Author(s): Bredeson C, LeRademacher J, Kato K, Dipersio JF, Agura E,
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Abstract We conducted a prospective cohort study testing the noninferiority of survival of ablative intravenous busulfan (IV-BU) vs ablative total body irradiation (TBI)-based regimens in myeloid malignancies. A total of 1483 patients undergoing transplantation 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 transplantation. 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\% confidence interval [CI]), were 56\% (95\% CI, 53\%-60\%) and 48\% (95\% CI, 43\%-54\%, P = .019) for IV-BU (relative risk, 0.82; 95\% CI, 0.68-0.98, P = .03) and TBI, respectively. Corresponding incidences of transplant-related mortality (TRM) were 18\% (95\% CI, 16\%-21\%) and 19\% (95\% CI, 15\%-23\%, P = .75) and disease progression were 34\% (95\% CI, 31\%-37\%) and 39\% (95\% CI, 34\%-44\%, P = .08). The incidence of hepatic veno-occlusive disease (VOD) was 5\% for IV-BU and 1\% with TBI (P < .001). There were no differences in progression-free survival and graft-versus-host disease. Compared with TBI, IV-BU resulted in superior survival with no increased risk for relapse or TRM. These results support the use of myeloablative IV-BU vs TBI-based conditioning regimens for treatment of myeloid malignancies.
This article was published in Blood
and referenced in Journal of Stem Cell Research & Therapy