title: Immunosuppressive therapy with anti-thymocyte globulin and cyclosporine A in selected children with hypoplastic refractory cytopenia.
abstract: It is currently unknown whether immunosuppressive therapy or hematopoietic stem cell transplantation is the most appropriate treatment strategy for children with refractory cytopenia and normal karyotype or trisomy 8. We report on 31 children with hypoplastic refractory cytopenia treated with immunosuppressive therapy consisting of antithymocyte globulin and cyclosporine. At 6 months, 22 of 29 evaluable patients had a complete or partial response; a total of ten patients achieved a complete response at varying time points. Six patients subsequently received a transplant because of non-response, progression to advanced myelodysplastic syndrome or evolution of monosomy 7. Overall and failure-free survival rates at 3 years were 88% and 57%, respectively.
authors.affiliation: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Germany.
authors.forename: Ayami
authors.initials: A
authors.lastname: Yoshimi
authors.forename: Irith
authors.initials: I
authors.lastname: Baumann
authors.forename: Monika
authors.initials: M
authors.lastname: Führer
authors.forename: Eva
authors.initials: E
authors.lastname: Bergsträsser
authors.forename: Ulrich
authors.initials: U
authors.lastname: Göbel
authors.forename: Karl-Walter
authors.initials: KW
authors.lastname: Sykora
authors.forename: Thomas
authors.initials: T
authors.lastname: Klingebiel
authors.forename: Ute
authors.initials: U
authors.lastname: Gross-Wieltsch
authors.forename: Marry M
authors.initials: MM
authors.lastname: van den Heuvel-Eibrink
authors.forename: Alexandra
authors.initials: A
authors.lastname: Fischer
authors.forename: Peter
authors.initials: P
authors.lastname: Nöllke
authors.forename: Charlotte
authors.initials: C
authors.lastname: Niemeyer
mesh_terms: D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D001803:Blood Transfusion; D002648:Child; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use*; D018450:Disease Progression; D018572:Disease-Free Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D007958:Leukocyte Count; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics / Q000401:mortality / Q000601:surgery; D011241:Prednisone / Q000627:therapeutic use; D011446:Prospective Studies; D012008:Recurrence; D012074:Remission Induction; D018709:Statistics, Nonparametric; D013601:T-Lymphocytes; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D011241:Prednisone