Favorable effect on acute and chronic graft-versus-host disease with cyclophosphamide and in vivo anti-CD52 monoclonal antibodies for marrow transplantation from HLA-identical sibling donors for acquired aplastic anemia

Vikas Gupta, Sarah E Ball, Qi-long Yi, Dedorah Sage, Shaun R McCann, Mark Lawler, Miguel Ortin, Mylene Freires, Geoff Hale, Hermann Waldmann, Edward C Gordon-Smith, Judith C W Marsh, Mark Lawler

Research output: Contribution to journalArticlepeer-review

44 Citations (Scopus)

Abstract

Between August 1989 and November 2003, 33 patients at our center with acquired aplastic anemia underwent bone marrow transplantation (BMT) from HLA-identical sibling donors with cyclophosphamide and in vivo anti-CD52 monoclonal antibodies (MoAb) for conditioning. The median age at BMT was 17 years (range, 4-46 years). Before BMT, 58% were heavily transfused (>50 transfusions), and 42% had previously experienced treatment failure with antithymocyte globulin-based immunosuppressive therapy. Unmanipulated bone marrow was used as the source of stem cells in all patients except 1. Graft-versus-host disease (GVHD) prophylaxis was with cyclosporine alone in 19 (58%) patients; 14 received anti-CD52 MoAb in addition to cyclosporine. The conditioning regimen was well tolerated without significant acute toxicity. Graft failure was seen in 8 patients (primary, n = 4; secondary, n = 4). Of those whose grafts failed, 4 survived long-term (complete autologous recovery, n = 2; rescue with previously stored marrow, n = 1; second allograft, n = 1). The cumulative incidence of graft failure and grade II to IV acute and chronic GVHD was 24%, 14%, and 4%, respectively. None developed extensive chronic GVHD. With a median follow-up of 59 months, the 5-year survival was 81% (95% confidence interval, 68%-96%). No unexpected early or late infectious or noninfectious complications were observed. We conclude that the conditioning regimen containing cyclophosphamide and anti-CD52 MoAb is well tolerated and effective for acquired aplastic anemia with HLA-matched sibling donors. The favorable effect on the incidence and severity of GVHD is noteworthy in this study and warrants further investigation.

Original languageEnglish
Pages (from-to)867-76
Number of pages10
JournalBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Volume10
Issue number12
DOIs
Publication statusPublished - Dec 2004

Keywords

  • Acute Disease
  • Adolescent
  • Adult
  • Anemia, Aplastic
  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Antibodies, Neoplasm
  • Antigens, CD
  • Antigens, Neoplasm
  • Bone Marrow Transplantation
  • Child
  • Child, Preschool
  • Chronic Disease
  • Cyclophosphamide
  • Female
  • Glycoproteins
  • Graft vs Host Disease
  • Histocompatibility Testing
  • Humans
  • Immunosuppressive Agents
  • Living Donors
  • Male
  • Middle Aged
  • Retrospective Studies
  • Siblings
  • Survival Analysis
  • Transplantation Chimera
  • Transplantation Conditioning

Fingerprint Dive into the research topics of 'Favorable effect on acute and chronic graft-versus-host disease with cyclophosphamide and in vivo anti-CD52 monoclonal antibodies for marrow transplantation from HLA-identical sibling donors for acquired aplastic anemia'. Together they form a unique fingerprint.

Cite this