title: Pretransplantation 5-azacitidine in high-risk myelodysplastic syndrome.
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).
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Taiga
authors.initials: T
authors.lastname: Nishihori
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Janelle
authors.initials: J
authors.lastname: Perkins
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Asmita
authors.initials: A
authors.lastname: Mishra
authors.affiliation: Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Rami
authors.initials: R
authors.lastname: Komrokji
authors.affiliation: Biostatistics Core, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Jongphil
authors.initials: J
authors.lastname: Kim
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Mohamed A
authors.initials: MA
authors.lastname: Kharfan-Dabaja
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Lia
authors.initials: L
authors.lastname: Perez
authors.affiliation: Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Lancet
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Hugo
authors.initials: H
authors.lastname: Fernandez
authors.affiliation: Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Alan
authors.initials: A
authors.lastname: List
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
authors.forename: Claudio
authors.initials: C
authors.lastname: Anasetti
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida. Electronic address: teresa.field@moffitt.org.
authors.forename: Teresa
authors.initials: T
authors.lastname: Field
mesh_terms: D000328:Adult; D000368:Aged; D001374:Azacitidine / Q000627:therapeutic use*; D003131:Combined Modality Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy / Q000628:therapy*; D010865:Pilot Projects; D011446:Prospective Studies; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D061349:Unrelated Donors
chemical_list: D001374:Azacitidine
keywords: 5-Azacitidine; Allogeneic hematopoietic cell transplantation; Hypomethylating agent; MDS