TY - JOUR
T1 - Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation
AU - Bredeson, Christopher
AU - LeRademacher, Jennifer
AU - Kato, Kazunobu
AU - DiPersio, John F.
AU - Agura, Edward
AU - Devine, Steven M.
AU - Appelbaum, Frederick R.
AU - Tomblyn, Marcie R.
AU - Laport, Ginna G.
AU - Zhu, Xiaochun
AU - McCarthy, Philip L.
AU - Ho, Vincent T.
AU - Cooke, Kenneth R.
AU - Armstrong, Elizabeth
AU - Smith, Angela
AU - Rizzo, J. Douglas
AU - Burkart, Jeanne M.
AU - Pasquini, Marcelo C.
PY - 2013/12/5
Y1 - 2013/12/5
N2 - 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.
AB - 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.
UR - https://www.scopus.com/pages/publications/84887535514
U2 - 10.1182/blood-2013-08-519009
DO - 10.1182/blood-2013-08-519009
M3 - Article
C2 - 24081656
AN - SCOPUS:84887535514
SN - 0006-4971
VL - 122
SP - 3871
EP - 3878
JO - Blood
JF - Blood
IS - 24
ER -