Trial Outcomes & Findings for Allogeneic HCT Using Nonmyeloablative Host Conditioning With TLI & ATG vs SOC in AML (NCT NCT00568633)
NCT ID: NCT00568633
Last Updated: 2019-09-24
Results Overview
Overall survival defined as the time interval between the date of attaining a first complete remission (CR) and the date of death from any cause. The outcome is reported as the number of participants alive (without dispersion).
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
58 participants
Primary outcome timeframe
2 years
Results posted on
2019-09-24
Participant Flow
Participant milestones
| Measure |
Allo-HSCT + TLI + ATG
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Overall Study
STARTED
|
25
|
33
|
|
Overall Study
COMPLETED
|
25
|
33
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Allogeneic HCT Using Nonmyeloablative Host Conditioning With TLI & ATG vs SOC in AML
Baseline characteristics by cohort
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
n=33 Participants
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
Total
n=58 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
46 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Age, Continuous
|
60 years
STANDARD_DEVIATION 5.1 • n=5 Participants
|
60 years
STANDARD_DEVIATION 6 • n=7 Participants
|
60 years
STANDARD_DEVIATION 5.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
25 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
55 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
25 participants
n=5 Participants
|
33 participants
n=7 Participants
|
58 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOverall survival defined as the time interval between the date of attaining a first complete remission (CR) and the date of death from any cause. The outcome is reported as the number of participants alive (without dispersion).
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
n=33 Participants
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Overall Survival (OS)
|
9 Participants
|
13 Participants
|
SECONDARY outcome
Timeframe: 2 yearsDisease-free survival is defined as the time interval between the date of attaining a first complete remission (CR) and the date of relapse. Disease free survival (DFS) will compared to conventional therapy vs Non-myeloablative Host Conditioning (NMA HCT). The outcome is reported as the number of participants which never experienced disease relapse (without dispersion).
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
n=33 Participants
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Disease-free Survival (DFS)
|
5 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: 2 yearsNon-relapse mortality is defined as death that occurs after therapy, from any cause except a cause associated with relapse. This will be reported as the number of participants experiencing non-relapse mortality (a number without dispersion).
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
n=33 Participants
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Non-relapse Mortality
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 2 yearsRelapse will be determined as ≥ 5% blast cells in the bone marrow, not secondary to regeneration after myelosuppressive therapy; OR emergence of extramedullary leukemia; OR the re-emergence of blasts in the peripheral blood. The outcome will be reported as the number and percentage of participants that meet these criteria (a number without dispersion).
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
n=33 Participants
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Relapse Rate
|
20 Participants
|
24 Participants
|
SECONDARY outcome
Timeframe: 100 days and 6 monthsPopulation: Participants in the Best Standard Care arm did not receive transplant, and are not evaluable for transplant outcomes.
Transplant-related mortality will be assessed as any death occurring within 6 months post-transplant, from any cause except relapse. It will be measured at 100 day and 6 months after transplant. The outcome is expressed as at the number of participants experiencing transplant-related mortality (a number without dispersion).
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Transplant-related Mortality
At 100 days
|
0 Participants
|
—
|
|
Transplant-related Mortality
At 6 months
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants in the Best Standard Care arm did not receive transplant, and are not evaluable for transplant outcomes.
Complete donor hematopoietic cell chimerism was evaluated in transplant recipients. Complete donor chimerism will be assessed as the presence of \> 95% donor T-cells (CD3+) in the blood. The outcome is reported as the percentage of participants that achieve complete donor chimerism, a number without dispersion.
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Complete Donor Hematopoietic Cell Chimerism
|
56 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants in the Best Standard Care arm did not receive transplant, and are not evaluable for transplant outcomes.
Early graft loss means a failure to achieve donor T-cell chimerism of \> 5% at any time after transplant. The outcome is reported as the percentage of participants that experience early graft loss, a number without dispersion.
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Early Graft Loss
|
4 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: 2 yearsThe assessment for completion of the intended therapy (in both arms) will be reported as the percentage of participants, a number without dispersion
Outcome measures
| Measure |
Allo-HSCT + TLI + ATG
n=25 Participants
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
n=33 Participants
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Patients Completing the Intended Therapy in Both Arms
|
100 percentage of participants
|
81.8 percentage of participants
|
Adverse Events
Allo-HSCT + TLI + ATG
Serious events: 20 serious events
Other events: 0 other events
Deaths: 16 deaths
Best Standard Care
Serious events: 24 serious events
Other events: 0 other events
Deaths: 20 deaths
Serious adverse events
| Measure |
Allo-HSCT + TLI + ATG
n=25 participants at risk
Participants achieving complete remission after consolidation therapy \& who have 5 of 6 HLA-match sibling donor to provide PBSC harvest for transplant. Pre-transplant subjects receive:
* Total lymphoid radiation (TLI) Days -11 to -7, and Days -4 to -1 (2 fractions on day -1)
* Anti-thymocyte globulin (ATG) Days -11 to -7
* Methylprednisolone Days -11 to -7
* Cyclosporine (CSP) Days -4 to +2
* 5+ of 6 HLA-matched CD34+ cells on Day 0
* Mycophenolate mofetil (MMF), Day 0 to Day +28
Allogeneic HSCT: Allogeneic, 5+ of 6 HLA-matched PBSC transplant from sibling, mobilized to target of 5 x 10e6 CD34+ cells/kg and \< 7 x 10e8 CD3+ cells/kg.
Anti-thymocyte globulin (ATG): 1.5 mg/kg for 5 days by IV
Cyclosporine (CSP): 6.25 mg/kg twice daily oral
Mycophenolate mofetil (MMF): 15 mg/kg twice daily oral
Total lymphoid irradiation (TLI): 80 cGy/fraction radiotherapy in 10 fractions.
Methylprednisolone sodium succinate: 1.0 mg/kg for 5 days by IV
|
Best Standard Care
n=33 participants at risk
Regular medical care for participants who achieve complete remission after standard consolidation therapy, but do not have a 5 of 6 HLA-match sibling donor. Treatment may consist of:
* Additional consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
Best standard care: Intervention consist of:
* Consolidation chemotherapy (3-4 cycles of cytarabine +/- an anthracycline agent, or other consolidation)
* Autologous transplantation
* Non-Myeloablative unrelated-donor transplant, +/- TLI and ATG conditioning
* Umbilical cord blood transplantation
* Haploidentical transplantation
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Relapse, leukemia
|
80.0%
20/25 • Number of events 20 • 2 years
Non-serious adverse events were not collected in this study.
|
72.7%
24/33 • Number of events 24 • 2 years
Non-serious adverse events were not collected in this study.
|
|
General disorders
Death not otherwise specified
|
4.0%
1/25 • Number of events 1 • 2 years
Non-serious adverse events were not collected in this study.
|
3.0%
1/33 • Number of events 1 • 2 years
Non-serious adverse events were not collected in this study.
|
|
Nervous system disorders
Altered mental status
|
4.0%
1/25 • Number of events 1 • 2 years
Non-serious adverse events were not collected in this study.
|
0.00%
0/33 • 2 years
Non-serious adverse events were not collected in this study.
|
Other adverse events
Adverse event data not reported
Additional Information
Robert Lowsky, Professor of Medicine (Blood and Marrow Transplantation)
Stanford University
Phone: 650-725-8950
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place