This phase II trial investigates two strategies and how well they work for the reduction of graft versus host disease in patients with acute leukemia or MDS in remission. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.
OUTLINE: Patients are randomized to 1 of 2 arms (Arms A and C). ARM A: Patients are assigned to 1 of 2 arms. ARM A1 (TBI BASED): Patients undergo total-body irradiation (TBI) twice daily (BID) on days -10 to -7, and receive thiotepa intravenously (IV) over 3 hours on days -6 and -5, fludarabine IV over 30 to 60 minutes on days -6 to -2, tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, CD34+ enriched CD45RA-depleted donor T-lymphocytes IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM A2 (BUSULFAN BASED): Patients receive fludarabine IV over 30 to 60 minutes on days -6 to -2, busulfan IV over 180 minutes on days -5 to -2, and undergo TBI BID on day -1. Patients also receive tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, CD34+ enriched CD45RA-depleted donor T-lymphocytes IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM C: Patients are assigned to 1 of 2 arms. ARM C1: Patients undergo TBI BID on days -4 to -2 or -3 to -1, and receive PBSC IV on day 0. Patients also receive cyclophosphamide IV over 1 to 2 hours on days 3 and 4, and tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day 5 approximately 24 hours after the end of the second dose of cyclophosphamide. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM C2: Patients receive fludarabine IV over 30 to 60 minutes on days -5 to -2, busulfan IV over 180 minutes on days -5 to -2, PBSC IV on day 0, cyclophosphamide IV over 1 to 2 hours on days 3 and 4, and tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day 5 approximately 24 hours after the end of the second dose of cyclophosphamide. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM D: (DISCONTINUED NOVEMBER 2021): Patients are assigned to 1 of 2 arms. ARM D1: Patients undergo TBI BID on days -6 to -4, and receive cyclophosphamide IV over 1 hour on days -3 to -2, tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, PBSC IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM D2: Patients receive busulfan IV over 180 minutes on days -8 to -5, cyclophosphamide IV over 1 hour on days -3 to -2, tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, PBSC IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). All patients also undergo bone marrow aspiration/biopsy, echocardiogram (ECHO) or multigated acquisition (MUGA) scan, and collection of blood samples throughout the trial. After completion of study treatment, patients are followed up at days 7, 14, 21, 28, 56, 80, 180, and 270 and at 1, 1.5, and 2 years.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
120
Undergo TBI
Given IV
Given IV
Given IV
Given IV
Given IV
Given IV
Given IV
Given IV
Given IV
Given IV
Undergo bone marrow aspiration/biopsy
Undergo ECHO
Undergo MUGA
Undergo blood sample collection
Moffitt Cancer Center
Tampa, Florida, United States
RECRUITINGUniversity of Pittsburgh Cancer Institute (UPCI)
Pittsburgh, Pennsylvania, United States
RECRUITINGFred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
RECRUITINGGraft versus host disease (GVHD)-free relapse-free survival (RFS)
Will be defined as survival-free of a history of: a) relapse after hematopoietic cell transplantation (HCT), b) grade III-IV acute GVHD after HCT, moderate or severe chronic GVHD meeting National Institutes of Health (NIH) criteria and requiring systemic pharmacologic immunosuppression for treatment of GVHD. RFS distribution will be estimated for each arm by the Kaplan-Meier curve, starting from the time of HCT. A 90% confidence interval (CI) will be constructed at 1 and 2-year post-HCT timepoints.
Time frame: At 2 years
Overall survival (OS)
Death will be considered as the event, and the OS distribution will be estimated for each group by the Kaplan-Meier method, starting from the time of HCT. A 90% CI will be constructed at the 1 and 2-year post-HCT time points.
Time frame: At 2 years
Relapse
Will be defined as recurrence of leukemia (\>= 5% leukemic blasts by flow or morphology) in bone marrow, circulating leukemic blasts or extramedullary disease (extramedullary disease definite i.e. proven by biopsy or probably based on clinical assessment if biopsy not feasible). Lower levels of leukemia in bone marrow or blood will be recorded and reported as 'measurable residual disease' but will not be considered relapse.
Time frame: At 2 years
Proportion of patients alive and off prednisone (or equivalent systemic corticosteroid) for treatment of GVHD
Time frame: At 3, 6, 9, 12, 15, 18, 21, 24 months post HCT
Graft rejection or irreversible graft failure (> 14 days duration)
Will be operationally defined as failure to achieve an absolute neutrophil count (ANC) \>= 0.5 x 10\^9/L before death or second HCT, or decrease to ANC \< 0.1 x 10\^9/L for 14 consecutive days (date of graft failure defined as the 14th day) after an established donor graft despite daily administration of granulocyte colony-stimulating factor (subcutaneously or intravenously) and =\< 20% bone marrow cellularity on bone marrow aspirate or biopsy any time in the first 2 years following HCT. If the delay or reduction in ANC is due to relapse (as determined by histopathology, flow cytometry or cytogenetic or molecular studies) this will not be considered graft failure. If a patient dies from organ toxicity and/or infection prior to day 28 without ANC \>= 0.5 x 10\^9/L this will not be considered graft failure.
Time frame: At 2 years
Incidence of chronic GVHD
Will be defined and graded based on NIH criteria and graded operationally as the occurrence of compatible symptoms.
Time frame: Up to 2 years
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