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Rebecca Olin, MD, MS


Busulfan Versus Melphalan: Long-Term Outcomes After Transplantation for AML

By: Celeste L. Dixon
Posted: Monday, June 29, 2020

Both similarities and differences in clinical outcomes were discovered when a Japanese research team performed a long-term nationwide retrospective study of adults with acute myeloid leukemia (AML) who received their first allogeneic hematopoietic cell transplantation (allo-HCT) after different reduced-intensity conditioning regimens between 2001 and 2010. “Fludarabine with reduced doses of busulfan and fludarabine with reduced doses of melphalan are widely used reduced-intensity conditioning regimens for AML,” explained Takuya Yamashita, MD, PhD, of St. Luke’s International Hospital, Tokyo, Japan, and colleagues. “Reduced-intensity conditioning has been facilitating allo-HCT for patients originally considered ineligible for HCT with myeloablative conditioning.”

The two regimens provided similar overall survival outcomes and both are “effective and useful,” the researchers reported in Bone Marrow Transplantation. They also noted that as of 2006 in Japan, busulfan could be administered intravenously with fludarabine, as an alternative to oral administration. Cumulative incidences of relapse did not significantly differ among the groups receiving fludarabine plus intravenous busulfan, fludarabine plus oral busulfan, and fludarabine plus melphalan (P = .29).

However, nonrelapse mortality was significantly lower in patients receiving fludarabine plus intravenous busulfan than in those receiving with fludarabine plus oral busulfan (P = .003) and fludarabine plus melphalan (P < .001). The combination of fludarabine and melphalan was associated with a significantly lower risk of relapse (P = .002), higher nonrelapse mortality (P = .013), and better leukemia-free survival (P = .015) than the combination of fludarabine plus intravenous busulfan. According to Dr. Yamashita and co-investigators, these results suggest that fludarabine plus melphalan may have more antileukemia potential than fludarabine plus intravenous busulfan, whereas this intravenous combination regimen appears to be less toxic than fludarabine plus melphalan.

Disclosure: The study authors reported no conflicts of interest.

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