Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation.

Publication Type:

Journal Article


Blood (2013)


2013, Clinical Research Division, October 2013


We conducted a prospective cohort study testing the non-inferiority of survival of ablative IV-BU versus ablative TBI-based regimens in myeloid malignancies. 1,483 patients transplanted for myeloid malignancies (IV-BU, N=1025, TBI, N=458) were enrolled. Cohorts were similar with respect to age, gender, race, performance score, disease and disease stage at transplant. Most patients had acute myeloid leukemia (68% IV-BU, 78% TBI). Grafts were primarily peripheral blood (77%) from HLA-matched siblings (40%) or well-matched unrelated donors (48%). Two-year probabilities of survival (95% CI), were 56% (53-60%) and 48% (43-54%, p=0.019) for IV-BU (Relative Risk 0.82, 0.68-0.98, p=0.03) and TBI, respectively. Corresponding incidences of transplant related mortality (TRM) were 18% (16-21%) and 19% (15-23%, p= 0.75) and disease progression were 34% (31-37%) and 39% (34-44%, p=0.08). The incidence of hepatic veno-occlusive disease (VOD) was 5% for IV-BU and 1% with TBI (p < 0.001). There were no differences in progression-free survival and graft-versus-host disease. Compared to TBI, IV-BU resulted in superior survival with no increased risk of relapse or TRM. These results support the use of myeloablative IV-BU over TBI-based conditioning regimens for treatment of myeloid malignancies.