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HLA Matching

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

Bone Marrow Transplantation volume 33, pages 1089–1095 (2004)Cite this article

Summary:

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.

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Acknowledgements

We are grateful to Colette Raffoux for critical reading of the manuscript. We thank Isabelle Hirsch for help in the collection of data, and Edith Audran, Patricia Przednowed and Christine Deshayes for excellent technical assistance.

This work was supported by Assitance Publique-Hôpitaux de Paris. We also thank France Greffe de Moelle for help in selection of donors, and Association Française des Myopathies for continuous support of the Laboratory of Cellular Therapy.

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Authors and Affiliations

  1. Laboratory of Immunology, Hopital Necker, Paris, France

    S Caillat-Zucman & M Gannagé

  2. Laboratory of Pediatric Immunology, Hopital Necker, Paris, France

    F Le Deist

  3. INSERM U429, Hopital Necker, Paris, France

    F Le Deist, S Hacein-Bey-Abina, A Fischer & M Cavazzana-Calvo

  4. Immunohematology Pediatrics Unit, Hopital Necker, Paris, France

    E Haddad, N Jabado, S Blanche, J-L Casanova & A Fischer

  5. Department of Biotherapy, Hopital Necker, Paris, France

    L Dal Cortivo, S Hacein-Bey-Abina & M Cavazzana-Calvo

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  1. S Caillat-Zucman
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Correspondence to S Caillat-Zucman.

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Caillat-Zucman, S., Le Deist, F., Haddad, E. et al. 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. Bone Marrow Transplant 33, 1089–1095 (2004). https://doi.org/10.1038/sj.bmt.1704510

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