Bone marrow transplantation from partially HLA-mismatched family donors for acute leukemia: single-center experience of 201 patients

Bone Marrow Transplant. 2004 Feb;33(4):389-96. doi: 10.1038/sj.bmt.1704391.


Between February 1993 and December 1999, 201 patients (1-59 years old, median 23) with acute leukemia (67% not in remission) underwent ex vivo T-cell-depleted (TCD) bone marrow transplants (BMT) from partially mismatched related donors (PMRD; 92% mismatched for 2-3 HLA A, B, DR antigens). Conditioning comprised total body irradiation, cyclophosphamide, cytarabine, etoposide, anti-thymocyte globulin (ATG), and methylprednisolone. Graft-versus-host disease (GVHD) prophylaxis comprised partial TCD with OKT3 (n=143) or T10B9 (n=58), steroids, ATG, and cyclosporine. The engraftment rate was 98%. The cumulative incidences of grades II-IV acute GVHD and chronic GVHD were 13 and 15%, respectively. The 5-year cumulative incidences of relapse and transplant-related mortality (TRM) were 31 and 51%, respectively. The actuarial 5-year overall survival (OS) and disease-free survival (DFS) probabilities were 19 and 18%, respectively. Patient age >15 years, active disease at transplant, donor age >25 years, and 3-antigen donor mismatch (host-versus-graft) affected the outcome adversely. The actuarial 5-year OS of four groups of patients identified based upon these risk factors was 39, 20, 13, and 0%, respectively (P<0.0001). We conclude that PMRD BMT is a potential treatment option for patients with high-risk acute leukemia who require an alternative donor transplant and fall into a group with a reasonable expected outcome.

MeSH terms

  • Acute Disease
  • Adolescent
  • Adult
  • Bone Marrow Transplantation / adverse effects
  • Bone Marrow Transplantation / immunology*
  • Bone Marrow Transplantation / mortality
  • Child
  • Child, Preschool
  • Female
  • Graft Survival
  • Graft vs Host Disease / drug therapy
  • Graft vs Host Disease / prevention & control
  • Histocompatibility Testing*
  • Histocompatibility*
  • Humans
  • Infant
  • Leukemia / complications
  • Leukemia / mortality
  • Leukemia / therapy*
  • Lymphocyte Depletion
  • Male
  • Middle Aged
  • Proportional Hazards Models
  • Retrospective Studies
  • Risk Factors
  • Survival Analysis