title: Favorable outcome in children and adolescents with a high proportion of advanced phase disease using single/multiple autologous or matched/mismatched allogeneic stem cell transplantations.
abstract: We determined the indication, outcome, and risk factors of single and multiple hematopoietic stem cell transplantation(s) (HSCT) in children and adolescents mostly with advanced disease. Forty-one out of 483 patients (8.5 %; median age 9 years) diagnosed at the University of Leipzig with hematological and oncological diseases required HSCT from 1999 to 2011. Patients had overall survival (OS) of 63 ± 10 and 63 ± 16 %, event-free survival (EFS) of 57 ± 10 and 42 ± 16 %, relapse incidence (RI) of 39 ± 10 and 44 ± 18 % and nonrelapse mortality (NRM) of 4 ± 4 and 13 ± 9 % at 10 years after one or more allogeneic and autologous HSCT, respectively. One patient in CR1 and five with advanced disease received two HSCT. Four of the six patients maintained/achieved CR for a median of 13 months. Three died of progression and one of NRM. Two patients had a third HSCT and one survived in CR +231 days after HSCT. Risk factors for OS and EFS were disease stage at HSCT and EBMT risk score. Center (pediatric or JACIE accredited pediatric/adult) was not a determinant for survival. Pediatric single and multiple HSCT are important curative approaches for high-risk malignant diseases with low NRM. Efforts to reduce high RI remain the major aim.
authors.affiliation: Department of Pediatric Hematology/Oncology/Hemostaseology, Children's Hospital, University of Leipzig, Liebigstrasse 20a, D-04103, Leipzig, Germany. Christian.Niederwieser@osumc.edu.|The Ohio State University, Arthur G. James Cancer Hospital and Richard J. Solove Research Institute C933 James Cancer Hospital, 460 West 10th Avenue, Columbus, OH, 43210, USA. Christian.Niederwieser@osumc.edu.
authors.forename: C
authors.initials: C
authors.lastname: Niederwieser
authors.affiliation: Department of Pediatric Hematology/Oncology/Hemostaseology, Children's Hospital, University of Leipzig, Liebigstrasse 20a, D-04103, Leipzig, Germany.
authors.forename: S
authors.initials: S
authors.lastname: Starke
authors.affiliation: Department of Pediatric Hematology/Oncology/Hemostaseology, Children's Hospital, University of Leipzig, Liebigstrasse 20a, D-04103, Leipzig, Germany.
authors.forename: L
authors.initials: L
authors.lastname: Fischer
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: R
authors.initials: R
authors.lastname: Krahl
authors.affiliation: Section for Hematology and Oncology, Department of Pediatrics, Jena University Hospital, Jena, Germany.
authors.forename: J
authors.initials: J
authors.lastname: Beck
authors.affiliation: Section for Hematology and Oncology, Department of Pediatrics, Jena University Hospital, Jena, Germany.
authors.forename: B
authors.initials: B
authors.lastname: Gruhn
authors.affiliation: Pediatric BMT Unit, Charité, Universitätsmedizin Berlin, Berlin, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Ebell
authors.affiliation: Department of Pediatrics, University Hospital, Halle (Saale), Germany.
authors.forename: D
authors.initials: D
authors.lastname: Körholz
authors.affiliation: Department of Pediatric Hematology and Oncology, Justus Liebig University, Giessen, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Wößmann
authors.affiliation: Stem Cell Transplantation and Immunology, Klinik für Kinder und Jugendmedizin, Frankfurt am Main, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Bader
authors.affiliation: Hematology / Oncology, University Children's Hospital Tuebingen, Tuebingen, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Lang
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: H-K
authors.initials: HK
authors.lastname: Al-Ali
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Cross
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: A-K
authors.initials: AK
authors.lastname: Eisfeld
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: S
authors.initials: S
authors.lastname: Heyn
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: V
authors.initials: V
authors.lastname: Vucinic
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: G-N
authors.initials: GN
authors.lastname: Franke
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Lange
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Pönisch
authors.affiliation: Department of Hematology and Oncology, University of Leipzig, Leipzig, Germany.
authors.forename: G
authors.initials: G
authors.lastname: Behre
authors.affiliation: Department of Pediatric Hematology/Oncology/Hemostaseology, Children's Hospital, University of Leipzig, Liebigstrasse 20a, D-04103, Leipzig, Germany.
authors.forename: H
authors.initials: H
authors.lastname: Christiansen
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D019337:Hematologic Neoplasms / Q000175:diagnosis* / Q000401:mortality / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D007223:Infant; D008297:Male; D015996:Survival Rate / Q000639:trends; D019172:Transplantation Conditioning / Q000379:methods / Q000401:mortality; D014184:Transplantation, Homologous / Q000379:methods / Q000401:mortality; D016896:Treatment Outcome; D055815:Young Adult
keywords: Allogeneic HSCT; Autologous HSCT; Donor cell chimerism; GvHD; Hematopoietic stem cell transplantation; Multiple HSCT; Pediatric