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Discovering the Optimal Combination of Anticancer Drug Administration for the Conditioning of Cord Blood Transplantation

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An analysis of Japanese hematopoietic stem-cell transplantation registry data showed that as the conditioning regimen for cord blood transplantation for myeloid malignancies, fludarabine with melphalan (140 mg/m2) and low-dose total-body irradiation resulted in the best survival rate after cord blood transplantation.

Tsukuba, Japan—Allogeneic hematopoietic stem-cell transplantation (allo-HSCT) can cure hematopoietic diseases, such as acute myeloid leukemia. Cord blood transplantation (CBT), which accounts for more than one-third of allo-HSCTs in Japan, uses hematopoietic stem cells contained in the blood in the umbilical cord and placenta. Moreover, it is a valuable option for patients lacking suitable HLA-matched donors.

Allo-HSCTs are expected to have antileukemia effects because of conditioning that contains a combination of anticancer drugs and total-body radiation performed before allo-HSCT and the allogeneic immune reaction of the transplanted cells. Various reduced-toxicity conditioning regimens exploring fludarabine, an anticancer drug, have been recently developed, and allo-HSCT is now conducted more safely. Thus, alkylating agents (e.g., melphalan and busulfan) and low-dose total-body irradiation are combined, but the optimal combination for CBT remains unclear.

Therefore, this study compared five different fludarabine-containing conditioning regimens commonly used in CBT for myeloid malignancies, such as myeloid leukemia and myelodysplastic syndrome, employing data from 1395 cases extracted from the Transplant Registry Unified Management Program. The results revealed that the combination of fludarabine, melphalan (140 mg/m2), and low-dose total-body irradiation offered the best survival rate (the 3-year survival rate was 67%) with the lowest relapse rate and nonrelapse mortality after CBT compared with that of other fludarabine-containing conditioning regimens. Researchers also found fewer deaths from infection using the combination.

Moreover, an appropriate combination of fludarabine and alkylating agents is expected to improve the outcome of future CBTs.

Original Paper

Title of original paper:
Comparison of fludarabine-based conditioning regimens in adult cord blood transplantation for myeloid malignancy: A retrospective, registry-based study
American Journal of Hematology


Professor CHIBA Shigeru
Assistant Professor KURITA Naoki

Institute of Medicine, University of Tsukuba

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