Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)
|Leukemia Myeloproliferative Disorders Myelodysplastic-Myeloproliferative Diseases||Biological: Allogeneic bone marrow transplantation Biological: Peripheral blood stem cell transplantation||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Phase III Randomized, Multicenter Trial Comparing G-CSF Mobilized Peripheral Blood Stem Cell With Marrow Transplantation From Human Leukocyte Antigen (HLA) Compatible Unrelated Donors (BMT CTN #0201)|
- Two-year Overall Survival [ Time Frame: Measured at 2 years ]Overall survival rate at 2 years according to an intention-to-treat analysis.
- Neutrophil Engraftment [ Time Frame: Measured at Day 28 ]
- Platelet Engraftment [ Time Frame: Measured at Day 180 ]
- Graft Failure [ Time Frame: Measured at 28 and 100 days ]
- Extensive Chronic Graft-versus-host Disease (GVHD) [ Time Frame: Measured at 730 days ]
- Chronic GVHD [ Time Frame: Measured at 2 years ]
- Relapse [ Time Frame: Measured at 2 years ]Analysis restricted to patients who received the transplant.
- Infections [ Time Frame: Measured at 1 and 2 years ]Number of infection reports per patient.
- Grades III-V Unexpected Adverse Events [ Time Frame: Measured by 2 years ]
- Acute GVHD Grade II-IV [ Time Frame: 100 days, 180 days ]
- Acute GVHD Grade III-IV [ Time Frame: 100 days, 180 days ]
- Current Immunosuppressive (IS) Free Survival [ Time Frame: Measured at 2 years ]This outcome measure takes into account subsequent immunosuppressive therapy that may occur following discontinuation of initial immunosuppressive therapy.
- Immune Reconstitution [ Time Frame: Measured at 100 days, 6 months, and 1 and 2 years ]
- Donor Recovery of Baseline Complete Blood Count (CBC) and White Blood Cell Count (WBC) Differential [ Time Frame: Measured at 1, 6, and 12 months ]
- Donor Recovery to Baseline Toxicity Scores [ Time Frame: Measured at 1, 6, and 12 months ]
- Donor Quality of Life [ Time Frame: Measured at 1, 6, and 12 months ]
- Patient Quality of Life [ Time Frame: Measured at baseline, 6 months, and 1, 2, and 5 years ]
|Study Start Date:||January 2004|
|Study Completion Date:||April 2014|
|Primary Completion Date:||April 2013 (Final data collection date for primary outcome measure)|
Active Comparator: Bone Marrow Transplant
Allogeneic bone marrow transplantation
Biological: Allogeneic bone marrow transplantation
Bone marrow transplant from HLA compatible unrelated donors.
Active Comparator: Blood Stem Cell Transplant
Peripheral blood stem cell transplantation
Biological: Peripheral blood stem cell transplantation
Peripheral blood transplant from HLA compatible unrelated donors.
Many studies of allogeneic marrow transplantation have shown that a higher dose of marrow cells correlates with more robust hematopoietic engraftment and lower mortality from infectious complications. Peripheral blood stem cells (PBSC) collected after mobilization with granulocyte colony stimulating factor (G-CSF) contain a larger number of CD34-positive (CD34) progenitors and total cells than bone marrow. These observations led to the hypothesis that transplantation of PBSC would lead to lower mortality compared to transplantation of marrow. In addition, PBSC grafts have a higher T cell content, predicting a possibly more powerful anti-leukemia effect. However, the higher T cell content of PBSC may also lead to increased incidence and severity of acute and chronic graft-versus-host disease (GVHD). This concern is especially serious when the donor is unrelated to the recipient. This prospective, randomized, multicenter clinical trial of unrelated donor transplantation will test the hypothesis that transplantation of PBSC leads to similar patient survival compared to transplantation of marrow.
This is a Phase III randomized, open label, multicenter clinical trial sponsored by the National Marrow Donor Program (NMDP) and the National Institutes of Health (NIH). The objective of the trial is to test the null hypothesis that there is no difference in overall survival after PBSC versus marrow transplants from HLA compatible unrelated donors. The study will compare G-CSF-mobilized PBSC transplantation with bone marrow transplantation from HLA-compatible unrelated donors for patients with leukemia, myelodysplastic or myeloproliferative syndromes. Conditioning and GVHD prophylaxis regimens will vary by center and within centers, however, the center must declare before randomization what regimens will be used for each patient. The primary endpoint of this trial is 2-year survival following randomization. Secondary analyses will consider neutrophil and platelet recovery, acute and chronic GVHD, time off all immunosuppressive therapy, relapse, infections, adverse events and immune reconstitution. The trial will include evaluation of patient and donor quality of life, composition of the graft, and immune reconstitution. Accrual is anticipated for 3 years with a follow-up period of 3 years.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00075816
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|Study Director:||Mary Horowitz, MD||Center for International Blood and Marrow Transplant Research|