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

Phase 3
2 Years
21 Years
Not Enrolling
Acute Lymphoblastic Leukemias, Acute Myelocytic Leukemia, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome, Hemoglobinuria, Paroxysmal, Non-Hodgkin Lymphoma

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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

- 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

Inclusion Criteria:

- All patients lacking an HLA identical sibling for whom an unrelated donor matched at
6/6 HLA loci is formally requested within about 3 months of search initiation

- Age greater than or equal to 24 months, but less than 21 years for new patients.

- Diagnosis of one of the following high risk hematological malignancies:

- Acute lymphoblastic leukemia (in second or subsequent remission or high risk in first

- Acute myeloid leukemia (in relapse or remission)

- Secondary AML/MDS

- Chronic myeloid leukemia

- Juvenile myelomonocytic leukemia

- Myelodysplastic syndrome

- Paroxysmal nocturnal hemoglobinuria

- Non-Hodgkin's lymphoma (in second or subsequent remission)

Exclusion Criteria:

- Patients with symptomatic cardiac disease, or evidence of significant cardiac disease
by echocardiogram, or cardiac shortening fraction below 25%

- Patients with renal creatinine clearance < 40ml/min/1.73m^2

- Patients with FVC < 40% predicted or pulse oximetry less than or equal to 92% on room
air if unable to perform pulmonary function tests

- Patients with direct bilirubin > 3 mg/dl

- Patients with SGPT > 500 U/L

- Patients with a Karnofsky or Lansky performance score < 70

- Patients who have received a previous allogeneic stem cell transplant

- Patients with a known allergy to rabbit or murine products

- Patients with isolated extramedullary leukemic relapse, including isolated CNS or
testicular recurrence

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To estimate the occurrence of acute graft versus host disease in patients who have received an unmanipulated hematopoietic stem cell transplant from a matched unrelated donor

Outcome Time Frame:

July 2005

Safety Issue:


Principal Investigator

Gregory Hale, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

St. Jude Children's Research Hospital


United States: Food and Drug Administration

Study ID:




Start Date:

May 2002

Completion Date:

January 2009

Related Keywords:

  • Acute Lymphoblastic Leukemias
  • Acute Myelocytic Leukemia
  • Chronic Myeloid Leukemia
  • Juvenile Myelomonocytic Leukemia
  • Myelodysplastic Syndrome
  • Hemoglobinuria, Paroxysmal
  • Non-Hodgkin Lymphoma
  • High risk hematologic malignancies
  • Allogeneic stem cell transplant
  • Matched unrelated donor transplantation
  • Unmanipulated stem cell graft
  • Bone marrow transplantation
  • Neoplasms
  • Hemoglobinuria
  • Hemoglobinuria, Paroxysmal
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Lymphoma
  • Lymphoma, Non-Hodgkin
  • Myelodysplastic Syndromes
  • Preleukemia
  • Leukemia, Myelomonocytic, Acute
  • Leukemia, Myelomonocytic, Chronic
  • Leukemia, Myelomonocytic, Juvenile
  • Hematologic Neoplasms



St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794