Trial Outcomes & Findings for Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome (NCT NCT00445744)

Results Overview

Number of patients with regimen-related liver toxicity. Diagnoses will be made according to the established criteria initially proposed in 1984 by McDonald et al.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

52 participants

Primary outcome timeframe

Up to day +20

Results posted on

2018-01-02

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Cyclophosphamide, Busulfan, Transplant) CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11. cyclophosphamide: Given IV busulfan: Given IV tacrolimus: Given IV or PO methotrexate: Given IV cytogenetic analysis: Correlative studies flow cytometry: Correlative studies pharmacological study: Correlative studies pharmacogenomic studies: Correlative studies peripheral blood stem cell transplantation: Undergo PBPC transplantation allogeneic hematopoietic stem cell transplantation: Undergo allogeneic transplantation
Overall Study STARTED	52
Overall Study COMPLETED	51
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Cyclophosphamide, Busulfan, Transplant) n=51 Participants CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11. cyclophosphamide: Given IV busulfan: Given IV tacrolimus: Given IV or PO methotrexate: Given IV cytogenetic analysis: Correlative studies flow cytometry: Correlative studies pharmacological study: Correlative studies pharmacogenomic studies: Correlative studies peripheral blood stem cell transplantation: Undergo PBPC transplantation allogeneic hematopoietic stem cell transplantation: Undergo allogeneic transplantation
Age, Continuous	55 years n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants
Sex: Female, Male Male	30 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to day +20

Number of patients with regimen-related liver toxicity. Diagnoses will be made according to the established criteria initially proposed in 1984 by McDonald et al.

Outcome measures

Outcome measures
Measure	Treatment (Cyclophosphamide, Busulfan, Transplant) n=51 Participants CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11. cyclophosphamide: Given IV busulfan: Given IV tacrolimus: Given IV or PO methotrexate: Given IV cytogenetic analysis: Correlative studies flow cytometry: Correlative studies pharmacological study: Correlative studies pharmacogenomic studies: Correlative studies peripheral blood stem cell transplantation: Undergo PBPC transplantation allogeneic hematopoietic stem cell transplantation: Undergo allogeneic transplantation
Effectiveness of Cyclophosphamide/Busulfan Regimen in Reducing Regimen-related Liver Toxicity	8 Participants

PRIMARY outcome

Timeframe: Up to day 200

Cumulative incidence rate with death as a competing risk, assessed at day 100.

Outcome measures

Outcome measures
Measure	Treatment (Cyclophosphamide, Busulfan, Transplant) n=31 Participants CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11. cyclophosphamide: Given IV busulfan: Given IV tacrolimus: Given IV or PO methotrexate: Given IV cytogenetic analysis: Correlative studies flow cytometry: Correlative studies pharmacological study: Correlative studies pharmacogenomic studies: Correlative studies peripheral blood stem cell transplantation: Undergo PBPC transplantation allogeneic hematopoietic stem cell transplantation: Undergo allogeneic transplantation
Non-relapse Mortality (NRM) (Patients With AML/MDS)	17 percent

Adverse Events

Treatment (Cyclophosphamide, Busulfan, Transplant)

Serious events: 19 serious events

Other events: 0 other events

Deaths: 19 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Cyclophosphamide, Busulfan, Transplant) n=52 participants at risk CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11. cyclophosphamide: Given IV busulfan: Given IV tacrolimus: Given IV or PO methotrexate: Given IV cytogenetic analysis: Correlative studies flow cytometry: Correlative studies pharmacological study: Correlative studies pharmacogenomic studies: Correlative studies peripheral blood stem cell transplantation: Undergo PBPC transplantation allogeneic hematopoietic stem cell transplantation: Undergo allogeneic transplantation
Immune system disorders graft-versus-host disease	3.8% 2/52 • Up to 3 years after enrollment Causes of death were recorded as adverse events. Other adverse events were not recorded in this study.
Blood and lymphatic system disorders Recurrent or progressive malignancy	17.3% 9/52 • Up to 3 years after enrollment Causes of death were recorded as adverse events. Other adverse events were not recorded in this study.
Infections and infestations Infection associated with graft-versus-host disease	7.7% 4/52 • Up to 3 years after enrollment Causes of death were recorded as adverse events. Other adverse events were not recorded in this study.
Infections and infestations infection not associated with graft-versus-host disease	3.8% 2/52 • Up to 3 years after enrollment Causes of death were recorded as adverse events. Other adverse events were not recorded in this study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Secondary malignancy	1.9% 1/52 • Up to 3 years after enrollment Causes of death were recorded as adverse events. Other adverse events were not recorded in this study.
Psychiatric disorders suicide	1.9% 1/52 • Up to 3 years after enrollment Causes of death were recorded as adverse events. Other adverse events were not recorded in this study.

Other adverse events

Adverse event data not reported

Additional Information

H. Joachim Deeg MD

FHCRC

Phone: 206 667 4409

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place