title: Impact of HLA matching on outcome of hematopoietic stem cell transplantation in children with inherited diseases: a single-center comparative analysis of genoidentical, haploidentical or unrelated donors.
abstract: Hematological inherited diseases can be cured by hematopoietic stem cell transplantation (HSCT) from an human leukocyte antigen (HLA)-identical sibling donor (MSD), but the outcome of unrelated donors (URD) or haploidentical donors (HMD) has been a cause of concern. In all, 94 children affected with inherited diseases underwent HSCT at a single center using MSD (group A, n=31), URD (group B, n=23) or HMD (group C, n=40). There was no difference in the rate of engraftment or in the incidence of grades III-IV acute graft-versus-host disease (GVHD) between the groups. Survival rate was 80.6% in group A, 62.5% in group B and 47.5% in group C (P=0.023). In group B, survival rate was 73.7% in the subgroup with zero or one class I mismatch, and 25% in the subgroup with two or more class I mismatches (P=0.04). In group C, survival rate was 83.3% in the 9/10-identical subgroup, 64.3% in the seven or 8/10 subgroup, and 25% in the five or 6/10 subgroup (P=0.0007). Thus, engraftment, incidence of GVHD and survival are similar in recipients of grafts from MSD, URD with 0-1 class I-mismatch, or HMD with at least 7/10 HLA matches. The low success of HSCT using more disparate donors suggests reserving them for patients with very poor prognosis.
authors.affiliation: Laboratory of Immunology, Hopital Necker, Paris, France. caillat@necker.fr
authors.forename: S
authors.initials: S
authors.lastname: Caillat-Zucman
authors.forename: F
authors.initials: F
authors.lastname: Le Deist
authors.forename: E
authors.initials: E
authors.lastname: Haddad
authors.forename: M
authors.initials: M
authors.lastname: Gannagé
authors.forename: L
authors.initials: L
authors.lastname: Dal Cortivo
authors.forename: N
authors.initials: N
authors.lastname: Jabado
authors.forename: S
authors.initials: S
authors.lastname: Hacein-Bey-Abina
authors.forename: S
authors.initials: S
authors.lastname: Blanche
authors.forename: J-L
authors.initials: JL
authors.lastname: Casanova
authors.forename: A
authors.initials: A
authors.lastname: Fischer
authors.forename: M
authors.initials: M
authors.lastname: Cavazzana-Calvo
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D030342:Genetic Diseases, Inborn / Q000150:complications / Q000401:mortality / Q000628:therapy*; D005838:Genotype; D006085:Graft Survival / Q000276:immunology; D006086:Graft vs Host Disease / Q000276:immunology; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality / Q000706:statistics & numerical data; D006648:Histocompatibility / Q000235:genetics*; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D009894:Opportunistic Infections / Q000276:immunology; D016019:Survival Analysis; D014019:Tissue Donors; D014184:Transplantation, Homologous; D014185:Transplantation, Isogeneic; D016896:Treatment Outcome