1. ¹Laboratory of Immunology, Hopital Necker, Paris, France
2. ²Laboratory of Pediatric Immunology, Hopital Necker, Paris, France
3. ³INSERM U429, Hopital Necker, Paris, France
4. ⁴Immunohematology Pediatrics Unit, Hopital Necker, Paris, France
5. ⁵Department of Biotherapy, Hopital Necker, Paris, France

Correspondence: Dr S Caillat-Zucman, Laboratory of Immunology, Hopital Necker, 161 Rue de Sèvres, Paris 75015, France. E-mail: caillat@necker.fr

Received 22 September 2003; Accepted 23 January 2004; Published online 12 April 2004.

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.