title: Unrelated alternative donor transplantation for severe acquired aplastic anemia: a study from the French Society of Bone Marrow Transplantation and Cell Therapies and the EBMT Severe Aplastic Anemia Working Party.
abstract: Unrelated allogeneic transplantation for severe aplastic anemia is a treatment option after immunosuppressive treatment failure in the absence of a matched sibling donor. Age, delay between disease diagnosis and transplantation, and HLA matching are the key factors in transplantation decisions, but their combined impact on patient outcomes remains unclear. Using the French Society of Bone Marrow Transplantation and Cell Therapies registry, we analyzed all consecutive patients (n=139) who underwent a first allogeneic transplantation for idiopathic severe aplastic anemia from an unrelated donor between 2000 and 2012. In an adjusted multivariate model, age over 30 years (Hazard Ratio=2.39; P=0.011), time from diagnosis to transplantation over 12 months (Hazard Ratio=2.18; P=0.027) and the use of a 9/10 mismatched unrelated donor (Hazard Ratio=2.14; P=0.036) were independent risk factors that significantly worsened overall survival. Accordingly, we built a predictive score using these three parameters, considering patients at low (zero or one risk factors, n=94) or high (two or three risk factors, n=45) risk. High-risk patients had significantly shorter survival (Hazard Ratio=3.04; P<0.001). The score was then confirmed on an independent cohort from the European Group for Blood and Marrow Transplantation database of 296 patients, with shorter survival in patients with at least 2 risk factors (Hazard Ratio=2.13; P=0.005) In conclusion, a simple score using age, transplantation timing and HLA matching would appear useful to help physicians in the daily care of patients with severe aplastic anemia.
authors.affiliation: Hematology Department, Institut Paoli Calmettes, Marseille, France.
authors.forename: Raynier
authors.initials: R
authors.lastname: Devillier
authors.affiliation: Pediatric Hematology Unit, Robert Debré University Hospital, Paris, France.
authors.forename: Jean-Hugues
authors.initials: JH
authors.lastname: Dalle
authors.affiliation: Hematology Department, King's College Hospital, London, UK.
authors.forename: Austin
authors.initials: A
authors.lastname: Kulasekararaj
authors.affiliation: Pediatric Hematology Unit, University Hospital, Nancy, France.
authors.forename: Maud
authors.initials: M
authors.lastname: D'aveni
authors.affiliation: Pediatric Hematology Unit, University Hospital, Nancy, France Hematology Department, Haut-Leveque Hospital and Bordeaux University Hospital, Pessac, France.
authors.forename: Laurence
authors.initials: L
authors.lastname: Clément
authors.affiliation: Hematology Department, Wroclaw Medical University, Poland.
authors.forename: Alicja
authors.initials: A
authors.lastname: Chybicka
authors.affiliation: Hematology Department, Haut-Leveque Hospital and Bordeaux University Hospital, Pessac, France.
authors.forename: Stéphane
authors.initials: S
authors.lastname: Vigouroux
authors.affiliation: Hematology Department, University Hospital, Nantes, France.
authors.forename: Patrice
authors.initials: P
authors.lastname: Chevallier
authors.affiliation: Hematology Department, St. George's Hospital, London, UK.
authors.forename: Mickey
authors.initials: M
authors.lastname: Koh
authors.affiliation: Pediatric Hematology Unit, University Hospital, Lyon, France.
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.affiliation: Hematology Department, University Hospital, Lyon, France.
authors.forename: Mauricette
authors.initials: M
authors.lastname: Michallet
authors.affiliation: Hematology Department, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.affiliation: Hematology Department, University Hospital, Lille, France.
authors.forename: Ibrahim
authors.initials: I
authors.lastname: Yakoub-Agha
authors.affiliation: Hematology Department, University Hospital, Grenoble, France.
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Cahn
authors.affiliation: Hematology Department, Karolinska University Hospital, Stockholm, Sweden.
authors.forename: Per
authors.initials: P
authors.lastname: Ljungman
authors.affiliation: Hematology Department, University Hospital, Rennes, France.
authors.forename: Marc
authors.initials: M
authors.lastname: Bernard
authors.affiliation: HLA Laboratory, Saint-Louis University Hospital, Paris, France French Society of Histocompatibility and Immunogenetics (SFHI), France.
authors.forename: Pascale
authors.initials: P
authors.lastname: Loiseau
authors.affiliation: HLA Laboratory, Etablissement Français du Sang, Lyon, France.
authors.forename: Valérie
authors.initials: V
authors.lastname: Dubois
authors.affiliation: Hematology Department, Henri Mondor Hospital, Créteil, France.
authors.forename: Sébastien
authors.initials: S
authors.lastname: Maury
authors.affiliation: BMT Department, Saint-Louis Hospital, Diderot Paris 7 University, Paris, France.
authors.forename: Gérard
authors.initials: G
authors.lastname: Socié
authors.affiliation: Clinical and Experimental Haematology Unit, Giannina Gaslini Children's Hospital, Genova, Italy.
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
authors.affiliation: BMT Department, Saint-Louis Hospital, Diderot Paris 7 University, Paris, France regis.peffaultdelatour@aphp.fr.
authors.forename: Regis
authors.initials: R
authors.lastname: Peffault de Latour
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000175:diagnosis* / Q000401:mortality / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000379:methods; D002648:Child; D002675:Child, Preschool; D005260:Female; D005602:France; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D007223:Infant; D008212:Lymphocyte Depletion; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D012720:Severity of Illness Index; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D061349:Unrelated Donors; D055815:Young Adult