title: Results and factors influencing outcome after fully haploidentical hematopoietic stem cell transplantation in children with very high-risk acute lymphoblastic leukemia: impact of center size: an analysis on behalf of the Acute Leukemia and Pediatric Disease Working Parties of the European Blood and Marrow Transplant group.
abstract: T cell-depleted haploidentical hematopoietic stem cell transplantation (haploHSCT) is an option to treat children with very high-risk acute lymphoblastic leukemia (ALL) lacking an HLA-identical donor. We analyzed 127 children with ALL who underwent haploHSCT in first (n = 22), second (n = 48), or third (n = 32), complete remission or in relapse (n = 25). The 5-year leukemia-free survival (LFS) was 30%, 34%, 22%, and 0%, respectively. A risk-factor analysis was performed for patients who underwent transplantation in remission (n = 102). Five-year nonrelapse mortality (NRM), relapse incidence (RI), and LFS were 37%, 36%, and 27%, respectively. A trend of improved LFS rate and decreased RI was observed for children given a graft with higher number of CD34(+) cells (adjusted P = .09 and P = .07, respectively). In a multivariate analysis, haploHSCT performed in larger centers (performing > or = 231 allotransplantations in the studied period) was associated with improved LFS rate and decreased RI (adjusted P = .01 and P = .04, respectively), adjusting for different patient-, disease-, and transplant-related factors such as number of previous autotransplantations, cytomegalovirus serology status, type of T-cell depletion, and use of total body irradiation and antithymocyte globulin. In conclusion, higher CD34(+) cell dose and better patient selection may improve outcomes of children with ALL who undergo a haploHSCT. Transplant centers initiating programs on haploHSCT for children may collaborate with more experienced centers.
authors.affiliation: Klinik fuer Kinder-und Jugendmedizin III, Johann Wolfgang Goethe Universitaet, Frankfurt, Germany. thomas.klingebiel@kgu.de
authors.forename: Thomas
authors.initials: T
authors.lastname: Klingebiel
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Cornish
authors.forename: Myriam
authors.initials: M
authors.lastname: Labopin
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Philippe
authors.initials: P
authors.lastname: Darbyshire
authors.forename: Rupert
authors.initials: R
authors.lastname: Handgretinger
authors.forename: Adriana
authors.initials: A
authors.lastname: Balduzzi
authors.forename: Joanna
authors.initials: J
authors.lastname: Owoc-Lempach
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.forename: Reuven
authors.initials: R
authors.lastname: Or
authors.forename: Christina
authors.initials: C
authors.lastname: Peters
authors.forename: Franco
authors.initials: F
authors.lastname: Aversa
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Polge
authors.forename: Giorgio
authors.initials: G
authors.lastname: Dini
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006776:Hospitals, Pediatric; D006801:Humans; D007223:Infant; D008297:Male; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000401:mortality / Q000628:therapy*; D012074:Remission Induction; D012307:Risk Factors; D015996:Survival Rate; D014184:Transplantation, Homologous