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Biol Blood Marrow Transplant. 2014 Jun;20(6):776-80. doi: 10.1016/j.bbmt.2014.02.008. Epub 2014 Feb 15.

Pretransplantation 5-azacitidine in high-risk myelodysplastic syndrome.

Author information

1
Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
2
Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
3
Biostatistics Core, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
4
Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida. Electronic address: teresa.field@moffitt.org.

Abstract

We prospectively evaluated the allogeneic hematopoietic cell transplantation (HCT) outcomes in high-risk myelodysplastic syndrome (MDS) patients who received pretransplantation 5-azacitidine. Twenty-five patients evaluated for allogeneic HCT consult and considered medically eligible for a donor search were enrolled. Azacitidine was administered at 75 mg/m(2) for 5 to 7 days every 4 weeks until a suitable donor was found. A median of 3 (range, 0 to 6) cycles of 5-azacitidine were administered. Preallogeneic HCT responses to 5-azacitidine, based on the International Working Group criteria, were 48% partial response, 33% stable disease, and 19% progressive disease. Four patients did not proceed to allogeneic HCT. Twenty-one patients, a median age of 55 (range, 25 to 67) years, received allogeneic HCT after myeloablative pharmacokinetically targeted i.v. busulfan and fludarabine conditioning regimen. Donors were either HLA-matched related or unrelated, except for 1 mismatch unrelated donor. With a median follow-up of 30 months, 1-year relapse-free and overall survivals were 52% (95% confidence interval [CI], 30% to 71%) and 62% (95% CI, 38% to 79%), respectively. Preallogeneic HCT 5-azacitidine administration was well tolerated and provided reasonable disease control before allogeneic HCT. (Registered at ClinicalTrials.gov as NCT00660400).

KEYWORDS:

5-Azacitidine; Allogeneic hematopoietic cell transplantation; Hypomethylating agent; MDS

PMID:
24534108
DOI:
10.1016/j.bbmt.2014.02.008
[Indexed for MEDLINE]
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