PMID- 15077132 OWN - NLM STAT- MEDLINE DCOM- 20050302 LR - 20140728 IS - 0268-3369 (Print) IS - 0268-3369 (Linking) VI - 33 IP - 11 DP - 2004 Jun TI - 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. PG - 1089-95 AB - 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. CI - Copyright 2004 Nature Publishing Group FAU - Caillat-Zucman, S AU - Caillat-Zucman S AD - Laboratory of Immunology, Hopital Necker, Paris, France. caillat@necker.fr FAU - Le Deist, F AU - Le Deist F FAU - Haddad, E AU - Haddad E FAU - Gannage, M AU - Gannage M FAU - Dal Cortivo, L AU - Dal Cortivo L FAU - Jabado, N AU - Jabado N FAU - Hacein-Bey-Abina, S AU - Hacein-Bey-Abina S FAU - Blanche, S AU - Blanche S FAU - Casanova, J-L AU - Casanova JL FAU - Fischer, A AU - Fischer A FAU - Cavazzana-Calvo, M AU - Cavazzana-Calvo M LA - eng PT - Comparative Study PT - Journal Article PT - Research Support, Non-U.S. Gov't PL - England TA - Bone Marrow Transplant JT - Bone marrow transplantation JID - 8702459 SB - IM MH - Adolescent MH - Child MH - Child, Preschool MH - Genetic Diseases, Inborn/complications/mortality/*therapy MH - Genotype MH - Graft Survival/immunology MH - Graft vs Host Disease/immunology MH - Haplotypes MH - *Hematopoietic Stem Cell Transplantation/adverse effects/mortality/statistics & numerical data MH - Histocompatibility/*genetics MH - *Histocompatibility Testing MH - Humans MH - Infant MH - Opportunistic Infections/immunology MH - Survival Analysis MH - Tissue Donors MH - Transplantation, Homologous MH - Transplantation, Isogeneic MH - Treatment Outcome EDAT- 2004/04/13 05:00 MHDA- 2005/03/03 09:00 CRDT- 2004/04/13 05:00 PHST- 2004/04/13 05:00 [pubmed] PHST- 2005/03/03 09:00 [medline] PHST- 2004/04/13 05:00 [entrez] AID - 1704510 [pii] AID - 10.1038/sj.bmt.1704510 [doi] PST - ppublish SO - Bone Marrow Transplant. 2004 Jun;33(11):1089-95. doi: 10.1038/sj.bmt.1704510.