Phase II Trial of Targeted Immune-Depleting Chemotherapy and Reduced-Intensity Allogeneic Hematopoietic Stem Cell Transplantation Using 8/8 and 7/8 HLA-Matched Unrelated Donors and Utilizing Two Graft-versus-Host Disease Prophylaxis Regimens for the Treatment of Leukemias, Lymphomas, and Pre-malignant Blood Disorders
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Steven Z. Pavletic, M.D., M.S.
Zetta Blacklock, R.N., B.S., B.S.N.
Sheila Phang, R.N., M.S.
Key Eligibility Criteria:
- Diagnosis of advanced or high-risk hematologic malignancies including acute myelogenous leukemia (AML), acute lymphocytic leukemia (ALL), myelodysplastic syndrome (MDS), chronic lymphocytic leukemia (CLL), prolymphocytic leukemia (PLL), non-Hodgkin lymphoma (NHL), Hodgkin lymphoma (HL), Burkitt or acute lymphoblastic lymphoma, chronic myelogenous leukemia (CML), multiple myeloma (MM), and myeloproliferative disorders (MPD)
- 18 to 74 years
- No suitable HLA-matched sibling
- An unrelated donor matched at a minimum of seven of eight alleles (HLA-A,-B,-C, and DRB1) by high-resolution typing, identified through the National Marrow Donor Program
- Life expectancy of at least 3 months
- ECOG ≤ 2
- Relatively normal major organ functions
- No active central nervous system (CNS) involvement by malignancy
- No other disease that would preclude study participation
- No active infections
- Patients receive one of two induction chemotherapy regimens (EPOCH-F/R or FLAG) based on diagnosis before transplant to treat the cancer and suppress immune function
- Conditioning regimen of cyclophosphamide for 4 days and fludarabine for 4 days before stem cell therapy (SCT) to prepare for the transplant
- Patients are randomly assigned to one of two combination drug treatments to prevent graft-versus-host disease GVHD as follows:
- Group 1: Tacrolimus starting 3 days before SCT and continuing for 6 months, plus methotrexate on days 1, 3, 6, and 11 post-SCT, plus sirolimus starting 3 days before the SCT and continuing through six months following SCT
- Group 2: Alemtuzumab for 4 days starting 8 days before SCT, plus cyclosporine starting 1 day before SCT and continuing for 6 months
- Patients receive the donor's stem cells and immune cells 2 days after the conditioning regimen
- Patients are followed at the clinic regularly for the first 6 months after SCT, and then less often for at least 5 years (some visits may include bone marrow aspirates and biopsies, blood draws, and other tests to monitor disease status)
- A skin biopsy, oral mucosa biopsy, and saliva collection are done to study chronic GVHD
- This trial will be conducted at the NIH Clinical Center in Bethesda, MD. It is open to patients who meet the eligibility requirements, regardless of where they live in the United States.
- There is no charge for medical care received at NIH Clinical Center.
- FAQs about this study - provides information for patients about the trial such as frequency and duration of visits, costs, how to enroll, and study outline.
- PDQ (Physicians Data Query) - provides additional details about this study for health care providers.