Allogeneic Bone Marrow Transplantation With Matched Unrelated Donors for Patients With Hematologic Malignancies Using a Preparative Regimen of Busulfan, Cyclophosphamide, and Fludarabine
Patients who have cancer of the bone marrow (leukemia) or lymph gland (lymphomas) are being
asked to take part in this study. This study uses a new chemotherapy regimen and matched
volunteer stem cell transplant to treat patients with cancers of this kind.
High dose chemotherapy, followed by a transplant of stem cells collected from either bone
marrow (BMT) or peripheral blood of an HLA (tissue type) matched unrelated donor, offers a
potential cure for several serious blood diseases including acute and chronic leukemia,
myelodysplastic syndrome and lymphoma.
However, the success of allogeneic (unrelated volunteer matched donor) transplant is limited
by treatment related illness, death, and relapse in patients with refractory (resistant)
disease and/or advanced age (older than 40 years).
Patients being sought for this study do not have an HLA-matched relative to donate stem
cell; however, through The National Marrow Donor Program or International Registries, an
HLA-matched unrelated donor has been found to donate stem cells. Preliminary data shows
that the outcome is comparable between bone marrow and peripheral blood stem cell
transplantations, and donor preference will determine which type of cells the patient
To carry out an unrelated transplant, a normal person with similar HLA type must be
available to donate cells. The patient must be prepared to accept the donor cells by
decreasing the body's immune systems. In addition, the malignant (cancerous) cells must be
destroyed to allow growth and repopulation of healthy donor cells. This is usually done by
giving total body irradiation and chemotherapy, which is associated with severe side effects
in patients over age 40.
In other studies, it has been found that treating the patient with two anticancer drugs,
Busulfan and Cyclophosphamide, without total body irradiation, is safe and sufficiently
immunosuppressive. High dose Busulfan and Cyclophosphamide are frequently used in
combination prior to matched related bone marrow transplant in case of acute leukemia,
myelodysplastic syndrome (MDS), Hodgkin's disease (HD), and chronic myeloid leukemia (CML).
Fludarabine (another anticancer drug) suppresses the immune system and has strong anticancer
effect against chronic lymphocytic leukemia (CLL) and non-Hodgkin's lymphoma (NHL).
Researchers are studying a new chemotherapy regimen that includes fludarabine, along with
busulfan and cyclophosphamide, to provide more immunosuppression and anti-cancer effect
without increasing the chemotherapy related toxicity in patients with CML, acute leukemia
and MDS, CLL, NHL, and HD.
The purposes of this study are: 1) to decrease the side effects due to the chemotherapy used
to treat the bone marrow; and 2) to increase the number of tumor cells killed. There will be
up to 55 patients enrolled in this study at Emory University. The expected duration of each
subject's participation in this study will be for two years.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Assess activity of preparative regimen in patients with CML, AML, RAEB, RAEB-t, CLL, NHL, HD, in all patients with refractory anemia, ringed sideroblasts, undergoing allogeneic stem cell transplantation from volunteer matched unrelated donor (VUD).
Amelia Langston, MD
Emory University Winship Cancer Institute
United States: Institutional Review Board
|Emory Universtiy Winship Cancer Institute||Atlanta, Georgia 30322|