Blood and Marrow Transplant Program

The Hyundai Cancer Institute at CHOC Blood and Marrow Transplantation (BMT) Program is made up of a team of highly trained oncology experts, including two dedicated nurse coordinators who assist patients and their families through the transplant process. The majority of children who may benefit from a blood or bone marrow transplant are those with leukemia, including acute lymphoblastic leukemia (ALL) and acute myelogenous leukemia (AML). Other conditions that may respond to transplantation include hematologic disorders such as immune deficiencies and aplastic anemia, Hodgkin’s and non-Hodgkin’s lymphoma, Ewing’s sarcoma and solid tumors including some brain tumors and neuroblastoma.

The Hyundai Cancer Institute BMT Program is the only pediatric blood and bone marrow transplant program in Orange County and offers a full range of transplant procedures and services. In addition to performing all necessary tests and procedures prior to the transplant, we test immediate family members (siblings and parents) to see if they are a match for the patient. (For more information on testing extended family members, please see the questions and answers below about the blood and marrow transplant process.)

What is a pediatric blood or bone marrow transplant?

A blood or marrow transplant (BMT) is a special therapy for patients with certain cancers or other diseases. A transplant replaces unhealthy blood-forming cells with healthy ones. To prepare for the transplant, patients may be given strong chemotherapy and/or radiation therapy. These treatments kill the unhealthy cells. Healthy blood-forming cells (stem cells) are then given to the patient in a vein much like a blood transfusion. The transplanted stem cells begin to grow and make the red blood cells, white blood cells and platelets the body needs. This is called engraftment. Engraftment typically takes two to four weeks. Until the transplanted cells have engrafted, patients stay in the hospital.

The goal of a blood or bone marrow transplant is to cure many diseases and types of cancer. When a child’s bone marrow has been damaged or destroyed due to a disease or intense treatments of radiation or chemotherapy for cancer, a transplant may be needed. A blood or marrow transplant can be used to:

• Replace diseased, non-functioning bone marrow with healthy functioning bone marrow for conditions such as leukemia, aplastic anemia and sickle cell anemia.
• Replace the bone marrow and restore its normal function after high doses of chemotherapy or radiation are given to treat a malignancy. This process is often called “rescue” and used for diseases including lymphoma and neuroblastoma.
• Replace bone marrow with genetically healthy functioning bone marrow to prevent further damage from a genetic disease process such as Hurler’s syndrome and adrenoleukodystrophy disorder.

Types of bone marrow transplants:

There are two different types of bone marrow transplants:

• Autologous BMT is an infusion of the patients’ stem cells.
• Allogeneic BMT is an infusion of a donor’s stem cells.

This person may be either a family member (related donor) or an unrelated donor. Related donors who are brothers or sisters and share the same parents have the highest chance of being a suitable donor since they may have inherited the same tissue type from their parents. Occasionally, a parent or an extended family member (half-siblings, cousins, etc.) is a donor match. Unfortunately, in many cases, parents may not be a good enough match to be their child’s donor.

Pediatric Blood and Bone Marrow Transplant services we offer:

• Autologous bone marrow transplant
  Transplant in which the patient receives his or her own bone marrow.
• Allogeneic bone marrow transplant
  Bone marrow transplant with marrow from a person other than the patient. The marrow may come from a related donor (such as a sibling) or an unrelated donor.
• CAR-T Cell Program
  Cellular immunotherapy that involves the collection and genetic modification of a patient’s own T-cells.
• Peripheral blood stem cell transplant
  In this process, the patient receives his or her own peripheral stem cells (autologous) or stem cells from a related donor (allogeneic).
• Umbilical cord blood transplant
  In this procedure, blood is collected from the placenta after the umbilical cord is separated from a newborn baby. This blood contains large numbers of bone marrow stem cells and can be used as a source of donor cells from a sibling or unrelated donor for a bone marrow transplant.

Blood and Bone Marrow Transplant Recovery

As with any procedure, such as bone marrow transplant, prognosis and long-term survival can vary greatly from child to child. The amount of transplants occurring for an increased number of diseases, coupled with medical developments, has greatly improved the outcome for bone marrow transplant in children and adults. Continuous follow-up care is essential for the child following a transplant. New methods to improve treatment and to decrease complications and side effects of transplant are continually being discovered.

Blood and Bone Marrow Transplant Risks

Blood and marrow transplants have risks involved, some of which are life threatening. The risks and benefits must be weighed in a thorough discussion with the transplant team prior to the procedure. Each child experiences diseases differently and a transplant may not be appropriate for everyone who suffers from these diseases. Some of the diseases that have been treated with blood and marrow transplant include the following:

• Leukemia
• Lymphomas
• Some solid tumors (i.e., neuroblastoma, rhabdomyosarcoma, brain tumors)
• Aplastic anemia
• Immune deficiencies (severe combined immunodeficiency disorder, Wiskott-Aldrich syndrome)
• Sickle cell disease
• Thalassemia
• Diamond-Blackfan anemia
• Metabolic/storage diseases (i.e., Hurler’s syndrome, adrenoleukodystrophy disorder)
• Cancer of the kidneys

Infections are common in children with severe bone marrow suppression. Blood tests are performed to prevent, detect and treat infections. Antibiotics, anti-fungal medications and anti-viral medications are often given to prevent serious infection. Bacterial infections are the most common and viral and fungal infections can be life threatening. Any infection can cause an extended hospital stay, prevent or delay engraftment, and/or cause permanent organ damage.

Preventative measures for common sources of infection are also a part of transplant. This may include any or all of the following: special air filtered rooms, diet restrictions, isolation requirements, restriction of visitors, strict hygiene regimen, frequent linen changes. Other complications can include: low platelets and low red blood cells, pain, fluid overload, respiratory distress, organ damage, graft failure, graft-versus-host disease.

CHOC’s BMT Team

A team of specially trained BMT experts guides each patient and his or her family throughout the BMT process. At CHOC, our BMT team is made up of:

• Blood and Marrow Transplant Medical Director Rishikesh Chavan, M.D., is the pediatric BMT physician who specializes in the care of children undergoing a BMT.
• A group of pediatric hematologists/oncologists assist in the day-to-day medical management of the children during the BMT process.
• BMT Coordinators Our BMT nurse coordinators organize all aspects of care provided to our patients before and after the transplant. They provide patient education, coordinate diagnostic testing and assist with follow-up care and home care needs.
• Social Workers Our social workers provide support to patients and their families to help them deal with the many issues that may arise, including lodging and transportation, finances and legal issues.
• Dietitians Our clinical dieticians assist patients in meeting their nutritional needs before and after the transplant. They work closely with patients and their families to provide nutritional education and suggestions if any nutritional problems arise. Our diet technicians help patients and their families select the patient’s daily food menu and work on any reasonable special requests while patients are in the hospital.
• Psychologists Specially trained psychologists help patients and their families with the emotional aspects of cancer. The psychology staff is dedicated to increasing opportunities for children with cancer and their families to live normal lives during and after hospitalization. Child Life Therapists The Child Life staff provides a wide variety of therapeutic programs to help the patients develop their social, emotional, and cognitive skills. Through play, children learn self-expression, decision-making, creativity and motor development.
• Blood and Donor Services Nursing Staff Registered nurses who are specially trained in apheresis technology and blood collection are with our patients every step of the way. They coordinate and provide nursing care for the blood donor during the entire donation process and for the patient undergoing apheresis procedures.
• Case Coordinator Our case coordinator nurses follow each child’s case and make recommendations for optimal utilization of resources.
• Classroom Teacher A credentialed teacher employed by the Orange Unified School District helps patients maintain their academics during hospitalization.
• Clinical Nurse Specialist A nurse that has a master’s degree in nursing and has specialized training in Pediatric Oncology/BMT works closely with the nursing staff and doctors to help develop a plan of care for the patient.
• Nurse Practitioner Under the direction of the oncologist, the nurse practitioner performs physical examinations as well as diagnostic and therapeutic procedures.
• Radiation Oncologist The radiation oncologist is the physician who evaluates each child’s need for radiation therapy.

Other team members will evaluate the child before transplantation and will provide follow-up care, as needed. These include, but are not limited to, the following: Nursing Staff, Pharmacists, Respiratory Therapists, Physical Therapists, Lab Technicians, Infectious Disease specialists, Dermatologists, Gastroenterologists, Chaplain

Israel, a standout athlete, spent his junior year of high school fighting cancer and returned home just before the onset of COVID-19. Now a senior, he’s working to get back on the football field and grateful to have his cancer journey behind him.

For Dr. Chavan, pediatric oncologist and director of CHOC’s Blood and Marrow Transplant Program, Israel’s excellent recovery from his BMT is a shining example of how most BMT patients eventually resume normal lives.

“A lot of people think of a transplant as a big, bad thing,” Dr. Chavan says.

With the proper medical care, it’s not.

“When you look at Israel today,” Dr. Chavan says, “you would not be able to tell that he had a bone marrow transplant just eight months ago.”