title: Influence of nucleated cell dose on overall survival of unrelated cord blood transplantation for patients with severe acquired aplastic anemia: a study by eurocord and the aplastic anemia working party of the European group for blood and marrow transplantation.
abstract: Information is scarce on outcomes after unrelated cord blood transplantation (UCBT) for patients with severe aplastic anemia (SAA). We retrospectively analyzed 71 patients (median age, 13 years; 28 adults) with SAA (9 with paroxysmal nocturnal hemoglobinuria [PNH]) who received a single-unit (n = 57; 79%) or double-unit UCBT (n = 14; 19%) in 32 centers between 1996 and 2009. A reduced-intensity conditioning regimen was provided in 68% of the patients. The cumulative incidence (CI) of neutrophil recovery was 51% ± 6% at day 60, with significantly better engraftment seen in recipients of higher prefreezing total nucleated cell (TNC) dose (>3.9 10(7)/kg; hazard ratio [HR], 1.5; P = .05). The CI of platelet engraftment at day 180 posttransplantation was 37% ± 7%, that of grade II-IV acute GVHD was 20% ± 5%, and that of chronic GVHD at 3 years was 18% ± 5%. At a median follow-up of 35 months (range, 3-83 months), the estimated probability of 3-year overall survival (OS) was 38% ± 6%. Significantly improved OS was seen in recipients of >3.9 10(7) TNCs/kg prefreezing (45%, compared with 18% for recipients of ≤ 3.9 10(7) TNC/kg; HR, 0.4; P = .007). These results highlight the fundamental role of cell dose for both engraftment and OS in patients with SAA undergoing UCBT.
authors.affiliation: Bone Marrow Transplant Unit, Saint Louis Hospital, Paris, France. regis.peffaultdelatour@sls.aphp.fr
authors.forename: Regis
authors.initials: R
authors.lastname: Peffault de Latour
authors.forename: Duncan
authors.initials: D
authors.lastname: Purtill
authors.forename: Annalisa
authors.initials: A
authors.lastname: Ruggeri
authors.forename: Guillermo
authors.initials: G
authors.lastname: Sanz
authors.forename: Gerard
authors.initials: G
authors.lastname: Michel
authors.forename: Virginie
authors.initials: V
authors.lastname: Gandemer
authors.forename: Sebastien
authors.initials: S
authors.lastname: Maury
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.forename: Carmen
authors.initials: C
authors.lastname: Bonfim
authors.forename: Mahmoud
authors.initials: M
authors.lastname: Aljurf
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
authors.forename: Gerard
authors.initials: G
authors.lastname: Socié
authors.forename: Jakob
authors.initials: J
authors.lastname: Passweg
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D002452:Cell Count; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006085:Graft Survival; D006086:Graft vs Host Disease; D006801:Humans; D010976:Platelet Count; D012189:Retrospective Studies; D015996:Survival Rate; D055815:Young Adult