Effect of Granulocyte Colony–Stimulating Factor–Combined Conditioning in Cord Blood Transplantation for Myelodysplastic Syndrome and Secondary Acute Myeloid Leukemia: A Retrospective Study in Japan

Highlights

• We conducted a retrospective nationwide study of 336 adult patients with myelodysplastic syndrome and secondary acute myeloid leukemia patients to determine the role of a granulocyte colony–stimulating factor combination in a conditioning regimen before cord blood transplantation.

• The granulocyte colony–stimulating factor–combined regimen showed significantly higher incidence of neutrophil engraftment and better survival results in cord blood transplantation for myelodysplastic syndrome and secondary acute myeloid leukemia.

Abstract

Granulocyte colony–stimulating factor (G-CSF) increases the susceptibility of dormant malignant or nonmalignant hematopoietic cells to cytarabine arabinoside (Ara-C) through the induction of cell cycle entry. Therefore, G-CSF–combined conditioning before allogeneic stem cell transplantation might positively contribute to decreased incidences of relapse and graft failure without having to increase the dose of cytotoxic drugs. We conducted a retrospective nationwide study of 336 adult patients with myelodysplastic syndrome (MDS) and secondary acute myeloid leukemia (sAML) after single-unit cord blood transplantation (CBT) who underwent 4 different kinds of conditioning regimens: total body irradiation (TBI) ≥ 8 Gy + Ara-C/G-CSF + cyclophosphamide (CY) (n = 65), TBI ≥ 8 Gy + Ara-C + CY (n = 119), TBI ≥ 8 Gy + other (n = 104), or TBI < 8 Gy or non-TBI (n = 48). The TBI ≥ 8 Gy + Ara-C/G-CSF + CY regimen showed significantly higher incidence of neutrophil engraftment (hazard ratio, 1.52; 95% confidence interval [CI], 1.10 to 2.08; P = .009) and lower overall mortality (hazard ratio, .46; 95% CI, .26 to .82; P = .008) rates compared with those without a G-CSF regimen. This retrospective study shows that the G-CSF–combined conditioning regimen provides better engraftment and survival results in CBT for adults with MDS and sAML.