title: Second Allogeneic Hematopoietic Cell Transplantation for Patients with Fanconi Anemia and Bone Marrow Failure.
abstract: A second allogeneic hematopoietic cell transplantation (HCT) is the sole salvage option for individuals who develop graft failure after their first HCT. Data on outcomes after second HCT in patients with Fanconi anemia (FA) are scarce. Here we report outcomes after second allogeneic HCT for FA (n = 81). The indication for second HCT was graft failure after the first HCT. Transplantations were performed between 1990 and 2012. The timing of the second HCT predicted subsequent graft failure and survival. Graft failure was high when the second HCT was performed less than 3 months from the first. The 3-month probability of graft failure was 69% when the interval between the first HCT and second HCT was less than 3 months, compared with 23% when the interval was longer (P < .001). Consequently, the 1-year survival rate was substantially lower when the interval between the first and second HCTs was less than 3 months compared with longer (23% vs 58%; P = .001). The corresponding 5-year probability of survival was 16% and 45%, respectively (P = .006). Taken together, these data suggest that fewer than one-half of patients with FA undergoing a second HCT for graft failure are long-term survivors. There is an urgent need to develop strategies to reduce the rate of graft failure after first HCT.
authors.affiliation: Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. Electronic address: mouhab@kfshrc.edu.sa.
authors.forename: Mouhab
authors.initials: M
authors.lastname: Ayas
authors.affiliation: Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Mary
authors.initials: M
authors.lastname: Eapen
authors.affiliation: Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin; Division of Biostatistics, Institute for Health and Society, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Le-Rademacher
authors.affiliation: Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Jeanette
authors.initials: J
authors.lastname: Carreras
authors.affiliation: Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California.
authors.forename: Hisham
authors.initials: H
authors.lastname: Abdel-Azim
authors.affiliation: Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
authors.forename: Blanche P
authors.initials: BP
authors.lastname: Alter
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas M.D. Anderson Cancer Center, Houston, Texas.
authors.forename: Paolo
authors.initials: P
authors.lastname: Anderlini
authors.affiliation: Hematology Branch, National Heart and Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
authors.forename: Minoo
authors.initials: M
authors.lastname: Battiwalla
authors.affiliation: Department of Pediatric Hematology, University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: Marc
authors.initials: M
authors.lastname: Bierings
authors.affiliation: Division of Pediatrics Hematology, Children's Hospital of Orange County, Orange, California.
authors.forename: David K
authors.initials: DK
authors.lastname: Buchbinder
authors.affiliation: Hospital de Clinicas, Federal University of Parana, Curitiba, Brazil.
authors.forename: Carmem
authors.initials: C
authors.lastname: Bonfim
authors.affiliation: Midwest Center for Cancer and Blood Disorders, Medical College of Wisconsin and Children's Hospital of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Bruce M
authors.initials: BM
authors.lastname: Camitta
authors.affiliation: Department of Pediatrics, University of Gothenburg, Gothenburg, Sweden.
authors.forename: Anders L
authors.initials: AL
authors.lastname: Fasth
authors.affiliation: Hematology Research Centre, Division of Experimental Medicine, Department of Medicine, Imperial College London, London, United Kingdom.
authors.forename: Robert Peter
authors.initials: RP
authors.lastname: Gale
authors.affiliation: Department of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts.
authors.forename: Michelle A
authors.initials: MA
authors.lastname: Lee
authors.affiliation: Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Medical Center, Minneapolis, Minnesota.
authors.forename: Troy C
authors.initials: TC
authors.lastname: Lund
authors.affiliation: Division of Bone Marrow Transplant and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
authors.forename: Kasiani C
authors.initials: KC
authors.lastname: Myers
authors.affiliation: Division of Therapeutic Immunology, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden; Centre for Clinical Research Sörmland, Uppsala University, Uppsala, Sweden.
authors.forename: Richard F
authors.initials: RF
authors.lastname: Olsson
authors.affiliation: Pediatric Blood and Marrow Transplant, Duke University Medical Center, Durham, North Carolina.
authors.forename: Kristin M
authors.initials: KM
authors.lastname: Page
authors.affiliation: Blood and Cancer Centre, Starship Children's Hospital, Auckland, New Zealand.
authors.forename: Tim D
authors.initials: TD
authors.lastname: Prestidge
authors.affiliation: Pediatric Hematology/Oncology/Stem Cell Transplantation, Children's Mercy Hospital, Kansas City, Missouri.
authors.forename: Mohamed
authors.initials: M
authors.lastname: Radhi
authors.affiliation: Division of Hematology/Oncology, Department of Pediatrics, Mattel Children's Hospital at UCLA, Los Angeles, California.
authors.forename: Ami J
authors.initials: AJ
authors.lastname: Shah
authors.affiliation: Department of Pediatric Hematology, Oncology and Bone Marrow Transplant, British Columbia's Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
authors.forename: Kirk R
authors.initials: KR
authors.lastname: Schultz
authors.affiliation: Division of Bone Marrow Transplant, Seattle Cancer Care Alliance, Seattle, Washington.
authors.forename: Baldeep
authors.initials: B
authors.lastname: Wirk
authors.affiliation: Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Medical Center, Minneapolis, Minnesota.
authors.forename: John E
authors.initials: JE
authors.lastname: Wagner
authors.affiliation: Clincal Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
authors.forename: H Joachim
authors.initials: HJ
authors.lastname: Deeg
mesh_terms: D000293:Adolescent; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D006084:Graft Rejection / Q000401:mortality / Q000628:therapy*; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000706:statistics & numerical data*; D006801:Humans; D007223:Infant; D007239:Infections / Q000401:mortality; D053208:Kaplan-Meier Estimate; D007958:Leukocyte Count; D008232:Lymphoproliferative Disorders / Q000209:etiology; D008297:Male; D009102:Multiple Organ Failure / Q000209:etiology / Q000401:mortality; D009504:Neutrophils; D016879:Salvage Therapy; D015996:Survival Rate; D055815:Young Adult
keywords: Fanconi anemia; Graft failure; Second transplantation