title: Double cord blood transplantation in patients with high risk bone marrow failure syndromes.
abstract: Patients with bone marrow failure syndromes (BMFS) who reject a first allogeneic transplant or fail immunosuppressive therapy (IST) have an especially grim prognosis. We report 14 patients (eight adults, six children) transplanted with double cord blood transplantation (dUCBT) for BMFS. Neutrophil recovery was observed in eight patients, with full donor chimerism of one unit, and acute GVHD in 10. With a median follow-up of 23 months, the estimated 2 years overall survival was 80 +/- 17% and 33 +/- 16% for patients with acquired and inherited BMFS, respectively. Transplantation of two partially HLA-matched UCB thus enables salvage treatment of high-risk patients with BMFS.
authors.affiliation: Service d'Hématologie-Greffe, Hôpital Saint Louis, APHP, Paris University 7, Paris, France.
authors.forename: A
authors.initials: A
authors.lastname: Ruggeri
authors.forename: R Peffault
authors.initials: RP
authors.lastname: de Latour
authors.forename: V
authors.initials: V
authors.lastname: Rocha
authors.forename: J
authors.initials: J
authors.lastname: Larghero
authors.forename: M
authors.initials: M
authors.lastname: Robin
authors.forename: C A
authors.initials: CA
authors.lastname: Rodrigues
authors.forename: R
authors.initials: R
authors.lastname: Traineau
authors.forename: P
authors.initials: P
authors.lastname: Ribaud
authors.forename: C
authors.initials: C
authors.lastname: Ferry
authors.forename: A
authors.initials: A
authors.lastname: Devergie
authors.forename: E
authors.initials: E
authors.lastname: Gluckman
authors.forename: G
authors.initials: G
authors.lastname: Socié
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy; D001855:Bone Marrow Diseases / Q000628:therapy*; D002648:Child; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D004812:Epidemiologic Methods; D005199:Fanconi Anemia / Q000628:therapy; D005260:Female; D006085:Graft Survival; D006801:Humans; D008297:Male; D016879:Salvage Therapy / Q000379:methods; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome; D055815:Young Adult