As the director of the Children's Hospital Blood and Marrow Transplant (BMT) Program, I care for patients who will not be cured without a BMT. I also am involved in clinical research to improve outcomes and decrease risks for our transplant patients.
Our bone marrow transplantation program at The Children's Hospital of Philadelphia is one of the largest and oldest pediatric bone marrow transplant programs in the U.S., with more than 1,200 transplants performed. Our program provides the unique combination of comprehensive patient treatment, multidisciplinary teamwork, innovative research and family support. We have 11 beds in HEPA-filtered rooms in a special HEPA-filtered unit which includes a playroom and family lounge. Our physicians, radiation oncologists, nurse practitioners, donor search coordinator, social worker, nutritionists and child life specialists all work together to provide comprehensive care for each patient.
At Children's Hospital, we are expert in everything related to blood and marrow transplants. We have the ability to collect autologous (self) hematopoietic stem cells from very small children for use in high dose chemotherapy protocols. We have expertise in allogeneic transplants, with matched related donors, unrelated donors and cord blood.
We have various clinical studies open. Our research efforts are directed toward providing better outcomes for children who receive bone marrow transplantation. We have developed special protocols to reduce the risks of complications from graft vs. host disease (GVHD) with an unrelated or partially matched related donor, which gives us the ability to use donors other institutions may not be able to use.
Translational research forms an important part of our program. A drug, sirolimus, was found to be effective in killing lymphoid leukemia in the lab of one of our physicians. This drug was added post transplant to patients with leukemia post transplant to improve outcomes. This pilot study was then taken to the Children's Oncology Group, and is now an important randomized protocol which may improve leukemia free survival. We participate with the University of Pennsylvania's Abramson Cancer Center, and studies have been done to show that donor cells activated by a special process and then given to the patient with relapsed leukemia or lymphoma may have a potent anti-cancer effect. These studies are being expanded.
Our program is accredited by the Foundation for Accreditation of Cell Therapy (FACT). All three components -- clinical, cell collection and stem cell laboratory -- are accredited. FACT accreditation is an extremely rigorous process, and there are few pediatric centers that have all three components on site.
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