Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation
Menée sur une cohorte de 1 483 patients présentant une malignité myéloïde et devant recevoir une greffe de cellules souches hématopoïétiques, cette étude prospective évalue, du point de vue du risque de récidive et de la survie, la non infériorité du busulfan IV par rapport à une radiothérapie de l'ensemble du corps
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.