title: Long-term outcome and prognostic factors of unrelated cord blood transplantation in children with haematological malignancies: a retrospective study using the Spanish Working Party for BMT in Children (GETMON) database.
abstract: Outcomes of unrelated cord blood transplants (UCBT) were assessed in 172 consecutive children, median age 5 years (range: 0.5-18), with haematological malignancies treated at nine Spanish hospitals between February 1996 and April 2009. Data were collected from the Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON) database. ALL was diagnosed in 125 patients, AML in 43 and myelodysplastic syndrome in 4. Myeloid engraftment (ANC⩾0.5 × 10(9)/L) occurred in 87.2% at a median of 22 days and was associated with the total nucleated cell (TNC) dose infused and use of a TT-containing conditioning regimen. Cumulative incidence of relapse was 20% at 1 year post transplant and 29% at 3 years, being higher in patients with a diagnosis of ALL, very high risk disease and GVHD grades 0-1. Cumulative incidence of non-relapse mortality (NRM) was 19% at 100 days post transplant and 39% at 1 year. BU-FLU-TT-ATG-conditioned patients had lower NRM. Disease-free survival (DFS) was 40% at 2 years post transplant (for patients transplanted since 2006). On multivariate analysis, TNC dose infused, AML and BU-FLU-TT-ATG-conditioning regimen increased the probability of DFS. It is of paramount importance to select cord blood units with the highest cell dose. As the BU-FLU-TT-ATG-conditioning regimen was associated with better DFS owing to lower NRM, further prospective studies testing this regimen are warranted.
authors.affiliation: Servicio de Oncología y Hematología Pediátricas, Hospital Universitario Vall d'Hebron, Universitat Autónoma de Barcelona, Barcelona, Spain.
authors.forename: C
authors.initials: C
authors.lastname: Díaz de Heredia
authors.affiliation: Unidad de Trasplante, Hospital Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain.
authors.forename: M
authors.initials: M
authors.lastname: González
authors.affiliation: Unidad de Oncología y TPH Pediátrico, Hospital Universitario Politécnico La Fe, Universidad de Valencia, Valencia, Spain.
authors.forename: A
authors.initials: A
authors.lastname: Verdeguer
authors.affiliation: Servicio de Oncología y Hematología Pediátricas, Hospital Universitario Vall d'Hebron, Universitat Autónoma de Barcelona, Barcelona, Spain.
authors.forename: I
authors.initials: I
authors.lastname: Elorza
authors.affiliation: Servicio de Hematología, Unidad de Hematología Infantil, Hospital Universitario Reina Sofía, Universidad de Córdoba, Córdoba, Spain.
authors.forename: A
authors.initials: A
authors.lastname: Rodriguez
authors.affiliation: Unidad de Hematología y Oncología Infantil, Hospital la Paz, Universidad Autónoma de Madrid, Madrid, Spain.
authors.forename: A
authors.initials: A
authors.lastname: Martinez
authors.affiliation: Servicio de Hematología, Hospital Virgen del Rocío, Universidad de Sevilla, Sevilla, Spain.
authors.forename: J M
authors.initials: JM
authors.lastname: Pérez
authors.affiliation: Unidad Pediátrica de Hematología, Oncología y TPH, Hospital de Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.
authors.forename: I
authors.initials: I
authors.lastname: Badell
authors.affiliation: Servicio de Hematología, Hospital Universitario Regional Carlos Haya, Universidad de Málaga, Málaga, Spain.
authors.forename: M E
authors.initials: ME
authors.lastname: Gonzalez
authors.affiliation: Servicio de Oncología y Hematología Pediátricas, Hospital Universitario Vall d'Hebron, Universitat Autónoma de Barcelona, Barcelona, Spain.
authors.forename: T
authors.initials: T
authors.lastname: Olivé
authors.affiliation: Unidad de Oncología y TPH Pediátrico, Hospital Universitario Politécnico La Fe, Universidad de Valencia, Valencia, Spain.
authors.forename: J M
authors.initials: JM
authors.lastname: Fernández
authors.affiliation: Unidad de Hematología Pediátrica, Servicio de Pediatría, Hospital Universitario Ramón y Cajal, Universidad de Alcalá, Madrid, Spain.
authors.forename: M S
authors.initials: MS
authors.lastname: Maldonado
authors.affiliation: Unidad de Trasplante, Hospital Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain.
authors.forename: M A
authors.initials: MA
authors.lastname: Díaz
authors.affiliation: Servicio de Oncología y Hematología Pediátricas, Hospital Universitario Vall d'Hebron, Universitat Autónoma de Barcelona, Barcelona, Spain.
authors.forename: J
authors.initials: J
authors.lastname: Sánchez de Toledo
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects; D016208:Databases, Factual; D018572:Disease-Free Survival; D005260:Female; D019337:Hematologic Neoplasms / Q000401:mortality / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D011379:Prognosis; D012189:Retrospective Studies; D013030:Spain / Q000453:epidemiology; D016896:Treatment Outcome; D061349:Unrelated Donors