Stem Cell Transplantation Services
The Pediatric Stem Cell Transplantation program at Penn State Children's Hospital provides high quality, comprehensive services to infants, children, adolescents and young adults whose treatment requires a stem cell transplant. The Stem Cell Transplant Program at Penn State was established two decades ago to allow patients and their families to receive state of the art care while remaining close to home. The pediatric transplant physicians have many years of experience caring for stem cell transplant patients, and much of your child's care is provided directly by the attending physician staff and nurse practitioner. In fact, a transplant doctor is available 24 hours a day, 7 days a week to answer your questions and to actually see your child if the need arises after hours. this hands-on approach by our experienced transplant staff results in high quality care, with potential problems being addressed immediately by staff who appreciate the special needs of transplant patients. We are accredited by the Foundation for the Accreditation of Hematopoietic Cell Therapy (FACT), are members of the Pediatric Blood and Marrow Transplant Consortium and the Children's Oncology Group. In addition, the Penn State Children's Hospital Pediatric Stem Cell Transplantation Program is considered a stem cell transplant center of excellence by many major insurance companies.
Children receiving stem cell transplants are admitted to our 15 bed, HEPA filtered west wing of the seventh floor of Children's Hospital. This unit is specially designed to care for high risk hematology-oncology and stem cell transplant patients. Care is provided by a dedicated team of pediatric stem cell transplant nurses, nurse practitioners, and physicians. New patient consultations and post-transplant care is provided in a separate pediatric stem cell transplant clinic, which is conveniently located on the same floor as the inpatient unit.
By participating in research studies performed by the Children's Oncology Group and the Pediatric Blood and Marrow Transplant Consortium, as well as Penn State transplant physician initiated protocols, our program provides patients with access to a number of cutting-edge research studies. We not only offer a full array of stem cell transplant services, but also a number of novel research studies, including allogeneic stem cell transplant for recurrent solid tumors, adoptive cellular immunotherapy for viral infections post transplant, cord blood transplantation, and cancer vaccines.
The Pediatric Stem Cell Transplant program at Penn State offers a variety of support services for our patients and families. The Four Diamonds Fund, which sponsors THON, the largest student run a charity in the nation, raises over $7 million per year, which goes directly to support our very own Pediatric Hematology-Oncology and Stem Cell Transplant Program here at Penn State Hershey. These funds help our families directly with the challenges they may face in caring for a child who has received stem cell transplant, as well as supporting our clinical and research programs. In addition, we have a team of dedicated nutritionists, pharmacist, child life specialists, psychologists, social workers, and a teacher who are available to help you and your child through this difficult time. Short and long-term follow-up care is provided by a multidisciplinary team, including pediatric pulmonology, cardiology, endocrinology, ophthalmology, dermatology, as well as other pediatric subspecialties.
Penn State Children's Hospital
Pediatric Hematology Oncology
Stem Cell Transplant Program
PO Box 850, M.C. H085
Hershey, PA 17033
Monday - Friday
8:00 a.m. - 4:30 p.m.
After Hours and Weekend:
Ask to speak with the Pediatric Stem Cell Transplant Attending Physician on-call
The Pediatric Stem Cell Transplant Program is composed of a multi-disciplinary team of specialists that are all dedicated to the overall health and well-being of the patient and their families. The team is comprised of transplant physicians, trained nurses, pharmacists, social workers, child life specialists and a dietician. For school age children, a school teacher is available. Our team participates in national research and are members of the Children's Oncology Group and Pediatric Blood and Marrow Consortium. All of these caring professionals are available and willing to assist the patient and families with any needs that arise.
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.
Research at Penn State's Pediatric Stem Cell Transplant program is involved in several clinical trials, many originating here at our hospital. We also actively participate with national groups studying stem cell transplant, and are member of the Children's Oncology Group and the Pediatric Blood and Marrow Transplant Consortium. Clinical research is being conducted on cord blood transplantation, graft versus host disease prevention, the use of allogeneic stem cell transplant for patients with therapy resistant solid tumors, as well as post transplant immunotherapy. Basic research in our program is focused on developing adoptive immunotherapy strategies that can be applied to pediatric stem cell transplant patients for leukemic relapse as well as viral infections.
Current Open Studies
|Principal Investigator||Title of Study|
|Kenneth Lucas, MD|| The Use of Umbilical Cord Blood as a Source of Hematopoietic Stem |
|Melanie Comito, MD|| |
Dose intensive Chemotherapy for children less than 10 years of age -
|Kenneth Lucas, MD|| A phase I study to examine the toxicity of allogeneic stem cell |
transplantation for Pediatric solid tumors with relapsed or therapy
|Melanie Comito, MD|| High dose temozolomide, thiotepa and carboplatin with autologous stem |
cell rescue (ASCR) followed by continuation therapy with 13-cis-retinoic
acid in patients with recurrent/refractory malignant brain tumors.
|Kenneth Lucas, MD|| A Phase I Trial to Examine the Safety, Clinical, Immunologic and |
Virologic Effects of CMV pp65 Specific Cytotoxic T Lymphocytes for
Recipients of Allogeneic Stem Cell Transplants with Persistent or
Therapy Refractory Infections.
|Kenneth Lucas, MD|| A Phase I-II Randomized Trial to Examine the Clinical, Immunologic, and |
Virologic Effects of CMV Specific Cytotoxic T Lymphocytes When Used for
Prophylaxis Against CMV Disease in Recipients of Allogeneic, T Cell
Depleted Stem Cell Transplants.
|John Neely, MD|| A Randomized Trial of Sirolimus-Based Graft versus Host Disease |
(GVHD) Prophylaxis after Hematopoietic Stem Cell Transplantation
(HSCT) in Relapsed Acute Lymphoblastic Leukemia (ALL).
Some Publications from Our Program:
- Lucas, KG, Salzman DE, Garcia A, sun Q. Adoptive Immunotherapy with allogeneic Epsterin Barr Virus-specific Cytotoxic T Lymphocytes for Relapsed, EBV-positive Hodgkin's Disease. Cancer 100: 1892-1901, 2004.
- Comito MA, Sun Q, Lucas KG. Immunotherapy for Epstein Barr Virus-associated Tumors. Leukemia and Lymphoma. 45:1981-1987, 2004.
- Lucas KG, Nelson JL, Erickson TD, Sun Q. Microchimerism detection by human leucocyte antigen-specific quantitative-polymerase chain reaction analysis in recipients of allogeneic Epstein-Barr virus-specific cytotoxic T lymphocytes. Br J Haematol. 129(3):443-4; 2005
- Lucas KG, Ungar D, Comito M, Bayerl M, Groh B. Submyeloablative cord blood transplantation corrects clinical defects seen in IPEX syndrome. Bone Marrow Transpl, 39: 55-6, 2007.
- Bao L, Sun Q, Lucas KG. Rapid Generation of CMV pp65 Specific T cells for Immunotherapy. Journal of Immunotherapy. 30: 557-561; 2007.
- Sun Q, Brewer N, Dunham K, Chen L, Bao L, Burton R, Lucas KG. Interferon-gamma expressing EBV LMP2A-specific T cells for cellular immunotherapy. Cellular Immunology, 246(2): 81-89, 2007.
- Lucas KG, Schwartz C, Kaplan J. Allogeneic Stem Cell Transplantation in a Patient with Relapsed Ewing's Sarcoma. Ped Blood and Cancer 51(1):142-4, 2008.
- Bao L, Dunham K, Stamer M, Mulieri K, Lucas KG. Expansion of CMV pp65 and IE-1 Specific Cytotoxic T Lymphocytes for CMV Specific Immunotherapy Following Allogeneic Stem Cell Transplant. Biology of Blood and Marrow Transplantation. 14(10):1156-62, 2008.
- Bao L, Lucas KG. Fusion of B lymphoblastoid and tumor cells expressing different antibiotic resistance genes facilitates selection of stable hybridomas. Hybridoma 27(5):401-5, 2008.
- Horn B, Dunham K, Stamer M, Cowan M, Lucas KG. Infusion of cytomegalovirus specific cytotoxic T lymphocytes from a sero-negative donor can facilitate resolution of infection and immune reconstitution. Ped Infectious Disease J 28: 65-67, 2009.
For questions about these or other studies, please contact Catherine Byrnes at 717-531-6012.
Why Stem Cell Transplant?
Stem cell transplantation in cancer treatment makes it possible for patients to receive higher doses of chemotherapy and/or radiation than would otherwise be possible. It is used most often as a treatment modality when conventional treatments have been unsuccessful in eradicating the cancer. Stem cell transplant can also be used as a type of immunotherapy, since some pediatric cancers can be successfully treated by taking advantage of the healthy donor’s immune response to the patient’s cancer.
There are several different sources of stem cells that are used in transplant, including unrelated adult donor bone marrow, unrelated umbilical cord blood, bone marrow or peripheral blood cells from a related donor, and stem cells collected from the patient prior to transplant (autologous transplant). Bone marrow, located in the center part of the bones, is where blood cells are made. Stem cells are produced in the marrow and mature to become red blood cells, white blood cells and platelets. As they mature, they are released into the blood where they perform their specific functions.
In the process of stem cell transplantation, high doses of chemotherapy and/or radiation are given that destroy the bone marrow's normal production of blood cells. Stem cells or bone marrow cells that have been collected are then given back to restore the body's production.
Stem cell transplantation is also used in children with deficiencies in the immune system, blood cell production, and with certain metabolic disorders. In these cases, transplanting the stem cells replaces a deficient component of the immune system or other components of the blood. Stem cell transplantation using the child’s own stem cells is also used for certain patients with malignant solid tumors that are at high risk for relapse, such as stage IV neuroblastoma and recurrent medulloblastoma. In these cases, stem cells are collected from the patient, frozen, and re-infused into the patient following high-dose chemotherapy.
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.
- Total body irradiation
- Anti-thymocyte globulin
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.
Mucositis (mouth and throat sores)
Nausea /inability to eat
Graft versus host disease (GVHD)
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.
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.