title: Hematopoietic cell transplantation in patients with myelodysplastic syndrome or acute myeloid leukemia arising from myelodysplastic syndrome: similar outcomes in patients with de novo disease and disease following prior therapy or antecedent hematologic disorders.
abstract: We analyzed outcomes after hematopoietic cell transplantation (HCT) in 257 patients, 3 to 72.7 years old (median, 43 y), with secondary myelodysplastic syndrome (MDS) including those with transformation to acute myeloid leukemia (tAML). Conditioning regimens included high-dose total-body irradiation (TBI)/chemotherapy (n = 83); busulfan (BU)/cyclophosphamide (CY) (BUCY, n = 122; with BU targeting [tBUCY], n = 93); fludarabine (Flu) with tBU (FLUtBU; n = 12); Flu plus 200 cGy TBI (n = 26); and miscellaneous regimens (n = 14). Donors were HLA-identical or partially mismatched family members in 135 and unrelated individuals in 122 patients. Five-year relapse-free survival was highest (43%) and nonrelapse mortality lowest (28%) among tBUCY-conditioned patients. Outcomes were compared with results in 339 patients who received transplants for de novo MDS/tAML, and a multivariate analysis failed to show significant differences in outcome between the 2 cohorts. Relapse probability and relapse-free survival correlated significantly with disease stage (P < .001) and karyotype (P < .001). Relapse incidence was lower (P = .003) and relapse-free survival superior (P = .02) with unrelated donor transplants. The data suggest that overall inferior outcome in patients with secondary MDS/tAML was related to the frequency of high-risk cytogenetics. For both cohorts, transplantation outcomes improved over the time interval studied.
authors.affiliation: Department of Hematology, Shanghai Sixth People's Hospital, Shanghai JiaoTong University, Shanghai, People's Republic of China.
authors.forename: Chunkang
authors.initials: C
authors.lastname: Chang
authors.forename: Barry E
authors.initials: BE
authors.lastname: Storer
authors.forename: Bart L
authors.initials: BL
authors.lastname: Scott
authors.forename: Eileen M
authors.initials: EM
authors.lastname: Bryant
authors.forename: Howard M
authors.initials: HM
authors.lastname: Shulman
authors.forename: Mary E
authors.initials: ME
authors.lastname: Flowers
authors.forename: Brenda M
authors.initials: BM
authors.lastname: Sandmaier
authors.forename: Robert P
authors.initials: RP
authors.lastname: Witherspoon
authors.forename: Richard A
authors.initials: RA
authors.lastname: Nash
authors.forename: Jean E
authors.initials: JE
authors.lastname: Sanders
authors.forename: Antonio
authors.initials: A
authors.lastname: Bedalov
authors.forename: John A
authors.initials: JA
authors.lastname: Hansen
authors.forename: Bruce E
authors.initials: BE
authors.lastname: Clurman
authors.forename: Rainer
authors.initials: R
authors.lastname: Storb
authors.forename: Frederick R
authors.initials: FR
authors.lastname: Appelbaum
authors.forename: H Joachim
authors.initials: HJ
authors.lastname: Deeg
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003520:Cyclophosphamide / Q000008:administration & dosage; D005260:Female; D006402:Hematologic Diseases / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D009369:Neoplasms / Q000150:complications* / Q000628:therapy; D016609:Neoplasms, Second Primary / Q000628:therapy*; D011379:Prognosis; D015996:Survival Rate; D019172:Transplantation Conditioning; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives; D014916:Whole-Body Irradiation
chemical_list: D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine