Our Blood and Marrow Transplant Program serves the needs of children who require a blood or marrow transplant to replace stem cells to treat their illness. Transplant is a treatment for a variety of diseases including certain types of pediatric cancer, disorders of the blood and immune system (the system that fights infection) as well as a wide variety of other rare disorders and syndromes.
Our comprehensive program is designed to support you through each step of the transplant process; starting with quick entry into the program. Our dedicated BMT nurse coordinator will manage the time-sensitive care coordination of critically ill children who require either an autologous or an allogeneic transplant as best chance for survival.
Families who decide to come to Cleveland Clinic Children’s for their child’s transplant will receive the highest level of innovative medical, nursing, and supportive care services from a dedicated and highly experienced team. The first bone marrow transplant at the Cleveland Clinic was completed in 1977, with our first pediatric transplant in 1984. This experienced team works together to provide comprehensive, individualized care for each patient. We work collaboratively with a variety of clinical experts for the specific management of your child’s condition before, during, and after transplant. We also have available to us the resources and expertise of Taussig Cancer Center’s Adult BMT program.
Our multidisciplinary team includes experts in the treatment of pediatric cancer as well as diseases and disorders of the blood and immune system. The team includes physicians, nurse practitioners, a nurse coordinator, social worker, psychologist, inpatient and outpatient registered nurses, nutritionists, and child life specialists.
Your child’s treatment group remains actively involved in the oversight of every patient’s care, discussing patients’ needs and treatment on a regular basis. Each week, the team reviews the list of patients currently receiving treatment and makes plans for the upcoming week and month to ensure well-coordinated care.
What We Treat
Bone marrow is the soft, spongy center of the bone that contains the stem cells that produce white blood cells, red blood cells, and platelets. Diseased or damaged blood cells interfere with the production of healthy blood cells and may build up in the body and invade other tissues. A bone marrow transplant can replace unhealthy cells with healthy cells, increasing the chances of a cure.
Cells used in blood and marrow transplant, called stem cells, are obtained from:
- Bone marrow – tissue inside the bone
- Peripheral blood – cells that make up the blood
- Cord blood - cells from the umbilical cord
Many diseases may be treated by transplant including those listed below. We can also treat myelodysplastic syndrome, inborn errors of metabolism, and other acute and chronic leukemias, bone marrow failure syndromes, and immunodeficiencies and immune dysregulation diseases.
- Acute lymphoblastic leukemia (ALL)
- Acute myelogenous leukemia (AML)
- Brain tumors
- Chronic myeloid leukemia (CML)
- Ewing’s tumor
- Hodgkin’s disease
- Juvenile myelomonocytic leukemia (JMML)
- Myelodysplastic syndrome (MDS)
- Non-Hodgkin’s lymphoma
- Relapsed germ cell tumors
- Soft tissue sarcoma
- Aplastic anemia
- Crohn’s disease
- Common variable immune deficiency (CVID)
- Diamond-Blackfan anemia
- Fanconi’s anemia
- Hemophagocytic Lymphohistiocytosis (HLH)
- Hurler’s syndrome
- IPEX syndrome
- Krabbe disease
- Langerhans cell histiocytosis (LCH)
- Multiple sclerosis
- Severe congenital neutropenia
- Sickle cell disease
- Systemic sclerosis
- Severe combined immunodeficiency (SCID)
- Wiskott Aldrich syndrome
The decision about the type of transplant that will be done depends on your child’s disease.
- Autologous transplant – If a child is to receive an autologous stem cell transplant, this means he or she will be getting their own cells for the transplant.
- Allogeneic transplant – If a child is to receive an allogeneic transplant, this means he or she is getting stem cells from either a family member or an unrelated donor. Allogeneic transplants may use bone marrow, cord blood, or peripheral stem cells depending on the donor and your child’s specific needs.
CAR T-Cell Therapy
The immunotherapy, called CAR T-cell therapy, was approved by the Food and Drug Administration in 2017 for use in children and young adults up to age 25 who have relapsed or refractory B cell acute lymphoblastic leukemia (ALL).
- The therapy involves removing certain immune system cells (T cells) from a patient’s blood and re-engineering those cells in a lab so that they can produce a gene that targets a particular receptor on cancer cells.
- Those engineered cells are then infused back into the patient’s blood through an IV, where they multiply and get to work finding and destroying cancer cells.
- Called Kymriah®, the treatment is the first of its kind available in the U.S and can only be administered by certain certified providers such as Cleveland Clinic Children's.
The BMT Process
Unlike organ transplants, blood and marrow transplants are not surgical procedures. They are more similar to blood transfusions. The challenging part of the BMT process is managing the complications following the transplant, when the patient is extremely susceptible to infection.
The typical BMT process is laid out below.
Several important steps take place long before the transplant occurs.
- Pre-transplant consultation: You and your child will meet with one of the transplant physician’s to discuss the potential role of bone marrow transplant in the treatment of your child’s illness.
- Finding a donor: The medical team will look for a relative, or a donor from the National Marrow Donor Program or cord blood registries. With certain diseases, we may utilize your own bone marrow for the transplant.
- Pre-transplant evaluation: Before the BMT process begins, your child will need to have an extensive evaluation to determine his or her eligibility for transplant. This evaluation will happen over several visits and includes lab testing, imaging, and consults with other services.
- Harvesting stem cells: Once a donor is identified, stem cells are harvested. If the donor is a relative, our team will meet with them separately to review the donation process.
- Pre-transplant conference: You will meet with one of the transplant physicians to discuss your child’s workup evaluation results and finalize a treatment plan.
When transplant time arrives:
- Preparation regimen: Your child will be admitted to the BMT unit a few days prior to transplant to receive a preparatory regiment that includes high doses of chemotherapy and/or radiation with the appropriate supportive treatment and diagnostic laboratory workup.
- Transplant day (Day 0): During the transplant day, stem cells that have been previously collected are given to the patient. The transplant itself occurs in the patient’s room and is similar to a blood transfusion.
During the weeks after transplant day, the patient’s body is unable to produce white blood cells increasing his or her risk of infection. Depending on the transplant type, it can take several weeks for the new stem cells to begin producing enough blood cells to protect the body from germs. During this period, patients are closely monitored in protective isolation on the BMT unit and receive medications to prevent infection until their new “healthy” cells can take over. While waiting on new stem cells to grow into new healthy blood cells, all patients will receive aggressive monitoring and supportive care.
Your child’s healthcare team will work with you to determine when it is time for your child to leave the hospital. This varies depending on the type of transplant and how patients are feeling and progressing after the BMT. Patients should expect frequent appointments with their transplant team for at least the first 100 days post-transplant. It is important to know that full recovery can take months or even years beyond that time. If your child is treated outside of Cleveland Clinic Children’s, our team will determine when it is safe to return to local care.
Depending on the type of transplant your child receives, you may need to stay within a one-hour commute to Cleveland Clinic Children’s for 60 to 100 days following the procedure. Some insurance companies provide a travel and lodging benefit that covers at least a portion of these expenses. If you need help arranging accommodations, our social worker is available to assist you in working with support services.
Our team will monitor your child’s health and well-being with comprehensive annual follow-ups after your child goes home.
Becoming a Bone Marrow Donor
Patients all over the world facing bone marrow transplant procedure need people like you to become a bone marrow donor, to donate blood stem cells or cord blood, or to help in other ways. All bone marrow transplant survivors have people like you to thank for their new opportunities. Learn more about how you can help.
Programs and Services
Pediatric Hematology Oncology provides support programs for patients and their families dealing with cancer and blood disorders. We are constantly striving to cure children while improving the quality of their lives. Learn more about our social service programs designed to improve patient outcomes. These programs and services can be facilitated through the work of our dedicated social workers. Learn more about our programs.
Nursing / Supportive Staff
- Lyndsay Pankratz, CNP
- Rose Miller, RN
Blood & Marrow Transplant Care Coordinator
- Milvon Wright, MSW, LSW
Blood & Marrow Transplant Social Worker
- Gwen Barron, RD
- Sarah Bewley, RD
- Lisa Bell, RN, BSN
Nurse Manager, Inpatient Unit
- Kristen Powaski, RN, BSN
Nurse Manager, Outpatient Unit
Below is a list of open bone marrow transplant trials available at Cleveland Clinic Children’s Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation. This list is constantly changing, so please contact Dr. Rabi Hanna at 216.445.4831.
- CBMT1Z15: Natural History and Biology of Long-Term Effect Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies
- FHCC1Z14: Multi-center, Open-label Randomized Study of Single or Double Myeloablative Cord Blood Transplantation with or without Infusion of Off-the-shelf Ex Vivo Expanded Cryopreserved Cord Blood Progenitor Cells in Patients with Hematologic Malignancies
- JAZZ1Z16: A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Preventative of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant
- MESO1Z16: A Single-arm, Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD
- THKO1Z15: Single-Arm Study to Assess the Efficacy of UVADEX (methoxsalen) Sterile Solution in Conjunction with the THERAKOS CELLEX Photopheresis System in Pediatric Patients with Steroid-Refractory aGVHD
Patients of our Blood and Marrow Transplant Program can be seen at Cleveland Clinic Main Campus:
Cleveland Clinic Main Campus
8950 Euclid Ave
Cleveland, OH 44195
Patients can also be seen via Virtual Visit. Patients use the Cleveland Clinic Express Care® Online tool to see our providers online from the comfort of their home. This service allows patients a fast, secure, and easy way to receive care from their healthcare team in a live virtual visit using a smartphone, tablet, or computer. The benefits of choosing a virtual visit include no travel or parking, less waiting, significant time savings, no facility fee and the convenience of seeing your physician from wherever you choose.
For information or to make an appointment for an in-person or virtual visit, please call 216.444.5517.
Note: All patients looking to schedule virtual visits must have a Cleveland Clinic Medical Record Number (MRN). If you do not have a Cleveland Clinic MRN, please call the appropriate number below and they will be able to assist you in getting your record created.
- USA: 216.444.5437
- International (including Canada): 216.444.8184