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In 2011, Dana-Farber/Children's Hospital Cancer Center's Pediatric Stem Cell Transplantation Program performed 93 stem cell transplant procedures, making the program one of the largest in the nation. The program is also among the country's safest, with one-year survival after transplant consistently exceeding 90 percent.
"Because of the intersection in expertise between our multispecialty children's hospital and National Center Institute -designated cancer center, our ability to help children navigate transplant is unparalleled," says Leslie E. Lehmann, MD, clinical director of the Pediatric Stem Cell Transplantation Program.
Established in the 1970s, the Pediatric Stem Cell Transplantation Program is one of the oldest and most experienced pediatric transplant centers. The program, at the forefront of transplantation research, has been designated as a Center of Medical Excellence by the Bone Marrow Transplant Quality Review Committee at UniCare, based on high care quality, high patient volume and a high number of positive long-term outcomes.
A range of conditions treated
Boston Children's 13-bed Stem Cell Transplant Unit treats children with a wide range of conditions, including many types of cancer and certain hematologic, immunologic and genetic disorders. The most common indications for stem cell transplant include acute lymphocytic leukemia (ALL) and other leukemias, severe combined immunodeficiency (SCID), bone marrow failure disorders (e.g. aplastic anemia), disorders of red blood cell production (e.g. sickle cell disease or thalassemia) and some solid tumors.
In malignant disorders, there are two primary reasons transplant is effective. First, it allows physicians to give higher doses of chemotherapy and then "rescue" patients with the stem cell transplant. Second, it's thought in some cases that the new bone marrow may exert a graft-versus-cancer effect on the recipient's immune system.
Stem cell transplant also makes it possible to correct certain genetic defects in the bone marrow using donor cells. For this reason, stem cell transplant represents a curative option for sickle cell disease, thalassemia, severe combined immune deficiency syndrome and various other genetic disorders.
Stem cell transplants may be either autologous (cells from the patient themselves) or allogeneic (cells from a compatible donor) depending on the condition treated. "We have a very strong donor search service, and we use both bone marrow donors and cord blood donors," says Dr. Lehmann. "This means that we are able to find an acceptable donor for virtually every patient."
Reducing the risks
The Stem Cell Transplantation Program's long history of transplant experience has made the procedure much safer for today's patients. "Twenty years ago," says Dr. Lehmann, "up to half of patients undergoing unrelated stem cell transplant unfortunately died of complications. Today, that number is fewer than 20 percent nationwide and less than 10 percent at Dana-Farber/Children's Hospital Cancer Center. Transplant has become a much safer and better process over the years."
Outstanding supportive care provided by a team of experienced specialists is one of the most important factors in guiding children safely through transplant. "At Dana-Farber/Children's Hospital Cancer Center, we have a true multidisciplinary team. Every patient is cared for by a combination of a primary care nurse, stem cell transplant attending, nutritionist, pain expert and pharmacist. This team rounds with us regularly," says Dr. Lehmann.
As a result of this multidisciplinary approach, children undergoing transplants today tend to have very positive outcomes overall. "The vast majority of children both psychologically and medically," explains Dr. Lehmann. For those patients who do experience complications of transplant, Dana-Farber/Children's Hospital Cancer Center has established the Graft Versus Host Disease Clinic, the only clinic in the country dedicated to following children with transplant complications.
A partnership with pediatricians
"Transplants are among the most complex medical procedures that are done today," says Dr. Lehmann." They require a multidisciplinary team of which the primary care provider is a key piece." Following the initial diagnosis, primary care providers play a critical role in informing the transplant team about the medical and psychosocial history of the patient.
During transplantation, pediatricians are an important source of continuity and support for patients and their families. "Providers and patients should be aware that transplant is a very long process, a marathon not a sprint," cautions Dr. Lehmann. "The psychological, emotional and financial impact of transplant on patients and families is enormous, and they need support throughout the process." The Stem Cell Transplantation Program coordinates regularly with primary care providers throughout treatment. "We take communication with the referring physicians as one of our highest priorities, and weekly updates are sent out to keep primary care providers up-to-date," says Dr. Lehmann. "Because we work so closely with our referrers, we are able to transition patients back home more quickly."
Finally, after transplantation, pediatricians will resume the bulk of patient care. Specialists at the Stem Cell Transplantation Program are available to answer any questions providers may have and to consult on any transplant-related needs or complications following treatment.
The Stem Cell Transplantation Program is committed to helping primary care providers learn more about the procedure. "We are delighted to provide pediatricians with a tour of the Stem Cell Transplant Unit at any time or to answer any questions they may have," says Dr. Lehmann.
More information:
childrenshospital.org/sct
Make a referral: 617-632-3961 | 1-888-PEDI-ONC (733-4662)
pedi_sct@dfci.harvard.edu
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