Pediatric Autologous Blood and Marrow Transplantation

About Autologous Transplantation

• What is an autologous blood and marrow transplantation?

  Autologous transplants are the most common of the BMT treatments. Autologous BMT at Children’s National is performed for patients with cancer. In this type of transplant, the patient is both donor⁽²⁾ and recipient of blood-forming stem cells, which are collected and frozen until needed following treatment.

• What conditions are treated with autologous blood and marrow transplantations in children?

  This therapy is commonly used to treat brain tumors which are difficult to cure with standard chemotherapy⁽³⁾ or radiation and in high-risk neuroblastoma⁽⁴⁾, a type of childhood cancer that usually starts in the abdomen or the chest. Autologous transplants are sometimes used in selected patients with other types of hard-to-treat cancer.

  Diseases treated by autologous BMTs are generally not disorders that start in or involve the bone marrow⁽⁵⁾. Instead, they are malignant (cancerous) tumors located elsewhere in the body that are responsive to high-dose chemotherapy and/or radiation treatment.

  The high doses of chemotherapy and/or radiation used to treat those cancers also destroy the patient's bone marrow. Without bone marrow, the body is unable to manufacture blood cells needed to defend against infection, carry oxygen, and stop bleeding.

  An autologous BMT enables Children’s National transplant doctors to "rescue" the patient from the effects of high-dose chemotherapy and/or radiation treatment by replacing the destroyed bone marrow.Future directions for the autologous transplant program at Children's National includes treatment of patients with severe autoimmune and rheumatologic disorders, such as systemic lupus erythematosus, juvenile rheumatoid arthritis and dermatomyositis.

Treatment

• Preparing for transplantation for a child

  • Medical history and physical evaluation: Up to 21 days before beginning the transplantation, the patient will come to the hospital to undergo a complete medical history and physician examination, including multiple tests to evaluate the child's blood and organ functions.
  • Stem cell collection: The Blood Donor Center team collects blood-forming stem cells from the patient's blood in a procedure called stem-cell apheresis. This takes place when a patient's blood counts are recovering after a cycle of chemotherapy and often involves placing a temporary central venous catheter in the femoral vein, which is located in the upper part of the leg. 
  • The catheter is connected to a machine that takes some blood out of the body, separates the white blood cells⁽⁶⁾ (or leukocytes, which contain the blood-forming stem cells) and returns the rest of the blood to the patient. Patients are typically connected to the machine for three to six hours in order to collect the needed number of stem cells. These cells are processed and frozen for later use by Children’s National's Stem Cell Laboratory.
  • Chemotherapy: Two to 10 days before transplantation, patients receive high-dose chemotherapy to treat the underlying disease, but which will often destroy the patient’s bone marrow. 

• Transplanting stem cells for a child

  After chemotherapy, your child will receive some of the thawed blood-forming stem cells that were previously removed and frozen. The stem cells are given intravenously, just like a regular blood transfusion.

• Engraftment for a child

  It takes one to two weeks for the stem cells to mature and begin to produce significant numbers of new blood cells, which is called engraftment⁽⁷⁾. Although engraftment begins one to two weeks after transplantation, it can take months or years for the entire immune system to fully recover. The first 100 days is a critical time in the transplant process. Children’s National's transplant team monitors patients closely for infections and low blood counts, which may require patients to remain in the Washington, D.C. area for frequent follow-up visits. Depending on a child’s condition, he or she may need to stay in the hospital.

  After a patient receives a transplant and passes the infection-risk period, he or she returns home to the care of his or her oncologist for further treatment or follow-up visits. Children’s National transplant doctors remain available to treat or help a child’s oncologist treat any transplant-related complications

• Complications or risks of transplantation for a child

  • Infections. Chemotherapy suppresses white blood cells, which normally fight and prevent infections. This puts the patient at risk of infections. Infections can be caused by bacteria, fungi or viruses⁽⁸⁾. Antibiotics, antifungal and antiviral drugs are given to prevent and treat infections. Infections that do not respond to the treatment may lead to death in approximately one in 30 patients.
  • Veno-occlusive disease (VOD)^(9). Blood vessels that lead into and pass through the liver are prone to damage after transplantation. This may result from chemotherapy and may lead to swelling and severe liver damage. The chances of severe VOD are less than 5%.
  • Nutrition problems. The stomach and intestines are sensitive to chemotherapy. Nausea, vomiting, mouth sores, diarrhea and loss of appetite may occur. Nutrition may be given intravenously or via nasogastric tube feedings until patients are able to eat.
  • Low blood counts. While waiting for new stem cells to make normal red blood cells and other blood cells, a patient usually needs transfusions of platelets⁽¹⁰⁾ and red blood cells⁽¹¹⁾, depending on the patient's clinical condition.
  • Social and emotional concerns. Transplantation is a challenge to both patient and family. They will encounter disruption of family relationships, absence from home and isolation from school, friends and relatives. The family is the key to supporting the child through the transplant period.
  • Infertility. Patients who receive autologous transplants are at risk for not being able to have children in the future. Depending on the disease being treated and the chemotherapy drugs and/or radiation therapy used for transplant, this risk can vary from very low to quite high. Sperm and egg donation and storage would be considered for any post-pubertal patients prior to the transplant procedure.