title: Hematopoietic stem cell transplantation in children and young adults with secondary myelodysplastic syndrome and acute myelogenous leukemia after aplastic anemia.
abstract: Secondary myelodysplastic syndrome and acute myelogenous leukemia (sMDS/sAML) are the most serious secondary events occurring after immunosuppressive therapy in patients with aplastic anemia. Here we evaluate the outcome of hematopoietic stem cell transplantation (HSCT) in 17 children and young adults with sMDS/sAML after childhood aplastic anemia. The median interval between the diagnosis of aplastic anemia and the development of sMDS/sAML was 2.9 years (range, 1.2 to 13.0 years). At a median age of 13.1 years (range, 4.4 to 26.7 years), patients underwent HSCT with bone marrow (n = 6) or peripheral blood stem cell (n = 11) grafts from HLA-matched sibling donors (n = 2), mismatched family donors (n = 2), or unrelated donors (n = 13). Monosomy 7 was detected in 13 patients. The preparative regimen consisted of busulfan, cyclophosphamide, and melphalan in 11 patients and other agents in 6 patients. All patients achieved neutrophil engraftment. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 47%, and that of chronic GVHD was 70%. Relapse occurred in 1 patient. The major cause of death was transplant-related complication (n = 9). Overall survival and event-free survival at 5 years after HSCT were both 41%. In summary, this study indicates that HSCT is a curative therapy for some patients with sMDS/sAML after aplastic anemia. Future efforts should focus on reducing transplantation-related mortality.
authors: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany. Electronic address: ayami.yoshimi@uniklinik-freiburg.de.
authors: Ayami
authors: A
authors: Yoshimi
authors: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors: Brigitte
authors: B
authors: Strahm
authors: Department of Pathology, Boeblingen Hospital, Clinical Centre South West, Boeblingen, Germany.
authors: Irith
authors: I
authors: Baumann
authors: Department of Pediatrics and Adolescent Medicine, University Hospital Ulm, Ulm, Germany.
authors: Ingrid
authors: I
authors: Furlan
authors: Department of Pathology, University Medical Center Erlangen, Erlangen, Germany.
authors: Stephan
authors: S
authors: Schwarz
authors: Department of General Pediatrics, Hematology and Oncology, Children's University Hospital, Giessen, Germany.
authors: Andrea
authors: A
authors: Teigler-Schlegel
authors: Dr von Haunersches Kinderspital, Children Hospital of the Ludwig-Maximilians-University of Munich, Munich, Germany.
authors: Joachim-Ulrich
authors: JU
authors: Walther
authors: Institute of Cell and Molecular Pathology, Hannover Medical School, Hannover, Germany.
authors: Brigitte
authors: B
authors: Schlegelberger
authors: Institute of Cell and Molecular Pathology, Hannover Medical School, Hannover, Germany.
authors: Gudrun
authors: G
authors: Göhring
authors: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors: Peter
authors: P
authors: Nöllke
authors: Dr von Haunersches Kinderspital, Children Hospital of the Ludwig-Maximilians-University of Munich, Munich, Germany.
authors: Monika
authors: M
authors: Führer
authors: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors: Charlotte M
authors: CM
authors: Niemeyer
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000401:mortality / Q000473:pathology; D006680:HLA Antigens / Q000276:immunology; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects; D015470:Leukemia, Myeloid, Acute / Q000209:etiology / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D019653:Myeloablative Agonists / Q000627:therapeutic use; D009190:Myelodysplastic Syndromes / Q000209:etiology / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation; D012720:Severity of Illness Index; D035781:Siblings; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous
chemical_list: D006680:HLA Antigens; D007166:Immunosuppressive Agents; D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D002066:Busulfan; D008558:Melphalan
keywords: Aplastic anemia; Hematopoietic stem cell transplantation; Myelodysplastic syndrome