Trial Information

Hematopoietic Stem Cell Transplantation Using Matched Unrelated Donor Peripheral Blood or Bone Marrow for Patients With Hematologic Malignancies

Secondary outcome evaluations for this clinical study include the following:

- To estimate the overall survival in patients with high risk hematological malignancies
who receive a HSCT with an unmanipulated marrow graft or a peripheral blood stem cell
graft

- To estimate disease-free survival and relapse rates

- To estimate the rates of chronic GvHD and graft failure

- To estimate the incidence of non-hematologic peri-transplant regimen-related toxicity
and regimen-related mortality in the first 100 days after transplantation

- To estimate the time to neutrophil and platelet engraftment after transplantation

- To determine the degree of NK cell and T-cell immune reconstitution at 30 days and 100
days post-transplant

- To estimate the incidence of EBV reactivation or post-transplant lymphoproliferative
disease (PTLPD)

- To determine the pharmacokinetics of anti-thymocyte globulin (rATG) in patients
receiving allogeneic transplantation and the development of rATG antibodies

Originally this study began as a randomized comparison between unmanipulated bone marrow and
T-cell depleted bone marrow utilizing the investigational CliniMACS selection system. The
hypothesis to be tested at the time was that the incidence of severe acute GvHD was
significantly reduced in children who received HSCT with a T-cell depleted bone marrow stem
cell graft as compared to those receiving an unmanipulated graft. Approximately midway
through the study the evidence indicated that although the incidence of severe acute GvHD
with T-cell depletion was low, it was not significantly lower than the standard treatment of
unmanipulated bone marrow. Therefore the study was amended to remove the T-cell depleted
arm and continue accrual to one arm providing all patients with an unmanipulated bone marrow
stem cell graft. The primary objective then being to determine if the true incidence of
severe acute GvHD was below 15% as reported. The observational group receiving PBPC
remained open for those patients whose donors or donor centers chose to provide PBPC in lieu
of bone marrow. Only one such patient was assigned to this group; therefore, no valid
conclusions can be formulated.

Intervention analysis was based on those patients who received an unmanipulated stem cell
product only. For this study, the investigators had requested bone marrow for all study
subjects. However, the final determination of the source of the hematopoietic stem cells,
bone marrow or peripheral blood, was at the discretion of the donor and the donor center.
Those participants who received a peripheral blood stem cell product were followed in the
observational group only. All participants, whether recipients of a bone marrow or blood
stem cell product, received the same preparative conditioning regimen