title: Impact of graft-versus-host disease prophylaxis on outcomes after myeloablative single-unit umbilical cord blood transplantation.
abstract: Myeloablative single-unit umbilical cord blood transplantation (sUCBT) using busulfan, thiotepa, fludarabine, and antithymocyte globulin (Grupo Español de Trasplante Hematopoyético [GETH]-2005 protocol) resulted in high rates of engraftment and high antitumor activity. We designed a new graft-versus-host disease prophylaxis, substituting long-term steroids with mycophenolate mofetil together with a slight reduction of antithymocyte globulin (GETH/Gruppo Italiano Trapianto Midollo Osseo [GITMO]-2008 protocol). The results in 145 consecutive patients were compared with those obtained in 88 patients from the previous GETH-2005 trial. The cumulative incidence (CI) of myeloid engraftment at 60 days for patients in the GETH-2005 and GETH/GITMO-2008 trials was 94% and 88%, respectively, at a median time to neutrophil recovery of 19 and 23 days, respectively (P < .0001). In the multivariable analyses, platelet engraftment, acute and chronic graft-versus-host disease, nonrelapse mortality, relapse, and event-free survival were not significantly different. The 3-year event-free survival rate in the GETH/GITMO-2008 trial was 66%, 31%, and 25% for patients transplanted in early, intermediate, and advanced stages of the disease, respectively (P < .0001). This study confirms that myeloablative sUCBT using busulfan-based conditioning is a valuable strategy for patients with hematological malignancies. The use of mycophenolate mofetil apparently had an adverse effect on myeloid engraftment, and therefore a cautious use of this agent is warranted in the UCBT setting.
authors.affiliation: Department of Hematology, Hospital Universitario La Fe, Valencia, Spain. sanz_jai@gva.es
authors.forename: Jaime
authors.initials: J
authors.lastname: Sanz
authors.forename: Alessandra
authors.initials: A
authors.lastname: Picardi
authors.forename: Juan C
authors.initials: JC
authors.lastname: Hernández Boluda
authors.forename: Carmen
authors.initials: C
authors.lastname: Martín
authors.forename: Christelle
authors.initials: C
authors.lastname: Ferrá
authors.forename: Chiara
authors.initials: C
authors.lastname: Nozzoli
authors.forename: Marta
authors.initials: M
authors.lastname: Gonzalez-Vicent
authors.forename: Alessandro
authors.initials: A
authors.lastname: Rambaldi
authors.forename: David
authors.initials: D
authors.lastname: Valcarcel
authors.forename: Amparo
authors.initials: A
authors.lastname: Verdeguer
authors.forename: David
authors.initials: D
authors.lastname: Serrano
authors.forename: Cristina Díaz
authors.initials: CD
authors.lastname: de Heredia
authors.forename: María Jesús
authors.initials: MJ
authors.lastname: Pascual
authors.forename: Raquel
authors.initials: R
authors.lastname: de Paz
authors.forename: Pau
authors.initials: P
authors.lastname: Montesinos
authors.forename: Benedetta
authors.initials: B
authors.lastname: Bartolozzi
authors.forename: Alessandra
authors.initials: A
authors.lastname: Algarotti
authors.forename: Miguel A
authors.initials: MA
authors.lastname: Sanz
authors.forename: William
authors.initials: W
authors.lastname: Arcese
authors.forename: Guillermo F
authors.initials: GF
authors.lastname: Sanz
mesh_terms: D000328:Adult; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D008297:Male; D012307:Risk Factors; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods*; D055815:Young Adult
keywords: Engraftment; GVHD prophylaxis; Umbilical cord blood