Blood Stem Cell Transplant
Conditions
Brief summary
This study will compare the outcomes of study participants who have had peripheral blood stem cell transplantation or with those who have had bone marrow transplantation from an unrelated donor for the treatment of serious hematological malignancies.
Detailed description
This study is a randomized, open-label, multicenter, prospective, comparative trial of granulocyte colony stimulating factor (G-CSF) mobilized peripheral blood stem cells verses marrow from unrelated donors for transplantation in patients with hematologic malignancies. The study will be comparing two-year survival probabilities between process has many steps after a matched donor is found. The donor also needs to give permission to join this study. Participants will be randomized to either peripheral blood stem cells (PBSC) or bone marrow. The kind of transplant the participant receives is random. Neither the participant nor the doctor may choose the type of transplant. Participants will receive vaccinations for diphtheria, tetanus, hepatitis B and pneumococcus. Blood samples will be drawn as part of a participant's normal care. Blood samples will also be collected to see if infection fighting cells are working. These samples will be collected at 12 weeks, one year and then yearly until three years post transplant. Information will be collected from the participant's medical history. Participants will also have phone interviews conducted and questions will be asked about their physical and emotional health. Participants taking part in this study may be followed for up to 5 years.
Interventions
PROCEDUREBlood stem cell versus bone marrow transplant
1. Cyclophosphamide and Total Body Irradiation (CY-TBI)-based regimens that include at least 120 mg/kg cyclophosphamide and at least 1200 cGy of fractionated TBI. 2. Busulfan and cyclophosphamide (BU-CY)-based regimens that include at least 14 mg/kg busulfan orally or 11.2 mg/kg busulfan intravenously (14 x 0.8 correction factor) or a targeted busulfan dosing strategy aimed at a serum concentration greater than 600 ng/mL at steady state and at least 120 mg/kg cyclophosphamide. 3. Fludarabine and melphalan (Flu-Mel)-based regimens that include a fludarabine dose of least 120 mg per m2 and a melphalan dose of at least 140 mg per m2. 4. Fludarabine, busulfan, and ATG (Flu-Bu-ATG)-based regimens that include a fludarabine dose of at least 120 mg/m2, at least 8 mg/kg busulfan orally or 250 mg/m2 busulfan intravenously, and at least 40 mg/kg equine ATG or 4 mg/kg rabbit ATG. Institutional standards should be followed for targeting plasma levels.
Sponsors
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI)
National Marrow Donor Program
University of Minnesota
University of Florida
Duke University
Ann & Robert H Lurie Children's Hospital of Chicago
Mayo Clinic
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 66 Years
Healthy volunteers
No
Inclusion criteria
Participants may be eligible if they are between the ages of 18 to 66 years and are undergoing unrelated blood stem cell transplantation for the first time for the treatment of their hematological malignancy. Additional eligibility criteria will be gathered and evaluated by the transplant physician to determine if an individual is eligible to take part.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Two-year survival | 2 years | The primary objective is to compare two-year survival probabilities between patients in the two study arms using an intent-to-treat analysis |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Two-year survival | 2 Years | Transplanted patients randomized to the two study arms will be compared for the following endpoints: survival, incidences of neutrophil and platelet engraftment, graft failure, acute graft-versus-host disease (GVHD), chronic GVHD, time off all immunosuppressive therapy, relapse, infections, adverse events, immune reconstitution, and quality of life. Donors in each arm of the study will be compared for time to return to baseline toxicity score, CBC and WBC differential values after donation and quality of life. |
| Neutrophil Engraftment > 500/mcl | 2 Years | — |
| Primary graft failure | 2 years | — |
| Platelet engraftment > 20,000 and 50,000/mcl transfusion independent | 2 years | — |
| Acute GVHD of grades II-IV and III-IV | 2 Years | — |
Countries
United States
Outcome results
None listed