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

Christopher Bredeson, Jennifer LeRademacher, Kazunobu Kato, John F. DiPersio, Edward Agura, Steven M. Devine, Frederick R. Appelbaum, Marcie R. Tomblyn, Ginna G. Laport, Xiaochun Zhu, Philip L. McCarthy, Vincent T. Ho, Kenneth R. Cooke, Elizabeth Armstrong, Angela Smith, J. Douglas Rizzo, Jeanne M. Burkart, Marcelo C. Pasquini

Research output: Contribution to journalArticlepeer-review

90 Scopus citations


We conducted a prospective cohort study testing the noninferiority of survival of ablative intravenous busulfan (4-BU) vs ablative total body irradiation (TBI)-based regimens in myeloid malignancies. A total of 1483 patients undergoing transplantation for myeloid malignancies (4-BU, N 5 1025; TBI, N 5 458) 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% 4-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 5 .019) for 4-BU (relative risk, 0.82; 95% CI, 0.68-0.98, P 5 .03) and TBI, respectively. Corresponding incidences of transplantrelated mortality (TRM) were 18% (95% CI, 16%-21%) and 19% (95% CI, 15%-23%, P 5 .75) and disease progression were 34%(95%CI, 31%-37%) and 39%(95%CI, 34%-44%, P5.08). The incidence of hepatic veno-occlusive disease (VOD) was 5% for 4-BU and 1% with TBI (P <.001). There were no differences in progression-free survival and graft-versus-host disease. Compared with TBI, 4-BU resulted in superior survival with no increased risk for relapse or TRM. These results support the use of myeloablative 4-BU vs TBI-based conditioning regimens for treatment of myeloid malignancies.

Original languageEnglish (US)
Pages (from-to)3871-3878
Number of pages8
Issue number24
StatePublished - Dec 5 2013

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology


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