title: Myelodysplastic syndrome evolving from aplastic anemia treated with immunosuppressive therapy: efficacy of hematopoietic stem cell transplantation.
abstract: A proportion of patients with aplastic anemia who are treated with immunosuppressive therapy develop clonal hematologic disorders, including post-aplastic anemia myelodysplastic syndrome. Many will proceed to allogeneic hematopoietic stem cell transplantation. We identified 123 patients with post-aplastic anemia myelodysplastic syndrome who from 1991 through 2011 underwent allogeneic hematopoietic stem cell transplantation, and in a matched-pair analysis compared outcome to that in 393 patients with de novo myelodysplastic syndrome. There was no difference in overall survival. There were no significant differences with regard to 5-year probabilities of relapse, non-relapse mortality, relapse-free survival and overall survival; these were 14%, 40%, 46% and 49% for post-aplastic anemia myelodysplastic syndrome, and 20%, 33%, 47% and 49% for de novo myelodysplastic syndrome, respectively. In multivariate analysis, relapse (hazard ratio 0.71; P=0.18), non-relapse mortality (hazard ratio 1.28; P=0.18), relapse-free survival (hazard ratio 0.97; P=0.80) and overall survival (hazard ratio 1.02; P=0.88) of post-aplastic anemia myelodysplastic syndrome were similar to those of patients with de novo myelodysplastic syndrome. Cytogenetic risk was independently associated with overall survival in both groups. Thus, transplant success in patients with post-aplastic anemia myelodysplastic syndrome was similar to that in patients with de novo myelodysplastic syndrome, and cytogenetics was the only significant prognostic factor for post-aplastic anemia myelodysplastic syndrome patients.
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; KonKuk University Medical Center, KonKuk University School of Medicine, Seoul, Republic of Korea;
authors.forename: Sung-Yong
authors.initials: SY
authors.lastname: Kim
authors.affiliation: Center for International Blood & Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA; Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA;
authors.forename: Jennifer
authors.initials: J
authors.lastname: Le Rademacher
authors.affiliation: Dana-Farber Cancer Institute, Boston, MA, USA;
authors.forename: Joseph H
authors.initials: JH
authors.lastname: Antin
authors.affiliation: The University of Texas MD Anderson Cancer Center, Department of Stem Cell Transplantation and Cellular Therapy, Houston, TX, USA;
authors.forename: Paolo
authors.initials: P
authors.lastname: Anderlini
authors.affiliation: Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia;
authors.forename: Mouhab
authors.initials: M
authors.lastname: Ayas
authors.affiliation: National Heart, Lung, and Blood Institute, Hematology Branch, National Institutes of Health, Bethesda, MD, USA;
authors.forename: Minoo
authors.initials: M
authors.lastname: Battiwalla
authors.affiliation: Center for International Blood & Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA;
authors.forename: Jeanette
authors.initials: J
authors.lastname: Carreras
authors.affiliation: Dept of Pediatrics/Pediatrics Blood & Marrow Transplantation, Duke University Medical Center, Durham, NC, USA;
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.affiliation: City of Hope National Medical Center, Duarte, CA, USA; and.
authors.forename: Ryotaro
authors.initials: R
authors.lastname: Nakamura
authors.affiliation: Center for International Blood & Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA;
authors.forename: Mary
authors.initials: M
authors.lastname: Eapen
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; jdeeg@fhcrc.org.
authors.forename: H Joachim
authors.initials: HJ
authors.lastname: Deeg
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D016022:Case-Control Studies; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000276:immunology* / Q000401:mortality / Q000473:pathology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000139:chemically induced / Q000401:mortality / Q000628:therapy*; D009367:Neoplasm Staging; D011379:Prognosis; D012008:Recurrence; D015996:Survival Rate; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents
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