of the patient and their family. At the Children’s Cancer Hospital, we try to keep life close to “normal” with a support system that includes child psychologists, child life specialists, social workers, teachers and chaplains to help the patient and family.
  • Organ damage: The functions of the liver, kidneys and lungs can be affected by a transplant. If this happens, your child may require dialysis or breathing assistance with a machine called a ventilator.
  • Graft versus host disease (GVHD): This occurs when the new stem cells (the graft) recognize the tissues of your body (the host) as foreign and mount an immune attack against them. Symptoms can range from mild and temporary to serious and chronic, and can be life-threatening in some cases. Read more about Graft versus host disease.

Survivors of bone marrow/stem cell transplantation are at increased risk for long-term side effects such as osteoporosis (weakening of the bones), cardiac or pulmonary abnormalities or endocrine problems, which include hypothyroidism and growth hormone insufficiency. For this reason,  patients are observed for a long time after transplant.

Pediatric Cell Therapy Team

Laurence Cooper

Dean Lee

Demetrios Patropoulos

Laura Worth

Susan Staba Kelly


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