Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease

Bone Marrow Transplant. 2000 Aug;26(4):445-9. doi: 10.1038/sj.bmt.1702518.

Abstract

Although allogeneic transplantation can be curative for patients with sickle cell disease, the toxicity of conditioning regimens has precluded its use in adults with significant end-organ damage. Newer conditioning regimens have been developed that are less toxic and that may broaden the applicability of allogeneic transplantation in this disorder. We report two adults with end-stage sickle cell disease, who underwent allogeneic transplantation from an HLA-identical sibling donor after conditioning with fludarabine/melphalan and ATG. Both patients had been extensively transfused and one had multiple RBC antibodies. One of the patients also had end-stage renal disease, and was dialysis dependent. Engraftment occurred promptly in both patients. Both achieved 100% donor chimerism and both were free of pain crises after transplant. The first patient died of a respiratory failure related to chronic graft-versus-host disease (GVHD) on day 335 after transplantation. The second patient developed severe gastro-intestinal GVHD and TTP and died on day 147 after transplantation. Conditioning with fludarabine/melphalan and ATG followed by allogeneic stem cell transplantation resulted in prompt and reliable engraftment in adults with end-stage sickle cell disease. The incidence of severe GVHD was unacceptably high and may be related to the ethnicity of the patients or to the inflammatory state associated with pre-existing sickle cell disease.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Anemia, Sickle Cell / therapy*
  • Fatal Outcome
  • Female
  • Graft Survival
  • Graft vs Host Disease
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Immunosuppressive Agents / toxicity
  • Middle Aged
  • Nuclear Family
  • Transplantation Chimera
  • Transplantation Conditioning / methods
  • Transplantation Conditioning / standards
  • Transplantation, Homologous / methods
  • Vidarabine / administration & dosage*
  • Vidarabine / analogs & derivatives*
  • Vidarabine / toxicity

Substances

  • Immunosuppressive Agents
  • Vidarabine
  • fludarabine