title: Comparable outcomes of matched-related and alternative donor stem cell transplantation for pediatric severe aplastic anemia.
abstract: Matched sibling donor (MSD) bone marrow transplantation is the treatment of choice for pediatric patients with severe aplastic anemia (SAA); however, only about 33% of patients will have an HLA-identical sibling. Alternative donor (AD) transplants may be an option for these patients, but such therapies have been associated with greater incidence of graft failure and graft-versus-host disease (GVHD). We retrospectively analyzed 36 pediatric patients who received 38 bone marrow or peripheral blood stem cell transplants (15 MSD and 23 AD) for SAA at our institution from April 1997 to October 2005. Nineteen AD recipients received reduced intensity conditioning with cyclophosphamide, low-dose total body irradiation, and antithymocyte globulin (ATG) or Campath. The 4-year overall survival for MSD recipients was 93% versus 89% for AD recipients treated with reduced intensity conditioning regimens at a median follow-up of 52 months (range, 6-99 months). No patient receiving Campath, compared with 3 of 9 patients receiving ATG, developed extensive, chronic GVHD. We conclude that, for children with SAA, AD transplantation is as effective as MSD transplantation. Further, compared with ATG, preparatory regimens containing Campath may be associated with a lower incidence of extensive, chronic GHVD.
authors.affiliation: Center for Cell and Gene Therapy, Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital, Houston, Texas 77030, USA. aakenned@txccc.org
authors.forename: Alana A
authors.initials: AA
authors.lastname: Kennedy-Nasser
authors.forename: Kathryn S
authors.initials: KS
authors.lastname: Leung
authors.forename: Anita
authors.initials: A
authors.lastname: Mahajan
authors.forename: Heidi L
authors.initials: HL
authors.lastname: Weiss
authors.forename: James A
authors.initials: JA
authors.lastname: Arce
authors.forename: Stephen
authors.initials: S
authors.lastname: Gottschalk
authors.forename: George
authors.initials: G
authors.lastname: Carrum
authors.forename: Shakila P
authors.initials: SP
authors.lastname: Khan
authors.forename: Helen E
authors.initials: HE
authors.lastname: Heslop
authors.forename: Malcolm K
authors.initials: MK
authors.lastname: Brenner
authors.forename: Catherine M
authors.initials: CM
authors.lastname: Bollard
authors.forename: Robert A
authors.initials: RA
authors.lastname: Krance
mesh_terms: D000293:Adolescent; D000074323:Alemtuzumab; D000741:Anemia, Aplastic / Q000601:surgery*; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm / Q000627:therapeutic use; D000961:Antilymphocyte Serum; D016026:Bone Marrow Transplantation / Q000276:immunology / Q000706:statistics & numerical data*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000517:prevention & control; D006648:Histocompatibility; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D007239:Infections / Q000453:epidemiology / Q000209:etiology; D053208:Kaplan-Meier Estimate; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D036102:Peripheral Blood Stem Cell Transplantation / Q000706:statistics & numerical data*; D011183:Postoperative Complications / Q000453:epidemiology / Q000209:etiology; D012189:Retrospective Studies; D035781:Siblings; D016019:Survival Analysis; D016176:T-Lymphocyte Subsets; D016559:Tacrolimus / Q000627:therapeutic use; D014019:Tissue Donors; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous / Q000276:immunology / Q000706:statistics & numerical data*; D016896:Treatment Outcome; D014916:Whole-Body Irradiation
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm; D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D000074323:Alemtuzumab; D016572:Cyclosporine; D016559:Tacrolimus; D008727:Methotrexate