Stem Cell Transplantation Services

A multidisciplinary team provides comprehensive care for pediatric stem cell transplant patients. The team forms a cohesive network to meet the complex requirements of the children and families going through stem cell transplant. This team consists of physicians, nurse practitioners, nurses, pharmacists, social workers, child life specialists, a dietician, and a psychologist who specialize in pediatrics and stem cell transplant.

Outpatient Clinic

For questions about these or other studies, please contact Catherine Byrnes at 717-531-6012.

The pre-transplant evaluation and donor search is a process that may take several weeks to months to complete. At the first step is to have you and your child come to Penn State for a consultation. During this visit, we will explain the reasoning behind stem cell transplant for your child's disorder, potential alternatives, as well as give information on chemotherapy/radiation and options for stem cell donors. The stem cell transplant physician will evaluate your child's past medical records and make sure that stem cell transplant is the best way to proceed. During his/her first visit blood work may be drawn, and we will provide you with written information and other resources for you to further understand the transplant process. If your child be receiving an allogeneic stem cell transplant, we will explain the donor search process, and different options we have for choosing a donor. If your child is receiving autologous transplant, we will discuss arrangements that will be made for collecting your child's stem cells before being admitted for the transplant. The stem cell transplant coordinator will be your primary contact person for the entire pre-transplant process, making appointments for additional evaluations /blood work as needed. The coordinator works closely with a financial counselor and social worker, to make sure all issues regarding your insurance company, timing of transplant, in any special needs your family should have are addressed prior to your transplantation admission. You should expect at least three discussions with the stem cell transplant staff at separate visits before being asked to sign a consent form to proceed with the stem cell transplant procedure. Please feel free to contact us anytime if questions or issues arise during the pre-transplant process.

Patients are admitted for stem cell transplant on 7 west in the Children's Hospital.  Most likely you will be admitted from clinic, potentially on the same day that you are asked to sign consent for the stem cell transplant, or having a central venous catheter placed. The stem cell transplant coordinator will make all arrangements for your child's admission.

The transplant preparative regimen

The preparative /conditioning regimen refers to the high-dose chemotherapy and/or radiation that patients receive before their stem cell transplant. There are several goals for this therapy prior to the stem cell transplant. For cancer patients, the primary goal of the chemotherapy and/or radiation is to destroy cancer cells.  Your child's immune system needs to be adequately suppressed, in space needs to be made for the new, healthy stem cells, in order for the cells to grow in your child's body. You will receive specific information on each of the chemotherapy drugs that your child will receive. Each of these agents is associated with its own unique side effects, and these potential problems will be explained to you in detail during the pre-transplant consultation. You should feel free to ask questions any time.

The day on which the stem cells are infused is considered day 0 of the stem cell transplant. All days before the stem cell transplant are referred to with a minus sign, and all days after the transplant are referred to with a “+” sign. The stem cells are given intravenously through your child's central venous catheter after the conditioning regimen is completed. These cells are infused like a blood transfusion. Your child will remain in a protective environment as an inpatient until the cells have grown and your child has recovered from the side effects of chemotherapy and/or radiation. The usual total time for hospitalization ranges from 25 to 50 days.

Your child is considered to have achieved engraftment when the absolute neutrophil count is greater than 500 for three days in a row. Generally platelets and red blood cell engraftment occur several weeks or even months later. While your child's white cell count may be nearly normal at the time of discharge from hospital, it is important to remember that several important components of the immune system, particularly those that fight viral and fungal infections, are very suppressed. For this reason, even after your child is discharge from the hospital, it is important to protect him/her from potential sources of infection.

Discharge planning

Prior to your child's discharge from the hospital, the stem cell transplant nurses, nurse practitioner, and attending will go over what you need to know to care for your child as an outpatient , as well as all of your child's medications, plan for follow-up, and what you need to do to get your home environment ready. Your child will require a restricted lifestyle after discharge, and will not be able to visit indoor public places, or attend school or other social functions until you're attending physician indicates to you that this would be safe. The timeframe during which this isolation is required will vary, based on the type of transplant and what degree your child's immune system is suppressed. In general, autologous transplant recipients are able to resume school and lead more of a normal life sooner (usually 3 to 4 months post-transplant). For allogeneic stem cell transplant recipients, the period of restriction usually lasts a bit longer, due to the fact that these children have lowered immune systems and are more susceptible to serious, potentially life-threatening infections. Your transplant doctor will discuss the appropriate timing for your child to resume school and other activities with you and your family, depending upon your child's clinical situation.

If your child is being considered for an allogeneic stem cell transplant, one of the first priorities is to determine who will serve as the stem cell donor. The chance of one of your child's siblings being a perfect match is roughly 25%, and the chances of another relative being suitably matched are much lower. The first step is to perform HLA typing on your child as well as any potential stem cell donors. HLA typing refers to the detection of specific proteins found on the surface of white blood cells. These proteins are different from those used to type red blood cells for transfusion.  It is not important that a patient's stem cell donor be matched for these red blood cell proteins. We will initially take blood from your child and any full siblings to see if they are a match. If they are not a match, we will search for an unrelated adult donor through the National Marrow Donor Program as well as look for matches in the the umbilical cord blood registries.

Unrelated Donor Transplantation

To the most commonly used sources of unrelated donor stem cells are from a living, unrelated donor or from umbilical cord blood cells that have been frozen and kept in a cord blood bank. The search for an unrelated adult donor can sometimes take one to two months, or sometimes longer if we have difficulty finding a match, or if additional testing is required. Unrelated adult donors can come from anywhere in the world. The bone marrow or peripheral blood stem cells are collected in the donor’s home area, and then transported under strict conditions  to Penn State Children's Hospital for transplantation. These cells are administered like a blood transfusion through your child's central venous catheter. The identity of this donor cannot be released until at least one year post-transplant, but you and your child are welcome to exchange letters with your unrelated donor, through the stem cell transplant coordinator and the National Marrow Donor Program. On year after your transplant, you will be able to meet your child's unrelated donor, if both parties wish to do so.

Many children receive unrelated donor cord blood transplants, since there are many of these units available worldwide.  The time required to acquire a cord blood unit is usually much shorter than with an unrelated adult donor. Limiting factors in determining whether a cord blood unit is suitable for transplantation include the HLA typing as well as the size of the cord blood unit. The donation process for the cord blood units is completely anonymous, and you will not be able to meet the donor or his/her family. As with an unrelated adult donor, cord blood units are screened for multiple types of infection, such as viruses. These units are shipped under strict conditions to Penn State Children's Hospital, thawed, and then infused into your child's central venous catheter like a blood transfusion.