Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas
Status:
Completed
Trial end date:
2011-12-14
Target enrollment:
Participant gender:
Summary
This study will examine the safety and effectiveness of stem cell transplantation for
treating patients with sarcomas (tumors of the bone, nerves, or soft tissue). Stem cells are
immature cells in the bone marrow and blood stream that develop into blood cells. Stem cells
transplanted from a healthy donor travel to the patient's bone marrow and begin producing
normal cells. In patients with certain cancers, such as leukemia and lymphoma, the donor's
immune cells attack the patient's cancer cells in what is called a "graft-versus-tumor"
effect, contributing to cure of the disease. This study will determine whether this treatment
can be used successfully to treat patients with sarcomas.
Patients between 4 and 35 years of age with a sarcoma that has spread from the primary site
or cannot be removed surgically, and for whom effective treatment is not available, may be
eligible for this study. Candidates must have been diagnosed by the age of 30 at the time of
enrollment. They must have a matched donor (usually a sibling). Participants undergo the
following procedures:
Donors: Stem cells are collected from the donor. To do this, the hormone granulocyte colony
stimulating factor (G-CSF) is injected under the skin for several days to move stem cells out
of the bone marrow into the bloodstream. Then, the cells are collected by apheresis. In this
procedure the blood is drawn through a needle placed in one arm and pumped into a machine
where the stem cells are separated out and removed. The rest of the blood is returned to the
donor through a needle in the other arm.
Patients: For patients who do not already have a central venous catheter (plastic tube), one
is placed into a major vein. This tube can stay in the body the entire treatment period for
giving medications, transfusing blood, , withdrawing blood samples, and delivering the
donated stem cells. Before the transplant procedure, patients receive from one to three
cycles of "induction" chemotherapy, with each cycle consisting of 5 days of fludarabine,
cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone followed by at least a
17-day rest period. All the drugs are infused through the catheter except prednisone, which
is taken by mouth. After the induction therapy, the patient is admitted to the hospital for 5
days of chemotherapy with high doses of cyclophosphamide, melphalan, and fludarabine. Two
days later, the stem cells are infused. The anticipated hospital stay is about 3 weeks, but
may be longer if complications arise. Patients are discharged when their white cell count is
near normal, they have no fever or infection, they can take sufficient food and fluids by
mouth, and they have no signs of serious graft-versus-host disease (GVHD)-a condition in
which the donor's cells "see" the patient's cells as foreign and mount an immune response
against them.
After hospital discharge, patients are followed in the clinic at least once or twice weekly
for a medical history, physical exam, and blood tests for 100 days. They receive medications
to prevent infection and GVHD and, if needed, blood transfusions. If GVHD has not developed
by about 120 days post transplant, patients receive additional white cells to boost the
immune response. After 100 days, follow-up visits may be less frequent. Follow-up continues
for at least 5 years. During the course of the study, patients undergo repeated medical
evaluations, including blood tests and radiology studies, to check on the cancer and on any
treatment side effects. On four occasions, white blood cells may be collected through
apheresis to see if immune responses can be generated against the sarcomas treated in this
study. Positron emission tomography (PET) scans may be done on five occasions. This test uses
a radioactive material to produce images useful in detecting primary tumors and cancer that
has spread.